Stem Cells & Gene Therapy

PROGRAMME BARCELONA 2019 CHANGING THE FACE ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS

OF MODERN MEDICINE: 16-19 OCTOBER 2018 ESGCT STEM CELLS & SWISSTECH LAUSANNE LAUSANNE 2018 ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 27 TH ANNUAL GENE THERAPY CONGRESS IN COLLABORATION WITH SETGyC

22-25 OCTOBER 2019 BARCELONA INTERNATIONAL CONVENTION CENTRE

XXVI ANNUAL CONGRESS 15th ANNUAL MEETING #CHANGINGMODERNMED WHAT, WHO, WHEN & WHERE, WELCOME AT YOUR FINGERTIPS...

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DOWNLOAD THE APP DOWNLOAD Create your SWISSTECH and its innovative technologies. the Gala Venue technology. CONVENTION CENTER The architecture of the building The west facade of the building own schedule The SwissTech Convention allows three auditoriums to is covered with panels made Center is one of the largest become one; the number of of organic dye-sensitized conference centres in the seats in each auditorium can solar cell, also called “Grätzel Lake Geneva region hosting also be changed in just a few cells” after Michael Grätzel, a Navigate the venue events of international fame. minutes. This modularity is physical chemistry professor This majestic building is based on two mechanisms: a at the EPFL and the inventor distinguished by its modernity system of sliding walls, and of this technology.

WEB: Search for Abstracts

And plenty more! REGISTRATION SPEAKER READY ROOM INFORMATION BOARD Registration is located on the The speaker ready room Delegates may post CVs, Campus floor, near the main is Office A/B on the employment opportunities or entrance to the SwissTech Garden Floor. When facing information on the designated Convention Center. the cloakroom, the Speaker board located near the GOOGLE PLAY: registration desk. We will be available: Ready Room is to your right. Tues 16th: 08.00 - 20.30 ABSTRACTS AND USEFUL CONTACTS DELEGATE LISTS Weds 17th: 08.00 - 19.00 HOW TO DOWNLOAD LOGIN Electronic copies can be Thurs 18th: 08.30 - 19.30 Ambulance ...... 144 THE APP Login with your Badge ID accessed through our congress Fri 19th: 08.30 - 19.30 • Option 1: Simply scan the relevant (printed underneath the QR Police ...... 117 app (please see inside front QR code (see left) code on your badge) WI-FI ACCESS Fire brigade ...... 118 cover) or in My Congress APP STORE: • Option 2: Search for EventPilot Wi-Fi is available Materials in your account NEED HELP TO GET Taxi: ...... +41 844 814 814 on the ESGCT website. Conference App in the App or STARTED? throughout the Google Play store Once you have downloaded the SwissTech Convention Center. [email protected] When prompted enter the event Login: Lausanne2018 Gaëlle Jamar, ESGCT manager app, click the banner for your +44 776 647 5379 code: ChangingModernMed app guide to get started Password: lausanne2018

ESGCT / ISSCR / SFTCG Collaborative Congress 2018 1 ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 CONTENTS

Welcome address ...... 4 Time to get social ...... 35

Partners ...... 7 Exhibitor floorplans ...... 38

Boards and committees 8 Partners ...... 46

Awards ...... 12 Exhibitors ...... 64

Venue maps ...... 16 Programme ...... 84

Congress locations on the metro 22 Poster sessions ...... 115

Lausanne – off the beaten track 32

ENGINEERING WITH SPECIAL THANKS TO: HOPE

AFFILIATED JOURNAL:

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Book Illustration & Design Book Print Production www.7thfloor.co.uk www.fineprint.co.uk © 2018 MeiraGTx Limited. All Rights Reserved. ESGCT / ISSCR / SFTCG Collaborative Congress 2018 3 WELCOME TO THE XXVI TH ESGCT CONGRESS, LAUSANNE 2018 The boards of ESGCT, ISSCR and SFTCG, social events during the meeting, including along with the Swiss gene and cell therapy the welcome reception, Molecular Mingle community, welcome you to Lausanne for networking evening and closing drinks. This the 2018 Annual Congress of ESGCT. The year the Molecular Mingle will be held at meeting will take place at the SwissTech the Olympic Museum in the centre of Convention Center in the centre of the Lausanne. The city has been the headquarters campus of the Ecole Polytechnique of the International Olympic Committee Federale de Lausanne (EPFL). The for more than a century, and the Olympic Center, characterised by its astonishing Museum has many fascinating exhibitions architecture and innovative technologies, on sport and the history of the games. The will provide a superb environment for museum will be for the sole use of our networking and sharing of exciting congress so we can enjoy each other’s advances. company whilst browsing. The scientific programme will highlight During this meeting you will be in the heart recent progress on ground-breaking of the Alps and on the shore of Lake Geneva. technologies, outstanding pre-clinical The region has an exceptionally strong proof-of-concept studies in gene and cell economic, cultural, sporting and academic therapy, and updates on on-going clinical momentum. The stunning landscape of the trials involving the wide range of conditions eleventh century terraced vineyards of the that are now targets for advanced Lavaux region, with the medieval village therapies. To support exchange of of St-Saphorin, has been recognised by information and expertise between various UNESCO as a World Heritage Centre. stakeholders and ensure dissemination of The amazing Chillon castle, the most visited knowledge and expertise, we have organised castle in Switzerland, is very nearby. a clinical trial & commercialisation The region is also rich in gastronomic workshop, along with educational sessions traditions and has many musical and artistic and public events. The meeting will attractions, including Chaplin’s World with also provide a unique opportunity to its magical journey through silent cinema, acknowledge the outstanding contributions and the Montreux Jazz Festival. of the members of the ESGCT and our th partnering societies for their pioneering As we look forward to the XXVI ESGCT achievements in the fields of gene and Congress and welcoming delegates from all For the first time, the ESGCT Congress is being cellular therapy. over the world to Lausanne, we hope that held in Switzerland. This year it is jointly organised you enjoy the meeting and thank you for We very much hope that you will attend the your being part of it. with the International Society for Stem Cell Research (ISSCR), the French Society of Gene and Cell Therapy (SFTCG), and the Swiss local organising committee.

Robin R Ali Doug Melton Pierre Cordelier Nicole Deglon President, ESGCT President, ISSCR President, SFTCG President, LOC

4 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 5 THANK YOU TO ALL OUR PARTNERS

DIAMOND PARTNERS

PLATINUM PARTNERS

GOLD PARTNERS C

CMY

SILVER PARTNERS

BRONZE PARTNERS

6 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 7 BOARDS ISSCR

Douglas A. Melton Roger A. Barker Haifan Lin ESGCT President Director Director (Harvard University, Cambridge, (University of Cambridge) (Yale Stem Cell Center, Yale MA) University School of Medicine, Marianne E. Bronner New Haven, CT) Hans C. Clevers Robin Ali Alessandro Aiuti Adrian Thrasher Director Past President (California Institute of Technology, Melissa H. Little President (HSR Tiget, Milan) (University College London) (Hubrecht Institute, Utrecht) Pasadena, CA) Director (University College London) Alberto Auricchio Joost van Haasteren (Murdoch Children’s Research Deepak Srivastava Hildegard Büning (TIGEM, Naples) Student Board Member Fiona Doetsch Institute and University of President Elect Melbourne, VI) Vice President (University of Oxford) Director (Gladstone Institutes, San (Biozentrum at the University of (Hannover Medical School) Nathalie Cartier-Lacave (INSERM/ CEA UMR1169, MIRCEN Larisa Condurat Fransisco, CA) Basel) Charles E. Murry Christof von Kalle CEA and University Paris-Sud, Student Board Member Director Amander T. Clark (Institute for Stem Cell and Treasurer University Paris Saclay) (University of Freiburg) Valentina Greco Regenerative Medicine, University (DKFZ / NCT, Heidelberg) Clerk Director Vincenzo Cerullo (University of California, Los (Yale Stem Cell Center, Yale of Washington, Seattle, WA) Juan Bueren (University of Helsinki) Angeles, CA) University School of Medicine, Martin F. Pera General Secretary New Haven, CT) Zoltan Ivics Director (CIEMAT / CIBERER-ISCIII, Madrid) Christine L. Mummery (Paul Ehrlich Institute, Langen) Vice President Konrad Hochedlinger (The Jackson Laboratory, Bar (Leiden University Medical Center) Director Harbor, ME) (Massachusetts General Hospital Arnold R. Kriegstein and Harvard University, MA) Hans R. Schöler Treasurer Director (University of California San Jane Lebkowski (Max Planck Institute for SFTCG Molecular Biomedicine, Münster) Francisco, CA) Director (Regenerative Patch Technologies, Santa Barbara, CA) Takanori Takebe Director Pierre Cordelier Eduard Ayuso Federico Mingozzi Ruth Lehmann (Yokohama City University, Japan; President (University of Nantes (IRT-UN) (Genethon, University Pierre & Director Cincinnati Children’s Hospital (Inserm UMR1037, INSERM UMR1089) Marie Curie, INSERM U974, Paris; (NYU School of Medicine, New Medical Center, OH) University Toulouse III – Paul Spark Therapeutics, Philadelphia, York City, NY) Joanna Wysocka Sabatier, ERL5294 CNRS) Karim Benihoud PN) (Institute of Cancer Gustave Urban Lendahl, Director Els Verhoeyen Roussy, CNRS UMR8203, François Moreau-Gaudry Director (Stanford University, CA) Vice President Paris) (University of Bordeaux, INSERM (Karolinska Institute, Stockholm) (EVIR, INSERM U758, ENS, Lyon) U1035) Ex Officio Members: Nicolas Boisgerault Olivier Nègre Patrick Midoux (Nantes – Angers (IRS-UN) CRCINA Sally Temple (Bluebird Bio, Cambridge MA) Treasurer – INSERM1232) (Neural Stem Cell Institute, New York City, NY) (Centre de Biophysique Moléculaire, Michel Pucéat Nathalie Cartier CNRS UPR4301, Orléans) (Aix Marseille University La Leonard I. Zon (MIRCen, INSERM U986, CEA, Timone, INSERM UMR1251) (Boston Children’s Hospital, MA) Leila Maouche-Chrétien Fontenay aux Roses) Secretary Matthias Titeux (Imagine Institute, INSERM U1163, John Devos (Imagine Institute, Inserm U1163, CNRS ERL8254, Paris) (University Hospital Montpellier) Paris)

8 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 9 BOARDS SAREPTA THERAPEUTICS ASPIRES TO BE ONE OF THE MOST

Continued IMPORTANT PRECISION GENETIC

PRESIDENTIAL LOC LOC MEDICINE COMPANIES, APPLYING

ITS EXPERTISE TO DEVELOP Nicole Déglon Yvan Arsenijevic President (HOJG, Lausanne) THERAPIES FOR A VARIETY (Lausanne University Hospital) Fabio Candotti Patrick Aebischer (Lausanne University Hospital) OF RARE DISEASES. (EPFL, Lausanne) Sonja Kleinlogel Georges Coukos (University of Bern) (Lausanne University Hospital) Bernard Schneider Martin Fussenegger (EPFL, Lausanne) (ETH, Zurich) Michael de Palma Didier Trono (EPFL, Lausanne) (EPFL, Lausanne)

ISSCR ORGANISING LEADERS IN PRECISION GENETIC MEDICINE COMMITTEE

Nissim Benvenisty (Hebrew University, Israel)

Robert Blelloch (University of California, San Francisco, CA)

Malin Parmar (Lund University)

Alex Meissner (Harvard University, Cambridge, MA and the Max Planck Institute for Molecular Genetics, Berlin)

PLEASE VISIT US AT BOOTH #46 10 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy AWARDS TRAVEL GRANTS

P254: Valentina Vavassori SR-TIGET, Milan Optimization of a CRISPR/ ESGCT OUTSTANDING ACHIEVEMENT Cas9-based strategy for the P060: Thomas Hartjes correction of CD40LG gene in Dept. of Neurosurgery, Erasmus human haematopoietic stem MC, Rotterdam cells and T cells P052: Carlos Carrascoso CIEMAT/CIBERER-ISCIII, Madrid INV029: Didier Trono (2018 Outstanding Organotypic multicellular spheres P255: Manel Llado (OMS) as a 3D model system Towards the gene therapy of the Achievement Award lecture) TIGEM, Naples bone marrow failure in patients to study oncolytic adenovirus Homology-independent targeted EPFL, Lausanne responses in glioblastoma with dyskeratosis congenita Retroelements, their polydactyl controllers integration for gene correction in tumours P027: Yari Gimenez Martinez and the specificity of human biology photoreceptors P023: Katelyn Masiuk CIEMAT/CIBERER-ISCIII, Madrid Department of Microbiology, Preclinical studies towards the Immunology and Molecular gene therapy of Diamond-Black- Genetics, University of California, & fan anemia ESGCT Young Investigator Los Angeles, CA OR031: Alessio Cantore (2018 Young dmPGE2 and poloxamer-F108 Investigator Award lecture) enhance transduction of human OR003: Micaela Harrasser SR-TIGET, Milan hematopoietic stem and University College London Shielding lentiviral vectors from phagocytosis progenitor cells with a Effective targeting of ROR1+ solid tumours with next- increases hepatocyte gene transfer in ß-globin lentiviral vector generation Chimeric Antigen P109: Marine Charrier non-human primates P357: Sem Aronson Receptor therapy INRA/Oniris UMR 703, Nantes UMC University of Amsterdam Demonstration of AAV-mediated liver directed gene P483: Weiheng Su immunomodulatory properties University of Oxford therapy corrects the cholestatic for human MuStem cell Exploiting adenovirus mechanisms phenotype in Abcb4-/- mice population, a promising for the enhanced production of candidate for cell therapy P298: Alessia De Caneva AAV vectors ICGEB, Trieste of muscular dystrophies Coupling AAV-mediated P435: Sarah Le Saux promoterless gene targeting to Institut Charles Gerhardt SaCas9 nuclease to efficiently Montpellier, UMR 5253 correct liver metabolic diseases CNRS-UM-ENSCM P396: Gabriele Ordazzo OR007: Kleopatra Rapti Exploring the potential of San Raffaele Scientific Institute, Heidelberg University Hospital extracellular vesicles as drug Milan Generation of novel delivery systems Novel gene therapy approaches immune-evading AAVs through for whole brain delivery of the identification and mutation of lysosomal GCase enzyme for wide immunogenic epitopes in the protection from alpha-synuclein variable capsid regions of toxic aggregates adeno-associated Virus 9 OR019: Anais Amaya P328: Razieh Monjezi The University of Sydney, NSW University Hospital Würzburg Successful in vivo editing of CRISPR/Cas9 unites with Sleeping patient-derived primary human Beauty to generate CAR-T cells hepatocytes with enhanced therapeutic index for fighting against immunosuppressive tumour microenvironment

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20 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 21 CONGRESS LOCATIONS ON THE METRO

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THURSDAY 18 OCTOBER AT 8PM

OLYMPIC MUSEUM, Curling competition @ 9pm LAUSANNE Live music & disco Photobooth Food Photobooth fun! Open bar until 23:00 Paying bar 23:00 - 02:00

Coaches will leave from SwissTech at 19:30 to the Olympic Museum Easily accessible by public transport, don’t forget to use your free travel pass! Make sure you join us at our (in) famous Molecular Mingle to network and socialise with friends old and new; and try your hand at curling...

curling kəːlɪŋ/ noun: a game played on ice in which large round flat stones are slid across the surface towards a target. Members of a team use brooms to sweep the surface MAP FROM of the ice in the path of the stone to control its speed and direction. OUCHY METRO POLITE NOTICE: We WILL take pictures....and targets are NOT to be human!

26 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 27 MAKE YOUR WORDS BLOOM!

PROUDLY SUPPORTING THE TOWNSHIP PROJECT IN SOUTH AFRICA INSTEAD OF OUR USUAL ESGCT PENCIL, we have this year opted to bring you pencils ESGCT IS NOW WORKING with a items are hand made, largely by you can - once used - plant and nurture into selection of handpicked suppliers to women-run co-ops, which means that your very own ESGCT plant. With various ensure we are sourcing products which every purchase of a Township® product options to choose from, which one did you are environmentally friendly, support directly translates into work for township get? Swap and trade with your colleagues, fair trade and offer sustainability and women. Materials are ethically sourced or come and see us at the ESGCT booth, community inclusion. and Township® was the first South African fashion brand to become a member of where we also have some pencil sharpeners Here at Lausanne 2018, we are proud the World Fair Trade Organization. going if you need to sharpen up. to be distributing congress bags from the Township Project in South Africa. ‘The co-operatives are at the heart Township® was founded in 1997 with of everything we do at Township®. My a commitment to creating meaningful aim was to bring economic opportunity and sustainable economic opportunities to women in their own communities. for women in South Africa’s township Listening to them and trying to answer communities. their needs has shaped my life radically. Working together, we have been on a Job creation and women’s economic journey of positive change.’ empowerment form the foundation of Township® founder & CEO, the Township® ethical fashion brand. All Nicole-Marie Iresch

WHERE WE CAN, we work with suppliers who themselves work in a sustainable and community-centred way; our printed matter is made from green stock, our lanyards from bamboo. Where we can’t, we aim to reuse and / or recycle as much as possible, so to this end remember to hand your badge wallets back into the registration desk – or drop them into the clearly marked boxes around the venue - before you head home after the congress! ASSOCIATION MANAGEMENT CONFERENCES & EVENTS OUTREACH & EDUCATION

At Editas Medicine, we’re committed to harnessing the power and potential of genome editing to develop transformative medicines for people suffering from serious diseases.

When it comes to your society’s success, we’re straight to the point Visit us at Check out our website for our hidden talents: www.wats-on.co.uk www.editasmedicine.com 3 villages are 3 of the most typical villages in the Lavaux UNESCO region. Composed CHÂTEAU DE of several compact groups of winegrowers’ CHILLON BEATEN E houses, the villages have a characteristic but H T not typical silhouette. With narrow alleys T R and terraces overlooking the vines, the Further out, very near Montreux, it is worth F A lake and the Alps, the experience is visiting this Mediaval Castle. This Castle is F C unforgettable. There are many walks the result of several centuries of constant K O between those villages one from Grandvaux building, adaptations, renovations and to Cully for example, you can take a train restorations, this site has been occupied from Lausanne to Grandvaux and could start since the Bronze Age. The rocky island on with a lunch at the Auberge de la Gare. which the castle is built, was both a natural | www.aubergegrandvaux.ch/restaurant | protection and a strategic location to control More details on the walk can be found the passage between northern and southern on the Lausanne Tourism website: www. Europe. The history of the castle was lausanne.ch/en/thematiques/loisirs-et-de- influenced by three major periods. tente/pour-les-lutins/balades-et-visites/ | www.chillon.ch/en/GP765/history balades-thematiques/vignoble-de-lavaux/ de-grandvaux-a-cully.html CHARLIE CHAPLIN hill overlooking Lausanne. The superb view MUSEUM IN LAUSANNE over the lake and the cathedral had already LUTRY been immortalised by Camille Corot during his stay in 1825. The villa, with part of the Meet Charlie Chaplin, family man, in the ART BRUT park, was given to the City of Lausanne by Even closer to Lausanne, just on the shores Manoir in Montreux; embark on a singular the descendants in 1976. The mansion has of the Lake this small medieval town is of journey through the sets of his greatest MUSEUM been carefully restored to its original great historical importance. The well- films at our Hollywood-style Studio; take splendour. preserved heart of the town, with its narrow a peaceful walk in the gardens, get ready alleyways and numerous merchants’ and for an unforgettable experience! The concept of Art Brut stems from the A private foundation was born at the same time. Its twofold mission is: noblemen’s houses dating from the 15th to | www.chaplinsworld.com French painter Jean Dubuffet who, from 18th centuries, is under a preservation order. • to keep a superb 19th-century Lausanne 1945, assembled a collection of objects There is a marked circular walk with eleven residence alive and open to the public created by the inmates of various information boards about the history psychiatric hospitals and prisons—solitary • to develop a high-quality museum of of Lutry, during the walk you or outcast persons. In their creations, he saw fine arts can stop to taste the wines “an entirely pure, raw artistic operation that | www.fondation-hermitage.ch at the different vaulted the creator fully reinvents in all its phases, cellars. as spurred uniquely by his own impulses.” JUST OUTSIDE LAUSANNE | www.caveau-du- The idea of Art Brut is thus based on certain singe-vert.ch social characteristics and aesthetic peculiarities. | www.artbrut.ch GRANDVAUX, HERMITAGE CULLY & LES FOUNDATION EPESSES

In 1841, the banker Charles-Juste Bugnion With their narrow streets and bought the land called The Hermitage, on a balconies over Lake Geneva, those Château de Chillon Lake Geneva 32 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy Vineyards ESGCT / ISSCR / SFTCG Collaborative Congress 2018 33 TIME TO GET SOCIAL HASHTAG TO WIN!

. · . . .Seeking cures. . ,

Stay on top of everything PRIZES ACTIVITIES . . . through gene.. . . that’s happening at the Win Molecular Mingle tickets, Drop by the ESGCT booth congress by following us Giant Microbes or one of our to join in the social media on Facebook, Twitter and other top secret prizes for: competitions, the vox populi · Instagram! Make sure you • The best Congress photo poster prize for young use the official hashtag investigators, or meet our therapy· • The best Lausanne photo #ChangingModernMed student board members. • The Molecular Mingle to find and share the latest DID YOU KNOW? selfie with the most people updates, get in touch or win a That you can contact other . prize in our competitions. . . . • The most retweeted delegates through our app (see Congress tweet Check out the ESGCT and page inside front cover) and get ISSCR booths as well, here you • The most liked Facebook tweeting, FBing and ‘gramming .Inspired by transformative innovation, post straight from the app? ' can find out about upcoming built on a sustainable and integrated events, join in competitions, Only posts tagged THE SMALL PRINT multi-platform approach play games, get more involved #ChangingModernMed Please be courteous and in YOUR society or just say will be considered and keep considerate in your use of hi! At the ESGCT booth you your privacy settings in social media and always make will also be able to buy mind - if we can’t see your sure that you don’t share Best -in-class curative gene and cell tickets for the Molecular posts, we can’t include them unpublished content without Mingle. in the competition! the author’s consent. therapiesf or patients with devastating diseases

Rocket Pharmaceuticals, Inc • The Empire State Building 350 Fifth Avenue, NY, NY 10118 • 646-440-9100 ESGCT: www.facebook.com/ESGCT/ ESGCT @ESGCT www.rocketpharma.com ISSCR: www.facebook.com/ISSCR/ ISSCR @ISSCR SFTCG: www.facebook.com/SFTCG/ SFTCG @SFTCG

ESGCT / ISSCR / SFTCG Collaborative Congress 2018 35 WWW.ESGCT.EU

ESGCT AWARDS BLOG STUDENT BOARD The ESGCT blog is written by MEMBERS members and guest bloggers and Our student board members Joost ESGCT outstanding achievement covers all themes related to gene van Haasteren and Larisa Condurat and cell therapy; from fundamental represent early career researchers at science to patient and public ESGCT travel grants for best scoring abstract ESGCT board level. If you have any engagement. Read it on our comments and ideas, please send website and get in touch if you them their way. You can email them: would like to contribute! ESGCT young investigator [email protected] and [email protected] (sponsored at national society meetings) or better yet, come see them at the ESGCT booth!

ESGCT young investigator FORUM Newly launched, the ESGCT Forum is the place to keep in touch before, Vox populi: best poster during and after congresses and spring schools, look and post opportunities for jobs and conferences NEW AWARDS but more importantly talk about gene and cell therapy. Discuss new papers, ask questions about techniques, Just launched – apply soon! and post your protocols! Please check the ESGCT website for full information and how to apply.

ESGCT mentor award A mentor is often the most overlooked, yet most valuable person in THE INTERACTIVE GENE & CELL THERAPY COMMUNITY HEATMAP ensuring a PhD student’s progression and survival! This is the chance for individuals who have benefited from effective mentoring to give back. €500 cash award, and complimentary registration to annual congress. Applications open in April. The interactive gene & cell therapy community heatmap on our website ESGCT public engagement award shows where in Europe a Finding ways to share your research and to get people from outside growing number of academic the scientific community involved is an increasingly important part of institutions, biotech and the research process. ESGCT wants to help promote excellent work. pharmaceutical companies, €500 cash award, and complimentary registration to annual congress. non-profits and other organisations Applications open now! are working to bring new treatments to patients. Learn who to ask for advice or find ESGCT travel grants have increased from your next collaboration in ten €300 grants to twenty €500 grants a few clicks! CAMPUS FLOOR EXHIBITORS 1-26 1 14

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42 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 43 www.bsgct.org

The British Society for Gene and Cell Therapy (BSGCT) & is dedicated to facilitating Upcoming education, communication events and the sharing of expertise BSGCT Autumn Conference 2018 and knowledge 23 November 2018, London Join us for this one-day conference as we explore genome editing Promoting Excellence | Education and Training for gene and cell therapy: technologies, models and translation Nurturing Early Career Researchers Public Engagement | Collaboration and Networking BSGCT Early Career Development and Collaboration Day & Joint ISCT Evening Networking Reception co-hosted by the NIHR GOSH BRC Junior Faculty 22 November 2018, London A daytime event for Early Career Researchers will be rounded off with a networking reception open to all. This is a FREE event, but places are limited!

BSGCT Annual Conference 2019 19 June - 21 June 2019, Sheffield Highlighting the latest research and techniques in gene and cell therapy, including presentations from Stephan Grupp, Kathrin Meyer and Michele de Luca

Join us! Become a BSGCT @_BSGCT member to receive reduced rates for conferences and events, and Images courtesy of Simon Callaghan Photography much more. View our membership benefits at: www.bsgct.org /BSGCT ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PARTNERS PARTNERS

DIAMOND PLATINUM

BioReliance | Merck Adverum is a clinical-stage gene therapy company targeting unmet medical needs As part of Merck KGaA, Darmstadt, Germany, BioReliance® Services is a key in serious rare and ocular diseases. Adverum has a robust pipeline that includes component of the life science services portfolio and the leading provider of product candidates designed to treat rare diseases alpha-1 antitrypsin (A1AT) contract services in the biopharmaceutical industry. BioReliance’s comprehensive deficiency and hereditary angioedema (HAE), as well as wet age-related macular and integrated services support every phase of the testing, development and degeneration (wAMD). By leveraging our core capabilities, which include manufacturing process. With locations worldwide, BioReliance offers more than clinical development and in-house manufacturing expertise, we are moving 1,000 tests and complementary services related to biologics safety testing and product candidates into the clinic that are designed to provide durable efficacy specialized toxicology. Our clients include the world’s top producers of traditional with the potential to greatly improve the quality of life for patients. Adverum has collaboration agreements with Regeneron Pharmaceuticals to research, develop, pharmaceuticals, medical devices and chemicals, as well as biopharmaceuticals and commercialize gene therapy products for ophthalmic diseases, and Editas www.bioreliance.com Medicine to explore the delivery of genome editing medicines for the treatment Oxford BioMedica (LSE:OXB) is a leading gene and cell therapy group focused on of inherited retinal diseases. www.adverumbio.com developing life changing treatments for serious diseases. Oxford BioMedica and bluebird bio is a clinical-stage company committed to developing potentially its subsidiaries (the “Group”) have built a sector leading lentiviral vector delivery transformative gene therapies for severe genetic diseases and T cell-based platform (LentiVector®), which the Group leverages to develop in vivo and ex immunotherapies. The company’s objective is to develop and bring to market the vivo products both in-house and with partners. The Group has created a valuable most advanced products based on the transformative potential of gene therapy proprietary portfolio of gene and cell therapy product candidates in the areas of to provide patients hope for a better life in the face of limited or no long-term oncology, ophthalmology and CNS disorders. The Group has also entered into a safe and effective treatment options. Headquartered in Cambridge, Massachusetts, number of partnerships, including with Novartis, Bioverativ, Sanofi, Axovant, bluebird bio has operations in the U.S. and Europe. www.bluebirdbio.com Orchard Therapeutics, Boehringer Ingelheim/UK Cystic Fibrosis Gene Therapy Consortium/Imperial Innovations, GC LabCell and Immune Design, through which The Human Induced Pluripotent Stem Cells Initiative (HipSci) is generating a large, it has long-term economic interests in other potential gene and cell therapy high-quality reference panel of human iPSC lines for the research community. products. Oxford BioMedica has world class facilities and capabilities to support HipSci stands apart from other iPSC projects in a number of respects: pre-clinical, research and bioprocessing development through to GMP production • Magnitude – HipSci is systematically generating iPSCs from hundreds of donors and supply of commercial and clinical trial materials. The production activities are using a standardised experimental pipeline. focused on the manufacture of lentiviral vectors from human cell lines in large • Cell line availability – HipSci’s cell lines are produced as a global iPSC resource. scale serum free suspension cultures. Oxford BioMedica is based across several The cell lines are readily available from two cell banks (ECACC and EBiSC), for locations in Oxfordshire, UK and employs more than 320 people. Further use by the wider research community. information is available at www.oxfordbiomedica.co.uk • Cohorts – HipSci’s reference panel comprises hundreds of cell lines from phenotypically healthy donors, plus several cohorts of donors with inherited genetic diseases. • The data – Each line generated is extensively characterised. Assays include genotyping by array, expression array, methylation array, RNA-seq, Exome-seq, proteomics mass spect www.hipsci.org

46 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 47 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PARTNERS PARTNERS

PLATINUM PLATINUM

Intellia Therapeutics is a leading gene editing company, focused on the REGENXBIO is a leading clinical-stage biotechnology company seeking to development of proprietary, potentially curative therapeutics using the CRISPR/ improve lives through the curative potential of gene therapy. REGENXBIO’s Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, transform medicine by permanently editing disease-associated genes in the human including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV body with a single treatment course. Our combination of deep scientific expertise Technology Platform Licensees are applying the NAV Technology Platform in the and clinical development experience, along with our leading intellectual property development of a broad pipeline of candidates in multiple therapeutic areas. portfolio, puts us in a unique position to unlock broad therapeutic applications of REGENXBIO is currently developing five product candidates, including RGX-314 for the CRISPR/Cas9 technology and create a new class of therapeutic products. the treatment of wet age-related macular degeneration (wet AMD), RGX-501 for Intellia was named as one of the top 10 biotech start-ups by Nature the treatment of homozygous familial hypercholesterolemia (HoFH), RGX-111 Biotechnology. In September 2015, Intellia was named a “Fierce 15” biotech for the treatment of mucopolysaccharidosis type I (MPS I), RGX-121 for the company by FierceBiotech. www.intelliatx.com treatment of mucopolysaccharidosis type II (MPS II) and RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis Type 2 (CLN2 Batten disease). Lonza offers world-class technology platforms in the areas of GMP cell culture and www.regenxbio.com viral-based therapeutic manufacturing, custom bio-therapeutic culture media, a large selection of primary and stem cells and a full line of custom bioassays. Our Rocket Pharma is a clinical-stage gene therapy company with a multi-platform extensive experience in cell therapy process optimisation and scale-up innovation approach for the treatment of patients with devastating diseases. We leverage helps clients to safely and effectively advance their products through all phases LV and AAV transduction strategies towards first-in-class programs. Hallmarks of of the commercial pipeline and maximise their return on investment. Our new Rocket’s vision include: 1) a high threshold for the selection of quality programs, viral-based therapeutics group provides viral vaccine manufacturing as well as 2) leverage of deep industry know-how through a world-class team and viral vector mediated gene therapies. Our staff can design, develop and implement manufacturing partnerships and 3) a laser focus on optimizing and innovating a manufacturing process that meets your autologous or allogeneic therapeutic gene therapy product parameters through a seasoned scientific approach that applications. de-risks programs as they enter clinical trials. www.rocketpharma.com www.pharma.lonza.com Sarepta Therapeutics is a commercial-stage biopharmaceutical company MeiraGTx is a vertically integrated, clinical stage gene therapy company with focused on the discovery and development of precision genetic medicines to four ongoing clinical programs and a broad pipeline of preclinical and research treat rare neuromuscular diseases. The Company is primarily focused on rapidly programs. MeiraGTx has core capabilities in viral vector design and optimization advancing the development of its potentially disease-modifying Duchenne and gene therapy manufacturing, as well as a potentially transformative gene muscular dystrophy (DMD) drug candidates and is proud to support the 2018 regulation technology. Led by an experienced management team, MeiraGTx has ESGCT Annual Meeting. For more information about Sarepta. www.sarepta.com taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTx’s initial At Spark Therapeutics, a fully integrated, commercial company committed to focus is on three distinct areas of unmet medical need: inherited retinal diseases, discovering, developing and delivering gene therapies, we challenge the inevita- severe forms of xerostomia and neurodegenerative diseases. Though initially bility of genetic diseases, including blindness, hemophilia and neurodegenerative focusing on the eye, salivary gland and central nervous system, MeiraGTx intends diseases. We have successfully applied our technology in the first FDA-approved to expand its focus in the future to develop additional gene therapy treatments gene therapy in the U.S. for a genetic disease, and currently have three programs for patients suffering from a range of serious diseases. in clinical trials, including product candidates that have shown promising early www.meiragtx.com results in patients with hemophilia. At Spark, we see the path to a world where no life is limited by genetic disease. For more information, visit www.sparktx.com, and follow us on Twitter and LinkedIn. 48 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 49 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PARTNERS PARTNERS

GOLD GOLD

Audentes Therapeutics is a clinical stage biotechnology company focused on Available Gene therapy relevant development and manufacturing capabilities developing and commercializing gene therapy products for patients living with and capacities can hardly support the strong growth of gene therapy product serious, life-threatening rare diseases. Audentes is currently developing four development activities across the globe. Long process development times and product candidates, including AT132 for the treatment of X-Linked Myotubular low yield manufacturing processes slow down product development and add Myopathy (XLMTM), AT342 for the treatment of Crigler-Najjar Syndrome, AT982 significant costs and risks to the development and licensure of gene therapy for the treatment of Pompe disease, and AT307 for the treatment of the CASQ2 products. subtype of Catecholaminergic Polymorphic Ventricular Tachycardia (CASQ2-CPVT). The Gene Therapy Center Austria (GTCA) covers all steps from vector to GMP We are a focused, experienced and passionate team committed to forging strong, production (200L, 500L, and 1000L) until early stage commercial including a broad global relationships with the patient, research and medical communities. range of AAV specific analytical methods. www.audentestx.com GTCA’s one-stop-shop concept is based on Shire’s proprietary AAV technology platform. The GTCA has successfully demonstrated lot-to-lot consistency and Brammer Bio is a best-in-class viral vector contract development and scalability across multiple programs. manufacturing organization (CDMO) for companies developing gene-modified cell Our partners benefit from our 30+ year experience in development, approval and therapies and in vivo gene therapies. Brammer offers end-to-end CDMO services global supply of complex biologics which reduces product development risks for from Phase I/II in Florida through commercial manufacturing in Massachusetts. our development partners and for our CMO clients. Brammer Bio provides clinical and commercial supply of viral vectors for advanced www.shire.at therapies, process and analytical development, and regulatory support, enabling large pharma and biotech clients to accelerate the delivery of novel medicines to Genethon, located in Evry, France, is a non-profit R&D organization dedicated to improve patient health. The Brammer team consists of extraordinary employees the design and development of gene therapy treatments for rare genetic diseases focused exclusively on serving the gene therapy needs of clients and their patients. from research to clinical validation. It was created by the AFM-Telethon (French We are Helping to Cure.® Muscular Dystrophy Association) which is its main funding source. www.brammerbio.com Genethon has multiple ongoing programs at clinical, preclinical and research stage for neuromuscular, blood, immune system, and liver diseases. Editas Medicine is a leading genome editing company focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 genome editing Several of these programs are pursued by Genethon as sponsor, others have been systems into a robust pipeline of treatments for people living with serious diseases licensed to leading biotech and pharma players in the gene therapy arena such as around the world. Editas Medicine aims to discover, develop, manufacture, and Audentes, Avexis/Novartis, Gensight Biologics, Orchard Therapeutics, Spark. commercialize transformative, durable, precision genomic medicines for a broad Genethon was awarded the “Prix Galien France” in 2012.. class of diseases. For the latest information and scientific presentations, please visit www.genethon.fr www.editasmedicine.com

50 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 51 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PARTNERS PARTNERS

GOLD GOLD

Human Gene Therapy is the premier peer-reviewed journal in the field and MolMed S.p.A. is a medical biotechnology company focused on research, includes HGT Methods and HGT Clinical Development, providing 22 issues of development and clinical validation of novel anticancer therapies. MolMed’s comprehensive, end-to-end coverage that is driving today’s explosion of gene pipeline includes antitumour therapeutics in clinical and preclinical development: therapy advances. Human Gene Therapy serves as the Official Journal of ESGCT. Zalmoxis® (TK), a cell-based therapy; NGR-hTNF, a novel therapeutic agent for Come visit us at booth 31 to pick up your copy of the special issue dedicated to solid tumours; CAR-CD44v6, an immuno-gene therapy project; MolMed also stem cell gene therapy, and meet Graham Parker, PhD, the Editor-in-Chief of conducts cell and gene therapy projects in collaboration with third parties, Stem Cells and Development, a premier source of clinical, basic, and translational offering resources and expertise covering preclinical to Phase III trials activities. research on stem cells of all tissue types and their potential therapeutic MolMed is listed on the main market (MTA) of the Milan stock exchange managed applications. Stem Cells and Development is an Official Journal of the British by Borsa Italiana. Society for Gene and Cell Therapy. www.molmed.com Mary Ann Liebert, Inc. publishers is pleased to announce the 2018 launch of The CRISPR Journal, stop by booth 31 or visit www.liebertpub.com/CRISPR Synthego is a leading provider of genome engineering solutions. The company’s for information. product portfolio includes software and synthetic RNA kits designed for CRISPR www.liebertpub.com/overview/human-gene-therapy genome editing and research. With next-generation informatics and machine learning, Synthego’s vision is to bring precision and automation to genome KCT is newly formed research center in Kuopio, Finland. We provide high quality engineering, enabling rapid and cost-effective research with consistent results basic and translational research. Professional team and laboratories with cutting for every scientist. edge devices are providing scientific know-how and modern technologies to www.synthego.com develop advanced therapies. We undertake science in collaboration with academic groups all over the world. Link to FinVector’s GMP manufacturing, quality and Voyager Therapeutics is a clinical-stage gene therapy company focused on regulatory teams ensures product path from research to patients. Currently KCT is developing life-changing treatments for severe neurological diseases. Voyager is searching for new projects on gene- and cell therapy. Come to visit our stand! committed to advancing the field of AAV gene therapy through innovation and www.kct.fi investment in vector engineering and optimization, manufacturing and dosing and delivery techniques. Voyager’s pipeline focuses on severe neurological diseases in MaxCyte is a global cell-based medicines and life sciences company applying its need of effective new therapies, including Parkinson’s disease, a monogenic form patented cell engineering technology to help patients with high unmet medical of ALS called SOD1, Huntington’s disease, Friedreich’s ataxia, neurodegenerative needs in a broad range of conditions. The company leverages its Flow Electropora- diseases related to defective or excess aggregation of tau protein in the brain tion® Technology to enable its partners across the biopharmaceutical industry to including Alzheimer’s disease and severe, chronic pain. Voyager has broad strategic advance the development of innovative medicines, particularly in cell therapy, in- collaborations with Sanofi Genzyme, the specialty care global business unit of cluding gene editing and immuno-oncology. MaxCyte has placed its cutting-edge Sanofi, AbbVie, and the University of Massachusetts Medical School. Founded by flow electroporation instruments worldwide, including with nine of the top 10 scientific and clinical leaders in the fields of AAV gene therapy, expressed RNA global biopharmaceutical companies, and has more than 55 partnered program interference and neuroscience, Voyager Therapeutics is headquartered in licenses in cell therapy including more than 25 licensed for clinical use. With its Cambridge, Massachusetts. robust delivery technology, MaxCyte helps its partners to unlock the full potential www.voyagertherapeutics.com of their products. For more information, www.maxcyte.com

52 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 53 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PARTNERS PARTNERS

SILVER SILVER

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company Delphi Genetics, located in Belgium, is a biotechnology company specialized in developing gene therapies for life-threatening rare genetic diseases. Abeona’s genetic engineering with a deep expertise in DNA and its applications. Certified lead programs include ABO-102 (AAV-SGSH), an adeno-associated virus GMP we provide technological and flexible biomanufacturing solutions. (AAV)-based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB- 101 Delphi Genetics is involved in several DNA vaccines and gene therapy programs, (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). including the development of CAR-T and collaborates on projects at all stages Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B of development, from R & D to clinical phases. Our complete, GMP-compliant, (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), flexible and single-use process for plasmid DNA manufacturing allows us to pro- ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), EB-201 vide R&D, High Quality and GMP grade plasmid DNA from a few mg up to several for epidermolysis bullosa (EB), ABO-301 (AAV- FANCC) for Fanconi anemia (FA) grams in our dedicated facility. disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona is developing its To support this manufacturing activity, Delphi Genetics also offers a proprietary proprietary vector platform, AIM™, for next generation product candidates. “antibiotic-free” technology, STABY®. The STABY® technology is implementable in www.abeonatherapeutics.com all plasmid DNA and provide a better plasmid stabilization, a production yield increase and a safer way to manufacture plasmid DNA without antibiotic resistance AFM (French Muscular Dystrophy Association) has a single objective: to defeat gene following regulatory agencies recommendations. neuromuscular diseases, which are devastating muscle-wasting diseases. Created in www.delphigenetics.com 1958 by a group of patients and their families, and recognised as being of public utility in 1976, it has set itself two missions: curing neuromuscular diseases and A pioneer in scholarly open access publishing, MDPI has supported academic reducing the disabilities they cause. communities since 1996. Based in Basel, Switzerland, MDPI has the mission to www.afm-france.org foster open scientific exchange in all forms, across all disciplines. Our 212 diverse, peer-reviewed, open access journals are supported by over 35,500 academic With six products on the market and a fully-integrated multinational organization editors. We serve scholars from around the world to ensure the latest research is in place, BioMarin is providing innovative therapeutics to patients with serious freely available and all content is distributed under a Creative Commons Attribu- unmet medical needs. The company is also currently conducting a clinical trial of tion License (CC BY). an AAV-based potential gene therapy for hemophilia A. www.mdpi.com www.biomarin.com

BioNTech Innovative Manufacturing Services – Experts in Cell and Gene Nature Technology Corporation (NTC) partners with biopharmaceutical and drug Therapy Manufacturing development companies to produce safe and effective non-viral vectors incorpo- BioNTech Innovative Manufacturing Services (IMFS) is a German Contract rating the best-in-class minimal Nanoplasmid TM vector backbone. Nanoplasmid Development and Manufacturing Organisation specialized in the industrialization TM vectors comprise a regulatory compliant antibiotic free selection marker (RNA- of cell and gene therapy products (viral vectors, cells and ivt mRNA). Based OUT) combined with a high-manufacturing-yield, host-restricted mini-origin of on extensive expertise in scientific, technical and regulatory prerequisites, we replication to deliver superior expression level and duration,and reduced transfec- develop and manufacture your products in a safe and cost-efficient way in our tion associated toxicity. Nanoplasmid TM vectors are ideal for gene and stem cell state-of-the-art GMP facility. We offer a complete service spectrum from process therapies, and as helper and packaging vectors for viral vector manufacture. NTC development through clinical trial to in-market supply. All services are fully provides outsourcing for custom vector design and synthesis, and plasmid manu- integrated and supervised by our QA department ensuring efficient and compliant facturing, using its leading HyperGRO TM process, resulting in rapid development manufacturing in our state-of-the-art GMP facility. of gene and stem cell therapeutics and DNA vaccines. www.biontech-imfs.de www.natx.com

54 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 55 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PARTNERS PARTNERS

SILVER SILVER

Novasep is a CDMO specialized in viruses & viral vectors production for tox, clinical Sangamo Therapeutics, Inc. is focused on translating ground-breaking science and commercial phases. Novasep offers global and integrated services from process into genomic therapies that transform patients’ lives using the company’s development to commercial manufacturing. For more than 15 years, we have industry leading platform technologies in genome editing, gene therapy, gene acquired experience in manufacturing viral vectors (lentivirus, AAV, ADV…) regulation and cell therapy. Sangamo is conducting Phase 1/2 clinical trials in for gene therapy and vaccination. Our investment in new commercial assets, Hemophilia A and Hemophilia B, lysosomal storage disorders MPS I and MPS II, addressing both drug substance and drug product manufacturing processes, can and beta thalassemia. Sangamo has an exclusive, global collaboration and license help to contribute to your project’s success. Come and meet us at booth 33. agreement with Pfizer Inc. for gene therapy programs for Hemophilia A and www.novasep.com gene regulation programs for C9ORF72-linked amyotrophic lateral sclerosis and frontotemporal lobar degeneration; with Kite, a Gilead Company, for next- Orchard Therapeutics is a fully integrated commercial-stage biotechnology generation autologous and allogeneic engineered cell therapies for the treatment company dedicated to transforming the lives of patients with rare diseases of cancer; with Bioverativ, a Sanofi Company, for hemoglobinopathies, including through innovative gene therapies. beta thalassemia and sickle cell disease; and with Shire International GmbH to Orchard’s portfolio of autologous ex vivo gene therapies includes Strimvelis, develop therapeutics for Huntington’s disease. www.sangamo.com the first autologous ex vivo gene therapy approved by the EMA for adenosine deaminase severe combined immunodeficiency (ADA-SCID). Additional programs Selecta Biosciences, Inc. is a clinical-stage biopharmaceutical company that is for primary immune deficiencies, inherited metabolic disorders and blood focused on unlocking the full potential of biologic therapies by mitigating disorders include three advanced registrational studies for ADA-SCID, unwanted immune responses. Selecta plans to combine its tolerogenic Synthetic metachromatic leukodystrophy (MLD) and Wiskott-Aldrich syndrome (WAS), Vaccine Particles (SVP™) to a range of biologics for rare and serious diseases that clinical programs for X-linked chronic granulomatous disease (X-CGD) and require new treatment options. The company’s current proprietary pipeline includes beta-thalassemia, as well as an extensive preclinical pipeline. SVP-enabled enzyme, oncology and gene therapies. SEL-212, the company’s lead Orchard currently has offices in the U.K. and the U.S., including London, candidate in Phase 2, is being developed to treat severe gout patients and resolve San Francisco and Boston. their debilitating symptoms, including flares and gouty arthritis. A Phase 1 trial is www.orchard-tx.com ongoing for a combination therapy consisting of SVP-Rapamycin and LMB-100 (Selecta’s SEL-403 product candidate) for the treatment of patients Paragon Bioservices is an award-winning Contract Development & with malignant pleural or peritoneal mesothelioma. Selecta’s proprietary gene Manufacturing Organization (CDMO). Our aim is to build strong client partnerships therapy product candidates are being developed for rare inborn errors of focusing on transformative technologies, including oncology, immunotherapies metabolism and have the potential to enable repeat administration. The use of (CAR-T cell therapies and oncolytic viruses), new generation vaccines (VLPs) and SVP also holds potential in the development of vaccines and treatments for gene therapies (AAV). Paragon’s cGMP facilities include microbial and mammalian allergies and autoimmune diseases. www.selectabio.com suites, aseptic fil-finish and full-segregated virus facilities. Responsibility to our clients, a passion for science and our collective need to uniQure is delivering on the promise of gene therapy, single treatments with contribute to better public heath—is what keeps us motivated and excited potentially curative results. We have developed a modular technology platform about the work that we perform. We are driven by our commitment to provide to rapidly bring new disease-modifying therapies to patients with CNS, liver/ exceptional quality scientific performance and customer service. metabolic and cardiovascular diseases. We are advancing a focused pipeline With over 25 years in the business, Paragon’s scientists, engineers, quality systems of innovative gene therapies and have entered late-stage clinical development personnel and project managers have many years of experience working with in our lead indication, hemophilia B, and established preclinical biologics—from research and process development services to GMP manufacturing proof-of-concept in Huntington’s disease. www.uniqure.com for clinical trials and eventual commercial launch. www.paragonbioservices.com/therapeutic-areas/gene-therapy/

56 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 57 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PARTNERS PARTNERS

SILVER BRONZE

Yposkesi is the largest European CDMO for gene therapy vector manufacturing. Cell Press is a leading publisher of biomedical and physical science research, Created in November 2016 in Corbeil Essonnes (France) as a spin off from the offering high-quality, cutting-edge research and resources. We drive science world-class gene therapy pioneer Genethon, Yposkesi provides a full-service offer forward and promote cross-pollination of ideas with our passion for excellence covering BioProcess development (USP & DSP), from small/pilot to large and commitment to innovation. Our aim is to engage the scientific community production scale,analytical development, GMP manufacturing of clinical by communicating important, exciting discoveries made today that will impact lots of lentiviral vectors and regulatory support. the future of research. We are also proud to publish Stem Cell Reports, the official Its current facility consists in a 50,000 sq ft building, operating 4 manufacturing journal of the ISSCR, and the Molecular Therapy family of journals, the portfolio suites for bulk drug substance, and 2 Fill & Finish suites. This capacity will be of gene and cell therapy research journals for the ASGCT. expanded in 2021 with a large scale facility (50,000 sq ft) equipped with 2,000L www.cell.com bioreactors, designed for commercial production and compliant to EMA and FDA. Capitalizing on more than 25 years of expertise, Yposkesi significantly invests in Generation Bio aims to do what no other gene therapy platform has yet innovation in bioprocessing to cost-effectively deliver on high quality projects. accomplished: to deliver titratable, re-dosable genetic medicines with drug-like www.yposkesi.com properties to patients with a wide range of genetic diseases. BRONZE www.generationbio.com

The Ontario Institute for Regenerative Medicine (OIRM) is a non-profit Anemocyte is a Biotech Manufacturing Organization (BMO): a biotech company that addresses CGT needs pro-actively offering one stop shop solutions and stem cell institute funded by the Ontario government and dedicated to fostering exciting innovations active in the field of Cell and Gene Therapies (CGTs). transforming discoveries into clinical trials and cures. Through our commitment Our experience and know-how come from far: to collaboration and partnerships, we leverage our resources to fund and support • 14 years of GMP manufacturing of CGTs and biological drugs; promising advances. OIRM is a passionate champion for investigators and their • 61 years of Contract Manufacturing within our Group (Holding F.I.S.) patients as we build a healthier future for Ontario, Canada, and the world. Our business includes: www.oirm.ca • Process development and GMP Manufacturing (Somatic Cells, Non-Viral Modi- fied Cells and Extracellular vesicles) TiGenix is an advanced biopharmaceutical company developing novel • Plasmid for Viral Vector Manufacturing therapies for serious medical conditions by exploiting the anti-inflammatory • Project 2020: up to commercial manufacturing capabilities (Including use of properties of allogeneic, or donor-derived, stem cells. TiGenix´ lead product, Viral Vectors for CGTs) Alofisel (darvadstrocel), previously Cx601, received European Commission (EC) www.anemocyte.com approval for the treatment of complex perianal fistulas in adult patients with Axon Medchem is a trusted supplier of high-value life science products, providing non-active/mildly active luminal Crohn’s disease, when fistulas have shown an Axon Ligands™ as world wide recognized drug reference standards for pharmaco- inadequate response to at least one conventional or biologic therapy. A global logical research. With more than 2000 excellent quality small molecule inhibitors Phase III trial intended to support a future U.S. Biologic License Application and modulators targeting more than 850 biological targets, we aim to facilitate (BLA) started in 2017. TiGenix has recently been acquired by Takeda, a global your scientific research and development. pharmaceutical company active in gastroenterology. Axon Medchem is also a leading European CRO in medicinal chemistry, specialized www.tigenix.com in contract research and high-quality synthesis of bio-active and/or drug-like molecules. We have the proven record in developing novel drug candidates and achieving excellence for a decade by providing our dedicated chemistry services for companies and research institutes around the world. www.axonmedchem.com

58 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 59 Book from 1 December 2018 to secure one of 150 seats. ESGCT SPRING SCHOOL 2019

in collaboration with 2019 The Oﬃ cial Journal of

the next milestone in cell and gene

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We want to be your partner Our scientists and engineers bring Visit pharma.lonza.com Special Issue on Gene Therapy and Stem Cells and add value to your therapy decade-long development experience North America development process. We invest across a broad spectrum of cell types and +1 301 898 7025 Guest Co-Editors: in enabling technologies and build technologies. This builds the backbone Europe & rest of world Thierry VandenDriessche and Graham Parker expertise to support the development of an extensive service offering, providing +41 61 316 81 11 and commercialization of new you with tailored process and analytical Email [email protected] innovative therapies. development, manufacturing and © 2018 Lonza. All rights reserved. regulatory services.

CellTherapy_Ad_Lausanne_A5.indd 1 9/14/18 11:38 AM ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 EXHIBITORS EXHIBITORS

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Booth 52: Ajinomoto Co., INC. is one of the largest amino acid companies in Japan Booth 13: The American Society of Gene & Cell Therapy (ASGCT) is the primary and is now focusing on cell therapy and stem cell research. We provide stem cell membership organization for scientists, physicians, professionals, and patient media and growth factors of clinical grade. advocates involved in gene and cell therapy. Our mission is to advance knowledge, www.ajinomoto.com awareness, and education leading to discovery and clinical application of gene and cell therapies to alleviate disease—we do that by serving as a catalyst to transform Booth 40: Aldevron serves the biotechnology industry with custom production of medicine by advancing gene and cell therapies to benefit patients and society. nucleic acids, proteins and antibodies. Featuring the largest and most modern GMP ASGCT’s programs include an annual scientific meeting in the United States each plasmid DNA manufacturing facility in the world, Aldevron provides thousands of spring welcoming more than 3,000 people as well as publication of the Molecular clients with plasmids, RNA, gene editing enzymes and more for biological research Therapy family of four journals. www.cell.com/molecular-therapy-family/home projects from discovery to clinical trials and commercial applications. These products are critical raw materials and key components in commercially Booth 5: ArcticZymes has a rich history leveraging its access to the marine available drugs and medical devices, helping accelerate development of treatments Arctic; identify novel cold-adapted enzymes for use in molecular research, for diseases such as cancer, infectious disease, pediatric disorders and rare diseases. diagnostics, and bioprocessing. ArcticZymes’ products are firmly entrenched in the molecular research and diagnostic arena; as both standalone enzymes and unique Our collaborative approach and commitment to providing quality materials allow components in the kits of our commercial partners. We have developed strong us to meet precise client requirements and provide innovative solutions to advance relationships with OEM partners and individual researchers alike with the science. Aldevron headquarters is in Fargo, North Dakota USA, and has facilities in strategic intent to expand our enzyme portfolio and the application of each Madison, Wisconsin and Freiburg, Germany. unique product. ArcticZymes has recently launched a novel Salt Active Nuclease www.aldevron.com that is successfully being used by producers of viral vaccines in gene therapy. This nonspecific, recombinant endonuclease has optimum activity at high salt Booth 38: ALS CellCelectorTM is the only system which enables automated concentrations, which can improve efficiency and yield in various workflows. isolation of single cells, clusters, adherent cell colonies or colonies grown in 3D www.arcticzymes.com semi-solid media. It’s an ideal system for (i) automated clonal picking of newly derived iPSC colonies, (ii) single cell or colony isolation for genome editing, and Booth 48: Booth 48: ASEPTIC TECHNOLOGIES provides the technology for cGMP (iii) automated picking of hematopoietic stem cell colonies. Isolated colonies or aseptic fill and finish for ATMP (AT-Closed Vial® Technology), combining: single cells can be deposited into a variety of destination plates for downstream culturing or molecular characterisation (qPCR, sequencing...). CellCelector • The Closed Vial, ensuring 100% of Container Closure Integrity during combines bright field, phase contract or fluorescence imaging, sensitive cryostorage and ultimate sterility assurance. Supplied ready-to-fill and cell/colony detection technology and patented robotics picking tools. The remaining closed through all process. system can be also used for stem cell culture monitoring and be integrated • The Filling Equipment, robust and scalable solution, performing only 3 into a fully automated stem cell production facility. operations: filling by piercing, sealing and capping. Available in manual and www.als-jena.com automated modifications. Our technology is widely used for cGMP filling of ATMPs for years, since it enables maximum safety during filling, cryostorage, shipping and administration; allows minimizing the Cost of Goods and an easy scaling-up. AT-Closed Vial® is compatible with liquid nitrogen-free controlled rate freezers by GE Asymptote. Automated thawing device is available for standardization of the administration. www.aseptictech.com 64 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 65 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 EXHIBITORS EXHIBITORS

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Booth 39: Asphalion is an international Scientific and Regulatory Affairs Booth 55: AMSBIO, a global provider of advanced life science technology. Experts consultancy, with offices in Barcelona, Madrid and Munich. We collaborate with in making oncology assays more physiologically relevant, our products and services Pharma and Biotech companies facilitating Drug Development and Regulatory include antibodies, peptides and recombinant proteins, tools for spheroid and Affairs projects for Drugs, Biologics, Biosimilars, ATMPs and Medical Devices. organoid culture as well as systems for studying cell motility, migration, Our involvement ranges from early development, through to registration and invasion and proliferation. New cancer drug screening and profiling service post-commercialization phases. Since the company was founded in 2000, we have also now available. consistently grown and now have a team of over 80 employees with backgrounds www.amsbio.com in all areas of life sciences. Our consultants are experts in their fields and are in direct contact with European agencies for the implementation of new Booth 62: BioLamina is a Swedish biotechnology company with a global regulatory standards. We provide global services and work for hundreds of presence. We offer an expansive portfolio of defined human recombinant laminin clients from around the world. Through collaborations with partners in all other proteins, Biolaminins, for a variety of applications, including reliable expansion of continents, we can accelerate your worldwide scientific and regulatory activities human pluripotent stem cells and differentiation and maintenance of different by using local expertise. specialised cell types, such as hepatocytes, cardiomyocytes and neural cells. By Services: providing the cells with their biologically relevant matrix proteins, the natural, • Regulatory and Scientific Strategy during Development cell-specific cell-matrix interaction can be mimicked, leading to improved cell • Medical and Scientific Writing functionality, robust culture systems and safe cells for therapy. BioLamina’s laminin • Global Submissions technology has been scientifically validated in many high impact journals. • eSubmission and RIM www.biolamina.com • Life-Cycle Outsourcing • Pharmacovigilance • Promoting the marketing registration of Medical Devices: Classification strategy, Booth 6: Founded in 1981, Biological Industries specializes in the development, CE Marking and compilation of technical file. manufacture, and distribution of IVD and research products for cell culture, www.asphalion.com focusing today on stem cell systems and media for cell therapy. BI offers a full range of xeno-free stem cell products and services, which includes stem cell Booth 59: AWST linear scalable, continuous flow, ultracentrifugation solutions culture media, freezing media, attachment factors, and cell dissociation solutions. reliably and efficiently separate, purify, and concentrate viral vectors and virus like BI products include: the Nutristem® range of cell media (for mesenchymal, induced particles in the development and manufacture of vaccines and other bio-prod- pluripotent, and embryonic stem cells), and BIOTARGETTM (optimized specifically ucts. Successful AAV purification has been achieved with yields exceeding 55%, for activation and expansion of T cells), serum-free, xeno-free media for research recovery rates to 50%, and dramatically reduced turnaround time as compared to and clinical applications, helping to advance stem-cell-based therapies. We are conventional column purification. Systems are available for research, pilot scale committed to a Culture of Excellence through our advanced manufacturing and production, and full scale manufacturing. quality-control systems, superior regulatory expertise, and extensive technical In addition to ultracentrifugation, AWST offers a fully automated system for fluid customer support, training, and R&D capabilities. handling and fractionation in a self-contained, mobile work station. System design www.bioind.com supports pinch valves for fluid routing, pumps for flow and direction, an inline refractometer for fractionation/monitoring and disposable flow, pressure and Booth 27: BioNTech temperature transducers for process monitoring. With over 60 years of experience See page 54. and service to the bioprocess community, AWST is the clear choice when looking www.biontech.de for an experienced supplier. www.awst.com 66 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 67 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 EXHIBITORS EXHIBITORS

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Booth 17: Bio-Techne brings together some of the most referenced brands in life Booth 12: CEVEC is a center of expertise for the production of biopharmaceuti- science - R&D Systems, Novus Biologicals, Tocris Bioscience, and ProteinSimple cals using a unique human cell-based production system. CAP-GT is a regulatory providing innovative, high-quality research tools, including: endorsed expression platform for scalable viral vector production. CEVEC has • Bioactive proteins – R&D Systems premiere bioactive proteins successfully developed CAP-GT suspension cell-derived viral packaging cell lines • Application-qualified Antibodies – a diverse and extensive analyte selection which enable better scale-up and competitive production costs when compared to from Novus and R&D Systems adherent cell culture systems. CAP-GT suspension cell lines grow to high cell den- sities and show a broad viral propagation spectrum. Gene therapy vectors such as • Immunoassays – Legendary R&D Systems Quantikine ELISAs, our huge selection lentivirus (LV), adenovirus (AV) and adeno-associated virus (AAV) can be produced of Luminex Assays and cost effective Proteome Profiler Arrays. at industrial scale. www.cevec.com • High quality small molecules – a unique collection of over 3,500 Tocris reagents Together we are Bio-Techne. Find out how we can be your partner and help you Booth 23: ChemoMetec develops, manufactures and sells high quality automated attain your research goals by visiting our stand. Image Cytometer’s within cell counters, which as the only ones on the market can www.bio-techne.com count and analyse aggregated cells, adipose derived stem cells, cells growing on microcarriers with the highest precision. We also offer advanced cell analysers to Booth 50: Bluebird Bio help streamline processes for maximum efficiency. Our instruments are widely used See page 47 in fields such as cancer research, stem cell research, production and quality control www.bluebirdbio.com of a number of products such as pharmaceuticals, beer, animal semen and milk. We have specialised assays for aggregated cells, cells growing on microcarriers and Booth 25: Brammer Bio adipose derived stem cells. 21 CFR Part 11 is also valued highly to have the highest See page 50 standards. Our products are held in high regard because of their high quality and precision as well as the ‘ease of use’ advanced cell analysis. We value our custom- www.brammerbio.com ers; Therefore our policy is “no hidden costs” - no service agreements, high level of support and free software updates. www.chemometec.com Booth 24: Caltag Medsystems provide Life Science and Drug Discovery scientists with a portfolio of over 200,000 high quality research and diagnostic reagents Booth 56: Over 5 years of partnership hundreds of GMP studies performed across Europe. Our Cell Culture portfolio ranges from primary cells, stem cells, cell through an integrated management scheme for recombinant proteins, gene thera- lines and media to skin, fresh animal brains, healthy and diseased products, py, and vaccine products. angiogenesis models and transfection reagents. Clean Cells capabilities: GLP/GMP certification for Quality Control and man- Through our suppliers, we are able to offer: ufacturing | Bacterial, Eukaryotic (producer or packaging cells)and Viral Bank • Human and Animal Tissue Sourcing Service GMP manufacturing | IMP Manufacturing (cell therapy products) | GMP storage | • Fresh and Frozen Bone Marrow Microbiological and viral testing | Identity – Karyotype - FISH | Adventitious virus • CD34+ Cells (Cord Blood, Peripheral Blood, Mobilised Peripheral Blood, Bone testing | Retrovirus detection | Specific contaminants detection (NAT methods) | Marrow) Genetic characterization | Residual DNA/HCP quantitation | RcAAV & RCL GMP certification for APIs and IMPs Manufacturing | • Leukapheresis Packs (mononuclear cells and autologous platelet rich plasma) Genibet capabilities: Biopharmaceuticals GMP Manufacturing (Drug Substance and Drug Product) | • Supplements and Media Bacterial and Yeasts Master/ Working Cell Bank GMP | Manufacturing | Eukaryotic We can source cells types according to specific donor requirements, such as age, Master /Working Cell Bank GMP Manufacturing | Master /Working Virus Seed sex etc. and there are a large number of cell types are available. With our fully Stock GMP Manufacturing | Cell and Gene Therapy GMP Manufacturing | Fill customised tissue procurement service, we are able to source tissue with specific and Finish | Customized offer | Quality Assurance Services | High flexibility and donor inclusion/exclusion criteria, all QC tested and pathology verified. versatile structure | Dedicated Project Manager and single contact point | Highly www.caltagmedsystems.co.uk qualified team www.clean-cells.com www.genibet.com 68 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 69 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 EXHIBITORS EXHIBITORS

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Booth 44: Delphi Genetics Booth 8: Flash Therapeutics is a new gene therapy company developing gene and See page 55 cell-based therapies leveraging its proprietary lentiviral production platform. www.delphigenetics.com The Company is built around the Vectalys lentiviral platform, which includes its patented non-integrative LentiFlash® technology, and integrative lentiviral vectors. Booth 7: In the early 80s’ the establishment of CELBIO represented a true mile- By providing efficient, transient and short-term RNA delivery, LentiFlash is suitable stone, as this Company quickly became a reference for a number of scientists, for gene editing and other advanced therapeutic approaches. When stable DNA giving them a valuable opportunity to gain access to a world of products and expression is needed (immunotherapies such as CAR T cells), integrative lentiviral equipment in Biotechnology. Later on, this name became familiar also in the Med- vectors will be the delivery method of choice. Both technologies benefit from ical and Diagnostic field. This leading approach remained unchanged and possibly novel production and purification processes developed and continually optimized improved throughout almost three decades, reinforcing the image of this Company since 2005. Flash Therapeutics is advancing two business lines: therapeutic devel- as a reliable and solid partner for the entire scientific community. At the beginning opment based on LentiFlash, with internal programs for blood and liver diseases; of the 90s’ a sister Company called Euroclone® started its activity with the goal development and manufacturing support to companies worldwide pursuing of giving more emphasis to the development of smart ideas which were felt to lentiviral-based therapies. Flash Therapeutics was established in 2018 through the evolve into nice products to sell. The success was such that in 2009 it was decided merger of FlashCell, a gene therapy company developing LentiFlash®; and Vectalys, to merge the two Companies, and CELBIO was fully incorporated into Euroclone®. a 13-year old lentiviral vector manufacturer. As a matter of fact, Euroclone® nowadays is bringing into the market the “passion” www.flashtherapeutics.com of the early days (since has inherited the Distribution of a panel of well known brands) along with a clear and growing interest in innovative and state-of-the art Booth 15: Gene Therapy Center Austria technologies originated by in-house R&D as well as obtained under private label See page 51 and OEM agreement with the most qualified manufacturers worldwide. In other www.shire.at words, Euroclone® continues the tradition of CELBIO, while evolving into a modern supplier of up-to-date and own-branded products, thus reconfirming its position as a key-provider for a number of laboratories and clinics. Booth 4: GeneWerk GmbH is a German startup company. The team has long-lasting www.euroclonegroup.it experience in the area of hematology, oncology and virology with focus on integration site analysis, sequencing and bioinformatics. GeneWerk provides quality controlled custom-tailored service based on 20 years of experience in the field of Booth 34: FinVector is a world leader in the research and development of Vi- ral-Based Gene Therapy products, with state-of-the-art facilities and a highly gene therapy, gene editing, immunotherapy and related areas. experienced scientific team working in the gene therapy market. We deliver a www.genewerk.de tailored service to meet and exceed our clients’ needs, and use our scientific expertise and industry knowledge to help clients take viral-based products from the pre-clinical phase, through clinical trials and to the market. Visit us on our stand! www.finvector.com

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Booth 37: GenoSafe is a CRO providing analytical testing services under GLP, GMP Booth 30: Lonza and GCP compliance. We help our clients through their drug development process See page 48 by caring out the laboratory evaluation of the quality, safety and efficacy of their www.lonza.com innovative products (AAV, Lentivirus, Retrovirus, CAR-T cell, Stem Cells). Booth 43: MaxCyte Working for the leaders in Gene and Cell therapy, GenoSafe has been involved in See page 52 immunogenicity studies, biodistribution-biodissemination studies and manufac- www.maxcyte.com tured batches characterization. Booth 36: MDPI Our proudest achievement was to be able to assist our clients from preclinical See page 55 research to clinical studies and Quality Control testing. www.mdpi.com We have a customizable approach with our clients, using our expertise to set up Booth 42: BioReliance | Merck the right method testing on their product that will go all the way through the See page 46 Drug Application www.bioreliance.com www.genosafe.com Booth 16: Miltenyi Biotec is a global provider of products and services that advance biomedical research and cellular therapy. Our innovative tools support Booth 31: Human Gene Therapy research at every level, from basic research to translational research to clinical See page 52 application. Used by scientists and clinicians around the world, our technologies www.liebertpub.com/overview/human-gene-therapy cover techniques of sample preparation, cell isolation, cell sorting, flow cytometry, and cell culture. Our more than 25 years of expertise spans research areas including immunology, stem cell biology, neuroscience, and cancer. Today, Miltenyi Bio- Booth 57: Environmental Risk Assessment Services Since 2001, Jenal & Partners tec has more than 2,000 employees in 28 countries – all dedicated to helping Biosafety Consulting provide Environmental Risk Assessments and Biologics researchers and clinicians make a greater impact on science and health. Assessment of gene and cell therapy products as well as recombinant vaccines www.miltenyibiotec.com and oncolytic viruses for both clinical trials and market authorisation applications submitted to EMA and FDA, respectively. One of the first hour ERAs has been the Booth 32: Molmed one for Glybera by UniQure. Jenal & Partners have extensive experience and back- See page 53 ground knowledge in the environmental risk assessment of gene and cell therapies www.molmed.com involving AAV, Adeno-, Lenti- and Retrovirus, HSV, MVA, NYVAC, VSV... Our clients Booth 58: Nature Technology are both small local and large international pharmaceutical companies. In the See page 55 context of the ERA design, services include responding to questions from www.natx.com competent authorities, compilation of ATMP safety data sheets, definition of ATMP safe administration procedures and safety training of medical personnel. Booth 9: NHS Blood and Transplant is a national organisation within the NHS dedicated to saving and improving lives. Our Cellular and Molecular Therapies www.jenalpartners.ch function offers broad experience and expertise in the provision and manufacture of cell and gene therapies through our national network of GMP grade and Booth 35: KCT MHRA licensed facilities. NHSBT partner with academic, commercial and NHS See page 52 organisations supporting novel cell and gene therapy programmes from concept www.kct.fi through to clinical trial, operating a ‘not for profit’ model. We welcome expressions of interest for partnerships with organisations striving to develop the future of medicine in the advanced therapies space. www.nhsbt.nhs.uk 72 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 73 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 EXHIBITORS EXHIBITORS

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Booth 33: Novasep Booth 29: Pall Biotech provides cutting-edge products and services to meet See page 56 the demanding needs of customers discovering, developing and producing www.novasep.com biologics and classic pharmaceuticals. The company’s membranes and membrane Booth 53: We focus and are highly specialized on applications in cell culture, stem devices optimize detection and sample preparation in the drug research, cell expansion and differentiation, cell counting, sample preparation and cell clinical diagnostics, genomics, and proteomics markets. Pall is a leading provider of assays. We continuously monitor the world’s most innovative life science markets automated systems and single-use solutions to pharmaceutical and biotechnology to expand our portfolio for your benefit. Visit our homepage www.ols-bio.ch for companies - from upstream, through downstream, to formulation and filling - an overview. With our customer-centric philosophy, we provide comprehensive and maintains certified ISO9001 manufacturing facilities worldwide. The support and advice before and after instrument installation. In fact, this is the company’s Scientific and Laboratory Services (SLS), Technical Services and starting point of a valuable relationship for the benefit of our customers. For us, Validation Laboratories have been a cornerstone of customer support for application support as well as service and maintenance of the systems is highly more than 30 years providing compatibility studies, extractable/leachable studies, important and our daily business. We maintain a close contact with the manufac- particulate validation and more. turers of our systems and our application specialists are continuously trained and www.pall.com/biotech kept up-to-date. This ensures optimal support with respect to all of your technical and applicative needs. Any of our system is available for demonstration in your laboratory. Get in touch with us, we look forward to meeting you! Booth 45: Paragon www.ols-bio.ch See page 56 www.paragonbioservices.com/therapeutic-areas/gene-therapy/ Booth 54: Orchard Therapeutics See page 56 www.orchard-tx.com Booth 41: PathoQuest, a leader in next-generation sequencing (NGS) based adventitious virus testing, is offering a game-changing NGS-based metagenomics Booth 26: Oxford BioMedica solution for Infectious Diseases Diagnostics and Biologics Genomic Services. See page 46 www.oxfordbiomedica.co.uk PathoQuest’s metagenomics NGS service is a fast, cost-effective and fully integrated high-throughput sequencing option for viral safety testing that enables Booth 61: Oxford Genetics is a leading synthetic biology company focused on the ability to do a single test instead of multiple assays. PathoQuests´s proprietary developing novel technologies to overcome the challenges associated with the sample-to-report process will help you to address the current limitations associat- discovery, development and production of biologics, gene therapies, cell therapies ed with pathogen detection across numerous biological samples types. In addition and vaccines. Our proprietary genomic system enables the precise engineering of to viral safety assessment, we offer microorganisms sequencing and genetic cell DNA, which through automated production platforms, analytical assessment and process development, drive the rational design of complex biological systems. bank characterization. Meet our expert Jean-Marie Charpin Ph.D., Chief Commer- Taking this approach we have developed several proprietary platform cial Officer, Biologics Genomic Service, and his team at stand #41 to learn more technologies, including: about the expertise we can bring to your testing needs, our advanced bioinfor- • Mammalian cell display system and humanization algorithm for the discovery matic platform and our state-of-the-art lab which meets the highest pharmaceu- and engineering of antibodies tical industry quality standards. • Viral vectors for transient expression of AAV and lentivirus www.pathoquest.com • Viral packaging and production cell lines for stable lentivirus and AAV manufacture • CRISPR pooled and arrayed libraries • CRISPR cell line engineering www.oxfordgenetics.com 74 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 75 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 EXHIBITORS EXHIBITORS

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Booth 10: Supporting life science research since 1988, PeproTech is a privately Booth 28: Polyplus-transfection applies its 15+ year expertise to the development owned biotechnology company focusing on the development and manufacture of novel transfection solutions for mammalian cells. We provide reagents for high of high quality cytokine products for the life-science and cell therapy markets. yield transient protein and antibody production in CHO and HEK-293 cells, as well Over the past 30 years the company has grown into a global enterprise with as for viral vector production for Gene and Cell Therapy (PEIpro product range). Our state-of-the-art manufacturing facilities in the US, and offices around the world. products meet the quality requirements for use in bioprocesses up to GMP grade. Our mission is to provide the highest quality products that address the needs www.polyplus-transfection.com of today’s scientists and researchers, and we pride ourselves on being a trusted partner within the scientific community. With over 2,000 products PeproTech has Booth 3: PROGEN, founded in 1983 and located in Heidelberg, Germany is developed and refined innovative protocols to ensure quality, reliability an established manufacturer and supplier of AAV Titration ELISA tests and and consistency. antibodies for gene therapy research and development. In the past 20 years, the Our product range includes: DIN EN ISO 13485 certified company has established a unique portfolio of AAV Research Grade Proteins and Antibodies | GMP-Compliant products for Cell, Gene tools for basic and clinical research as well as for pharmaceutical applications. and Tissue Therapy | Animal Free Cytokine Range | ELISA kits | Cell Culture Media Among them are specific antibodies to study AAV assembly, capsid formation Kits / Supplements or the course of an infection as well as a line of reliable AAV quantification Please contact PeproTech to discuss your research requirements:+ 44 (0)20 7610 ELISAs for different serotypes utilizing PROGEN’s portfolio of capsid-specific AAV 3062 www.peprotech.com antibodies. PROGEN offers its broad expertise in antibody and ELISA technologies, protein interaction and purification and welcomes collaborations with academic Booth 1: PlasmidFactory GmbH & Co. KG is a globally active biopharmaceutical and industrial institutions to advance basic AAV research. In addition, the company, founded in Bielefeld, Germany, in 2000. As a leading contract man- company aims to establish partnerships with academic, pharmaceutical or ufacturing organization (CMO) for plasmid and minicircle DNA, it has a strong medical institutions for the development of AAV-based tools and therapies, customer base in the fields of cancer research, gene and cell therapy, CAR-T-cell e.g. for standardization and validation steps in clinical trials. development, and vaccination. PlasmidFactory produces plasmids and minicir- www.progen.com cles according to customer’s requirements in modern laboratories with high quality standards. In addition to its In Stock products, e.g. for the worldwide exclusively Booth 60: RoweMed AG – Medical 4 Life is an innovative MedTech company. offered pDG/pDP AAV 2-plasmid-system, and custom manufacturing of plasmid Our focus is on developing and producing customized medical plastic systems, and minicircle DNA, PlasmidFactory focuses its R&D efforts on its core competen- especially for the handling of sensitive pharmaceuticals. cies in the production, analysis, application, and storage of DNA. R&D: The development of systems is closely coordinated with the customer, www.plasmidfactory.com from first sketch up to a market-ready product, including CAD construction, prototyping, first series and product tests. Production: We offer injection molding, assembling and packaging under one-roof. In the cleanroom production facility (ISO class 7) in Parchim our employees carry out single-part, small series and automated mass production. At the assembling department all common welding and gluing technologies are implemented. Regulatory Affairs: In addition to the technical services we offer the complete technical documentation according to the requirements of the Medical Device Directive 93/42/EEC. www.rowemed.de

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Booth 46: Sarepta Therapeutics Booth 21: SYNENTEC provides tailored solutions to solve your individual cell See page 49 culture challenges, like the proof of monoclonality for regulatory approval of cell www.sarepta.com lines, or viability assays and cell density measurements in process development. We develop automated cell culture microscopes such as CELLAVISTA and NYONE Booth 64: Sartorius Stedim – Regenerative Medicine to offer a way to answer your individual cell culture questions in a fast and easy Sartorius Stedim Biotech is a global solution provider to the biologics industry way. Our YT image analysis software is constantly being expanded with new and is well positioned to support regenerative medicine companies by applications and our products are used in the fields of cell line development, applying technologies adapted for these sectors. With a broad range of unique and innovative technologies and services, Sartorius supports the development, stemcell research, immunology, cell and gene therapy as well as in scientific analysis and manufacture of a range of different types of regenerative medicines research. Applications like… 1) Single Cell Cloning (SCC), High Resolution Cloning with single-use automated systems. www.sartorius.com as well as Fluorescence activated SCC (FASCC), 2) Trypan Blue Viability Count, 3) Suspension Cell Count, Confluence Measurement and Colony Detection, 4) Booth 2: SIRION Biotech is a world leader in innovating virus vector technologies FASC Seeding Control, 5) Transfection Efficiency …provide only a part of all the and provides custom services to academic and industrial partners worldwide. possibilities amongst which we facilitate your daily lab use and make it a little SIRION is the only company mastering all 3 major virus types (LV, AV and AAV) more professional! You like going more into the depth? Discover your cells with that are used regularly for genetic manipulation of cell systems, whether for in applications like… 1) Cell Nuclei Count and Characterization, 2) Immuno-stainings, vitro or in vivo purposes. Within the biotechnology sector SIRION cater to a 3) Antibody Internalization, 4) CD-marker Analysis, 5) iPS-cell Detection, 6) focus number of research and development projects, most prominently in therapy forming assays, 7) Toxicity Studies, 8) Apoptosis Monitoring, 9) Total Well Intensity development, e.g. gene and CAR-T cell therapy fields. For development of new Quantitation. The imagers support a wide range of cell culture systems in the SBS cancer immunotherapies, SIRION Biotech has developed a universal adjuvant – format, e.g. mikrotiter plates, transwell plates and microscope slides as well as TM LentiBOOST that can increase T- and B-cell transduction significantly. SIRION culture dishes and T-Flasks. offers full customized services for gene therapy R&D by optimizing AAV tissue www.synentec.com specificity and improving the immunogenic profile. SIRION’s project oriented focus on optimization of vector technologies makes us the perfect partner to strengthen your R&D of new therapies. Booth 18: Synthace is a London-based biotech/software company whose aim is www.sirion-biotech.com to make life science research more efficient. Our sophisticated software allows scientists to easily and flexibly control their automation. This opens up the Booth 14: STEMCELL Technologies Inc. is a Canadian biotechnology company that possibility for rapid DoE studies, media optimisation, assay setup, data analytics, develops specialty cell culture media, cell isolation systems and accessory products and more. for life science research. Our specialty media include cGMP grade mTeSR™1 and 3D www.synthace.com culture kits, supporting the transition from bench to bedside for cancer research, cell therapy and regenerative medicine research, immunotherapy and more. Rigorous quality control processes and raw material selection are critical to the optimal performance of STEMCELL’s entire portfolio of products, ensuring that batch-to-batch variability is minimized. Driven by science and a passion for quality, STEMCELL delivers over 2500 products worldwide with consistent, unfailing quality and one-on-one assistance to help you succeed. If there is a product or service that we do not currently offer, we are happy to work with you to customize media or cell isolation products to meet your specific needs. www.stemcell.com

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EXHIBITORS EXHIBITORS

Booth 11: Takara Bio Europe is a wholly owned subsidiary of Takara Bio Inc., Booth 63: VectorBuilder, established in 2014, is a revolutionary online platform who develops, manufactures, and distributes a wide range of life science reagents that allows researchers to design custom vectors/viruses and simply order them. under the Takara™, Clontech®, and Cellartis® brands. Key products include SMART We possess a massive collection of ORFs since we have generated over 165,000 cDNA synthesis kits for a variety of samples and applications, including NGS, vectors so far meaning we do not synthesize, which in turn means low cost. high-performance qPCR and PCR reagents (including the Takara Ex Taq®, Takara VectorBuilder in addition offers exceptionally high quality viruses, viral packaging, LA Taq®, Titanium®, and Advantage® enzymes), Cellartis stem cells and stem shRNA/gRNA CRISPR libraries and BAC modification as well as CRISPR/Cas9 trans- cell reagents, RT enzymes and SMART library construction kits, the innovative fection/microinjection kits. In-Fusion® cloning system, Guide-it gene editing tools, Tet-based inducible gene www.vectorbuilder.com expression systems, and Living Colors® fluorescent proteins. Recently, Takara Bio acquired Rubicon Genomics and WaferGen Bio-systems. As part of the Takara Booth 20: Vironova is a Swedish biotechnology company providing Bio family, they expand our NGS portfolio as well as add R&D, manufacturing, comprehensive, hardware, software, and services for the analysis of nanoparticles. and support for automation systems for NGS and qPCR applications. Takara Bio’s Vironova revolutionizes access to transmission electron microscopy–based image portfolio supports applications including NGS, gene discovery, regulation, and analysis in biopharmaceutical development. Our solution enables automated function studies, as well as genetic analysis, protein expression and purification, analyses for faster and better informed decisions to secure robust bioprocessing gene editing, stem cell studies, and plant and food research. and final product quality. www.takarabio.com www.vironova.com

Booth 19: The Gene Therapy Program is a complex R&D organization with the Booth 22: VIVEbiotech is a company fully specialized in lentiviral vectors with two internal capacity to perform gene transfer studies from basic research through areas of expertise: phase 1/2 proof-of-concept human trials. We partner with industry leaders to advance the field of gene therapeutics. The Penn Vector core is a full service core GMP CDMO: VIVEbiotech is GMP Contract Development and Manufacturing with over a decade of experience in the production in viral-based vectors. The Organization focused on the manufacture of lentiviral vectors from early stages to core’s main objective is to provide investigators access to state-of-the-art vector GMP. Working with European and US-based companies, our main aim is to adapt technology for preclinical studies and other basic research applications. to your own requirements from a technical and timeline perspective. www.gtp.med.upenn.edu Innovation: VIVEbiotech´s technology development strategy is focused on providing a response to the main hurdles that currently exist within the gene therapy field: • The need for cost-effective processes. • The enhancement of the safety profile of viral vectors. VIVEbiotech is working on improved producer cell line development, new pseudo-typing strategies and the development of its own worldwide licensed technology: a non-integrative episomal stable lentiviral vector (LENTISOMA). www.vivebiotech.com

80 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 81 ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 EXHIBITORS STEM CELL

EXHIBITORS REPORTS EDITOR-IN-CHIEF Booth 47: Xendo is a leading consultancy and project management organisation Christine Mummery, PhD, in the fields of (bio)pharmaceutical products, medical devices and healthcare. We Leiden University Medical Center bring our palette of services to companies, ranging from start-ups to multinational organizations, to provide them with robust solutions. Whether they are a (bio)pharmaceutical or medical device company, a hospital or a pharmacy, a OPEN ACCESS manufacturer or a laboratory, we match their colour. For over 25 years, we have successfully completed thousands of national and international assignments Stem Cell Reports for start-ups as well as for the largest, established multinational companies and organisations. Over 220 experienced and highly educated professionals offer their offers rapid consideration expertise ranging from strategic advice and project management to auditing, of your research operational support and training; providing a full-colour spectrum. www.xendo.com At Stem Cell Reports our editorial team provides you with fast and fair consideration of your research. Booth 49: Yposkesi Read some of the most recently published research: See page 58 • Inactivation of PLK4-STIL Module Prevents www.yposkesi.com Self-Renewal and Triggers p53-Dependent Differentiation in Human Pluripotent Stem Cells • Cell Surface N-Glycans Influence Electrophysiological Properties and Fate Potential of Neural Stem Cells • Defining Lineage-Specific Membrane Fluidity Signatures that Regulate Adhesion Kinetics • Activation of Intrinsic Growth State Enhances Host AAV needed? Axonal Regeneration into Neural Progenitor Cell Grafts • Rapid Mast Cell Generation fromGata2Reporter Pluripotent Stem Cells GTCA – Gene Therapy Center Austria • A Chemical Recipe for Generation of Clinical-Grade Striatal Neurons from hESCs Covering all steps • Organoids from Nephrotic Disease-Derived iPSCs from vector to product Identify Impaired NEPHRIN Localization and Slit Diaphragm Formation in Kidney Podocytes co-located in the Vienna area

For more information visit cell.com/stem-cell-reports

Your AAV One-Stop-Shop in the Heart of Europe

Visit booth 15 or contact: [email protected] INTERNATIONAL SOCIETY FOR STEM CELL RESEARCH 82 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy GTCA_shire_134x94,5mm.indd 1 02.08.18 17:10 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 EDUCATION SESSION EDUCATION SESSION

TUESDAY 16 OCTOBER TUESDAY 16 OCTOBER 09:15-09:30 Organiser and Chair: 11:30-12:30 EDUC 3: CANCER – WHERE DO WE STAND Hildegard Büning Hannover Medical School AND HOW TO MOVE FORWARD? GARDEN FLOOR: GARDEN FLOOR: CONFERENCE EDUC 1: OPENING WORDS CONFERENCE IINV019: Vincenzo Cerullo University of Helsinki ROOM 2/3 INV013: Hildegard Büning Hannover Medical School ROOM 2/3 Dressing viruses in tumor’s clothing: welcome to the Gene and cell therapy - a brief update cloning-free oncolytic vaccine era INV021: Delphine Fessart INSERM U913, University of EDUC 2: TECH. SPEED DATING 09:30-11:00 Bordeaux GARDEN FLOOR: (15 minutes each and 15 for questions) Cancer stem cells and organoids development: towards a CONFERENCE better understanding of the biology behind organoids ROOM 2/3 INV014: Els Verhoeyen CIRI, INSERM U1111 Lyon, C3M, INSERM U1065, Nice 12:30-13:15 Lunch Novel lentiviral pseudotypes for natural killer based cancer CAMPUS FLOOR immunotherapies and ‘nanoblades’ for efficient gene editing in T, B, iPS cells and blood stem cells 13:15-14:45 EDUC 4: CNS SESSION INV015: Karim Benihoud INV021: Nicole Déglon Lausanne University Hospital CNRS UMR 8203, University Paris-Sud, Villejuif Introduction to gene and cell therapy in the CNS INV013: Hildegard Büning INV022: Nathalie Cartier INSERM/ CEA UMR1169, MIRCEN Hannover Medical School CEA and University Paris-Sud, University Paris Saclay Insights into adenovirus and AAV vectorology Gene therapy for Huntington’s disease INV023: Ron Crystal Weill Cornell Medical College, New INV016: Eduard Ayuso York City, NY INSERM UMR1089, University of Nantes Gene therapy for Alzheimer’s disease Manufacturing and quality control of viral vectors EDUC 5: HSC STEM CELLS - WHERE DO WE INV017: Claudio Mussolino 14:45-16:45 STAND AND HOW TO MOVE FORWARD University of Freiburg INV025: Adrian Thrasher University College London How to re-write or epigenetic control our genetic Institute of Child Health information Primary immunodeficiency, gene therapy vs. alternative strategies how do we move into the “real” clinical reality INV018: Michel Pucéat INSERM U1251, Marseille INV026: Juan Bueren CIEMAT/CIBERER-ISCIII, Madrid A brief overview on iPSC and embryonic stem cell Gene therapy in bone marrow failure syndromes technology INV027: Olivier Nègre BlueBirdBio, Cambridge, MA 11:00-11:30 Coffee break Haematopoietic stem cell gene therapy CAMPUS FLOOR INV020: Luigi Naldini SR-TIGET, Milan HSC gene therapy: from lentiviral gene transfer to gene editing 16:45-17:00 Coffee break 84 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy CAMPUS FLOOR ESGCT / ISSCR / SFTCG Collaborative Congress 2018 85 Advance your gene and cell therapy manufacturing

• Integrated cell processing solutions • Process innovation and development • cGMP viral vector production • Cell banking and analytical services

SELECTA BIOSCIENCES IS PROUD TO SPONSOR ESGCT 2018 Selecta’s SVP platform programs the immune system to elicit selective tolerance, mitigating formation of anti-drug antibodies against biologic drugs and gene therapies – enabling life-saving and life-enhancing therapies to do the work intended.

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Copyright © 2017 Merck KGaA. All Rights Reserved. Merck is a registered trademark and the vibrant M is a trademark of Merck KGaA. A-0640717 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 CLINICAL TRIAL AND CLINICAL TRIAL AND COMMERCIALISATION WORKSHOP COMMERCIALISATION WORKSHOP

TUESDAY 16 OCTOBER TUESDAY 16 OCTOBER

Organisers: 13:15-14:15 Lunch Alessandro Aiuti SR-TIGET, Milan Robin Ali UCL, Institute of Opthalmology, London 14:15-16:30 PATIENT ACCESS, REGULATORY AND PLANNING AND RUNNING A CLINICAL TRIAL REIMBURSEMENT CHALLENGES 9:30-11:15 CLOUD FLOOR: CLOUD FLOOR: INV001: Kim Champion AUDITORIUM C INV008: Ron Philip AUDITORIUM C University College London Clinical Trials Centre Spark Therapeutics, Philadelphia, PA General considerations for setting up a clinical trial Patient access worldwide

INV002: Paola Albertini INV009: Ilona Reischl SR-TIGET, Milan Federal Office for Safety in Health Care, Vienna Quality requirements for GLP tox testing and GCLP Update on new ATMP guidelines for GMP production: focus clinical testing on the risk based approach

INV003: Marco Anelli INV010: Martina Schüssler-Lenz ProductLife Group, Milan CAT Chair, Paul Ehrlich Institute, Langen Pharmacovigilance from a sponsor, CRO point of view Risk management planning in the development of ATMP

INV004: Chiara Bonini INV011: Michela Gabaldo SR-TIGET, Milan Telethon, Milan Registry platform for gene and cell therapy: the Market access for ATMP in Europe EBMT approach INV012: Eric Auger Coffee 11:15-11:45 Putnam Associates, Boston, MA CAMPUS FLOOR Market access for ATMP in US MANUFACTURING OF GENE AND CELL 16:30-17:00 Coffee 11:45-13:15 PRODUCTS CLOUD FLOOR: CAMPUS FLOOR AUDITORIUM C INV005: Jean-François Brunet Lausanne University Hospital Building academic GMP facility

INV006: Xin Swanson Lonza, Houston, TX Scaling GMP AAV production

INV007: James Miskin Oxford Biomedica Scaling GMP lentiviral vector production

88 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 89 Helping to Cure®

BEST-IN-CLASS CONTRACT MANUFACTURING Brammer Bio is dedicated to providing clinical and commercial supply of viral vectors for in vivo gene and ex vivo gene-modified cell therapies, process and GENE THERAPY FOR RARE DISEASES analytical development, and regulatory Advancing Therapies From Research support, enabling the delivery of novel medicines and improving patient health. to Patient Treatment Genethon, created by AFM-Telethon, has the mission to make innovative gene therapy treatments available to patients affected with rare genetic www.brammerbio.com diseases, and in particular for neuromuscular disorders. Having played a pioneering role in deciphering the human genome, Genethon is today BRAM71_Half_AD_ESGCT.indd 1 8/20/18 11:53 AM one of the leading organizations for the development of gene therapy treatments. The pipeline of products out of Genethon’s R&D includes gene therapies GMP Solutions for Your C&G Therapy currently in clinical trials and at preclinical stages, for muscular dystrophies (Duchenne Muscular Dystrophy, Limb Girdle Muscle Dystrophies, Myotubular Myopathy, MolMed S.p.A. is a world leading cGMP CDMO offering end-to-end services for Cell & Spinal Muscular Atrophy), immune deficiencies, blood and liver diseases. Gene therapy projects. We bring our extensive experience to meet your needs at every stage of product lifecycle with our two GMP facilities of 5,000 SQM combined area approved for clinical and commercial applications. These products are developed either with Genethon as sponsor, or in partnership with private companies and academic institutions. Plasmid GMP Vector Cell QA and Analytics manufacturing supply transduction Regulatory Network of 48L and 200L Tech transfer and Over 100 in- Commercial level Member of certified batches for development of house methods quality FOR MORE INFORMATION and details: subcontractors retroviral and clients processes for vector and DP management for managing HQ lentiviral vectors and/or release by system standards www.genethon.fr or GMP plasmid manufacturing proprietary internal QPs manufacturing based on process for Support to file proprietary CD34+ and T-cell EMA and FDA MolMed offers processes transduction procedures proprietary HQ packaging plasmids for LVV manufacturing

www.molmed.com For info: [email protected] © C. Hargoues ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PROGRAMME PROGRAMME

TUESDAY 16 OCTOBER WEDNESDAY 17 OCTOBER 17:00-19:00 2018 OPENING SESSION Proffered papers: Chairs: OR001: Jamal Alzubi CAMPUS FLOOR: Institute for Transfusion Medicine and Gene Therapy, AUDITORIUM A Robin Ali, Pierre Cordelier, Nicole Deglon, Nancy Witty Freiburg INV028: Grégoire Courtine A novel chimeric antigen receptor (CAR) T cell approach EPFL, Lausanne eliminates prostate cancer in a mouse tumor model Targeted neurotechnologies enabling walking after paralysis OR002: Saskia König Institute for Transfusion Medicine and Gene Therapy, INV029: Didier Trono (2018 Outstanding Freiburg Achievement Award lecture) Allele-specific editing of STAT3 mutations with CRISPR/Cas EPFL, Lausanne in primary cells of hyper-IgE syndrome patients Retroelements, their polydactyl controllers and the specificity of human biology Presented posters: P166: Kathrin Haake WELCOME RECEPTION Hannover Medical School 19:00-20:00 Hematopoietic stem cell gene therapy for Ifnyr1 deficiency CAMPUS FLOOR protects mice from mycobacterial infections and paves way for macrophage transplantation therapy P167: Viviane Dettmer Institute for Transfusion Medicine and Gene Therapy, WEDNESDAY 17 OCTOBER Freiburg Retroviral UNC13D transfer restores cytotoxic function in T cells derived from familial hemophagocytic 09:00-10:40 S1A: DISEASE MODELLING lymphohistiocytosis type 3 patients Chair: GARDEN FLOOR: CONFERENCE Amy Wagers 09:00-10:40 SESSION 1B: CANCER GENE AND ROOM 2/3 CELL THERAPY INV030: Eva Hedlund CAMPUS FLOOR: AUDITORIUM A Chairs: Karolinska Institute, Stockholm Vincenzo Cerullo, Nicolas Boisgérault Elucidating early disease mechanisms in ALS using stem cells INV032: Waseem Qasim INV031: Holger Willenbring University College London University California San Francisco, CA Genome engineered T cell immunotherapies for leukaemia A mouse model of a human cholestatic liver disease reveals extent and therapeutic potential of mammalian INV033: Kah Whye Peng transdifferentiation Mayo Clinic, Rochester, MN VSV-IFNb-NIS, an armed and trackable oncolytic vesicular stomatitis virus

92 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 93 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PROGRAMME PROGRAMME

WEDNESDAY 17 OCTOBER WEDNESDAY 17 OCTOBER 09:00-10:40 Proffered papers: Proffered papers: OR003: Micaela Harrasser Travel grant awarded by: OR005: Samiah Al Zaidy CAMPUS FLOOR: Centre for Gene Therapy, Nationwide Children’s Hospital, AUDITORIUM A University College London Effective targeting of ROR1+ solid tumours with & Columbus, OH next-generation chimeric antigen receptor therapy AVXS-101 phase 1 gene replacement therapy clinical trial OR004: Catia Traversari in spinal muscular atrophy type 1 (SMA1): 24-month MolMed S.p.A., Milan event-free survival and achievement of developmental In vivo antitumour activity of a hCD44v6-specific chimeric milestones antigen receptor in syngeneic models of solid tumours OR006: Stylianos Michalakis Ludwig Maximilian University, Munich Presented posters: Gene supplementation therapy for CNGA3-linked achromatospia P093: Chin Yan Chang Osaka University Presented posters: RIG-I pathway stimulation polarizes neutrophils to P222: Rui Nobre anti-tumor type neutrophil and suppress tumor growth University of Coimbra P019: Silke Uhrig-Schmidt Non-invasive allele-specific silencing therapy and University Hospital Heidelberg biomarkers for Machado-Joseph Disease CARAAVs - a novel class of CAR-antagonists in cancer P223: Ruslan Grishanin immunotherapy Adverum Biotechnologies, Menlo Park, CA P328: Razieh Monjezi Travel grant awarded by: Long-term aflibercept expression levels in non-human University Hospital Würzburg primates following intravitreal administration of CRISPR/Cas9 unites with Sleeping Beauty to generate ADVM-022, a potential gene therapy for wet agerelated CAR-T cells with enhanced therapeutic index for fighting macular degeneration against immunosuppressive tumour microenvironment 10:40-11:10 Coffee break 09:00-10:40 SESSION 1C: CNS AND EYE DISEASES GENE AND CELL THERAPY I PLENARY SESSION 2: FROM BENCH CLOUD FLOOR: 11:10-13:10 Chairs: Alberto Auricchio, Yvan Arsenijevic TO BEDSIDE I AUDITORUM C CAMPUS FLOOR: INV034: Ian MacDonald AUDITORIUM A Chair: Luigi Naldini University of Alberta, Edmonton, AB INV036: Stuart Forbes Gene therapy for choroideremia: what have we learned Edinburgh University, Centre of regenerative medicine from the clinical trials to date Macrophage therapy for liver disease- preclinical and INV088: Alberto Auricchio clinical TIGEM, Naples INV037: Amy Wagers Expanding AAV transfer capacity in the retina Harvard University, Cambridge, MA In vivo gene editing in tissues and tissue stem cells

94 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 95 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PROGRAMME PROGRAMME

WEDNESDAY 17 OCTOBER WEDNESDAY 17 OCTOBER

INV038: Bev Davidson SESSION 2B: VECTOR DEVELOPMENT I 11:10-13:10 The Children’s Hospital of Philadelphia and The University 15:10-16:50 CAMPUS FLOOR: of Pennsylvania CAMPUS FLOOR: Chairs: AUDITORIUM A Emerging therapies for neurodegenerative diseases AUDITORIUM A Els Verhoeyen, Axel Schambach

INV039: Wing Yen Wong INV043: Jay Chiorini BioMarin, Novato, CA MPTB, NIDCR, NIH, Bethesda, MD Gene therapy in haemophilia: from vision to reality Characterisation of AAV44.9 13:10-15:10 Lunch INV044: Dirk Grimm Bioquant, Heidelberg GARDEN & CAMPUS FLOORS POSTER SESSION I : ODD NUMBERS (see p116) Small but increasingly mighty - latest advances in AAV biology and vector optimization GARDEN FLOOR 13:10-13.30 SFTCG AGM Proffered papers: CAMPUS FLOOR: AUDITORIUM A OR007: Kleopatra Rapti Travel grant awarded by: Heidelberg University Hospital 13:45-14.45 REGULATORY WORKSHOP Generation of novel immune-evading AAVs through GARDEN FLOOR: CONFERENCE ROOM 2/3 identification and mutation of immunogenic epitopes in Alison Armstrong the variable capsid regions of adeno-associated virus 9 SESSION 2A: GENOMIC CHARACTERISATION OR008: Monica Volpin 15:10-16:50 OF PLURIPOTENT STEM CELLS SR-TIGET, Milan GARDEN FLOOR: The impact of vector integration on chromatin Chair: Ludovic Vallier CONFERENCE architecture ROOM 2/3 INV040: Fiona Watt King’s College, London Presented posters: Identifying extrinsic and intrinsic drivers of variation in cell P483: Weiheng Su Travel grant awarded by: behaviour in human iPS cell lines University of Oxford

Exploiting adenovirus mechanisms for the enhanced & INV041: Kevin Eggan production of AAV vectors Harvard University, Cambridge, MA Reducing noise and bias from studies of disease- P484: Jihad El Andari implicated genetic variation through massively-mosaic stem University Hospital Heidelberg cell systems Identification of new muscle-tropic adeno-associated virus (AAV) capsids for treatment of rare hereditary muscular INV091: Tenneille Ludwig disorders WiCell, Madison WI Identification of recurrent genetic variants in hPSCs; a changing landscape

96 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 97 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PROGRAMME PROGRAMME

WEDNESDAY 17 OCTOBER WEDNESDAY 17 OCTOBER

SESSION 2C: EMA/CAT PLENARY SESSION 3: CANCER 15:10-16:50 REGULATORY ASPECTS OF 17:20-18:50 IMMUNOTHERAPY AND CANCER CLOUD FLOOR: ADVANCED THERAPY MEDICINAL CAMPUS FLOOR: STEM CELLS AUDITORIUM C PRODUCTS (ATMPS) AUDITORIUM A Chairs: Chairs: Chiara Bonini, Robert Blelloch Martina Schüssler-Lenz, Ilona Reischl INV049: Hinrich Abken INV045: Martina Schüssler-Lenz University Hospital Regensburg CAT Chair, Paul Ehrlich Institute, Langen CARs and TRUCKs: next generation adoptive cell therapy Introduction to the committee for advanced therapies (CAT) and its tasks in the evaluation of advanced therapies INV050: Yasuhiro Yamada University of Tokyo INV046: Marcos Timón Dissecting cancer biology with iPS cell technology Medicines Agency AEMPS, Madrid EU regulatory aspects of CAR-T cells INV051: Robert Blelloch University of California, San Francisco, CA INV047: Hans Ovelgönne Exosomal PD-L1 as an immune-modulator in cancer Medicines Agency MEB, Utrecht EU regulatory aspects of rAAV vectors INVITED SPEAKER DINNER 20:00-20:30 (INVITATION ONLY) INV048: Matthias Renner Coaches will leave SwissTech at 19.00 back to the Paul Ehrlich Institute, Langen speaker dinner venue EU regulatory aspects of genome editing

ROUND TABLE DISCUSSION 16:50-17:20 Coffee break

98 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 99 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PROGRAMME PROGRAMME

THURSDAY 18 OCTOBER THURSDAY 18 OCTOBER

SESSION 3A: BIOENGINEERING SESSION 3B: MUSCLE & CARDIOVASCULAR 9:00-10:40 9:00-10:40 DISEASES CLOUD FLOOR: Chair: GARDEN FLOOR: AUDITORIUM C Molly Stevens CONFERENCE Chairs: ROOM 2/3 Michel Pucéat, Uta Griesenbach INV052: David Schaffer University of California, Berkeley, CA INV054: Ana Buj Bello Molecular elucidation and engineering of stem cell fate Genethon, Evry decisions Gene therapy of myotubular myopathy: from preclinical studies to a clinical trial INV053: Penney Gilbert University of Toronto, ON INV055: Silvia Priori Making 3D models that matter: engineering skeletal muscle Istituti Clinici Scientifici Maugeri, Pavia tissue in a dish Gene therapy to prevent chaotic behaviours in cardiac electrophysiology Proffered papers: OR009: Pauline Schmit INV056: Antoine de Vries Harvard University, Cambridge, MA Leiden University Medical Centre Cross-packaging control in multiplexed AAV libraries Shining light on cardiac tachyarrhythmias

OR010: François du Plessis Presented posters: uniQure, Amsterdam Development of a next generation synthetic promoter for P392: Capucine Trollet liver directed gene therapy Sorbonne University, Paris BB-301: a single “silence and replace” AAV-based vector Presented posters: for the treatment of oculopharyngeal muscular dystrophy (OPMD) P168: Ekaterina Naumenko Kazan Federal University P247: Francesco Tedesco Enchanced dark-field microscopy for histological detection University College London of nanostructured scaffolds after implantation into bone High-fidelity disease modelling of skeletal muscle defects laminopathies using LMNA-mutant human iPS cells and bioengineered muscles P169: Albert Rizvanov Kazan Federal University Migration ability of human polymorphonuclear leukocytes loaded with synthetic microcapsules

100 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 101 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PROGRAMME PROGRAMME

THURSDAY 18 OCTOBER THURSDAY 18 OCTOBER

SESSION 3C: BLOOD DISORDERS I PLENARY SESSION 4: FROM BENCH 9:00-10:40 11:10-12:40 TO BEDSIDE II CAMPUS FLOOR: Chairs: CAMPUS FLOOR: AUDITORIUM A Juan Bueren, Waseem Qasim AUDITORIUM A Chairs: Adrian Thrasher, Malin Parmar

INV057: Marina Cavazzana INV059: Deepak Srivastava Imagine Institute, Paris Gladstone Institutes, San Francisco, CA Gene therapy of hemoglobinopathies Cardiac development: basis for disease and regeneration

INV058: Bernhard Gentner INV060: Kristin Baldwin SR-TIGET, Milan Scripps Research, San Diego, CA Towards next-generation gene therapy with ex vivo- Precision reprogramming approaches to cardiovascular and engineered haematopoietic stem and progenitor cells neurologic disease

Proffered papers: INV061: Lorenz Studer Memorial Sloan Kettering Cancer Center, New York, NY OR011: Frank Staal Towards a pluripotent-based cell therapy for Parkinson’s Leiden University Medical Centre disease Developing stem cell-based gene therapy for RAG1 deficient-SCID Lunch OR012: Paula Rio 12:40-14:40 CIEMAT-CIBERER-IIS/FJD, Madrid POSTER SESSION II : EVEN NUMBERS (see p138) Gene therapy trial in non-conditioned Fanconi anemia patients GARDEN FLOOR 13.15-14.15 WORKSHOP ON THE USE OF LENTIVIRAL Presented posters: VECTORS FOR IN VIVO GENE THERAPY GARDEN FLOOR: CONFERENCE ROOM 2/3 P052: Carlos Carrascoso Travel grant awarded by: CIEMAT-CIBERER-IIS/FJD, Madrid Speakers: Luigi Naldini, SR-TIGET, Milan Towards the gene therapy of the bone Liver-directed lentivral gene therapy of hemophilia marrow failure in patients with dyskeratosis congenita Deborah Gill, University of Oxford Lung-targeted SIV gene therapy for cystic Fibrosis P053: Pamela Quaranta Kyri Mitrophanous, Oxford Biomedica SR-TIGET, Milan Clinical experience of local administration of lentiviral Role of peripheral blood circulating haematopoietic stem/ vectors in retinal disorders and Parkinson’s disease progenitor cells during physiological hematopoietic maturation and after gene therapy 10:40-11:10 Coffee break

102 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 103 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PROGRAMME PROGRAMME

THURSDAY 18 OCTOBER THURSDAY 18 OCTOBER

SESSION 4A: MOLECULAR BASIS OF Presented posters: 14:40-16:50 DEVELOPMENTAL POTENTIAL P485: Pasqualine Colella GARDEN FLOOR: Chair: Amander Clark University of Paris-Saclay CONFERENCE Tandem promoter design confers tolerogenic and ROOM 2/3 INV062: Jennifer Erwin persistent transgene expression to AAV gene therapy in The Lieber Institute for Brain Development, Baltimore, MD neonate Pompe mice Repetitive elements in stem cells 14:40-16:50 SESSION 4C: GENE EDITING INV063: Ludovic Vallier Chairs: Keith Joung, Paula Rio Wellcome Sanger Institute, Cambridge CAMPUS FLOOR: AUDITORIUM A Mechanisms controlling cell fate decisions in human INV067: Toni Cathomen pluripotent stem cells University of Freiburg New insights in CRISPR/Cas specificity, DNA repair Proffered papers: dynamics and DNA repair outcomes in gene edited OR026: Alberto De Iaco human haematopoietic stem cells EPFL, Lausanne DPPA2 and DPPA4 regulate expression of Dux in mouse INV068: Yong Chang embryonic stem cells Intellia Therapeutics, Cambridge, MA Delivering on the therapeutic potential of CRISPR/Cas9: SESSION 4B: VECTOR DEVELOPMENT II development of an LNP-mediated genome editing 14:40-16:50 therapeutic for the treatment of ATTR CLOUD FLOOR: Chairs: Hildegard Büning, Karim Benihoud AUDITORIUM C Proffered papers: INV064: Rob Kotin OR016: Gerald Schwank University of Massachusetts Medical School, Worcester, MA ETH, Zürich Orthologous dependoparvovirus molecular fossils may pro- Correction of autosomal recessive disorders via vide a source of novel structural motifs and capsids for rAAV CRISPR-associated base editors in adult animals OR017: Jae Young Lee INV065: Jude Samulski ToolGen, Seoul University of North Carolina, Chapel Hill, NC CRISPR/Cas9-mediated downregulation of PMP22 Gene therapy for DMD: from bench to bedside ameliorates Charcot-Marie-Tooth disease 1A in mice INV066: Eduard Ayuso, University of Nantes OR018: Jonathan Finn Starting from the end: analytics driving the manufacturing Intellia Therapeutics, Cambridge, MA Supra-therapeutic levels of transgene expression achieved process of viral vectors in vivo by CRISPR/Cas9 mediated targeted gene insertion Proffered papers: OR019: Anais Amaya Travel grant awarded by: The University of Sydney, NSW OR014: Mathieu Mevel, University of Nantes Successful in vivo editing of patient-derived NextGenAAV: a mix of organic chemistry and vectorology primary human hepatocytes OR015: Sonja Kleinlogel, University of Bern Evolution of recombinant adeno-associated viral vectors for favorable retinal penetration properties

104 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 105 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PROGRAMME PROGRAMME

THURSDAY 18 OCTOBER THURSDAY 18 OCTOBER Presented posters: 20:00 MOLECULAR MINGLE AT P298: Alessia De Caneva Travel grant awarded by: THE OLYMPIC MUSEUM ICGEB, Trieste Coaches will leave from SwissTech at 19.30 Coupling AAV-mediated promoterless gene to the Olympic Museum targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases P299: Antonio Casini FRIDAY 19 OCTOBER University of Trento evoCas9, a highly specific SpCas9 variant from a yeast in SESSION 5A: CELL THERAPY AND vivo screening 09:00-10:40 REPLACEMENT P300: Els Verhoeyen CLOUD FLOOR: CIRI, INSERM U1111 Lyon, C3M, INSERM U1065, Nice AUDITORIUM C Chair: Efficient genome editing in primary human T, B and HSCs Stuart Forbes using Baboon envelope gp pseudotyped virus derived “Na- noblades” loaded with Cas9/sgRNA ribonucleoproteins INV073: Asuka Morizane University of Kyoto Coffee break Cell therapy for Parkinson’s disease with induced 16:50-17:20 pluripotent stem cells PLENARY SESSION 5: NEW TOOLS AND 17:20-19:20 TECHNOLOGIES INV074: Hanna Mikkola CAMPUS FLOOR: Chairs: Robin Ali, Gordon Keller University of California, Los Angeles, CA AUDITORIUM A MLLT3 governs human hematopoietic stem cell self-renewal INV069: Keith Joung Harvard Medical School, Cambridge, MA Proffered papers: In vivo CRISPR gene editing with no detectable OR020: Andrea Schejtman genome-wide off-target mutations University College London Towards clinical application of a lentiviral gene therapy INV070: David Russell protocol for p47phox deficient chronic granulomatous University of Washington and Universal Cells, Seattle, WA disease Universal donor stem cells OR021: Ilaria Meloni University of Siena INV71: Botond Roska Toward gene editing in Rett syndrome Friedrich Miescher Institute for Biomedical Research, Basel The human retina and its organoids at single cell resolution Presented posters: INV072: Alex Meissner P170: Immacolata Brigida Max Planck Institute for Molecular Genetics, Berlin SR-TIGET, Milan Differential regulation of Oct4 targets facilitates Gene therapy for adenosine deaminase 2 deficiency reacquisition of pluripotency

106 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 107 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PROGRAMME PROGRAMME

FRIDAY 19 OCTOBER FRIDAY 19 OCTOBER

SESSION 5B: METABOLIC AND LYSOSOMAL SESSION 5C: BLOOD DISORDERS II 09:00-10:40 DISEASES 09:00-10:40 CAMPUS FLOOR: GARDEN FLOOR: Chairs: AUDITORIUM A Chairs: CONFERENCE Olivier Nègre, Thierry VandenDriessche Federico Mingozzi, Fatima Bosch ROOM 2/3 INV077: Isabelle André-Schmutz INV075: Juan Ruiz Institute Imagine, Paris Abeona Therapeutics, Dallas, TX Ex vivo generated lymphoid progenitors for immune Treatment of lysosomal storage diseases (MPS-IIIA and IIIB) reconstitution in the context of allogeneic transplantation by intravenous administration of AAV vectors and gene therapy

INV076: Michel Zerah INV078: Basil Sharrack Necker Hospital, Paris University of Sheffield Intracerebral gene therapy: neurosurgical point of view Autologous hematopoietic cell transplantation in multiple sclerosis Proffered papers: OR023: Brian Bigger Proffered papers: University of Manchester OR024: Elena Almarza Novoa Brain targeted stem cell gene therapy corrects CIEMAT, Madrid mucopolysaccharidosis type II via multiple mechanisms? Comprehensive preclinical studies for the gene therapy of OR022: Joseph Lillegard patients with leukocyte adhesion deficiency type I (LAD-I) Mayo Clinic, Rochester, MN OR025: Ilana Moscatelli In utero liver-directed lentiviral gene therapy cures a pig Lund University model of hereditary tyrosinemia type 1 Haematopoietic stem cell targeted neonatal gene therapy by a clinically applicable lentiviral vector corrects Presented posters: osteopetrosis in oc/oc mice P357: Sem Aronson Travel grant awarded by: Presented posters: University of Amsterdam Brain targeted stem cell gene therapy corrects P054: Elena Barbon Mucopolysaccharidosis type II via multiple mechanisms University of Evry The potential use of nanobodies delivered via AAV vectors ROUND TABLE in the treatment of haemophilia P171: Cristina Olgassi University of Eastern of Piedmont Orientale, Novara Patient-specific iPSC-derived endothelial cells provide long-term phenotypic correction of haemophilia A 10:40-11:10 Coffee break

108 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 109 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PROGRAMME PROGRAMME

FRIDAY 19 OCTOBER FRIDAY 19 OCTOBER

PLENARY SESSION 6: ORGANOIDS AND 13.45-14.45 GENE THERAPY FOR THE 21ST 11:10-13:10 CELL ENGINEERING CENTURY PATIENT CAMPUS FLOOR: Chairs: Robin Ali, Deepak Srivistava CLOUD FLOOR AUDITORIUM AUDITORIUM A Chairs: INV079: Molly Stevens Nathalie Cartier, MIRCen, INSERM U986, CEA, Fontenay Imperial College London aux Roses Exploring and engineering the cell-material interface for Samantha Parker, Lysogene, Paris regenerative medicine and mechanobiology Speakers: Annie Hubert, Alliance for Regenerative Medicine (ARM) INV080: Rick Livesey Nicole Boice, Global Genes University of Cambridge Cara O’Neill, Cure Sanfilippo Foundation Engineering stem cell-derived human neural tissues to study Elin Haf Davies, Aparito brain development and disease 15:00-16:30 SESSION 6A: REGULATION AND CONTROL OF INV081: Hiromitsu Nakauchi PURIPOTENCY AND LINEAGE SPECIFICATION? University of Tokyo GARDEN FLOOR: CONFERENCE Chair: Teneille Ludwig From cells to organs: exploiting the organ niche for ROOM 2/3 interspecies organogenesis INV083: Keisuke Kaji Edinburgh University INV082: Gordon Keller Molecular mechanisms of cellular reprogramming University of Toronto Translating human development to new therapies with INV084: Amander Clark pluripotent stem cells University of California Los Angeles, CA Unique control of naïve pluripotency in human stem cells 13:10-15:00 Lunch and the germline POSTER SESSION III : ALL POSTERS Proffered papers: GARDEN FLOOR OR013: Julien Pontis EPFL, Lausanne 13.45-14.45 EARLY CAREER RESEARCHERS CAREER’S Evolutionarily recent transposable elements and their WORKSHOP GARDEN FLOOR: CONFERENCE ROOM 2/3 controllers regulate human early embryonic transcriptional Vincenzo Cerullo, University of Helsinki network Deniz Kirik, Lund University Presented posters: P371: Pavel Makarevich Moscow State University Role of paracrine factors secreted by mesenchymal stromal cells in spermatogonial stem cell niche regulation

110 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 111 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018 PROGRAMME PROGRAMME

FRIDAY 19 OCTOBER FRIDAY 19 OCTOBER 15:00-16:30 SESSION 6B: IMMUNE RESPONSES FOLLOWING Proffered papers: GENE THERAPY: FROM VACCINES TO VECTOR OR029: Vania Broccoli CLOUD FLOOR: OPTIMISATION AUDITORIUM C San Raffaele Hospital, Milan Chairs: Anne Galy, Jude Samulski Modeling functional and dysfunctional brain circuits with human iPSC-derived neurons in microfluidic chambers INV085: Maria Croyle OR030: Lee Ni-Chung University of Texas, Austin National Taiwan University Hospital, Taipei Reaching beyond the cold chain: formulation design of Gene therapy for AADC deficiency results in de novo vaccines to improve potency, enhance distribution and dopamine production and supports durable improvement modulate other biological processes in major motor milestones

INV086: Ying Kai Chan Coffee break Harvard University, Cambridge, MA 16:30-17:00 Engineering AAV vectors to evade innate immune and inflammatory responses 17:00-19:00 PRESIDENTIAL SYMPOSIUM ESGCT AGM Proffered papers: CAMPUS FLOOR: AUDITORIUM A Chairs: OR027: Dimitrios Laurin Wagner Robin Ali, Pierre Cordelier, Nicole Deglon, Nancy Witty Charité, Berlin T cell immunity towards CRISPR-associated nucleases INV089: Juergen Knoblich OR028: Tim Beissert Institute of Molecular Biotechnology, Vienna TRON, Mainz Cerebral organoids: modelling human brain development Alphaviral trans-replicating RNA is a low dose vaccine and tumorigenesis in stem cell derived 3D culture vector INV090: Rudolf Jaenisch 15:00-16:30 SESSION 6C: CNS AND EYE DISEASES II MIT, Cambridge, MA Epigenetic regulation in development, ageing and disease CAMPUS FLOOR: Chairs: AUDITORIUM A Nathalie Cartier, Nicole Deglon Proffered papers: INV087: Shin-Ichi Muramatsu OR031: Alessio Cantore (Young Investigator Jichi Medical University Award lecture) Gene therapy for Parkinson’s disease, implications from a SR-TIGET, Milan clinical study of AADC deficiency Shielding lentiviral vectors from phagocytosis increases hepatocyte gene transfer in non-human primates INV035: Sandro Alves Brainvectis, Paris 19:00-19:30 CLOSING DRINKS CYP46A1-gene therapy alleviates spinocerebellar ataxia in mouse models 112 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 113 ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 POSTER SESSIONS

n P001-P018 GARDEN FLOOR: Bioengineering Challenging the n P023-p054 Blood disorders n P060-P093 inevitability of Cancer n P100-P172 Cell therapy and genetic disease replacement n P177-P223 CNS and ocular diseases Spark Therapeutics is building a leading n P226-P247 integrated gene therapy platform as we strive Disease modelling to turn genes into medicines for patients with n P250-P302 inherited diseases, including inherited forms of Gene editing blindness, hemophilia, and other progressive and n P305-P306 debilitating diseases. Genomic characterisation of pluripotent stem cells We seek to challenge the inevitability of genetic n P307-P328 disease by striving to discover, develop and deliver Immunotherapy and treatments in ways unimaginable – until now. genetic vaccines n P330-P357 Metabolic and lysosomal Find out more at sparktx.com, and follow us diseases on Twitter and LinkedIn. n P360-P371 Molecular basis of development potential n P373-P392 Muscle, cardiovascular and pulmonary diseases n P395-P422 Other n P425-P430 Regulation and control of puripotency and lineage specification © 2018, Spark Therapeutics n P435-P492 N-SPK-US-680006 Vector develpment

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P027 P037 P001 P013 Yari Gimenez Martinez CIEMAT/ Kavitha Rajavel Shire, Dublin Philippe Véron Genethon, UMR_S951, Ekaterina Naumenko Kazan Federal CIBERER-ISCIII, Madrid Safety and dose escalation of BAX 888 Inserm, Univ Evry, Université Paris Saclay, University Preclinical studies towards the gene therapy (SHP654), an AAV8 vector expressing EPHE Identification of nano particle mixtures in of Diamond-Blackfan anemia B-domain deleted factor VIII in patients with Development of an AAV-specific human cell culture using dark-field and severe haemophilia A: design of a global, plasmapheresis device for the selective hyperspectral imaging P029 open-label, multicentre Phase 1/2 study removal of anti-capsid antibodies CIRI, INSERM U1111 Lyon, P015 Els Verhoeyen P039 P003 C3M, INSERM U1065, Nice Ekaterina Naumenko Kazan Federal BaEV pseudotyped LVs confer FVIII gene Laura Garcia Perez Leiden University Mohamed Zoughaib Kazan Federal University transfer in HSCs, allowing secretion of Medical Center University Silver-infused halloysite nanotubes as an functional factor FVIII from a B-cell specific Developing SIN lentiviral vectors to correct Interaction of implanted zinc-containing antibacterial nanocomposite in cell therapy promoter in vivo in NSG FVIII -/- mice RAG2 deficiency cryogel with tissue environment P017 P031 P041 P005 BIOENGINEERING Kazan Federal University Albert Rizvanov Oscar Quintana Bustamante CIEMAT/ Daniela Cesana SR-TIGET, Milan Jean-Francois Brunet Lausanne University Effect of titanium nickelide reticular CIBERER-ISCIII, Madrid Addressing the impact of vector genotoxicity Hospital membranes on mesenchymal stem cells Preclinical biosafety studies of lentiviral on the dynamics of hematopoietic Cell manufacturing facility at Lausanne in vitro vector-mediated gene therapy in erythrocyte reconstitution by integration site analyses university hospital: large experience of P019 pyruvate kinase deficiency autologous skin cells for burns patients BLOOD DISORDERS P043 Silke Uhrig-Schmidt NCT University P033

P007 BLOOD DISORDERS Carlos Carrascoso-Rubio CIEMAT/ BIOENGINEERING Hospital Heidelberg Cristina Mesa CIEMAT/CIBERER-ISCIII, CIBERER-ISCIII, Madrid Tomas Kostelec BIA Separations, CARAAVs - a novel class of CAR-antagonists Madrid The engraftment of lentivirally transduced Ajdovšcina in cancer immunotherapy Efficient and cost effective transduction hCD34+ cells in non-conditioned NSG mice is HEK-derived AAV purification: comparison P023 of hematopoietic stem cells with lentiviral not altered due to a mobilization regimen of small scale laboratory production towards vectors for the treatment of leukocyte with G-CSF/AMD3100 industrial format using monoliths Katelyn Masiuk Department of adhesion deficiency type I Microbiology, Immunology and Molecular P045 P009 Genetics, University of California, P035 Juan A. Hernandez Bort Shire, Gene Jacek Lubelski uniQure, Amsterdam Los Angeles, CA Eleanor Luce Inserm UMR 1193, Villejuif Therapy Center Austria, Vienna rAAV large scale manufacturing using BEVS dmPGE2 and poloxamer-F108 enhance Proof of concept for autologous cell/gene Lyophilisation cycle development of AAV technology transduction of human hematopoietic therapy of hemophilia B using patient’s gene therapy product stem and progenitor cells with a ß-globin specific iPSC-derived hepatocytes after P011 lentiviral vector genetic correction with CRISPR/Cas9 P047 Wioletta Lech Mossakowski Medical technology BLOOD DISORDERS P025 Juan A. Hernandez Bort Shire - Gene Research Centre, Polish Academy of Sciences Therapy Center Austria, Vienna Riccardo Biavasco SR-TIGET, Milan Molecular evaluation of neuroprotective Biological activity of additional AAV properties of WJ-MSC in different Oncogene-induced senescence in subpopulation in AAV gene therapy product microenvironmental conditions haematopoietic progenitors features myeloid-restricted hematopoiesis and histiocytosis

116 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 117 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 1 / 17 OCT 13.10 - 15.10 POSTER SESSION 1 / 17 OCT 13.10 - 15.10

P049 P067 P081 P101 Ning Wu DKFZ and NCT, Heidelberg Johann Foloppe Transgene, Strasbourg Albert Rizvanov Kazan Federal University Catarina Miranda CNC, University of Exploring the HIV-1 integration sites with TG6002: a novel oncolytic and vectorised Generation of genetically engineered canine Coimbra different methods gene-prodrug therapy approach to target mesenchymal stem cells co-expressing Repeated mesenchymal stromal cells and treat cancer immunomodulating cytokines and tumour treatment sustainably alleviates Machado- P051 suppressors Joseph disease/spinocerebellar ataxia type-3 P069 Kazan Federal Mariya Tikhomirova P083 P103 University Pierre Cordelier Inserm U1037, Toulouse Effect of dexamethasone on autophagy Role of components of microRNA machinery Albert Rizvanov Kazan Federal University Raffaella Di Micco SR-TIGET, Milan induction in T-lymphocytes of patients with in carcinogenesis: targeting DGCR8 impairs Self-organization and cell proliferation of Impact of DNA damage response activation mild bronchial asthma pancreatic tumors growth adipose derived stem cells, HeLa and in haematopoietic stem and progenitor cells BLOOD DISORDERS P071 mononuclear cells after co-culture upon gene-targeting P053 Tomoyuki Nishikawa Osaka University P085 P105 Pamela Quaranta SR-TIGET, Milan Development of RNA drug to induce Role of peripheral blood circulating Mariya Tikhomirova Kazan Federal Marianna Paulis National Research Council antitumour immunity and cancer selective haematopoietic stem/progenitor cells during University (CNR) – IRGB/UOS of Milan apoptosis physiological hematopoietic maturation and Non-small lung cancer cells exhibit signs of Chromosome transplantation in human iPS after gene therapy P073 EMT in response to cisplatin treatment and cells as a possible approach to treat disorders after development of cisplatin resistance due to X chromosome abnormalities

P061 Mariangela Garofalo University of Milan CANCER Extracellular vesicles enhance the targeted P087 P107 Pierre Cordelier Inserm U1037, Toulouse CANCER delivery of immunogenic oncolytic The antitumoral activity of TLR7 ligands is adenovirus in immunocompetent mice Angelina Titova Kazan Federal University Julia Hanauer Paul Ehrlich Institute, corrupted by the microenvironment of Possibilities of application of photodynamic Langen pancreatic tumors P075 therapy for the treatment of squamous cell In vivo generation of CAR-T cells by carcinoma of the oesophagus and bronchus CD4- and CD8-targeted lentiviral vectors

Shuji Kubo Hyogo College of Medicine, CELL THERAPY AND REPLACEMENT P063 Nishinomiya P089 P109 Philippe Erbs Transgene, Strasbourg Human mesenchymal stem cells as cellular Vaccinia virus shuffling: deVV5, a novel vehicles to deliver retroviral replicating Albert Rizvanov Kazan Federal University Marine Charrier INRA/Oniris UMR 703,

CANCER chimeric poxvirus with improved oncolytic vectors for cancer gene therapy IL-2 genetically modified mesenchymal stem Nantes potency cells demonstrate increased VEGF, MMP2 and Demonstration of immunomodulatory P077 TGF-ß1 genes expression properties for human MuStem cell P065 Sunil Arora PGIMER, Chandigarh population, a promising candidate for cell P093 Olakunle Oladimeji University of KwaZulu- Breast cancer stem cells (BCSCs) associate therapy of muscular dystrophies Natal, Westville with aggressive tumors and cause field Osaka University Chin yang Chang P111 Laminin receptor dependent gold cancerization in breast cancer RIG-I pathway stimulation polarizes nanoparticles for mitochondrial targeted P079 neutrophils to anti-tumor type neutrophil Walter Habeler I-STEM, Corbeil-Essonnes delivery in cancer and suppress tumor growth Establishment of a tissue engineered product Zaneta Slyk Medical University of Warsaw consisting of RPE derived from clinical grade Topical administration of rAAV/ human embryonic stem cell line cultured on carboxymethylocellulose formulations – human amniotic membrane for clinical in vitro and in vivo tests applications

118 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 119 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

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P113 P125 P137 P149 Alice Rannou INRA/Oniris UMR 703, Nantes Antonio Lo Nigro Hubrecht Institute, Pavel Makarevich Lomonosov Moscow Ekaterina Naumenko Kazan Federal Myocardial infarct repair with human adult Utrecht State University University muscle-derived stem cells “MuStem” Dissecting pancreatic exocrine heterogeneity Optimization of mesenchymal stromal cells “Smart” nanostructured scaffolds for to identify a bi-potent progenitor culture in serum-free media using cell-free osteoregeneration in veterinary P115 extracellular matrix biomaterial P127 P151 University of Piemonte Cristina Olgasi P139 Orientale, Novara Luiza de Macedo Abdo Brazilian National Maxim Baltin Kazan Federal University Cancer Institute, Rio de Janeiro Application of combined gene and cell Albert Rizvanov Kazan Federal University Micellar formulation of methylprednisolone Chimeric antigen receptor modified therapy within an implantable therapeutic Cell mediated gene therapy for succinate for local therapy of spinal cord T cells using Sleeping Beauty system by device for the treatment of severe neurodegenerative diseases, neurotrauma injury in rats electroporation and combination with haemophilia A and stroke co-culture expansion has cytotoxic activity P153 P117 in vitro and in vivo P141 Yeonmi Lee University of Ulsan College of Anna Rafiei Hannover Medical School P129 Els Verhoeyen CIRI, INSERM U1111 Lyon, Medicine, ASAN Institute for Life Sciences, Generation of human iPSC-derived Nuttha Klincumhom Chulalongkorn, C3M, INSERM U1065, Nice ASAN Medical Center, Seoul macrophages using a GMP-compliant Bangkok Baboon retrovirus envelope pseudotyped Therapeutic potential of iPSCs derived from process pipeline The effect of Toll-like receptor 3 (TLR3) lentivectors permit robust transduction of a patient with mitochondria disease on regenerative properties of human NK cells and represent and improved tool for P119 periodontal ligament stem cells (PDLSCs) cancer immunotherapy P155 University College London RASA Center, Kazan Giulia Ferrari P131 P143 Albert Rizvanov Combining iPS cell-derived myogenic Federal University progenitors and human artificial Liliana Mendonça University of Coimbra Albert Rizvanov Kazan Federal University Ultrastructure of human adipose-derived Generation and transplantation of CELL THERAPY AND REPLACEMENT chromosomes for genomic-integration-free, Analysis of the interaction of mesenchymal multipotent mesenchymal stromal cells patient-specific iPSC-derived neuroepithelial CELL THERAPY AND REPLACEMENT systemically deliverable cell and gene CELL THERAPY AND REPLACEMENT stem cells with artificial human loaded with synthetic microcapsules CELL THERAPY AND REPLACEMENT therapy of Duchenne muscular dystrophy stem cells for Machado-Joseph disease neuroblastoma cell-derived microvesicles treatment using imaging flow cytometry P157 P121 P133 National Research P145 Alessandra Castelli Chiara Cugno Sidra Medicine, Qatar Chuti Laowtammathron SiSCR, Mahidol Council (CNR) – IRGB/UOS of Milan Clinical grade production of mesenchymal University Pavel Makarevich Lomonosov Moscow Chromosome transplantation to correct the stromal cells from adipose tissue and Enhanced generation of human embryonic State University chronic granulomatous disease defect in foreskin stem cells with minimized trophoblast cell Development of novel antifibrotic therapy mouse pluripotent stem cells proliferation for clinical application based on human MSC secretome P123 P159 P135 P147 Martin Wolf Medical University, Salzburg Artur Fedianin Kazan Federal University Extracellular vesicles of placental expanded Amina Ahmad The University of Sydney Ekaterina Naumenko Kazan Federal Immunoexpression of PSD95 and stromal cells have immunomodulatory and Sydney Children’s Hospitals Network, University synaptophysin in the motononeurons of the capacity and pro-angiogenic function Westmead, NSW Influence of magnetic nanoparticles lumbosacral spinal segments of mouse Reduced activity of the complement system stabilized with polyelectrolytes on 2D and during experimental hypogravity through elevation of complement factor I 3D cell cultures formation using AAV mediated gene delivery to the liver

120 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 121 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 1 / 17 OCT 13.10 - 15.10 POSTER SESSION 1 / 17 OCT 13.10 - 15.10

P161 P177 P189 P201 Diane Seimetz Biopharma Excellence, Rosario Sánchez-Pernaute Andalusian Rajashekhar Gangaraju University of Kyriacos Mitrophanous Oxford BioMedica Munich Initiative for Advanced Therapies, Seville Tennessee Health Science Center, Memphis, TN Prosavin®, a dopamine gene therapy for How to find the right dose for successful Isolation and characterization of CSF-NSCs Adipose stem cells and their paracrine advanced Parkinson’s disease: 5 years phase transition of ATMPs from the nonclinical to from premature infants with intraventricular factors are therapeutic for early retinal I/II clinical update the clinical development stage haemorrhage complications of diabetes in the Ins2Akita mouse P203 P163 P179 Kazan Federal University P191 Albert Rizvanov Da young Chang Ajou University, Suwon Serena Giannelli San Raffaele Scientific Effects of VEGF and FGF2 gene therapy on Assessment of bystander effects of Institute, Milan Gabriel Vachey Lausanne University sciatic nerve regeneration in rats mesenchymal stem cells carrying a cytosine A novel AAV design improving efficiency and Hospital deaminase suicide gene on glioma cells safety in a gene therapy approach for Rett Allele-specific gene editing for Huntington’s P205 syndrome in a mouse model disease mediated by a self-inactivating University Hospitals P165 Thais Bascuas Castillo CRISPR/Cas9 system of Geneva P181 Department of Neurosurgery, Andrew Crane P193 Non-viral transfections with the University of Minnesota, Minneapolis, MN Patrizia Tornabene TIGEM, Naples neuroprotective factors PEDF and GM-CSF Non-hematopoietic umbilical cord blood AAV-mediated protein trans-splicing in the Gaia Colasante San Raffael Scientific reduces oxidative stress in human pigment stem cells exert a neuroprotective effect via retina Institute, Milan epithelium cells in vitro offering a promising reduced numbers of infiltrating immune cells dCas9-based Scn1a gene activation restores approach to treat avascular age-related in chronic stroke and traumatic brain injury P183 inhibitory interneuron excitability and macular degeneration CNS AND OCULAR DISEASES CNC, University of Coimbra attenuates seizures in Dravet syndrome mice P167 Sonia Duarte P207 Let-7 microRNA overexpression mediated by P195 Viviane Dettmer University Hospital lentivirus alleviates neuropathology, balance Albert Rizvanov Kazan Federal University

Freiburg CNS AND OCULAR DISEASES and motor deficits in Machado-Joseph Heikki Turunen AGTC, Cambridge, MA Application of autologous peripheral blood

Retroviral UNC13D transfer restores disease mouse models CNS AND OCULAR DISEASES Effect of neutralizing anti-AAV antibodies mononuclear cells into the area of spinal CELL THERAPY AND REPLACEMENT cytotoxic function in T cells derived from on vector transduction following intravitreal cord injury of pigs in subacute period familial hemophagocytic lymphohistiocytosis P185 administration of AAV in non-human P211 type 3 patients Holger Patzke Voyager Therapeutics, primates Cambridge, MA CNC, University of Coimbra P169 P197 Rui Nobre Robust SOD1 knockdown in large mammal Assessing cerebellar neurometabolic Albert Rizvanov Kazan Federal University spinal cord using a novel delivery paradigm Liliane Tenenbaum Lausanne University biomarkers by in vivo magnetic resonance Migration ability of human with AAV gene therapy targeting SOD1 for Hospital spectroscopy in a transgenic mouse model of polymorphonuclear leukocytes loaded the treatment of SOD1-ALS Long-term uninterrupted high dose Machado-Joseph disease with synthetic microcapsules treatment reduces the beneficial effects of P187 GDNF gene delivery P171 uniQure, Amsterdam Astrid Valles Sanchez P199 Cristina Olgasi University of Piemonte Sustained mutant Huntingtin lowering in Orientale, Novara the brain and cerebrospinal fluid of Ellie Crompton Royal Holloway, University Patient-specific iPSC-derived endothelial Huntington disease minipigs mediated by of London cells provide long-term phenotypic AAV5-miHTT gene therapy Using integration-deficient lentiviral vectors correction of hemophilia A as a therapeutic strategy for spinal muscular atrophy 122 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 123 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 1 / 17 OCT 13.10 - 15.10 POSTER SESSION 1 / 17 OCT 13.10 - 15.10

P213 P227 P239 P253 Ludovico Arcuri CNR National Research Elisa Di Pasquale Humanitas Research Marina Pavlou Ludwig Maximilian Jungmin Lee ToolGen, Seoul Council, Institute of Neuroscience, Milan Hospital, Rozzano University, Munich CRISPR/Cas9-mediated therapeutic editing AAV.PHP.eB-mediated OPA1 gene expression Lentiviral-mediated SCN5A delivery rescues In vitro evaluation of AAV capsid variant of Rpe65 ameliorates the disease phenotypes in a mouse model of Parkinson’s disease as a electrophysiological defects in iPSC-based transduction efficiency in a mouse model of Leber congenital valuable strategy for neuroprotection cardiac models of laminopathy amaurosis P241 P215 P229 P255 Ryota Watano Jichi Medical University Corinne Kostic University of Lausanne Angela Gritti SR-TIGET and San Raffaele AAV8-mediated gene expression in pigs with Manel Llado Santaeularia TIGEM, Naples Gene therapy in Rho-/- mice, a model to Hospital, Milan neutralizing antibody against vector capsid Homology-independent targeted integration validate rod-specific vectors and to identify Human iPSC-based models reveal defective for gene correction in photoreceptors biomarkers of irreversible function neuronal and glial differentiation from P243 P257 restoration neural progenitor cells (NPCs) in globoid Albert Rizvanov Kazan Federal University cell leukodystrophy Editas Medicine, Cambridge, MA P217 Analysis of ectopic dysferlin expression John Zuris P231 effect on the proliferative activity of Highly efficient single and multi-gene Arpad Palfi Trinity College Dublin HEK293A cells after electroporation knockout with CRISPR/Cpf1 in T cells for the AAV-mediated RP2 replacement in a Ulrich Koller EB House Austria, Medical development of improved cell therapies patient-derived in vitro disease model University Salzburg DISEASE MODELLING P245 P259 Keratin 14 labelling with a fluorescence University College P219 Anai Gonzalez Cordero molecule facilitates efficiency analysis of London Kunwoo Lee GenEdit, Berkeley, CA RNA therapeutics in epidermolysis bullosa GENE EDITING S kleine Holthaus University College Using hiPSC-derived retinal organoids to Efficient in vivo delivery of CRISPR simplex London model Ush2a pathophysiology ribonucleoprotein using polymer AAV-mediated gene therapy to treat ocular DISEASE MODELLING P233 nanoparticles failure in NCLs caused by transmembrane P247 CNS AND OCULAR DISEASES Gloria Gonzalez-Aseguinolaza FIMA, P261 protein deficiencies Francesco Saverio Tedesco University Pamplona College London Pietro Genovese SR-TIGET, Milan P221 A new mouse model of HDV infection based High-fidelity disease modelling of skeletal Single cell transcriptomic analysis of gene on AAV vector mediated delivery of the Joshua Bartoe Michigan State University, muscle laminopathies using LMNA-mutant edited HSPC uncovers molecular targets to HDV genome reproduces important East Lansing, MI human iPS cells and bioengineered muscles improve long-term cell repopulation ability characteristics of human HDV infection Submacular injection without vitrectomy P251 P263 of a photoreceptor-targeted AAV5 in P235 cynomolgus macaques Michael Stefanidakis Editas Medicine, Thomas Kocher EB House Austria, Medical Asha Recino University of Cambridge Cambridge, MA University Salzburg P223 Restoration of normoglycemia in diabetic Preclinical assessment of in vivo gene editing A minicircle-based screening system models via insulin gene therapy Ruslan Grishanin Adverum Biotechnologies, efficiency, specificity, and tolerability of facilitates the selection of promising CRISPR/

Menlo Park, CA GENE EDITING P237 EDIT-101, an investigational CRISPR Cas9 double nicking pairs for homology- Long-term aflibercept expression levels in treatment for Leber congenital amaurosis directed repair of genetic mutations non-human primates following intravitreal Massimiliano Caiazzo Utrecht University 10 (LCA10) administration of ADVM-022, a potential Generation of a 3D human neural system for gene therapy for wet age-related macular Parkinson’s disease modeling degeneration

124 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 125 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 1 / 17 OCT 13.10 - 15.10 POSTER SESSION 1 / 17 OCT 13.10 - 15.10

P265 P277 P291 P305 Daniela Benati University of Modena e Giulia Maule CIBIO, University of Trento Yumi Kanegae Jikei University Elias Friman EPFL, Lausanne Reggio Emilia Allele specific genome editing rescues cystic Efficient genome editing system using OCT4 and SOX2 are required at the M-G1 Specific knock-down of C-terminal fibrosis splicing mutations in patients short-term and strong expression of Cas9 transition to re-establish chromatin dominant mutation in Rhodopsin gene by organoids accessibility at enhancers in pluripotent P293 CRISPR/Cas9 system P279 stem cells OF PLURIPOTENT STEM CELLS OF PLURIPOTENT CIEMAT/CIBERER-ISCIII, GENOMIC CHARACTERISATION P267 Maria Garcia Bravo Carsten Lederer The Cyprus Institute of Madrid P307 Neurology and Genetics Marshall Huston Sangamo Therapeutics, ssODN design is essential to ensure an Reversal of fetal globin silencing in primary Alexandre Juillerat Cellectis, Paris Richmond, CA accurate gene correction in fibroblasts cells through isoform-specific knockout of Empowering CAR-T-cell immunotherapies by Enhancing ZFN expression and nuclease derived from primary hyperoxaluria type 1 the BCL11A transcription factor precise genome editing activity in a genome editing construct leads patients to improvement of an in vivo genome P281 P309 editing platform P295 Dina Glazkova Central Research Institute of Sonia Guedan Carrrio The University of Yunus Emre Eksi Akdeniz University Center P269 Epidemiology, Moscow Pennsylvania, Philadelphia, PA Guide RNA selection and homology for Gene and Cell Therapy, Antalya Alteration of CD28 signaling motifs increases Afroditi Avgerinou University College arms design influence the efficiency of Generation of insulin deficient pancreatic anti-tumour activity and persistence of London CRISPR-mediated integration of donor DNA beta cells for insulin gene replacement chimeric antigen receptor-modified T cells Cultivation and characterisation of human therapy P283 airway epithelial cells with potential for P311 P297 CFTR editing for the development of cystic Alessia Cavazza Great Ormond Institute of GENE EDITING Eliana Ruggiero San Raffaele Scientific GENE EDITING fibrosis therapies Child Health, University College London Taeyoung Koo Institute for Basic Science,

GENE EDITING Institute, Milan Targeted gene correction for the treatment Seoul P271 Hunting novel WT1-specific T-cell receptors of severe combined immunodeficiency Adenine base editing in a mouse model of for immune gene therapy of acute myeloid Beatrice Claudia Cianciotti San Raffaele caused by mutations in the IL7R gene Duchenne muscular dystrophy leukaemia Scientific Institute, Milan P285 Generation of memory stem T cells specific P299 Amy Walker University College London P313 for tumour antigens and resistant to Antonio Casini CIBIO, University of Trento inhibitory signals by genome editing Development of a nanoparticle formulation Davide Grandolfo Cell Therapy Catapult, for delivery of a CRISPR/Cas9 gene therapy evoCas9, a highly specific SpCas9 variant AND GENETIC VACCINES IMMUNOTHERAPY London from a yeast in vivo screening P273 for cystic fibrosis Development of analytical strategy to ensure Nerea Zabaleta Lasarte CIMA, IdiSNA, P287 P301 production efficiency, and consistency of a University of Navarra, Pamplona WT1-TCR immunotherapy Nisha Nair VUB, Brussels Jessica Carmen MaxCyte, Gaithersburg, MD Precise in vivo genome editing using two Truncated gRNAs result in efficient in vivo CRISPR-mediated gene editing using a P315 gRNAs targeting nearby genomic regions liver-directed gene inactivation using cGMP-compliant non-viral cell engineering: Gabriele Antonarelli SR-TIGET, Milan CRISPR/Cas9 from iPSC disease modelling to clinically- P275 Enhancing activity of established meaningful correction of monogenic disease P289 anti-myeloma regimens by gene Marisa Cappella Sorbonne University, mutations in patient cells therapy-driven Interferon-a delivery INSERM U974, AIM, Paris Matias I. Autio Genome Institute of Singapore AAV-mediated gene therapy for fALS Characterisation of novel putative human safe harbour loci for controlled transgene expression 126 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 127 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 1 / 17 OCT 13.10 - 15.10 POSTER SESSION 1 / 17 OCT 13.10 - 15.10

P317 P331 P341 P353 Brian Busser Cellectis Therapeutics, Paris Randy Chandler National Institutes of Umut Cagin Genethon, UMR_S951, Inserm, Sereina Deplazes University Children’s Engineering CAR-T cells with an integrated Health, Bethesda, MD Univ Evry, Université Paris Saclay, EPHE Hospital Zürich on/off switch Liver directed gene therapy using AAV9 to Functional, biochemical and transcriptional Naked-DNA minicircle-vector based treat a new murine model of propionic rescue of advanced Pompe disease with liver gene-therapy for ornithine transcarbamylase P319 acidemia caused by Pccb deficiency expression of secretable GAA (OTC)-deficient spf-ash mice GeneWerk, Heidelberg Peter Ulrich P333 P343 P355 Lentivirus integration site analysis characterization of CTL019 Fulvio Mavilio Audentes Therapeutics, San Giulia De Sabbata ICGEB, Trieste Ruben van Helden Leiden University Francisco, CA Development of a novel AAV-based gene Medical Center P321 Gene therapy for Crigler-Najjar syndrome therapy in combination with tolerogenic Metabolic characterization of hiPSC-derived Albert Rizvanov Kazan Federal University with AT342, a liver-targeted AAV8-UGT1A1 nanoparticles for sustained treatment of cardiomyocytes from patients suffering from vector: preliminary results from a Phase ornithine transcarbamylase deficiency infantile cardiomyopathy Anti-CD19 CAR-T cells are efficient against 1/2 study (VALENS) CD19-positive 3D bioprinted solid tumor P345 P357 models

P335 DISEASES AND LYSOSOMAL METABOLIC Sean Armour Spark Therapeutics, Sem Aronson UMC University of P323 Saori Miwa Jikei University Philadelphia, PA Amsterdam Yaling Shi Lonza, Basel Intravenous AAV9-mediated gene Optimization and scale-up of a liver-directed AAV-mediated liver directed gene therapy therapy ameliorates neuronal disease of approach for Pompe disease based on corrects the cholestatic phenotype in Automated end-to-end manufacturing mucopolysaccharidosis type ll (MPSll) secretable GAA Abcb4-/- mice solutions for CAR-T immunotherapies P337 P347 P361 P325

IMMUNOTHERAPY AND GENETIC VACCINES IMMUNOTHERAPY Randy Chandler National Institutes of Salih Sanlioglu Gene and Cell Therapy Daniela Gnani SR-TIGET, Milan Josefina Nilsson Vironova, Stockholm METABOLIC AND LYSOSOMAL DISEASES AND LYSOSOMAL METABOLIC Health, Bethesda, MD Center of Akdeniz University, Antalya A pre-senescence program in aged Automated determination of the purity Anc80 and AAV8 vectors mediate Intraperitoneal delivery of LentiINS vector mesenchymal stromal cells contributes and packaging of gene therapy delivery equivalent long-term hepatic correction of lowered fasting plasma glucose and to inflammation in the hematopoietic platforms using transmission electron DISEASES AND LYSOSOMAL METABOLIC methylmalonyl-CoA mutase deficiency in a improved glucose tolerance of type 1 compartment microscopy murine model of isolated methylmalonic diabetic Wistar rats P363 P327 acidemia (MMA) P349 Ilya Grigoryev Kazan Federal University Valerie Kedinger Polyplus Transfection, P339 Robert Kaiser Children’s Hospital of Illkirch Triazole and pirazole p-tert- Valentina Poletti Dana-Farber Cancer Minnesota, Minneapolis, MN butylthiacalixarene derivatives - new DNA Alternatives to viral vectors for nucleic Institute - Harvard Medical School, A toxicological assessment of in vivo condensing agents for gene therapy acid-mediated therapies Cambridge, MA lentiviral vector administration in a mouse Enhanced lentiviral transduction coupled to model of liver injury P365 cell homeostasis preservation in human Angelina Titova Kazan Federal University hematopoietic stem/progenitor cells P351 Influence of long-term cultivation and Sem Aronson UMC University of Amsterdam POTENTIAL MOLECULAR BASIS OF DEVELOPMENTAL cryopreservation on phenotype of rat Crigler-Najjar World Registry: shortcomings hepatic stellate cells of current treatments underscore the potential benefit of gene therapy

128 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 129 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 1 / 17 OCT 13.10 - 15.10 POSTER SESSION 1 / 17 OCT 13.10 - 15.10

P367 P377 P389 P403 Mikhail Mavlikeev Kazan Federal University Angelina Titova Kazan Federal University Camille Vaubourg Genethon, UMR_S951, Bryan Piras St. Jude Children’s Research VEGF-VEGFRs axis role in reparative Biosynthesis of DYSF protein in Bla/J Inserm, Univ Evry, Université Paris Saclay, Hospital, Memphis, TN osteogenesis in paraossal haematomas dysferlin-deficient mice after administration EPHE Evaluation of multiple adherent and of recombinant adenovirus encoding Evaluation in mice of two genetic modifiers suspension 293-based cells for production of P369 dysferlin cDNA of muscular dystrophy on the phenotypic AAV Institute of Fundamental severity of FKRP deficiency Angelina Titova P379 P405 Medicine and Biology, KFU, Kazan P391 Parenchymal and nonparenchymal cellular Astrid Brull Sorbonne University, Paris Bryan Piras St. Jude Children’s Research proliferation dynamics after partial Gene therapy via trans-splicing for Albert Rizvanov Kazan Federal University Hospital, Memphis, TN hepatectomy with and without LMNA-related congenital muscular Gene therapy for the treatment of injuries of Optimization of transfection conditions to 2-acetylaminofluorene injection in rats dystrophy the superficial digital flexor tendons and maximize AAV production in suspension branch of suspensory ligament lesions in 293-based cells P371 P381 MUSCLE, CARDIOVASCULAR AND PULMONARY DISEASES horses P407 Lomonosov Moscow Laboratory of Genetics Pavel Makarevich Thayane Crestani P395 State University and Molecular Cardiology, Heart Institute, Pavel Makarevich Institute for Regenerative

MOLECULAR BASIS OF DEVELOPMENTAL POTENTIAL MOLECULAR BASIS OF DEVELOPMENTAL Role of paracrine factors secreted University of São Paulo Medical School Omar Dabbous AveXis, Bannockburn, IL Medicine, Medical Research and Education by mesenchymal stromal cells in Urine progenitor cells as an alternative Indirect treatment comparison of AVXS-101 Centre, Lomonosov Moscow State University spermatogonial stem cell niche regulation source to generate iPSC-derived to nusinersen for the treatment of type 1 Antifibrotic environment of stromal cells cardiomyocytes (iPSC-CMs) and electrical spinal muscular atrophy (SMA1) drives scar-free healing of human P373 stimulus as an important mechanism for OTHER endometrium during menstruation P397 Sarepta Therapeutics, iPSC-CMs maturation Andrea Sarasin P409 Cambridge, MA INSERM UMR 1193, P383 Antonietta Messina PPMO treatment results in widespread Villejuif Omar Dabbous AveXis, Bannockburn, IL muscle delivery and efficacy in mice Patrice Vidal Genethon, UMR_S951, Inserm, Spheroid formation and evaluation Number needed to treat in spinal muscular and cynomolgus monkeys: a potential Univ Evry, Université Paris Saclay, EPHE of human induced pluripotent stem atrophy type 1 with AVXS-101 relative to therapeutic platform for the treatment AAV vector-based gene therapy for glycogen cell-derived hepatocytes through nusinersen of Duchenne muscular dystrophy storage disease type III self-assembly process MUSCLE, CARDIOVASCULAR AND PULMONARY DISEASES OTHER P411 P375 P385 P399 Omar Dabbous AveXis, Bannockburn, IL Alberto Malerba Centre of Biomedical Fabien Dorange Genethon, UMR_S951, Ji Hyun Kim Rznomics, Gwangju Economic burden of infant-onset (type 1) Sciences, School of Biological Sciences, Inserm, Univ Evry, Université Paris Saclay, Deactivation of hepatic stellate cells by spinal muscular atrophy: a retrospective Royal Holloway University of London EPHE down-regulation of exosomal miRNAs in claims database analysis Intranuclear inclusion body (INI) pathology Comparison of immunodetection HCV-induced liver fibrosis and muscle atrophy in oculopharyngeal based-technologies for HCP testing for gene P413 P401 muscular dystrophy can be reversed by therapy products Caroline Odenwald PROGEN Biotechnik, “silence and replace” AAV-mediated gene MUSCLE, CARDIOVASCULAR AND PULMONARY DISEASES Yen-Hui Chan National Taiwan University Heidelberg therapy P387 Hospital, Taipei (r)evolution of AAV2 titration ELISA – from Tiziana Coradin Oxford BioMedica Anc80-mediated gene therapy rescues the monoclonal to recombinant Targeting lung diseases with pseudotyped hearing and balance dysfunctions in the Pjvk lentiviral vectors mutant mice

130 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 131 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 1 / 17 OCT 13.10 - 15.10 POSTER SESSION 1 / 17 OCT 13.10 - 15.10

P415 P435 P447 P459 Julie Chassagne UMRS974, UPMC Sorbonne Sarah Le Saux Institute Charles Gerhardt Xiaoxia Shi UMC University of Amsterdam Jens Gruber German Primate Center, University, Inserm / AIM, Paris Montpellier, UMR 5253 CNRS-UM-ENSCM Insertion of a liver-specific promoter in Göttingen RFX1 and RFX3 transcription factors interact Exploring the potential of extracellular an SV40 gene delivery vector results in Improving viral and non-viral vectors: with the D sequence of adeno-associated vesicles as drug delivery systems improved expression of therapeutic genes adopting exosome biology for efficiency and virus inverted terminal repeat and regulate in hepatoma cell lines hydrogels for controlled release AAV transduction P437 P449 P461 P417 Matthias Bozza DKFZ and NCT, Heidelberg A novel non-viral, non-integrative Claire Domenger Heidelberg University Ram Shankar PlasmidFactory, Bielefeld Quentin Enjalbert Anaquant, Villeurbanne nano-DNA vector system for T-cell Hospital AAV vectors are going viral in gene and cell A universal analytical tool for protein engineering Post-transcriptional fine-tuning of AAV therapy identification and quantification in gene or vector gene expression for hemophilia A P439 P463 cell therapies development gene therapy OTHER Selecta Biosciences, Kerstin Hein Cevec Pharmaceuticals, Cologne P419 Petr Ilyinskii P451 Watertown, MA Generation of helper virus-free adeno- John Murray University of Florida, Tolerogenic nanoparticles enhance Lori Karpes Voyager Therapeutics, associated viral vector packaging/producer Jacksonville, FL transgene expression and intracellular vector Cambridge, MA cell lines based on a human suspension cell Paramagnetic immunobead isolation of copy number after initial and repeated Challenges and successes in high line human adipose-derived stem cells: a proof administrations of AAV-based gene therapy concentration AAV drug product P465 of concept study vectors through immunological and formulation development CHA Bundang Medical P421 non-immunological mechanisms Kyung-Ju Choi P453 center, CHA University Ekaterina Naumenko Kazan Federal P441 Anti-tumor activity of GM-CSF and IL-12 VECTOR DEVELOPMENT VECTOR Heidi Hynynen Kuopio Center for Gene and University Betty Maepa University of the Cell Therapy expressing oncolytic HSV-1

Kaolin clay alleviates toxicity of graphene DEVELOPMENT VECTOR Witwatersrand, Johannesburg Development of AAV full/empty capsid ratio DEVELOPMENT VECTOR P467 oxide in aqueous environments

Application of a synthetic adeno-associated analysis using semi-automated transmission Voyager Therapeutics, P425 viral vector for the delivery of anti-hepatitis electron microscopy Jeffrey Slack B virus gene therapeutics Cambridge, MA David Suter École Polytechnique Fédérale P455 Production of Caltech AAV capsids with de Lausanne P443 BEVS: challenges and solutions Diana Schenkwein A.I.Virtanen Institute, Endogenous fluctuations of OCT4 and SOX2 Alissa Bray Oxford Genetics University of Eastern Finland, Kuopio P469 bias pluripotent cell fate decisions Reconfiguration of AAV Rep-Cap coding Protein transduction of genome editing Pavel Makarevich Lomonosov Moscow P427 sequences significantly increases viral vector enzymes with new lentivirus vector derived State University yield and enables inducible AAV production particles Louise Bullen Manchester Metropolitan Development of a plasmid construct in HEK293 cells University P457 encoding HGF and VEGF165 for gene AND LINEAGE SPECIFICATION Interrogation into the molecular role of p62 P445 therapy in iPS reprogramming Giuliana Vallanti MolMed, Milan P471 REGULATION AND CONTROL OF PURIPOTENCY AND CONTROL OF PURIPOTENCY REGULATION Youna Coquin Genethon, UMR_S951, Lentiviral/retroviral vector large scale Inserm, Univ Evry, Université Paris Saclay, manufacturing Lionel Galibert Kuopio Center for Gene and EPHE Cell Therapy New lentiviral pseudotypes based on murine Large-scale optimisation and production of syncytin-A or -B are efficient tools for the rAAV vector encoding VEGF in iCELLis transduction of murine and human B cells bioreactors

132 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 133 ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018

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P473 P485 Simon Chanas GSK, Stevenage Pasqualina Colella Genethon, Evry, Comparison of recombinant AAV vector yield UMR_S951, Inserm, Univ Evry, University using transient transfection of producer cells Paris Saclay with 2- and 3-plasmid systems Tandem promoter design confers tolerogenic and persistent transgene expression to AAV P475 gene therapy in neonate Pompe mice PlasmidFactory, Bielefeld Marco Schmeer P487 Minicircle DNA as starting material for development of ATMPs Simone Merlin University of Piemonte Orientale, Novara P477 FVIII expression driven by its native promoter Andre Sobczyk Brain and Spine Institute, allowed phenotypic correction in hemophilic Paris mice iVector, a core facility for bioproduction of P489 viral vectors (lentivirus, AAV & CAV-2) used in neurosciences research at the Brain and Anna Kajaste-Rudnitski SR-TIGET, Milan Spine Intitute Cyclosporine H overcomes IFITM3-mediated

VECTOR DEVELOPMENT VECTOR innate immune restriction to lentiviral P479 transduction and gene editing in human Pascale Belguise Polyplus Transfection, haematopoietic stem cells VECTOR DEVELOPMENT VECTOR Illkirch P491 Addressing large-scale manufacturing of clinical grade viral vectors using an Petr Ilyinskii Selecta Biosciences, optimized PEI-based transfection process Watertown, MA Combination of an engineered AAV vector P481 Anc80 and tolerogenic nanoparticles Roland Leathers Thermo Fisher Scientific, encapsulating rapamycin enables efficient Waltham, MA transgene expression in mice with pre-existing neutralizing antibodies and April 29 - May 2, 2019 A novel suspension-based lentiviral provides a therapeutic benefit in a mouse production platform to achieve model of methylmalonic acidemia cost-effective clinical manufacturing Washington, D.C. P483 Weiheng Su University of Oxford Exploiting adenovirus mechanisms for the annualmeeting.asgct.org enhanced production of AAV vectors

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ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 2 / 18 OCT 12.40 - 14.40 POSTER SESSION 2 / 18 OCT 12.40 - 14.40

P002 P016 P032 P044 Sylvain Cecchini Voyager Therapeutics, Mohamed Zoughaib Kazan Federal Luca Basso-Ricci SR-TIGET, Milan Sara Deola Sidra Medicine, Qatar Cambridge, MA University Kinetics and quality assessment of mobilized Flow-cytometry platform for intracellular Engineering of 5’ UTR to control the In vitro optimization of a cryogel based stem cell product upon G-CSF and Plerixafor detection of FVIII in blood cells: a new tool expression and incorporation level of VP1 tumour model administration in patients with hematologi- to assess gene therapy efficiency for during rAAV vector production using a cal disorders for gene therapy approaches haemophilia-A baculovirus system P018 P034 P046 BIOENGINEERING Kazan Federal P006 Ekaterina Naumenko University Aphrodite Georgakopoulou G.Papanicolaou Akbar Farjadfar Fasa University of Medical Mohamed Zoughaib Kazan Federal Magnetic nanomaterials for 3D spheroids Hospital, Thessaloniki Sciences University formation Efficient ex vivo gene transfer of y-globin in Gene therapy of breast cancer related Development of nanosized human thalassemic CD34+ cells using an anemia by delivering of erythropoietin in hydroxyapatite-containing cryogels P024 integrating hybrid adenoviral vector system mice model as a matrix for osteogenic cells G.Papanicolaou P048 Aphrodite Georgakopoulou P036 Hospital, Thessaloniki P008 Sergei Abramov Kazan Federal University Reversal of the thalassemic phenotype in Diego Leon-Rico University College London The risk allele A of rs200395694 associated Omid Mashinchian Nestlé Institute of mice post in vivo transduction of mobilized Institute of Child Health with SLE in Swedish patients affects on Health Sciences, Lausanne hematopoietic stem cells (HSCs) with an Systematic comparison of culture media and MEF2D gene regulation and alternative 3D-derivation of uncommitted human integrating hybrid adenovirus vector system transduction enhancers for optimised CD34+ splicing muscle stem cells from iPSCs cell-based retroviral gene therapy protocols P026 P050 P010 P038 BIOENGINEERING Wolfgang Miesbach University Hospital, Mariya Tikhomirova Kazan Federal BLOOD DISORDERS Ekaterina Naumenko Kazan Federal Frankfurt BLOOD DISORDERS Kavitha Rajavel Shire, Dublin University University Surgery and bleed management in patients The prevalence of pre-existing humoral The role of autophagy expression in Effects of curcumin-loaded halloysite on receiving AMT-060 in a Phase I/II trial: immunity to AAV in adults with severe T-lymphocytes of patients with severe C.elegans nematodes in host-microbiome evaluation of the safety of exogenous FIX haemophilia: interim results from an asthma interactions treatment after gene transfer ongoing global epidemiology study P052 P012 P028 P040

BLOOD DISORDERS Carlos Carrascoso-Rubio CIEMAT/ Ekaterina Naumenko Kazan Federal Maria Ester Bernardo San Raffael Scientific Jose Antonio Casado Olea CIEMAT/ CIBERER-ISCIII, Madrid University Institute, Milan CIBERER-ISCIII, Madrid Towards the gene therapy of the bone The mechanism of the nanoparticles Impairment in the hematopoietic supportive The ex vivo transduction of human marrow failure in patients with dyskeratosis penetration into human cells capacity of bone marrow stroma in hematopoietic stem cells induces the congenita beta-thalassemia patients is associated with expression of NKG2D ligands P054 P014 niche iron overload and oxidative stress P042 Kazan Federal Elena Barbon Genethon, UMR_S951 Mohamed Zoughaib P030 University Sophie Ramadier Imagine Institute, Inserm, Univ Evry, University Paris Saclay, EPHE Characterization of microelement-doped Gülen Güney Hacettepe University, Anuara INSERM UMR1163, Paris hydrogels as a bioactive cellular matrix The NPY-Y1 receptor for neuropeptide Y is Combination of lentiviral and genome The potential use of nanobodies delivered highly expressed on hematopoietic stem cells editing technologies for the treatment via AAV vectors in the treatment of from bone marrow and cord blood of sickle cell disease haemophilia

138 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 139 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 2 / 18 OCT 12.40 - 14.40 POSTER SESSION 2 / 18 OCT 12.40 - 14.40

P060 P072 P086 P106 Thomas Hartjes Dept. of Neurosurgery, Sung Soo Kim Department of Anatomy, Ahter Dilsad Sanlioglu Akdeniz University María Rosario Hervás Salcedo CIEMAT/ Erasmus MC, Rotterdam Ajou University School of Medicine, Suwon Center for Gene and Cell Therapy, Antalya CIBERER-ISCIII, Madrid Organotypic multicellular spheres (OMS) as a Synergistic antitumour effects of HuR knockdown decreases membrane Improved haematopoietic engraftment 3D model system to study oncolytic temozolomide in combination with suicide expression of DR5 and reduces apoptosis due to the intrabone or intravenous adenovirus responses in glioblastoma gene expressing mesenchymal stem cells in levels co-transplantation of human tumours orthotopic xenograft glioma models haematopoietic stem cells and mesenchymal P088 stromal cells in immunodeficient mice P062 P074 Albert Rizvanov Kazan Federal University P108 Pedro Leite Azevedo Stem Cell Laboratory, Akbar Farjadfar Fasa University of Medical Rac1 and Bcl-2 expression changes in Bone Marrow Transplantation Unit, National CANCER Sciences co-culture of mesenchymal stem cells and Tatjana Cornu University Medical Center Cancer Institute (INCA), Rio de Janeiro Evaluation of proapoptotic effects of MSC neuroblastoma cells after incubation with Freiburg WNT signaling pathway regulates Bmp4 expressing endostatin and TRAIL on SVEC cisplatin A highly efficient and GMP-compliant expression in mesenchymal stromal cells and 4T1 cell lines and mouse model protocol to manufacture CCR5-edited cells from acute myeloid leukemia patients P091 to treat HIV infection P064 P076 Angelina Titova Kazan Federal University P110 Ai-Li Shiau National Cheng Kung Chang Ho Lee Dankook University, Yongin The results of treatment of patients with Tarekegn Hiwot University of Birmingham University, Tainan metastatic melanoma without a primary Gene replacement based on RNA A phase 1/2 clinical trial for AAV8-mediated Adenovirus-mediated transfer of shRNA focus being detected reprogramming as an effective approach for liver-directed gene therapy in adults with against Elovl6 reduces the progression of personalized cancer theranostics late-onset OTC deficiency hepatocellular carcinoma P100 CANCER CANCER P078 Academia Sinica, Taipei P112 P066 Hung-Chih Kuo Angelina Titova Kazan Federal University Transplantation of somatic cell-induced Andrey Pulin Federal State Budgetary Filippo Birocchi San Raffaele University, The search for the primary tumor in patients neural progenitors enhances functional Scientific Institution “Institute of General Milan with metastases of malignant tumors recovery after stroke CELL THERAPY AND REPLACEMENT Pathology and Pathophysiology”, Moscow without identified primary tumor Development of a chimeric form of IFNa for Comparative analysis of therapeutic efficacy “on demand” in vivo cancer gene therapy P080 P102 of mesenchymal stromal cells isolated from Patricia Peking SCI-TReCS, Paracelsus different sources on rat model of thermal P068 Laia Simó-Riudalbas EPFL, Lausanne skin burn Transposable element-driven transcripts as Medical University, Salzburg Hrvoje Miletic Haukeland University new cancer biomarkers Generation of immunosuppressive iPSC- P114 Hospital, Bergen derived stromal cells for tissue regeneration HSV-tk mediated suicide gene therapy P082 Pavel Makarevich Lomonosov Moscow P104 State University leads to an immunogenic cell death of Lukasz Kuryk Targovax, Helsinki Cell sheets as a platform for therapeutic glioblastoma cells and a T-cell mediated Quantification and functional evaluation of Serena Scala SR-TIGET, Milan immune response CELL THERAPY AND REPLACEMENT delivery and tissue modelling CD40L production from an adenovirus vector Comparison of hematopoietic reconstitution ONCOS-401 P070 dynamics of MPB- and BM-derived P116 hematopoietic stem/progenitor cells gene P084 Jumi Park University of Ulsan College of Pierre Cordelier Inserm U1037, Toulouse therapy in Wiskott-Aldrich syndrome Medicine, Seoul H-1 parvovirus inhibits both primary tumor Sung Yong Ahn Yonsei University, Seoul patients treated with lentiviral gene therapy and metastatic growth of human pancreatic Mitofusin-2 expression is implicated in Mitochondrial genome mutations in induced tumours cervical cancer pathogenesis pluripotent stem cells

140 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 141 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 2 / 18 OCT 12.40 - 14.40 POSTER SESSION 2 / 18 OCT 12.40 - 14.40

P118 P128 P140 P152 Pavel Makarevich Faculty of Medicine, Keerang Park Chungbuk Health & Science María Rosario Hervás Salcedo CIEMAT/ Albert Rizvanov Kazan Federal University Lomonosov Moscow State University University, Cheongwon-gun CIBERER-ISCIII, Madrid Analysis of secretome of umbilical cord MSC self-organization in vitro is concordant Development of testing methodologies to In vivo enhanced anti-inflammatory effects blood mononuclear cells after gene with elevation of regenerative potential and detect residual host genomic DNA for of human mesenchymal stromal cells modification characteristics related to stem cell niche lentivirus vector-based gene therapy transfected with CXCR4 and IL10 mRNAs P154 function products P142 Yaowalak U-Pratya Siriraj Hospital, P120 P130 Martina Kropp University of Geneva Mahidol University, Bangkok Sergio Lopez Manzaneda CIEMAT/ Chanchao Lorthongpanich Siriraj Hospital, GMP-grade production of tIPE, a cell-based The study of spectroscopic signature of CIBERER-ISCIII, Madrid Mahidol University, Bangkok gene therapy product to treat neovascular human pluripotent stem cell-derived Analysis of the threshold of corrected cells Promotion effects of LPA on the osteogenic age-related macular degeneration (nvAMD) hepatocytes using synchroton FTIR required for the phenotypic correction of differentiation of human umbilical cord developed in the TargetAMD project P156 erythrocyte pyruvate kinase deficiency blood-derived mesenchymal stem cells P144 Tomas Kostelec BIA Separations, P122 P132 Ajdovšcina Michelle O’Doherty GSK, Stevenage Rapid high sensitivity detection of extra- Alicia Roig Merino DKFZ and NCT, Olga Gurvich Kuopio Center for Gene and Comparison of scale down model cellular vesicles by PATfix™ HPLC equipped Heidelberg Cell Therapy performance to at-scale to facilitate with multi-angle light scattering (MALS) A novel non-integrative and autonomously Analyzing effect of process changes on gene biopharmaceutical process development replicating DNA vector system for the expression in regulatory macrophages using P158 persistent genetic modification of stem RNAseq profiling P146 Keerang Park Chungbuk Health & Science cells and transgenesis Kazan Federal University P134 Albert Rizvanov University, Cheongwon-gun CELL THERAPY AND REPLACEMENT P124 Membrane vesicles as biocompatible vectors Development of advanced in vitro assays for Andrey Pulin Federal State Budgetary for bioactive molecules and drugs delivery

CELL THERAPY AND REPLACEMENT biological products to detect adventitious Ewa Janosz Hannover Medical School Scientific Institution “Institute of General CELL THERAPY AND REPLACEMENT

CELL THERAPY AND REPLACEMENT bovine viruses utilizing TCID50 and Pulmonary macrophage Pathology and Pathophysiology”, Moscow P148 next-generation sequencing transplantation-based therapy for Induction of myogenic differentiation in Albert Rizvanov Kazan Federal University alpha-1 antitrypsin deficiency spheroids from oral mucosa derived P160 Gene modification of fibroblasts with FGF2 mesenchymal stromal cells Pavel Makarevich Institute for Regenerative P126 increases the efficiency of the cell therapy of Medicine, Medical Research and Education P136 thermal skin burn in rabbits Ulf Geumann apceth Biopharma, Munich Center, Lomonosov Moscow State University Human mesenchymal stem cells genetically Hyunjeong Kim Asan Medical Center, Seoul P150 Decellularized extracellular matrix of human mesenchymal stromal cells as a novel engineered to express alpha-1 anti-trypsin Therapeutic effect of mesenchymal stem Ersin Akinci Akdeniz University, Department biomaterial for regenerative medicine (apceth-201) confer a long-term survival cells derived from the human umbilical cord of Enzyme and Microbial Biotechnology, benefit in lethal mouse models of in a rabbit temporomandibular joint model Antalya P164 graft-vs-host-disease of osteoarthritis Optimization of transfection conditions and Christine Baldeschi I-STEM, reagents to improve the transfection P138 Corbeil-Essonnes efficiency of dCas9-Activator plasmid into Albert Rizvanov Kazan Federal University human cells Differentiation of non-human primate Influence of mesenchymal stem cell-derived pluripotent stem cells into functional microvesicles on skin regeneration keratinocytes

142 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 143 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 2 / 18 OCT 12.40 - 14.40 POSTER SESSION 2 / 18 OCT 12.40 - 14.40

P166 P182 P194 P206 Kathrin Haake Hannover Medical School Ivana Trapani TIGEM, Naples Olivier Goureau Institute of Vision, Paris Akbar Farjadfar Fasa University of Medical Hematopoietic stem cell gene therapy Genome editing to generate a pig model of Characterization and transplantation of Sciences for Ifnyr1 deficiency protects mice from Stargardt disease type 1 CD73-positive photoreceptors isolated from Evaluation of gene therapy in spinal cord mycobacterial infections and paves way human iPS cell-derived retinal organoids injury using of neurotrophic factor BDNF P184 for macrophage transplantation therapy and regulation with miR-9/124 in SCI rat Alexander Smith University College London P196 model P168 Institute of Ophthalmology AGCTlab.org, Royal Rafael J. Yáñez-Muñoz P208 Ekaterina Naumenko Kazan Federal Development and efficacy assessment of Holloway, University of London University AAV2/8-hG1.7p.coCNGA3, a CNGA3 gene Induced pluripotent stem cell-based Albert Rizvanov Kazan Federal University Enchanced dark-field microscopy for therapy vector endothelial cell models of the human Genetically engineered umbilical cord blood histological detection of nanostructured P186 blood-brain barrier to screen gene and mononuclear cells for therapy of spinal cord scaffolds after implantation into bone other therapies for CNS entry injury in combination with epidural defects Steven Lee University of Ulsan stimulation Intravitreal injection of AAV expressing P198 P170 P210 soluble VEGF receptor-1 variant induces Kelly Glajch Biogen, Cambridge, MA anti-VEGF activity and suppresses choroidal Immacolata Brigida SR-TIGET, Milan AAV-GBA1 gene therapy for Parkinson’s João Brás University of Porto neovascularisation CELL THERAPY AND REPLACEMENT Gene therapy for adenosine deaminase 2 disease Exploring the role of miRNAs on microglia deficiency P188 activation P200 P172 Douglas Sproule AveXis, Bannockburn, IL P212 Zero incidence of adeno-associated virus Stefano Espinoza Istituto Italiano di Albert Rizvanov Kazan Federal University serotype 9 (AAV9) antibodies in a cohort Tecnologia (IIT), Genova Luis Pereira de Almeida CNC, University of Effect of genetic modification with reporter SINEUP for GDNF rescues motor deficits and Coimbra

of spinal muscular atrophy (SMA) type 1 CNS AND OCULAR DISEASES

genes on mesenchymal stem cell differential neurodegeneration in a mouse model of CNS AND OCULAR DISEASES Gene and cell therapy for brain CNS AND OCULAR DISEASES patients screened in STR1VE, a pivotal phase ability into osteogenic, adipogenic and 3 study of AVXS-101 Parkinson’s disease disorders: the case of the polyglutamine chondrogenic lineages Machado-Joseph disease P190 P202 P178 P214 Pengcheng Zhou Voyager Therapeutics, Konstantin Yarygin Institute of Biomedical Giridhar Murlidharan Voyager Cambridge, MA Chemistry, Moscow Ning Chang Ophthalmological Hospital, Therapeutics, Cambridge, MA Robust Huntingtin knockdown in cortex and Effects of mesenchymal stromal cells and Lausanne Intravenous administration of engineered putamen in large mammals using a novel neural progenitor cells derived from them by Developing gene therapy for FAM161A AAV gene therapy capsid demonstrates dosing paradigm with VY-HTT01, an AAV direct reprogramming in experimental associated retinitis pigmentosa in a murine improved CNS transduction in adult mice gene therapy targeting Huntington for the ischemic stroke model treatment of Huntington’s disease P180 P204 P216 P192 Valérie Vilmont EPFL, Lausanne Albert Rizvanov Kazan Federal University Sandra Johnen University Hospital RWTH

CNS AND OCULAR DISEASES An miRNA-based gene therapy approach to Andrew Grande University of Minnesota, Tubulation of rat’s sciatic nerve and Aachen target mutated SOD1 in key cell types in Minneapolis, MN stimulation of vascularisation using VEGF Genetic modification of freshly isolated amyotrophic lateral sclerosis (ALS) Viral reprogramming of reactive astrocytes and FGF2 encoding gene therapy plasmid primary human pigment epithelial cells to into neurons as a regenerative therapy in treat nvAMD stroke

144 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 145 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 2 / 18 OCT 12.40 - 14.40 POSTER SESSION 2 / 18 OCT 12.40 - 14.40

P218 P234 P250 P262 Albert Rizvanov Kazan Federal University Yvan Arsenijevic University of Lausanne Petros Patsali The Cyprus Institute of Gianluca Petris CIBIO, University of Trento Cytokine profile in blood serum and Towards high throughput drug screening for Neurology and Genetics, Thessaloniki Novel vector systems for transient delivery cerebrospinal fluid in human traumatic human retina organoids CRISPR/Cas9- and TALEN-mediated of CRISPR-nucleases spinal cord injury patients disruption of aberrant regulatory elements P236 restores normal splicing and gene function P264 P222 Maria Ines Almeida University of Porto Flash Therapeutics, P252 Pascale Bouillé Rui Nobre CNC, University of Coimbra Linking non-coding RNAs to osteogenic Toulouse

CNS AND OCULAR DISEASES Non-invasive allele-specific silencing therapy differentiation Mirella Lo Scrudato Genethon, UMR_S951 All-in-one delivery of gene-editing and biomarkers for Machado-Joseph disease P238 Inserm, Univ Evry, University Paris Saclay, system into primary cells and in vivo using EPHE LentiFlash®, a MS2-chimeric viral RNA P226 Gülen Güney Hacettepe University In vivo genomic deletion of expanded CTG delivery tool designed for clinical Diogo Mosqueira University of Nottingham Hematopoietic differentiation of induced repeats reduces pathological signs of applications pluripotent stem cells derived from patients myotonic dystrophy type 1 CRISPR/Cas9 gene editing in human with Griscelli syndrome type 2 P266 pluripotent stem cell-cardiomyocytes P254 provides a platform for modeling P240 Joost van Haasteren University of Oxford hypertrophic cardiomyopathy Valentina Vavassori SR-TIGET, Milan Demonstrating therapeutic applicability of Albert Rizvanov Kazan Federal University Optimization of a CRISPR/Cas9-based homology-independent targeted integration P228 MyoD-directed reprogramming of fibroblasts strategy for the correction of CD40LG gene (HITI) in a fluorescent reporter cell line with DYSF gene mutation for human Hans-Peter Kiem Fred Hutchinson Cancer in human haematopoietic stem cells and dysferlinopathy modeling T cells P268

Research Center, Seattle, WA GENE EDITING

P242 GENE EDITING Great Ormond Street Immunotoxin-based conditioning facilitates P256 Alessia Cavazza Institute of Child Health, University College autologous haematopoietic stem cell DISEASE MODELLING Capucine Trollet Inserm U974, Paris engraftment and multi-lineage development Janice Stricker-Shaver University of London Human skeletal muscle xenograft as a tool in a Fanconi anemia mouse model Tuebingen Haematopoietic stem cell gene editing for to assess transduction efficiency of AAV Silencing Huntingtin in the hypothalamus of the treatment of Wiskott-Aldrich syndrome serotypes P230 a transgenic Huntington disease rat model P270 Pakpoom Kheolamai Thammasat University P244 using AAV-mediated microRNA strategy DISEASE MODELLING Sara Regio LNTM, Lausanne University Interleukin-25 restores vessel-forming Olivier Humbert Fred Hutchinson Cancer P258 capacity of dysfunctional endothelial Research Center, Seattle, WA Hospital progenitor cells under high glucose Persistence of CRISPR/Cas9-edited Caitlin VanLith Mayo Clinic, Rochester, MN A new generation self-inactivating editing condition hematopoietic stem and progenitor cells In utero AAV-based genome-editing to cure system with improved delivery and reactivation of fetal hemoglobin in a mouse model of human hereditary P272 P232 nonhuman primates tyrosinemia type 1 FCV Centro de Investigación Paola Solanes EPFL, Lausanne Dunja Lukovic P246 P260 Príncipe Felipe , National Stem Cell Bank, Cas9/gRNA selective targeting of the Valencia Sylvain Perriot University Hospital, Paula Rio CIEMAT/CIBERER-ISCIII, Madrid Beethoven tmc-1 mutant allele for treating Human iPSC derived retinal organoids Lausanne NHEJ-mediated gene editing phenotypically progressive hearing loss by AAV-based display synaptic contacts and follow native Human induced pluripotent stem cell-derived corrects Fanconi anemia A patients’ delivery retina layer patterning astrocytes are differentially activated by haematopoietic stem and progenitor cells multiple sclerosis-associated cytokines

146 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 147 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 2 / 18 OCT 12.40 - 14.40 POSTER SESSION 2 / 18 OCT 12.40 - 14.40

P274 P288 P300 P318 Sumitava Dastidar VUB, Brussels David Gray University of California Los Els Verhoeyen CIRI; INSERM U1111; Lyon Margit Jeschke Novartis, Basel Reversal of spliceopathy in cardiomyocytes Angeles, CA Efficient genome editing in primary human Characterization of tisagenlecleucel, a CAR-T derived from myotonic dystrophy patient- Comparing methods of targeted integration T, B and HSCs using Baboon envelope gp cell product manufactured from patients specific iPS cells by gene editing with to correct Bruton’s tyrosine kinase defects pseudotyped virus derived “Nanoblades” with pediatric ALL CRISPR/Cas9 ribonuclear protein complexes loaded with Cas9/sgRNA ribonucleoproteins P290 P320 P276 P302

Omer Anakok East Anatolia High GENE EDITING Vania Lo Presti UMC Utrecht Steffen Panzner Lipocalyx, Halle Technology Application and Research Center Fernando Larcher Laguzzi CIEMAT/ Highly efficient multiplex genome editing Delivery of Cas/gRNA RNP using the Viromer (DAYTAM), Yakutiye, Erzurum CIBERER-ISCIII, Madrid and lentiviral transduction in cord blood platform New generation of the minimised UCOEs for Clinically-relevant correction of recessive derived CD8+ T cells; towards a novel P278 direct transgene expression from the innate dystrophic epidermolysis bullosa by dual cellular treatment for acute myeloid HNRPA2B1 promoter sgRNA CRISPR/Cas9-mediated gene editing leukemia relapse in pediatric patients Leigh Brody Celixir, Stratford-upon-Avon P306 A high-throughput deep sequencing P292 P322

approach for CRISPR off-target assessment Subashika Govindan EPFL, Lausanne Julia Reichelt EB House Austria, Salzburg Peter Ulrich Novartis, Basel in therapeutic genome editing applications Role of OCT4 in establishing and maintaining

TALEN-mediated inactivation of GENOMIC Consequences of maternal microchimerism chromatin architecture during stem cell P280 dominant-negative keratin alleles for upon CAR-T cell treatment self-renewal general phenotypic alleviation of OF CHARACTERISATION Kunwoo Lee GenEdit, Berkeley, CA STEM CELLS PLURIPOTENT Extension of the crRNA enhances Cpf1 gene epidermolytic ichthyosis P324 GENE EDITING P308 editing and in vivo delivery with polymer Novartis, Basel P294 Peter Ulrich nanoparticle Stephen Hyde University of Oxford GENE EDITING Analysis of lentivirus integration site P282 Kevin Holden Synthego, Menlo Park Antibody gene transfer for prophylaxis of distributions in CTL019 immunotherapy An optimized platform for efficient CRISPR respiratory syncytial virus (RSV) infection SuCheong Yeom Seoul National University P326

editing of iPSCs using synthetic sgRNA AND GENETIC VACCINES IMMUNOTHERAPY In vivo multiple gene targeting for P312 Mariya Tikhomirova Kazan Federal pancreatic cancer modeling with adeno P296 Deborah Gill University of Oxford associate virus and CRISPR/Cas9 University Enrico Mastrobattista Utrecht University Lung-targeted lentiviral vector mediates The comparative analysis of Treg-cells and P284 Cellular delivery of CRISPR/Cas9 passive immunisation against influenza transcription factor Foxp3 in lung diseases Oscar Quintana Bustamante CIEMAT/ ribonucleoproteins via biomimic lipid P314 P328 CIBERER-ISCIII, Madrid nanoparticles Catia Traversari MolMed, Milan Gene editing of PKLR gene in hematopoietic University Hospital P298 Razieh Monjezi cells for the efficient correction of pyruvate CD44v6 CAR-T cells display antitumor Würzburg activity against CD44v6+ human solid tumors kinase deficiency Alessia De Caneva ICGEB, Trieste CRISPR/Cas9 unites with Sleeping Beauty to P286 Coupling AAV-mediated promoterless gene P316 generate CAR-T cells with enhanced targeting to SaCas9 nuclease to efficiently therapeutic index for fighting against

Lorna FitzPatrick Manchester Metropolitan correct liver metabolic diseases AND GENETIC VACCINES IMMUNOTHERAPY Henk-Jan Prins Amsterdam UMC, Free immunosuppressive tumour University University of Amsterdam, Hematology, microenvironment CRISPR/Cas9-mediated genome edited Cancer Center Amsterdam human embryonic stem cells as in vitro Generation of universal “off-the-shelf” models of Batten disease chimeric antigen receptor (CAR)-engineered T cells 148 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 149 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 2 / 18 OCT 12.40 - 14.40 POSTER SESSION 2 / 18 OCT 12.40 - 14.40

P330 P342 P354 P374 Nerissa Kreher AVROBIO, Cambridge, MA Ralph Laufer Lysogene, Neuilly-sur-Seine Piotr Kopinski Howard Hughes Medical Michiko Tanaka Kyushu University, Fukuoka Gene therapy for Fabry disease AAV gene therapy LYS-SAF302 demonstrates Institute, Chevy Chase, MD Current status of clinical development for widespread sulfatase distribution in primate Mitochondrial DNA mutation causes nuclear gene therapy with Sendai virus vector P332 brain and correction of disease pathology in chromatin changes and makes glutamine a expressing the human FGF-2 gene substrate for histone acetylation (DVC1-0101) to treat peripheral arterial Andrea Calabria SR-TIGET, Milan MPSIIIA mice disease Characterization of hematopoietic system P344 P356 reconstitution in vivo in metachromatic Mukerrem Hale Tasyurek Akdeniz P376 leukodystrophy gene therapy patients Seemin Ahmed Homology Medicines, Bedford, MA University Center for Gene and Cell Therapy, Martina Marinello Genethon, P334 Durable correction of phenylketonuria in Antalya UMR_S951 Inserm, Univ Evry, University vivo following a single intravenous dose GLP-1 gene delivery stimulates cellular Paris Saclay, EPHE Maria Cristina Fillat IDIBAPS, Barcelona of AAVHSC15 packaging a human DISEASES AND LYSOSOMAL METABOLIC proliferation and trans-differentiation of Single stranded AAV-mediated gene therapy AAV9-GCDH gene therapy for glutaric phenylalanine hydroxylase transgene pancreatic cells in type 2 diabetes corrects spinal muscular atrophy in mice. aciduria type I P360 P346 P378 P336 Mahé Raccaud EPFL, Lausanne Kyle Chiang LogicBio Therapeutics, Foster Fulvio Mavilio Audentes Therapeutics, San Mitotic chromosome association predicts Randy Chandler National Institutes of City, CA Francisco, CA genome-wide transcription factor Health, Bethesda, MD Early dose response data with homologous Gene therapy for X-linked myotubular occupancy and impact on local chromatin Targeted integration of MUT into Albumin recombination-based genome editing myopathy with AT132 (rAAV8-Des-hMTM1): accessibility using a promoterless AAV vector approach using a promoterless AAV vector preliminary results from the ASPIRO (GeneRideTM) confers a hepatocellular in mice with methylmalonic acidemia P362 phase-1/2 study growth advantage in mice with P348 methylmalonic acidemia (MMA) Ilya Grigoryev Kazan Federal University P380 Julien Pichon UMR 703 INRA/Oniris, Nantes A thiacalixarene-modified polydiacetylene-

METABOLIC AND LYSOSOMAL DISEASES AND LYSOSOMAL METABOLIC based DNA sensor for potential application Ellie Crompton AGCTlab.org, Centre for P338 FoxO3a overexpression prevents both in gene therapy Gene and Cell Therapy, Centre for Biomedical METABOLIC AND LYSOSOMAL DISEASES AND LYSOSOMAL METABOLIC Marshall Huston Sangamo Therapeutics, glycogen overload and autophagic buildup MUSCLE, CARDIOVASCULAR AND PULMONARY DISEASES Sciences, School of Biological Sciences, Richmond, CA in Pompe disease P364 Royal Holloway, University of London Liver-based expression of the human P350 Ilya Grigoryev Kazan Federal University Integration-deficient lentiviral vectors for in alpha-galactosidase A gene in a murine Gold nanoparticles, capped by amphiphilic utero gene therapy in spinal muscular Hiu Man Grisch University Children’s Fabry model results in continuous amidoamino calixresorcinarenes as a atrophy Hospital Zurich therapeutic levels of enzyme activity potential non-viral vectors for gene delivery and effective substrate reduction Liver-directed gene therapy for CBS P382 P366 deficiency in mice using a minicircle-based Jozef Dulak Jagiellonian University, Krakow P340 naked DNA vector Angelina Titova Kazan Federal University Heme oxygenase-1 modulates the properties

Stuart Ellison University of Manchester P352 POTENTIAL MOLECULAR BASIS OF DEVELOPMENTAL CD133-positive cells in prenatal development of human induced pluripotent stem Pre-clinical safety and efficacy evaluation of of human pancreas cells-derived cardiomyocytes Markus Peters Agilis Biotherapeutics, haematopoietic stem cell gene therapy for Lyanfield, MA P368 MPSIIIA Estimated prevalence of aromatic l-amino Ilya Grigoryev Kazan Federal University acid decarboxylase (AADC) deficiency in the Amphiphilic octacationic calixresorcines as United States, European Union, and Japan non-toxic potential non-viral vectors for gene delivery 150 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 151 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 2 / 18 OCT 12.40 - 14.40 POSTER SESSION 2 / 18 OCT 12.40 - 14.40

P384 P396 P410 P426 Mark Yen Adverum Biotechnologies, Menlo Gabriele Ordazzo San Raffaele Scientific Tomas Kostelec BIA Separations, Alexandra Iouranova EPFL, Lausanne Park, CA Institute, Milan Ajdovšcina KRAB-ZFPs and transposable elements rewire Preliminary safety data from ADVANCE Novel gene therapy approaches for whole One-step monolith purification of oncolytic early human embryonic development clinical study (ADVM-043-01) in alpha 1 brain delivery of the lysosomal GCase Influenza virus produced in Vero cells transcriptional networks antitrypsin (A1AT) deficiency enzyme for wide protection from alpha-synuclein toxic aggregates P412 P428 P386 Inserm U1089, Nantes George Papanikolaou P398 Célia Couzinié Ioanna Vallianou Audrey Bourdon Nantes Gene Therapy Prevalence of AAV 1, 2, 3, 5, 6, 8, 9 and 10 Hospital, Thessaloniki Laboratory, UMR 1089, INSERM & University Chao-Liang Wu Department of neutralizing factors in non-human primates Efficient expansion of immunophenotypically of Nantes Biochemistry and Molecular Biology, primitive, genetically modified haematopoietic Impact of the inclusion of the Dystrophin College of Medicine, National Cheng Kung P414 stem cells derived from mobilized peripheral University, Tainan C-terminal domain in a micro-Dystrophin Marlene Rosa do Carmo GSK, Stevenage blood using small molecules transgene designed for gene therapy of shRNA-mediated knockdown of prothymosin HSC transduction protocol improvement Duchenne muscular dystrophy a ameliorates experimental colitis in mice P430 – RetroNectin or no RetroNectin, that is the Steve Pells ReNeuron, Bridgend P388 P402 question IPSCs derived from inducibly-immortalised Jérôme Poupiot Genethon, UMR_S951 Anna Baoutina National Measurement P416 adult stem cells as a source for clinical-scale Inserm, Univ Evry, University Paris Saclay, Institute, West Lindfield, NSW Caroline Odenwald Progen Biotechnik, manufacture of allogeneic cell therapies EPHE Improved analysis of transgenes in blood for OTHER Heidelberg Regulatory T cells and effector T cell P436 gene therapy and gene doping testing OTHER AAV titration ELISA for standardized gene exhaustion are essential to establish and delivery Conrad Vink GSK, Stevenage maintain liver-mediated immunological P404 Bacterial artificial chromosomes for rapid tolerance to muscle-expressed antigens

Omar Dabbous AveXis, Bannockburn, IL P418 AND LINEAGE SPECIFICATION AND CONTROL OF PURIPOTENCY REGULATION generation of high titre stable suspension P390 Early diagnosis and speed to effect in type 1 Maria Fe Medina McMaster University, producer cell lines for lentiviral vector spinal muscular atrophy (SMA1) Hamilton, ON manufacture Albert Rizvanov Kazan Federal University MUSCLE, CARDIOVASCULAR AND PULMONARY DISEASES Chromatography purification of adenoviral Transcriptional activation of mutant P406 P438 vectors dysferlin gene expression in human skin Alex Abadie Takara Bio Europe SAS, Kousaku Ohno University of California San fibroblasts using CRISPR/Cas9 SAM Saint-Germain-en-Laye P420 Francisco technology Novel human iPSC-derived hepatocytes with Matthew Smart Cell Therapy Catapult, Axonal transport of a modified AAV2 in the P392 advanced functionality and long-term 2D London non-human primate brain cultures of human primary hepatocytes for Automated analysis of quality attributes of Capucine Trollet Sorbonne University, Paris metabolic disease studies P440 pluripotent stem cell morphology in BB-301: a single “silence and replace” adherent and suspension culture Mark Shearman AGTC, Cambridge, MA

AAV-based vector for the treatment of P408 DEVELOPMENT VECTOR Comparable efficiency of photoreceptor oculopharyngeal muscular dystrophy Nick Veringmeier XENDO, Leiden P422 transduction by AAV2tYF, AAV5 and AAV8 in (OPMD) Planning for successful and efficient ATMP Nick Veringmeier XENDO, Leiden non-human primates development Strategic planning and regulatory landscape for the development of complex biologics

152 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 153 ESGCT / ISSCR / SFTCG ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 2018 COLLABORATIVE CONGRESS 2018

GARDEN FLOOR GARDEN FLOOR POSTER SESSION 2 / 18 OCT 12.40 - 14.40 POSTER SESSION 2 / 18 OCT 12.40 - 14.40

P442 P456 P468 P482 Jessika Ceiler DKFZ and NCT, Heidelberg Kristoffer Riecken University Medical Zsuzsanna Izsvak MDC, Berlin Chiara Crosta Anemocyte, Gerenzano Novel insights into wild-type and Centre Hamburg-Eppendorf, Hamburg While, no barrier insulator function is Analytical characterization of plasmid DNA recombinant AAV mitochondrial integration Efficient pseudotyping of different retroviral required for stable transgene expression for viral vector from Sleeping Beauty transposon, 6CTCF P444 vectors using a novel, codon-optimised gene for chimaeric GALV envelope enhancer blocker improves its biosafety P484 for therapeutical applications Pedro E Cruz University of Florida, University Hospital P458 Jihad EL Andari Gainsville, FL P470 Heidelberg Evaluating central nervous system promoters MoonKyung Kang Chungnam National Identification of new muscle-tropic using double strand adeno-associated virus Alisa Nousiainen A.I.Virtanen Institute, University, Daejeon adeno-associated virus (AAV) capsids for 6 triple mutants (T492V-Y705F-Y731F) virus University of Eastern Finland, Kuopio Human codon-optimization of Inclusion of HR arms into LVs improves treatment of rare hereditary muscular P446 GaLV-pseudotyped semi-retroviral rDNA-directed integration with an disorders replicating vector system improved genomic IN-I-PpoIN119A fusion protein Izabela Kraszewska Jagiellonian University, stability and anti-cancer activity P486 Krakow P472 Recruitment of MRN complex to the ITRs Juan Manuel Iglesias Gonzalez Synpromics P460 Maria Ontiveros Autonomous University of limits permissivity of the cells to scAAV Ltd. Barcelona transduction Tom Payne Oxford Genetics A novel genomics-based platform for the UPV: production of customized high Generation and characterisation of creation of synthetic promoters for cell and quality viral vectors at a joint UAB-VHIR P448 cGMP-compliant suspension HEK293-TetR gene therapy technological platform Adrian Westhaus Children’s Medical cell line for adenoviral vaccine manufacture Research Institute, Westmean, NSW P474 P488 The influence of AAV shuffled library P462

Friedrich Koch-Nolte University Medical DEVELOPMENT VECTOR Eduard Ayuso INSERM UMR1089, University platform design on the outcome of directed VECTOR DEVELOPMENT VECTOR Gloria Gonzalez-Aseguinolaza FIMA, Center Hamburg-Eppendorf of Nantes VECTOR DEVELOPMENT VECTOR evolution Pamplona DEVELOPMENT VECTOR Using nanobodies to target AAV vectors to MicroRNAs, overlooked contaminants of AAV P450 Analysis of the impact of NAbs on the specific cells vector batches transduction efficiency of the synthetic P476 Giridhara R Jayandharan Indian Institute hepatotropic AAV-Anc80 vector in P490 of Technology (IIT) Kanpur Albert Rizvanov Kazan Federal University non-human primates Anne-Kathrin Herrmann Heidelberg Deciphering the role of hepatic cell Construction of plasmid vectors encoding University Hospital, Cluster of Excellence microRNAome during AAV infection P464 pro-angiogenic factors for gene therapy CellNetworks, Heidelberg using picornoviral structural elements P452 Win Cheung REGENXBIO., Rockville, MD DNA family shuffling has no detrimental P478 Albert Rizvanov Kazan Federal University Relative infectivity as a reliable method for impact on the functionality of the Adenoviral-mediated lactoferrin gene quantifying differences in the infectivity of Yuanhong Li uniQure, Amsterdam assembly-activating protein (AAP) from AAV therapy for abscesses of the maxillofacial AAV vectors in vitro uniQure down stream purification process P492 area in rat model shows excellent viral clearance capabilities P466 Michela Milani SR-TIGET, Milan P454 P480 Qian Liu Oxford Genetics Liver-directed gene therapy in newborn mice Bas Bosma uniQure, Amsterdam Generation of cGMP-compliant stable Albert Rizvanov Kazan Federal University with lentiviral vectors Comprehensive comparative evaluation of packaging and producer cell lines for No evidence of ectopic bone formation in the qualitative attributes of AAV5 batches inducible lentiviral vector production soft tissues of rats injected with plasmid produced in mammalian and insect cells DNA encoding human VEGF165 and BMP2 cDNA 154 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy ESGCT / ISSCR / SFTCG Collaborative Congress 2018 155 NOTES Your Experts in GMP Manufacturing

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156 Changing the Face of Modern Medicine: Stem Cells & Gene Therapy NOTES

Promote cell and gene-based therapies Establish a forum Help decision makers

The Spanish Society for Gene and Cell Help decision makers Therapy (SETGyC) is the main forum evaluate technical and scientific that represents scientists, healthcare aspects of regulations that affect professionals and companies actively cell and gene-based therapies working in advanced therapies in Spain. We aim to: If you want to meet the most Promote research and development relevant experts in the basic and of cell and gene-based therapies clinical aspects of advanced therapies, join us! You will receive reduced rates Establish a forum for the exchange for conferences and events, qualify of information and to enhance the for travel grant applications and knowledge and application of novel much more! therapeutic strategies, both among dedicated professionals and to More info at www.setgyc.es society at large The European Society of Gene and Cell Therapy has, as its objective, the promotion of science and research. We achieve this in part through scientific and educational activities, in particular through measures aimed at the promotion and the exchange of information Spend your time on discovery, and ideas with regard to gene therapy, cell therapy, genetic vaccination, the encouragement of research fields, and clinical applications. As such we would not optimizing CRISPR. like to support the activities of national societies that share this goal – please see below for information and contact details of some national societies to Synthego’s Engineered Cells take the complexity out of CRISPR. help you, should you wish to get in touch with any of them. We’ll deliver your edit in your cell line with guaranteed results.

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[email protected] GERMANY [email protected] For further information and future events: For further information and future events:

UNITED KINGDOM www.bsgct.org www.dg-gt.de ONE-TIME DELIVERY. FRANCE

FINLAND BENEFITS FOR A LIFETIME. For further information, contact details [email protected] and future events: For further information and future events: www.fsgt.fi www.sftcg.fr Voyager’s mission is to develop and deliver life-changing gene therapies to people around the world living with severe neurological diseases. This begins with a disciplined commitment to discovery, research, development and manufacturing, and extends to our

GREECE thoughtful engagement with patients, healthcare [email protected] For further information, contact details For further information and future events: and future events: providers, caregivers and advocacy organizations.

www.generegther.gr/en/ THE NETHERLANDS www.nvgct.nl Voyager’s pipeline focuses on severe neurological diseases in need of effective new therapies, including Parkinson’s disease, a monogenic form of ALS called SOD1, Huntington’s disease, Friedreich’s ataxia, neurodegenerative diseases related to defective or excess aggregation of tau protein in the brain including Alzheimer’s disease and severe, chronic pain. SPAIN [email protected] Learn more at www.VoyagerTherapeutics.com For further information and future events: www.setgyc.es Autumn 2019: Leipzig Spring 2020: Hannover Spring 2021: Munich

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An exciting line-up of meetings with Yo I ur Go Vector or internationally renowned speakers and the latest developments in gene and cell therapy. Our Nanoplasmid™ platform Nanoplasmids are ideal for gene Keep an eye out for satellite sessions combines superior expression, and stem cell therapies, and in dedicated to early career researchers, duration and manufacturing yield viral vector manufacture. policy and public engagement. with antibiotic-free selection, improving regulatory compliance. Join us! Email: [email protected] Become a DG-GT member to receive reduced rates for conferences See us at the Congress Exhibit 58 Visit us on the web at www.natx.com Twitter: @DGGTeV and events, and much more. View our membership benefits at: Facebook: /DGGTeV www.dg-gt.de SPRING 2020 LYON

KEEP WATCHING FOR DETAILS – IN COLLABORATION WITH WWW.SFTCG.FR

The SFTCG wants to function as a platform to: 1. Encourage the communication between scientists in the field of gene and cellular therapy through the organization of its yearly meeting and exchange between labs in of students and novel technologies. 2. And provide and interface between scientist, clinicians and patients 3. Inform in a comprehensive and responsible way the general public about gene and cellular therapy in France and Europe

BECOME A MEMBER AND...

...you will benefit from heavily discounted and regulatory affairs, links to journals in fees for SFTCG events. In addition, you will the field and particularly to Human Gene have a unique chance to enter the “who is Therapy journal family. This will also be who” online database of gene and cellular a unique occasion to network with expert therapy labs in France. You will have full scientists and clinicians, to grow your access to job and grant proposals, but also professional network, expand business to up-to-date news and views on gene and opportunities, and advance your career cellular therapy programs, clinical trials in the field of gene and cell therapy. #CHANGINGMODERNMED WHAT, WHO, WHEN & WHERE, SPRINGAT YOUR 2020 FINGERTIPS... LYON

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...you will benefitHOW TO from DOWNLOAD heavily discounted andLOGIN regulatory affairs, links to journals in fees for SFTCGTHE events. APP In addition, you will theLogin field with and yourparticularly Badge ID to Human Gene have a unique• Option chance 1: to Simply enter scanthe “who the relevant is Therapy(printed journal underneath family. the This QR will also be who” online databaseQR code (see of gene left) and cellular a uniquecode on occasion your badge) to network with expert APP STORE: therapy labs• inOption France. 2: SearchYou will for have EventPilot full scientistsNEED HELPand clinicians, TO GET to grow your access to job Conferenceand grant proposals, App in the but App also or professionalSTARTED? network, expand business to up-to-dateGoogle news and Play views store on gene and opportunities,Once you have and downloaded advance your the career cellular therapyWhen programs, prompted clinicalenter the trials event in app,the field click theof gene banner and for cell your therapy. code: ChangingModernMed app guide to get started PROGRAMME BARCELONA 2019 CHANGING THE FACE ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS

OF MODERN MEDICINE: 16-19 OCTOBER 2018 ESGCT STEM CELLS & SWISSTECH LAUSANNE LAUSANNE 2018 ESGCT / ISSCR / SFTCG COLLABORATIVE CONGRESS 27 TH ANNUAL GENE THERAPY CONGRESS IN COLLABORATION WITH SETGyC

22-25 OCTOBER 2019 BARCELONA INTERNATIONAL CONVENTION CENTRE

XXVI ANNUAL CONGRESS 15th ANNUAL MEETING