Centric Registry for Strimvelis in ADA-SCID
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CADTH ISSUES in EMERGING HEALTH TECHNOLOGIES Informing Decisions About New Health Technologies
CADTH ISSUES IN EMERGING HEALTH TECHNOLOGIES Informing Decisions About New Health Technologies Issue Mar 171 2018 Gene Therapy: An Overview of Approved and Pipeline Technologies CADTH ISSUES IN EMERGING HEALTH TECHNOLOGIES 1 Authors: Alison Sinclair, Saadul Islam, Sarah Jones Cite as: Gene therapy: an overview of approved and pipeline technologies. Ottawa: CADTH; 2018 Mar. (CADTH issues in emerging health technologies; issue 171). Acknowledgments: Louis de Léséleuc, Jeff Mason, Teo Quay, Joanne Kim, Lesley Dunfield, Eftyhia Helis, Iryna Magega, Jane Hurge ISSN: 1488-6324 (online) Disclaimer: The information in this document is intended to help Canadian health care decision-makers, health care professionals, health systems leaders, and policy- makers make well-informed decisions and thereby improve the quality of health care services. While patients and others may access this document, the document is made available for informational purposes only and no representations or warranties are made with respect to its fitness for any particular purpose. The information in this document should not be used as a substitute for professional medical advice or as a substitute for the application of clinical judgment in respect of the care of a particular patient or other professional judgment in any decision-making process. The Canadian Agency for Drugs and Technologies in Health (CADTH) does not endorse any information, drugs, therapies, treatments, products, processes, or services. While CADTH has taken care to ensure that the information prepared by it in this document is accurate, complete, and up-to-date as at the applicable date the material was first published by CADTH, CADTH does not make any guarantees to that effect. -
Twenty-Five Years of Gene Therapy for ADA-SCID: from Bubble Babies to an Approved Drug
Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug Francesca Ferrua1,2 and Alessandro Aiuti1,2,* 1San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy;2Vita-Salute San Raffaele University, Milan, Italy. Twenty-five years have passed since first attempts of gene therapy (GT) in children affected by severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) defect, also known by the general public as bubble babies. ADA-SCID is fatal early in life if untreated. Unconditioned hematopoietic stem cell (HSC) transplant from matched sibling donor represents a curative treatment but is available for few patients. Enzyme replacement therapy can be life-saving, but its chronic use has many drawbacks. This review summarizes the history of ADA-SCID GT over the last 25 years, starting from first pioneering studies in the early 1990s using gamma-retroviral vectors, based on multiple infusions of genetically corrected autologous peripheral blood lymphocytes. HSC represented the ideal target for gene correction to guarantee production of engineered multi-lineage progeny, but it required a decade to achieve thera- peutic benefit with this approach. Introduction of low-intensity conditioning represented a crucial step in achieving stable gene-corrected HSC engraftment and therapeutic levels of ADA-expressing cells. Recent clinical trials demonstrated that gamma-retroviral GT for ADA-SCID has a favorable safety profile and is effective in restoring normal purine metabolism and immune functions in patients >13 years after treatment. No abnormal clonal proliferation or leukemia development have been observed in >40 patients treated experimentally in five different centers worldwide. -
Introducing FSI-174 – Anti-Ckit Antibody 22 January 2019 This Presentation Contains Forward-Looking Statements That Involve Substantial Risks and Uncertainties
NASDAQ: FTSV Helping Patients Defeat Their Cancer KOL Breakfast to Discuss Forty Seven´s Expanding Pipeline: Introducing FSI-174 – Anti-cKIT Antibody 22 January 2019 This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements other than statements of historical facts contained in this presentation, including statements regarding our future financial condition, results of operations, business strategy and plans, and objectives of management for future operations, as well as statements regarding industry trends, are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potentially” “predict,” “should,” “will” or the negative of these terms or other similar expressions. We have based these forward-looking statements largely on our current expectations and projections about future events and trends that we believe may affect our financial condition, results of operations, business strategy and financial needs. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including, among other things: the success, cost and timing of our product development activities and clinical trials; our expectations about the timing of achieving regulatory approval and the cost of our development programs; our ability to obtain funding for our operations, including funding necessary to complete further development -
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R&D Day NOVEMBER 2020 Disclaimer This presentation has been prepared by AVROBIO, Inc. trials and gene therapy programs; the expected safety profile of will not materialize as expected; risks associated with our (“AVROBIO”) for informational purposes only and not for any other our investigational gene therapies; the potential impact of the dependence on third-party suppliers and manufacturers; risks purpose. Certain information contained in this presentation and COVID-19 outbreak on our clinical trial programs and business regarding the accuracy of our estimates of expenses and future statements made orally during this presentation relate to or are generally, as well as our plans and expectations with respect to revenue; risks relating to our capital requirements and needs for based on studies, publications, surveys and other data obtained the timing and resumption of any development activities that may additional financing; risks relating to clinical trial and business from third-party sources and AVROBIO’s own internal estimates be temporarily paused as a result of the COVID-19 outbreak; and interruptions resulting from the COVID-19 outbreak or similar and research. While AVROBIO believes these third-party sources the market opportunity for and anticipated commercial activities public health crises, including that such interruptions may to be reliable as of the date of this presentation, it has not relating to our investigational gene therapies;. Any such materially delay our development timeline and/or increase our independently verified, and AVROBIO makes no representation as statements in this presentation that are not statements of historical development costs or that data collection efforts may be impaired to the adequacy, fairness, accuracy or completeness of any fact may be deemed to be forward-looking statements. -
Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: a Journey from Mouse Cage to Bed Side
pharmaceutics Review Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side Laura Garcia-Perez 1 , Anita Ordas 1 , Kirsten Canté-Barrett 1 , Pauline Meij 2, Karin Pike-Overzet 1, Arjan Lankester 3 and Frank J. T. Staal 1,* 1 Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, 2333ZA Leiden, The Netherlands; [email protected] (L.G.-P.); [email protected] (A.O.); [email protected] (K.C.-B.); [email protected] (K.P.-O.) 2 Department of Clinical Pharmacy and Toxicology, Leiden University Medical Center, 2333ZA Leiden, The Netherlands; [email protected] 3 Willem-Alexander Children’s Hospital, Leiden University Medical Center, 2333ZA Leiden, The Netherlands; [email protected] * Correspondence: [email protected] Received: 30 April 2020; Accepted: 9 June 2020; Published: 13 June 2020 Abstract: Recent clinical trials using patient’s own corrected hematopoietic stem cells (HSCs), such as for primary immunodeficiencies (Adenosine deaminase (ADA) deficiency, X-linked Severe Combined Immunodeficiency (SCID), X-linked chronic granulomatous disease (CGD), Wiskott–Aldrich Syndrome (WAS)), have yielded promising results in the clinic; endorsing gene therapy to become standard therapy for a number of diseases. However, the journey to achieve such a successful therapy is not easy, and several challenges have to be overcome. In this review, we will address several different challenges in the development of gene therapy for immune deficiencies using our own experience with Recombinase-activating gene 1 (RAG1) SCID as an example. We will discuss product development (targeting of the therapeutic cells and choice of a suitable vector and delivery method), the proof-of-concept (in vitro and in vivo efficacy, toxicology, and safety), and the final release steps to the clinic (scaling up, good manufacturing practice (GMP) procedures/protocols and regulatory hurdles). -
EFPIA Pipeline Review 2021 Update
EFPIA Pipeline Review 2021 Update Project Report February 2021 Disclaimer • Information contained within this document is based on publicly available information and Primary Market Research • Findings and recommendations are based on the views of IQVIA, and do not necessarily represent those of EFPIA • Please note: the research to inform this report was conducted between August and Nov 2020 © 2020. All rights reserved. IQVIA® is a registered trademark of IQVIA Inc. in the United States, the European Union, and various other countries. This document contains a pipeline overview, deep dives of the eight areas, as well as considerations for innovation access Hyperlinks for Content Navigation Pipeline overview Benefit of Innovation From Innovation to Access 1 IQVIA_EFPIA Pipeline Review 2021 - Full Report Hyperlinks to detailed content allow for easy reference and navigation of the document Slides Slides numbers numbers Pipeline overview 8-22 4. Adapting Financing Models……………………………………... 194-201 5. Developing infrastructure to support care delivery……..……202-209 Benefits of Innovation …………………………………………. 23-24 6. Optimising patient management/treatment strategies………..210-216 1. NSCLC Checkpoint Inhibitor Combinations…………………..... 25-39 7. Enabling data science and technology partnerships………….217-232 2. Disease Modifying Therapies for Alzheimer’s Disease…… …40-58 8. Horizon scanning and stakeholder dialogue………………….…233-235 3. Gene Therapies ….… 59-70 4. Cell Therapies (including CAR-Ts) 71-86 5. PPAR / FXR Agonists for NASH…………………………………… 87-102 Appendix…………………………………………………………........236-318 6. Remyelinating CNS Therapies …………… …103-120 Pipeline product details 7. mRNA vaccines for Glioblastoma ………………………………121-130 Glossary 8. Curative therapies for HBV and HIV 131-154 Project methodology Pipeline overview details From Innovation to Access…………… . -
The 45Th Annual Meeting of the European Society for Blood and Marrow Transplantation: Physicians – Oral Session
Bone Marrow Transplantation (2019) 54:16–141 https://doi.org/10.1038/s41409-019-0562-9 MEETING ABSTRACTS The 45th Annual Meeting of the European Society for Blood and Marrow Transplantation: Physicians – Oral Session © Springer Nature Limited 2019 24–27 March 2019 ● Frankfurt, Germany Modified and published with permission from https://www.ebmt.org/annual-meeting Sponsorship Statement: Publication of this supplement is sponsored by the European Society for Blood and Marrow Transplantation. All content was reviewed and approved by the EBMT Committee, which held full responsibility for the abstract selections. Presenting author names are bold type in the contributor lists. 1234567890();,: 1234567890();,: Acute leukaemia Methods: Study aim was to evaluate the schedule of IST given in combination with PT-Cy as GVHD-prophylaxis O010 post-haplo for acute leukemia (AL) and reported to the The impact of the starting day of graft-versus-host ALWP/EBMT registry. Patients were divided into 3 groups: disease prophylaxis on haploidentical transplantation received cyclosporine A-mycofenolate-mofetil(CSA- with post-transplant cyclophosphamide: A retrospective +MMF) initiated at day+1 (group-1, n=124) or CSA study on behalf of acute leukaemia working party- +MMF both started at day+5 (group-2, n=170) and EBMT tacrolimus + MMF from day+5 (group-3, n=215). Transplants were performed from 2006-2017 and median Annalisa Ruggeri1, Myriam Labopin2, Johanna Tischer3, follow up is 21 months (range 11-36). PT-CY was given on Jean - Luis Diez Martin4, Benedetto Bruno5, Simona Day+3 and Day+5 in group-1 and on day+3 and day+4in Sica6, Luca Castagna7, Boris Afanasyev8, Antonin Vitek9, group-2 and 3. -
Gene Therapy Using Haematopoietic Stem and Progenitor Cells
REVIEWS Gene therapy using haematopoietic stem and progenitor cells Giuliana Ferrari1,2, Adrian J. Thrasher3,4 and Alessandro Aiuti 1,2,5 ✉ Abstract | Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective treatment modality for monogenic disorders of the blood system such as primary immunodeficiencies and β- thalassaemia. Medicinal products based on autologous HSPCs corrected using lentiviral and gammaretroviral vectors have now been approved for clinical use, and the site- specific genome modification of HSPCs using gene editing techniques such as CRISPR–Cas9 has shown great clinical promise. Preclinical studies have shown engineered HSPCs could also be used to cross- correct non-haematopoietic cells in neurodegenerative metabolic diseases. Here, we review the most recent advances in HSPC gene therapy and discuss emerging strategies for using HSPC gene therapy for a range of diseases. 7 Allogeneic Haematopoietic stem cell (HSC) transplantation copies of a therapeutic gene . Once reinfused, genetically Relating to or denoting that (HSCT) has been a routine procedure for treating inborn modified HSPCs undergo self- renewal and establish a the source of cells, tissues or errors of metabolism and the blood system for more population of modified cells, which pass the transgene organs for transplant is from an than 50 years1,2. The first successful transplantations for to daughter blood cells on differentiation (Fig. 1). The individual genetically different from the recipient. the treatment of immune disorders were conducted in first proof- of- concept HSPC gene therapy studies were 1968 using allogeneic stem cells to treat X- linked severe conducted in the 1990s to address adenosine deami- Primary immunodeficiencies combined immunodeficiency (SCID- X1), an inherited nase (ADA) deficiency SCID (ADA- SCID); although (PIDs).