2021 Rnatx Program & Abstract Book
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1 Dear Friends and Colleagues, Welcome to the RNA Therapeutics Institute’s third annual symposium, RNA Therapeutics: From Concept to Clinic, held virtually from the University of Massachusetts Medical School in Worcester, MA. What a year it has been for all of us, but especially for RNA! Throughout the latter half of the 20th century, RNA was thought mainly to intermediate between DNA and protein. Over the last three decades, that view has given way to the understanding that RNA plays rich and complex roles previously ascribed only to proteins. From ncRNAs to RNAi, from CRISPR to circular RNAs, we have witnessed astonishing discoveries and equally amazing translation of these breakthroughs to therapeutics and now mRNA vaccines. We are delighted to feature speakers whose fascination with RNA biology has inspired them to explore the possibilities of harnessing RNA to create therapeutics. This exceptional virtual experience will keep with our tradition of providing high-quality scientific sessions, an exhibit hall that will offer one-on-one opportunities to meet with our business partners, and social events to allow attendees to mingle, catch up with old friends, and forge new collaborations. It also promises two dynamic and inspiring keynote addresses, as well as a fun party that we know you will enjoy - all on a digital platform. To stimulate dialogue, we’ve included time in the program for discussion during our welcome reception on Wednesday afternoon, after each talk, as well as during the poster sessions, coffee networking breaks, and our RNATx party on Thursday afternoon. The scientific sessions and posters, drawn from our international call for abstracts, offer a taste of the newest work in the field and provide new opportunities to forge collaborations with current and future thought-leaders in the RNA community. We would like to thank everyone who has worked diligently to create this symposium, particularly the talented staff at the RTI. We especially thank our speakers for their enthusiastic participation. Finally, we are most grateful to our sponsors and exhibitors for their generous support. Sincerely, Phillip D. Zamore, PhD Angela Messmer-Blust, PhD RNA Therapeutics Institute UMass Medical School Symposium Co-Organizers 2 2021 RNA Therapeutics: From Concept to Clinic Contents: • Virtual Venue…………..………….………………………………………….……………….……4 • Sponsors……………………………….………………………………….……………….……….6 • Oral Program…………………………….…………………………………….…………….……10 • On-Demand Short Talk Abstracts……….………………………………………….….………..12 • Live Short Talk Abstracts…..………………………………….………….……………...………20 • Poster Program………………………………………………………………….………………..25 • Keynote & Invited Speaker Biographies…………………………………………………….….31 • Panelist & Organizer Biographies………………..………………………………….………….41 • Symposium Survey…………..………………………………….………….……………………44 • Poster Abstracts…………..………………………………….………….………..………..…….45 ___________________________________________________________________________ Cover Image: This image of the mouse cerebellum in a murine model of ALS (C9ORF BAC) shows selective uptake of antisense oligonucleotides (ASOs; green) into purkinji neurons at the interface of grey and white matter. Nuclei are shown in blue. Image by Dr. Michael Moazami, Jonathan Watts Lab, RTI, UMass Medical School. 3 Virtual Venue VENUE: RNATx2021.vfairs.com POSTERS & NETWORKING: https://spatial.chat/s/rnatx21 Tutorial & Info on Spatial Chat REGISTRATION: You will receive an email on how to activate your account for the virtual platform. Please email [email protected] if you have not received or are having trouble logging in. We recommend logging in a day prior to ensure connectivity. TECHNICAL SUPPORT: For technical support during the symposium [email protected] or call 508-380-6077. CAREER WORKSHOP, EXHIBITORS, POSTER & SCIENTIFIC SESSIONS INFORMATION Session URL Careers Under the Microscope Workshop (pre-registration required) RNATx2021.vfairs.com Wednesday, June 23rd Enter Auditorium (if registered) Keynote Sessions: Michael Rosbash and Kathy High RNATx2021.vfairs.com Wednesday, June 23rd and Friday June 25th Enter Auditorium Welcome Reception, RNATx & Sirnaomics Lobbies, sponsored by Sirnaomics https://spatial.chat/s/rnatx21 Wednesday, June 23rd Scientific Sessions RNATx2021.vfairs.com Wednesday - Friday, June 23-25th Enter Auditorium RNATx 2021 Virtual Party sponsored by ChemGenes GO REMOTE LINK Thursday, June 24th (TURN OFF YOUR VPN) Poster Sessions sponsored by Agilent Wednesday - Friday, June 23-25th h"ps://spa(al.chat/s/rnatx21 Spatial chat poster session info Exhibitors RNATx2021.vfairs.com Wednesday - Friday, June 23-25th Enter Exhibit Hall Coffee & Networking Breaks, sponsored by TriLINK h"ps://spa(al.chat/s/rnatx21 Wednesday - Friday, June 23-25th 2021 RNATx VIRTUAL PARTY Please join the RNATx party to play an interactive Game Show sponsored by ChemGenes. Teams will play against each other in mini-games of trivia, puzzles, Pictionary, and more! Who will be the ultimate RNATx Game Show Champion? EXHIBITORS We are very happy to welcome our exhibitors who kindly support this meeting. Please take the time to visit them during the networking breaks and poster sessions. 4 POSTER SESSIONS Poster Numbers Session Date/Time VOTE for the Note best poster Wednesday, June 23rd These presentation times are for a Posters: 1 - 42 & 46 Session 1 * 3.30 - 4.15pm Q&A session via Spatial Chat. Thursday, June 24th Posters: 43 - 86 Session 2 * The poster pdfs & 2 minute videos 1.15 - 2.00pm that you uploaded will stay up for the entirety of the meeting. Posters: 87 - 125 Friday, June 25th Session 3 1.15 - 2.00pm *NOTE: All posters will stay up for the entire symposium. The presentation times refer to the Q&A sessions in Spatial Chat only. SPEAKERS All oral presenters are reminded to be in their respective sessions no later than 30 minutes before the start of the session in order to meet with the session chair and to finalize their setup of presentation visuals. You will receive ALL links to your email via Zoom AND in a calendar invite. SOCIAL MEDIA Official symposium hashtag: #RNATx2021 Symposium website: www.RNATx2021.vfairs.com Twitter Account: @UMassMedRTI The organizers encourage attendees to tweet about the remarkable science that they experience at the meeting. Please respect the following rules in your posts and tweets: 1. Be polite and respectful of others 2. Be respectful of those presenters that ask attendees to refrain from tweeting content from talks and posters. 3. If you’re enjoying the conference, share it with your social media network! 4. Never post/tweet about unpublished data, and look for these icons on the speakers’ slides: Ok to screenshot, Not ok to screenshot, photograph, photograph, and/or share: and/or share: CONFERENCE HARASSMENT POLICY 2021 RNATx is dedicated to providing a harassment-free conference experience for everyone, regardless of gender, gender identity & expression, sexual orientation, disability, physical appearance, body size, race, age, or religion. We do not tolerate harassment of conference participants in any form. Conference participants violating these rules may be expelled from the conference (without a refund) at the discretion of the conference organizers. Our anti-harassment policy and how to report any violations can be found at: https://bit.ly/30AMkQz 5 With Gratitude to our Sponsors GOLD SPONSORS 6 SILVER SPONSORS 7 BRONZE SPONSORS ® SUPPORTERS 8 EXHIBITORS Eclipse GUAG CG A CU A U UC U GA U U AC G AC C C UA GU GA C BioInnovations C Sanderson Center for Optical Experimentation 9 ORAL PROGRAM Wednesday, June 23, 2021 (times listed in ET) 1.00 – 2.30pm Careers Under the Microscope Workshop (pre-registration required; separate agenda) 2.40 – 2.45pm Welcome and Keynote introduction, Phillip D. Zamore, HHMI, RNA Therapeutics Institute, UMass Medical School Keynote: Michael Rosbash, Brandeis University 2.45 – 3.30pm More than 50 years of RNA: Plus ça change... Virtual Poster Session and Exhibit Hall- VOTE for the best poster 3.30 – 4.15pm Poster Session Sponsored by Agilent Technologies RNATx Kickoff, Virtual Happy Hour via Spatial Chat Lobby, 4.15 – 5.00pm Sponsored by Sirnaomics Progr Thursday, June 24, 2021 8.55 – 9.25am Virtual Coffee & Networking, Spatial Chat Lobby Session I: Editing Session Chair: Xiaolong Dong, RTI, UMass Medical School 9.25 – 9.30am Welcome and Introduction, Phillip D. Zamore Genome editing and single cell approaches to study early human development 9.30 – 9.55am Kathy Niakan, Crick Institute In vivo gene editing in muscles and muscle stem cells 10.00 – 10.25am Amy Wagers, Harvard University 10.30 – 10.55am Development of gene-drives and a method to block them in A. gambiae Chrysanthi Taxiarchi, Imperial College of London Site-directed RNA editing: current strategies and future directions 11.00 – 11.25am Joshua Rosenthal, Marine Biological Laboratory ON DEMAND Short Talks from abstracts – Vote for your favorite talk within the conference platform! Virtual Networking & Coffee Break, Spatial Chat Lobby 11:30 – 12:00pm Sponsored by TriLink BioTechnologies Session II: RNAs in Immunity Session Chair: Kexin Zhang, RTI, UMass Medical School Innate Immune nucleic acid sensing pathways 12.15 – 12.40pm Kate Fitzgerald, UMass Medical School N6-Methyladenosine in Immunity 12.45 – 1.10pm Ye Grace Chen, Yale University ON DEMAND Short Talks from abstracts – Vote for your favorite talk within the conference platform! Virtual Poster Session and Exhibit Hall - VOTE for the best poster 1.15 – 2.00pm Poster Session Sponsored by Agilent Technologies