2021 Pre-Meeting Workshops: May 10, 2021
All meeting content is available through August 13 on the meeting platform and on ASGCT.org.
Registration for the Pre-Meeting Workshops is separate from registration for the Annual Meeting. For more information on Pre-Meeting Workshop registration rates, visit the Registration page.
Register for a Pre-Meeting Workshop
Morning Workshops
The morning Pre-Meeting Workshops will be held from 10 a.m.-2 p.m. ET (7-11 a.m. PT, 8 a.m.-12 p.m. MT, 9 a.m.-1 p.m. CT)
Planned by the Patient Outreach Committee
For individuals with genetic disorders, their families, and other advocates, this workshop will address topics such as the clinical trials process; the role of patient advocates in fostering gene therapy research; the development of effective partnerships; and challenges and opportunities in gene therapy development.
You may be eligible for free registration for this workshop if you are an individual with a rare disease, the caregiver of a person with a rare disease, or a volunteer advocate.
Introduction: Overview of gene therapy, research, and development
- Kevin Flanigan, M.D., Director, Center for Gene Therapy, Nationwide Children's Hospital
Session I: Lessons Learned from Initiating Gene Therapy Research
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Gina Hann, Co-Founder, The Rare Village Foundation, President & Founder, Foundation for Batten Hope
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Christine Waggoner, President, Cure GM1 Foundation
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Sue Wilson, President, The Children's Medical Research Foundation, Inc.
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Amber Olsen, Executive Director & Founder, United MSD Foundation
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Matt Wilsey, President & Co-Founder, Grace Science Foundation
Session II: Understanding the Research Process: Being Equipped for Success
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Steven Gray, Ph.D., Associate Professor, UT Southwestern
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Jill Wood, Chief Financial Officer, Phoenix Nest Biotech
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Michelle Berg, President, GMP Nucleic Acids, Aldevron
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P.J. Brooks, Ph.D., Program Director, Office of Rare Disease Research, NCATS
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Planned by the Regulatory Affairs Committee in collaboration with the European Society of Gene and Cell Therapy
This workshop will include discussion of progress toward convergence in regulatory practices in gene therapy development between the U.S. and EU, as well as the use of reliance on established regulatory frameworks by regulators globally. Speakers will focus on recent efforts to address GMO requirements in EU member states, and companies will share their experiences in developing across multiple regions.
Session I: Regulatory Convergence in GMO Requirements and Environmental Risk Assessment
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Alessandro Aiuti, M.D., Ph.D., Deputy Director of Clinical Research, San Raffaele Telethon Institute
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Stuart Beattie, Ph.D., Clinical Lead, Gene Therapy Regulatory CMC, Biogen
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Gentaro Tajima, M.S., Japan Regulatory Lead, Pfizer
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Michael Havert, Ph.D., Senior Director of Regulatory CMC, bluebird bio
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Tomáš Boráň, M.D., Head of Marketing Authorisation Branch, State Institute for Drug Control (SUKL), Czech Republic
Session II: Hot Topics and Emerging Trends in the Regulation of Gene Therapies
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Wilson Bryan, M.D., Director of Office of Tissues and Advanced Therapies, FDA
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Martina Schüssler-Lenz, M.D., Deputy Head of the Advanced Therapy Medicinal Products Section at the Paul-Ehrlich Institute
Session III: Industry Experience in the Global Development of Gene Therapies
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Dan Takefman, Ph.D., Principal, Takefman Gene Therapy Advisors LLC
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Keith Wonnacott, Ph.D., Executive Director of Regulatory Affairs, Pfizer
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Michael Ladd, PharmD., VP Regulatory Science, Orchard Therapeutics
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Katie Picone, PharmD., Senior Global Program Regulatory Director, Novartis
Session IV: Use of Regulatory Reliance for Regenerative Medicines/Advanced Therapies
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Lawrence Liberti, Ph.D., Head, Regulatory Collaborations, Centre for Innovation in Regulatory Science
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Murray Lumpkin, M.D., Deputy Director, Regulatory Affairs, Lead Global Regulatory Systems Initiatives, The Bill and Melinda Gates Foundation
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Michael Shum, Director, Application and Advisory Management, Prescription Medicines Authorisation Branch, Australian Therapeutic Goods Administration
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The Oncolytic Viruses workshop will feature overviews of the recent advances in oncolytics, including oncolytic viruses, cancer vaccines, and novel combination immuno-oncologic therapies.
Session I: Successes and Challenges
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Gregory Friedmann, M.D., University of Alabama at Birmingham
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E. Antonio Chiocca, M.D., Ph.D., Brigham and Women’s/Faulkner Hospital
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Joseph Jackson, M.S., Ph.D., University of Pittsburgh
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Parker Dryja, University of New Mexico
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Ji Young Yoo, Ph.D., University of Texas, McGovern Medical School
Session II: In Pursuit of Synergy
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Jacob van Vloten, Ph.D., Mayo Clinic
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Laura Elvgin, Ph.D., University of British Columbia
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Maria Eugenia Davola, Ph.D., McMaster University
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John Christie, Arizona State University
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Bangxing Hong, Ph.D., University of Texas, Health Science Center
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The Gene Editing workshop will provide an overview of current gene editing technologies and approaches, emerging uses and applications, with sessions highlighting junior investigators and clinical gene editing programs.
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Shengdar Tsai, Ph.D., St. Jude Children’s Hospital
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Ben Kleinstiver, Ph.D., Massachusetts General Hospital
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Alexander Marson, M.D., Ph.D., University of California, San Francisco
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Deepak Reyon, Ph.D., Editas Medicine
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Hari Jayaram, Ph.D., Spotlight Therapeutics
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Nicole Gaudelli, Ph.D., Beam Therapeutics
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Anna Kwilas, Ph.D., FDA
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Daniel O’Connell, Ph.D., Intellia Therapeutics
Panel – Regulatory Approach to Genome Editing for Clinic
Moderator – Fyodor Urnov, Ph.D., University of California, Berkeley
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Daniel O’Connell, Ph.D., Intellia Therapeutics
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Anna Kwilas, Ph.D., FDA
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Deepak Reyon, Ph.D., Editas Medicine
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Nicole Gaudelli, Ph.D., Beam Therapeutics
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Planned by the Bio-Industry Committee
This workshop will explore the practical aspects of working in the biopharma industry including getting that first job, what to expect in industry, and how to thrive and develop your career in the industry’s matrix environment. The workshop is case-based with each presenter recounting their personal journey with key insights gleaned that can be directly applied to your career development needs. The workshop will be hyperinteractive, with the majority of time set aside for Q&A and personal perspective sharing.
Session I: Working & Thriving in Industry - What You Need to Know to Prepare and Succeed
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Steven Gray, Ph.D., Associate Professor, UT Southwestern
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Gallia Levy, M.D., Ph.D., Chief Medical Officer, Spark Therapeutics
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Kamal Bharucha, M.D., Vice President, Clinical Development, BridgeBio
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Adora Ndu, PharmD, J.D., Vice President, Regulatory Affairs, Global Head of Policy, Research, Engagement & International, BioMarin
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Dan Takefman, Ph.D., Principal, Takefman Gene Therapy Advisors LLC
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Nisha Jain, M.D., Takeda Pharmaceuticals
Session II: Learning from Experience - Case Studies of Transitions from Academia to Industry
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Federico Mingozzi, Ph.D., Chief Scientific Officer, Spark Therapeutics
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Kevin Williams, M.D., J.D., Chief Medical Officer, Pfizer Rare Disease
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Leonard Valentino, M.D., Chief Executive Officer, National Hemophilia Foundation
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Katherine Beaverson, M.S., Senior Director, Patient Advocacy Lead, Pfizer Rare Disease
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Fred Porter, Ph.D., Chief Technical Officer, Taysha Gene Therapies
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Chat Lounge Networking
The Chat Lounge break will be held 2-3 p.m. ET (11 a.m.-12 p.m. PT, 12-1 p.m. MT, 1-2 p.m. CT)
Afternoon Workshops
The afternoon Pre-Meeting Workshops will be held 3-7 p.m. ET (12-4 p.m. PT, 1-5 p.m. MT, 2-6 p.m. CT)
Planned by the Government Relations Committee
This workshop will address how key stakeholders impact development of, and patient access to, gene and cell therapies. Perspectives from investors, payers, policymakers and patients on the growth of the industry, learnings from initial gene and cell therapy launches, and potential solutions to facilitate access will be explored. In addition, the workshop will delve into recent policy changes that impact our industry.
Keynote: Attracting Capital and Building a Company in the Gene Therapy Space
- R.A. Session II, President, CEO & Founder, Taysha Gene Therapies
Session I: Investor State of Play
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Michael Gladstone, Partner, Atlas Venture
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Ritu Baral, Managing Director, Health Care-Biotechnology, Cowen
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Craig Gordon, M.D., Partner & Investment Analyst, Capital Group
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Aaron Schwimmer, Managing Director, Barclays Capital
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Josh Schimmer, M.D., Senior Managing Director, Biotech, Evercore ISI
Session II: What's New in Federal Health Policy?
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Conor Sheehy, Deputy Legislative Director, Senator Tim Scott
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Greg Mathis, Jr., Health Policy Advisor, Senator Mark R. Warner
Session III: How the Payer Community is Adapting to Support Gene & Cell Therapy Access
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Dave McLean, Ph.D., Chief Executive Officer, Emerging Therapies Solutions
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Matthew Fickie, M.D., Medical Director of Clinical Services, Highmark Inc.
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Bruce Pyneson, Principal & Consulting Actuary, Milliman
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Brooks Wildasin, Associate Vice President of Strategy, Outcome Rx, CareMetx
Session IV: Tales from the Trenches – Navigating Coverage Challenges for Gene Therapy: A Patient Perspective
- Katee and Jason DellaMaggiora, parent of a son with SMA Type 1
View the Full Program
ASGCT is a 501(c)3 tax-exempt organization and does not participate in any political activity, either directly or indirectly, including support for or against campaigns for public office. The participation of speaker(s) who are elected officials does not indicate support for or against any political party, political party agenda, or candidate for public office. Any elected official or political appointee who is also a speaker at an ASGCT-sponsored event is permitted to discuss topics related exclusively to the mission of the Society and is expressly prohibited from discussing any campaign for public office or other political activity.
Planned by the Commercialization Committee
Intended primarily for investigators in translational research looking to partner with industry or venture capital (VC). The Early-Stage Development workshop will provide guidance on successfully advancing preclinical research to an IND application and initiation of clinical trials. Speakers will address topics such as requirements for pre-clinical proof-of-concept, product testing and characterization, considerations for first-in-human studies, and tips for successful partnering or collaborations with VCs and industry.
Session I: Challenges and Successes in Preparing for Clinical Trials
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David Schaffer, Ph.D., Director, UC Berkley Stem Cell Center
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Kim Noonan, Ph.D., Executive Vice President & Chief Science Officer, WindMIL Therapeutics
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Joe Gold, Ph.D., Director of Manufacturing, Center for Biomedicine and Genetics, City of Hope
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Florence Salmon, Ph.D., formerly Portfolio Head, Regulatory Affairs, CMC, Cell & Gene Therapies, Novartis
Session II: Funding and Investment for a Promising New Therapy
- Barbara Lueckel, Ph.D., Global Head of Research Technologies Partnering, Roche
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Anna French, D.Phil., Partner, Qiming Venture Partners USA
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Deborah Palestrant, Ph.D., Partner and Head of 4:59 Initiative, 5 AM Ventures
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Debra Miller, Founder & CEO, CureDuchenne
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Hosted by ASGCT, NIST, SCB, and USP
This workshop will address the significance of standards in the field to allow flexible regulation and high-quality products. Presenters will share recent progress in standards setting for gene therapy and editing; updates on current projects under development; and ways to provide input.
Session I: State of Standards in the Field
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Samantha Maragh, Ph.D., Leader, Genome Editing Program, NIST
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Dawn Henke, Ph.D., Senior Technical Program Manager, SCB
Session II: Filling the Gaps: Collaborative Standards Advancement Projects
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Wyatt Vreeland, Ph.D., Chemical Engineer, NIST
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Kelvin Lee, Ph.D., Director, NIIMBL
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Fouad Atouf, Ph.D., VP of Global Biologics, USP
Session III: Regulatory and Developer Perspectives
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Judy Arcidiacono, International Regulatory Expert, FDA
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Diane McCarthy, Ph.D., Director, Biologics Pipeline Development, USP
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Tony Ho, M.D., Executive Vice President, Research and Development, CRISPR Therapeutics
Session IV: Summary of the Most Pressing Standards Needs and Next Steps
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The Expanding the Success of Immune Effector Cell Therapies for Cancer Workshop will feature discussions of the current state of CAR T therapeutic approaches in light of recent FDA approvals with updates on clinical long term follow-up gene editing and going beyond autologous CAR-T cells for cancer.
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The Viral Vector Development workshop will provide an in-depth discussion of the advances as well as the challenges in vector development and manufacturing, including GMP production.
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Brian Bigger, Ph.D., University of Manchester
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Christian Brendel, Ph.D., Boston Children’s Hospital
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Semih Tareen, Ph.D., Sana Biotechnology
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Philip Tai, Ph.D., University of Massachusetts Medical School
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Leszek Lisowski, Ph.D., MBA, Children’s Medical Research Institute
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Hiroyuki Nakai, M.D., Ph.D., Oregon Health and Science University
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