2021 Pre-Meeting Workshops: May 10, 2021

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.

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)

Patient Advocates’ Role in Advancing Gene Therapy

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

Gina Hann, Co-Founder, The Rare Village Foundation, President & Founder, Foundation for Batten Hope
Christine Waggoner, President, Cure GM1 Foundation
Sue Wilson, President, The Children's Medical Research Foundation, Inc.
Amber Olsen, Executive Director & Founder, United MSD Foundation
Matt Wilsey, President & Co-Founder, Grace Science Foundation

Session II: Understanding the Research Process: Being Equipped for Success

Steven Gray, Ph.D., Associate Professor, UT Southwestern
Jill Wood, Chief Financial Officer, Phoenix Nest Biotech
Michelle Berg, President, GMP Nucleic Acids, Aldevron
P.J. Brooks, Ph.D., Program Director, Office of Rare Disease Research, NCATS

Recent Developments in Global Regulation of Gene Therapies

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

Alessandro Aiuti, M.D., Ph.D., Deputy Director of Clinical Research, San Raffaele Telethon Institute
Stuart Beattie, Ph.D., Clinical Lead, Gene Therapy Regulatory CMC, Biogen
Gentaro Tajima, Japan Regulatory Lead, Pfizer
Michael Havert, Ph.D., Senior Director of Regulatory CMC, bluebird bio
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

Wilson Bryan, M.D., Director of Office of Tissues and Advanced Therapies, FDA
Martina Schussler-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

Dan Takefman, Ph.D., Principal, Takefman Gene Therapy Advisors LLC
Keith Wonnacott, Ph.D., Executive Director of Regulatory Affairs, Pfizer
Michael Ladd, PharmD., VP Regulatory Science, Orchard Therapeutics
Katie Picone, PharmD., Senior Global Program Regulatory Director, Novartis

Session IV: Use of Regulatory Reliance for Regenerative Medicines/Advanced Therapies

Lawrence Liberti, Ph.D., Head, Regulatory Collaborations, Centre for Innovation in Regulatory Science
Murray Lumpkin, M.D., Deputy Director, Regulatory Affairs, Lead Global Regulatory Systems Initiatives, The Bill and Melinda Gates Foundation
Michael Shum, Director, Application and Advisory Management, Prescription Medicines Authorisation Branch, Australian Therapeutic Goods Administration

Oncolytic Viruses: Successes, Challenges, and a Promising Future

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

Gregory Friedmann, M.D., University of Alabama at Birmingham
E. Antonio Chiocca, M.D., Ph.D., Brigham and Women’s/Faulkner Hospital
Joseph Jackson, M.S., Ph.D., University of Pittsburgh
Parker Dryja, University of New Mexico
Ji Young Yoo, Ph.D., University of Texas, McGovern Medical School

Session II: In Pursuit of Synergy

Jacob van Vloten, Ph.D., Mayo Clinic
Laura Elvgin, Ph.D., University of British Columbia
Maria Eugenia Davola, Ph.D., McMaster University
John Christie, Arizona State University
Bangxing Hong, Ph.D., University of Texas, Health Science Center

Moving Genome Editing to the Clinic: From Technology to Therapeutics

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.

Shengdar Tsai, Ph.D., St. Jude Children’s Hospital
Ben Kleinstiver, Ph.D., Massachusetts General Hospital
Alexander Marson, M.D., Ph.D., University of California, San Francisco
Deepak Reyon, Ph.D., Editas Medicine
Hari Jayaram, Ph.D., Spotlight Therapeutics
Nicole Gaudelli, Ph.D., Beam Therapeutics
Anna Kwilas, Ph.D., FDA
Daniel O’Connell, Ph.D., Intellia Therapeutics

Panel – Regulatory Approach to Genome Editing for Clinic

Moderator – Fyodor Urnov, Ph.D., University of California, Berkeley

Daniel O’Connell, Ph.D., Intellia Therapeutics
Anna Kwilas, Ph.D., FDA
Deepak Reyon, Ph.D., Editas Medicine
Nicole Gaudelli, Ph.D., Beam Therapeutics

Transitioning from Academics to Industry

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

Steven Gray, Ph.D., Associate Professor, UT Southwestern
Gallia Levy, M.D., Ph.D., Chief Medical Officer, Spark Therapeutics
Kamal Bharucha, M.D., Vice President, Clinical Development, BridgeBio
Adora Ndu, PharmD, J.D., Vice President, Regulatory Affairs, Global Head of Policy, Research, Engagement & International, BioMarin
Dan Takefman, Ph.D., Principal, Takefman Gene Therapy Advisors LLC
Nisha Jain, M.D., Takeda Pharmaceuticals

Session II: Learning from Experience - Case Studies of Transitions from Academia to Industry

Federico Mingozzi, Ph.D., Chief Scientific Officer, Spark Therapeutics
Kevin Williams, M.D., J.D., Chief Medical Officer, Pfizer Rare Disease
Leonard Valentino, M.D., Chief Executive Officer, National Hemophilia Foundation
Katherine Beaverson, M.S., Senior Director, Patient Advocacy Lead, Pfizer Rare Disease
Fred Porter, Ph.D., Chief Technical Officer, Taysha Gene Therapies

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)

Early-Stage Development

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

David Schaffer, Ph.D., Director, UC Berkley Stem Cell Center
Kim Noonan, Ph.D., Executive Vice President & Chief Science Officer, WindMIL Therapeutics
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
Anna French, D.Phil., Partner, Qiming Venture Partners USA
Deborah Palestrant, Ph.D., Partner and Head of 4:59 Initiative, 5 AM Ventures
Debra Miller, Founder & CEO, CureDuchenne

Standards in Gene Therapy and Genome Editing

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

Samantha Maragh, Ph.D., Leader, Genome Editing Program, NIST
Dawn Henke, Ph.D., Senior Technical Program Manager, SCB

Session II: Filling the Gaps: Collaborative Standards Advancement Projects

Wyatt Vreeland, Ph.D., Chemical Engineer, NIST
Kelvin Lee, Ph.D., Director, NIIMBL
Fouad Atouf, Ph.D., VP of Global Biologics, USP

Session III: Regulatory and Developer Perspectives

Judy Arcidiacono, International Regulatory Expert, FDA
Mehrshid Alai-Safar, Ph.D., Vice President, Regulatory CMC, Kite Pharma, Inc.
Tony Ho, M.D., Executive Vice President, Research and Development, CRISPR Therapeutics

Session IV: Summary of the Most Pressing Standards Needs and Next Steps

CAR T and Related Immune Effector Cell Therapies

The CAR T and Related Immune Effector Cell Therapies 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.

Viral Vector Development

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.

Brian Bigger, Ph.D., University of Manchester
Christian Brendel, Ph.D., Boston Children’s Hospital
Semih Tareen, Ph.D., Sana Biotechnology
Philip Tai, Ph.D., University of Massachusetts Medical School
Leszek Lisowski, Ph.D., MBA, Children’s Medical Research Institute
Hiroyuki Nakai, M.D., Ph.D., Oregon Health and Science University

Emerging Issues in Market Access

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 GCT launches, and potential solutions to facilitate access will be explored. In addition, the workshop will delve into recent policy changes that impact our industry and relevant priorities of the new Administration.

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

Bruce Booth, D.Phil., Partner, Atlas Venture
Ritu Baral, Managing Director, Health Care-Biotechnology, Cowen
Craig Gordon, M.D., Partner & Investment Analyst, Capital Group
Josh Schimmer, M.D., Senior Managing Director, Biotech, Evercore ISI

Session II: Tales from the Trenches – Navigating Reimbursement Challenges for Gene Therapy: A Patient Perspective

Katee and Jason DellaMaggiora, parent of a son with SMA Type 1

Session III: How the Payer Community is Adapting to Support Gene & Cell Therapy Access

Dave McLean, Ph.D., Chief Executive Officer, Emerging Therapies Solutions
Matthew Fickie, M.D., Medical Director of Clinical Services, Highmark Inc.
Bruce Pyneson, Principal & Consulting Actuary, Milliman
Brooks Wildasin, Associate Vice President of Strategy, Outcome Rx, CareMetx

Session IV: What’s New in Federal Health Policy

Important Dates

Call for Abstracts Opens
November 16, 2020

Registration Opens
December 8, 2020

Call for Abstracts Closes
January 31, 2021

Abstract Notifications Sent
April 5, 2021

Abstract Withdrawal Date
April 12, 2021

Abstracts Released to the Public
April 27, 2021

Early Registration Closes
May 1, 2021

24th ASGCT Annual Meeting
May 11 – 14, 2021

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