2022 Policy Summit Agenda

(subject to change)

This year’s Policy Summit will offer a variety of stakeholder perspectives on hot topics and how they inform policies that impact the cell and gene therapy field.

Check back to this page for more agenda information as speakers are confirmed!

Policy Summit Co-Chairs: Diane Berry, PhD, Sarepta Therapeutics & Keith Wonnacott, PhD, LEXEO Therapeutics

Monday September 26, 2022

9:00 – 10:30 a.m.: Supporting Innovative CGT Development

• RMAT in 2022: Updates and Learnings
  Wilson Bryan, MD, FDA OTAT

• New Endpoints and Biomarkers in Supporting CGT Development
  Adora Ndu, PharmD, JD, BridgeBio

• The Bespoke Gene Therapy Consortium: Increasing Accessibility of Gene Therapies for Rare Diseases
  Courtney Silverthorne, Foundation for the National Institutes of Health

• Moderator: Daniela Drago, PhD, Aurion Biotech

10:30 – 10:45 a.m.: Break

10:45 a.m. – 12:00 p.m.: Panel on PDUFA Reauthorization

• PDUFA VII: CBER Updates
  Wilson Bryan, MD, FDA OTAT

• Moderator: Keith Wonnacott, PhD, LEXEO Therapeutics

12:00 – 1:30 p.m.: Lunch

1:30 – 2:45 pm: Panel on Diversity in Cell and Gene Therapy Development

• Moderator: Rayne Rouce, MD, Texas Children's Hospital & Baylor College of Medicine

2:45 – 3:15 p.m.: Break

3:15 – 4:30 p.m.: Panel on Accelerated Approval

• Developer Viewpoint on Accelerated Approval
  Diane Berry, PhD, Sarepta Therapeutics

• Moderator: Jeremy Allen, Spark Therapeutics

Tuesday September 27, 2022

9:00 – 10:15 a.m.: Panel on Coverage and Reimbursement for Cell and Gene Therapies

• Payer Decisions that Impact Patient Access
  Kollet Koulianos, National Hemophilia Foundation

• Moderator: Francesca Cook, REGENXBIO

10:15 – 10:30 a.m.: Break

10:30 a.m. – 12:00 p.m.: CMC Applications and Hurdles for Gene and Cell Therapies

• Moderator: Megan Zoschg Canniere, Spark Therapeutics

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