2021 Policy Summit Agenda

(All times and topics tentative)

This year’s Policy Summit will offer a variety of stakeholder perspectives on hot topics in the gene therapy field and how they inform regulatory, legislative, and payment policies for diagnosis and treatment.

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Wednesday September 22

9 a.m. – 4:30 p.m. ET

  • Moving the Field to the Next Level: New Developments in Technology

    • Introduction and Overview of the State of the Science: Preclinical and Clinical Pipeline of Molecular Therapeutics
      David Barrett, JD, American Society of Gene & Cell Therapy

    • The Role of Policy in the Development of New Gene Editing Tools
      Krishanu Saha, Ph.D., NIH Somatic Cell Genome Editing Consortium

    • RNA-Based Gene Writing: New Approached to Genome Editing
      Jacob Rubens, Ph.D., Tessera Therapeutics

    • CAR-Macrophages (CAR-M): A Novel Approach to Solid Tumor Immunotherapy
      Debora Barton, M.D., CARISMA Therapeutics

    • Prime and Base Editing: What it Means for the Field
      Guiseppe Ciaramella, Ph.D., Beam Therapeutics

    • Moderator: Hans-Peter Kiem, M.D., Ph.D., Fred Hutchinson Cancer Research Center 

  • The Status of Medicaid Coverage and Access for Gene and Cell Therapies​

    • Advocating for Medicaid Coverage
      Diane Berry, Ph.D., Sarepta Therapeutics

    • The Medicaid Pipeline: Gene Therapy Impacts & Precision Financing Options
      ​Mark Trusheim, MIT FoCUS Program

    • The State Innovation Landscape
      Josh Trent, Leavitt Partners

    • Moderator: Cathy Kelly, Pink Sheet

  • Policy on The Ground: Medicaid Coverage and Reimbursement

    • MACPAC Recommendations to Congress: Gene and Cell Therapy Payment Systems
      Anne Schwartz, Ph.D., Medicaid and CHIP Payment and Access Commission (MACPAC)

Thursday September 23

9 a.m. – 4:30 p.m. ET

  • Prioritizing Patients in Gene Therapy Development

    • Centering the Patient Voice
      Erin Ward, MTM-CNM Family Connection (Myotubular and Centronuclear Myopathy)

    • Patient Opportunities and Challenges in Gene Therapy
      Pat Furlong, Parent Project Muscular Dystrophy

    • Patient Registries for Rare Disease
      Benjamin Forred, Coordination of Rare Diseases at Sanford (CoRDS) Project at Sanford Research

    • Moderator: Lesha Shah, M.D., Icahn School of Medicine at Mount Sinai

  • Issues in Diagnostic Testing

    • Developing Companion Diagnostics
      Robert Haynes, REGENXBIO

    • Access to Specialized Testing: Medicaid Coverage of Genetic Testing
      Christina Hartman, The Assistance Fund 

    • Moderator: Deepa Chand, M.D., Novartis

  • Vector Integration

    • Readout from the ASGCT AAV Integration Round Table
      Kevin Eggan, Ph.D., BioMarin

    • How FDA's Thinking is Evolving: Integration, Risk/Benefit, and Long-Term Follow-up Considerations
      Rosa Sherafat-Kazemzadeh, M.D., Office of Tissues and Advanced Therapies (OTAT), FDA

    • Risk and Benefit: Ensuring and Supporting Informed Decision-Making by Clinicians and Patients
      Graham Foster, Ph.D., FRCP, Queen Mary University of London

  • Enhancing Gene Therapy Development Through FDA User Fee Support

    • Industry Perspective: PDUFA Impacts on Innovative Product Development
      Karen Midthun, M.D., Greenleaf Health

    • Future PDUFA Proposals: Impact on Gene Therapy Development
      Cartier Esham, Ph.D., Biotechnology Innovation Organization (BIO)

    • Moderator: Scott McGoohan, JD, Vertex

Friday September 24

9 a.m. – 12 p.m. ET

  • Keynote: Key Issues in Regulatory Policy

    • Andi Lipstein Fristedt, Deputy Commissioner for Policy, Legislation, and International Affairs, FDA

  • Workforce Development Challenges in Gene Therapy

    • Recruiting and Maintaining the Manufacturing Workforce
      Vijay Surapaneni, Aldevron

    • Moderator: Kaye Spratt, Ph.D., BridgeBio  

  • From Accelerated Approval to Reimbursement

    • Achieving Success: Advancing a Product Through the Accelerated Approval Process
      Renu Vaish, Kite Pharma

    • The Merits of Separating Accelerated Approval From Reimbursement
      Rachel Sher, JD, National Organization for Rare Disorders (NORD)

    • Moderator: Jocelyn Ulrich, Pharmaceutical Research and Manufacturers of America (PhRMA)

Register Here


ASGCT Policy Summit

September 22-24, 2021

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