2021 Policy Summit

This year’s Policy Summit offered 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.

Download the PDF Program

Wednesday September 22

All times listed are ET (View today's event in your time zone) 

• Moving the Field to the Next Level: Looking to the Future
  9-9:50 a.m.

  • 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 Government in the Development of New Gene Editing Tools
    PJ Brooks, PhD, Working Group Coordinator, Somatic Cell Genome Editing Program; Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS)

• Break
  9:50-10:10 a.m.

• Moving the Field to the Next Level: New Developments in Technology
  ​10:10-11:35 a.m.

  • RNA-Based Gene Writing: New Approaches to Genome Editing
    Jacob Rubens, PhD, Tessera Therapeutics

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

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

  • Moderator: Hans-Peter Kiem, MD, PhD, Fred Hutchinson Cancer Research Center

• Lunch break
  11:35 a.m.-12:45 p.m.

• Fireside Chat: High-Cost, High-Value Therapies: Lessons Learned From Previous Payment Debates 
  12:45-1:15 p.m.

  •  Demetrios Kouzoukas, JD, Board Member, Clover Health; Former Director, Center for Medicare, and Principal Deputy Administrator, Centers for Medicare & Medicaid Services (CMS)

  • Moderator: Anna Griffin, Gilead Sciences

• The Status of Medicaid Coverage and Access for Gene and Cell Therapies
  1:15-2:45 p.m.​

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

  • Advocating for Medicaid Coverage 
    Diane Berry, PhD, Sarepta Therapeutics 

  • The State Innovation Landscape
    Josh Trent, Leavitt Partners

  • Moderator: Cathy Kelly, Pink Sheet

• Break
  2:45-3:15 p.m.

• Policy on The Ground: Medicaid Coverage and Reimbursement
  3:15-3:45 p.m.

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

  • Moderator: Dan Farmer, BGR Group

Thursday September 23

All times listed are ET (View today's event in your time zone)

• Prioritizing Patients in Gene Therapy Development
  9-10:15 a.m.

  • 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, MD, Icahn School of Medicine at Mount Sinai

• Break
  10:15-10:45 a.m.

• Issues in Diagnostic Testing
  10:45 a.m.-12:15 p.m. 

  • Current US Regulatory Requirements for Development of Companion Diagnostics for Oncology Therapies 
    Peter Bross, MD, Office of Tissues and Advanced Therapies (OTAT), FDA

  • Developing Companion Diagnostics
    Nina Hunter, PhD, Vice President, Regulatory Affairs & Science Policy, REGENXBIO

  • Companion Diagnostics and IVDs: Policy Issues and Potential for Reforms
    Jeff Allen, PhD, Friends of Cancer Research

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

  • Moderator: Deepa Chand, MD, Novartis Gene Therapies

• Lunch break
  12:15-1:15 p.m.

• AAV Vector Integration
  1:15-2:45 p.m.

  • Readout of ASGCT’s AAV Integration Roundtable
    Doug McCarty, PhD

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

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

  • Moderator: John Gray, PhD, Vertex

• Break
  2:45-3 p.m.

• Enhancing Gene Therapy Development Through FDA User Fee Support
  3-4:30 p.m.

  • Efforts at CBER to Support Gene Therapy Development
    Chris Joneckis, PhD, Center for Biologics Evaluation and Research, FDA

  • Industry Perspective: PDUFA Impacts on Innovative Product Development
    Karen Midthun, MD, Greenleaf Health

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

  • Moderator: Scott McGoohan, JD, Vertex

Friday September 24

All times listed are ET (View today's event in your time zone)

• Keynote: Key Issues in Regulatory Policy
  9-9:30 a.m.

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

  • Moderator: Remy Brim, PhD, BGR Group

• Workforce Development Challenges in Gene Therapy
  9:30-10:20 a.m.

  • Recruiting and Maintaining the Manufacturing Workforce
    Vijay Surapaneni, Aldevron

  • Attracting and Retaining Diversity in Biotech
    Donia Slimani, MilliporeSigma

  • Moderator: S. Kaye Spratt, PhD, BridgeBio Gene Therapy 

• Break
  10:20-10:35 a.m.

• From Accelerated Approval to Reimbursement
  10:35 a.m.-12 p.m.

  • Accelerated Approval Standards: Application in Cell and Gene Therapy
    Nancy Bradish Myers, JD, Catalyst Healthcare Consulting

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

  • A Payer Perspective on Coverage and Reimbursement of Treatments with Accelerated Approval
    Ani Khachatourian, PharmD, Highmark Blue Cross Blue Shield

  • The Merits of Separating Accelerated Approval From Reimbursement
    Richard White, National Organization for Rare Disorders (NORD)

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

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 or political appointees 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.