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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.
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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
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
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.
January 22-23, 2025 | Virtual
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