2020 Policy Summit Agenda

(Subject to change)

Wednesday September 23

Ethical and regulatory issues in gene and cell therapy

8:30 – 9 a.m. Keynote speaker: George Daley, M.D., Ph.D., Dean of the Faculty of Medicine & Caroline Shields Walker Professor of Medicine, Harvard Medical School
Science Advocacy: From Stem Cells to COVID

9 – 10:45 a.m. Responsible development and use of technology

• Update on international efforts on oversight of clinical use of germline gene editing
• Use of somatic cell gene therapy/editing for prevention of disease
• Sandy Macrae, M.B., Ch.B, Ph.D., Chief Executive Officer of Sangamo Therapeutics
  Bioethical issues in gene editing
• Panel discussion

10:45 – 11 a.m. Break

11 a.m. – 12:10 p.m. Nontherapeutic uses of gene editing

• Renee Wegrzyn, Ph.D., Program Manager, Defense Advanced Research Projects Agency (DARPA)
  Transient gene therapies for prophylactic use
• David A Relman, M. D., Thomas C. and Joan M. Merigan Professor in Medicine, Stanford University
  National security implications of gene editing
• Panel discussion

12:10 – 1 p.m. Lunch break

1 – 2:40 p.m. Challenges and solutions in large-scale manufacturing

• Andrew Byrnes, Ph.D., Chief, Gene Transfer and Immunogenicity Branch, Division of Cellular and Gene Therapies, FDA
  Standardization in vector manufacturing
• Christopher Murphy, M.Sc., Chief Operations Officer, Brammer Bio
  Developing manufacturing facility readiness: CMO perspective
• Robert A. Baffi, Ph.D., Special Advisor to the Chairman and Chief Executive Officer, BioMarin
  Developing in-house manufacturing facility readiness
• Panel discussion

2:40 – 3:10 p.m. Break

3:10 – 5 p.m. CMC and regulatory issues

• Herb Runnels, Ph.D., BioTx Pharmaceutical Sciences, Analytical R&D, Pfizer
  Identifying critical quality attributes for AAV vector products
• Identifying critical quality attributes for CAR T-cell therapies
• Streamlining CMC requirements globally
• Panel discussion

Thursday September 24

Regulatory, legislative, and access issues in gene therapy

8:30 – 9 a.m.  Keynote speaker: Peter Marks, M.D., Ph.D., Director of FDA CBER
Streamlining Development by Leveraging Applications on File

9 – 10:10 a.m. Trial design considerations for gene therapy for rare diseases

• Brad Glasscock, PharmD, Group Vice President, Head of Global Regulatory Affairs, BioMarin
  Utilizing increased regulatory flexibilities in gene therapy trial design
• Daniel Chung, D.O., Medical Strategy Lead- Ophthalmology, Spark Therapeutics
  Significance of patient-focused trial design: A case study
• Panel discussion

10:10 – 10:30 a.m. Break

10:30 a.m. – 12 p.m. Overcoming regulatory challenges in gene therapy trials

• Ying Huang, Ph.D., Pharmacologist, Pharmacology/Toxicology Branch, FDA
  Selecting appropriate animal models for clinical success
• Becky Schweighardt, Ph.D., Senior Director, Immunogenicity Assessment, BioMarin
  Addressing immunogenicity in clinical trials
• Increased reliance on remote data collection: Effects on long-term follow-up
• Panel discussion

Noon – 12:30 p.m. Lunch break

12:30 – 1 p.m. Keynote speaker: Michelle McMurry-Heath, M.D., Ph.D., President and CEO of the Biotechnology Innovation Organization (BIO)
Science as Social Justice, Scientists as Crusaders for Truth

1 – 2:30 p.m. Evidence generation for regulators and payers

• Eric Faulkner, Vice President, Precision and Transformative Medicine, Evidera
  Evidence-generation solutions to support regulatory and payer requirements
• Opportunities for parallel consultation and evidence generation in Europe (EMA and HTAs)
• FDA and CMS parallel review
• Panel discussion

2:30 – 3 p.m. Break

3 – 5 p.m. Policy in the Pipeline: Spotlight on Sickle Cell Disease (SCD)

• Beverley Francis-Gibson, M.A., President & Chief Executive Officer, Sickle Cell Disease Association of America
  Policy implications of gene therapies for SCD: Patient advocacy group viewpoint
• William Chou, M.D., Chief Executive Officer, Aruvant Sciences
  Bringing new therapies to patients: Academic to commercial manufacturing and reimbursement considerations
• Policy implications of gene therapy and gene editing for SCD: Developer viewpoints
• Tech transfer: Contributions of academic research to gene therapy development for SCD
• Enhancing global transportability of gene therapy for SCD
• Panel discussion

Friday, September 25

Access to testing and treatment

8:30 – 9 a.m. Keynote speaker (to be confirmed)

9 – 10:30 a.m. Patient and market access to approved products in the EU

• Dan and Jodi Symes
  The value of gene and cell therapy: A patient perspective
• Adam Hutchings, Managing Director, Dolon
  An overview of patient and market access to approved gene therapies in the EU
• Nicola Heffron, Head of Europe, bluebird bio
  Case study: Market access to Zynteglo in the EU
• Market access to approved products for rare diseases in the EU
• Panel discussion

10:30 – 11 a.m. Break

11 a.m. – noon Genetic testing and screening policies

• Diane Berry, Ph.D., Senior Vice President, Global Health Policy, Government, and Patient Affairs, Sarepta Therapeutics
  The significance of newborn screening policies on access to gene therapy:
• Annie Kennedy, Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases
  Patient advocate perspectives on newborn screening policies
• Panel discussion