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



ASGCT Policy Summit

September 23-25, 2020