2020 Policy Summit Agenda

(Subject to change - all times are CDT)


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Wednesday, September 23

Ethical and regulatory issues in gene and cell therapy

8:30 – 9 a.m. (CDT) Keynote speaker: George Q. 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 – 10:30 a.m. (CDT) Responsible development and use of technology

  • Eric Juengst, Ph.D., Director of the UNC Center for Bioethics & Professor of Social Medicine and Genetics, University of North Carolina at Chapel Hill
    Anticipating preventive somatic cell gene editing: “incidental enhancements” and their governance implications  
  • R. Alta Charo, J.D., Warren P. Knowles Professor of Law and Bioethics, University of Wisconsin Law School
    Global law and policy on germline editing
  • Sandy Macrae, M.B., Ch.B, Ph.D., Chief Executive Officer of Sangamo Therapeutics
    Addressing bioethical challenges in genomic medicine
  • Panel discussion moderated by Tim Hunt, J.D., formerly Chief Corporate Affairs Officer, Editas Medicine

10:30 – 11 a.m. Break

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

  • Renee Wegrzyn, Ph.D., Vice President, Business Development, Ginkgo Bioworks; formerly 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
    Security implications of genome engineering 
  • Panel discussion moderated by Pilar Ossorio, Ph.D., J.D., Professor of Law and Bioethics, University of Wisconsin-Madison

12:10 – 1 p.m. Lunch break

1 – 2:40 p.m. (CDT) 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 K. Murphy, Vice President and General Manager of Viral Vector Services in Thermo Fisher Scientific’s Pharma Services Group
    Developing manufacturing facility readiness: CMO perspective
  • Robert A. Baffi, Ph.D., Special Advisor to the Chairman and Chief Executive Officer, BioMarin
    Strong Process Development for Gene Therapy Products
  • Panel discussion moderated by Joanne S. Eglovitch, Senior Editor, Pink Sheet

2:40 – 3:10 p.m. Break

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

  • Herb Runnels, Ph.D., BioTx Pharmaceutical Sciences, Analytical R&D, Pfizer
    Identifying critical quality attributes for AAV vector products
  • Katie Miller, Ph.D., Principal, Dark Horse Consulting 
    Identifying critical quality attributes for gene-modified cell therapies
  • Denise Gavin, Ph.D., Chief, Gene Therapies Branch, FDA
    Global CMC Convergence: an FDA Perspective
  • Panel discussion moderated by Jan Thirkettle, Ph.D., Chief Development Officer, Freeline Therapeutics Limited

Thursday, September 24

Regulatory, legislative, and access issues in gene therapy

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

9 – 10:10 a.m. (CDT) 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 moderated by Andrea Spezzi, M.D., Chief Medical Officer and Co-Founder, Orchard Therapeutics

10:10 – 10:30 a.m. Break

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

  • Anne-Virginie Eggimann, Senior Vice-President, Regulatory Science, bluebird bio
    Regulatory considerations to improve gene therapy development throughout the life cycle
  • Ying Huang, Ph.D., Pharmacologist, Pharmacology/Toxicology Branch, FDA
    Selecting appropriate animal models to inform clinical development
  • Becky Schweighardt, Ph.D., Executive Director, Translational Sciences, BioMarin
    Addressing immunogenicity in clinical trials
  • Panel discussion moderated by Nancy Bradish Myers, J.D., President/Founder, Catalyst Healthcare Consulting, Inc.

12 – 12:30 p.m. Lunch break

12:30 – 1 p.m. (CDT) 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. (CDT) Evidence generation for regulators and payers

  • Eric Faulkner, Vice President, Precision and Transformative Medicine, Evidera
    Evidence-generation solutions to support regulatory and payer requirements
  • Americo Cicchetti, Professor of Business Organization, Faculty of Economics, Universit√° Cattolica del Sacro Cuore
    Evidence requirements for health technology assessment in Italy
  • Jeet Guram, M.D., Senior Advisor in the Office of the Commissioner, FDA
    FDA & CMS parallel review process, expedited approvals, and outcome-based payment models
  • Panel discussion moderated by Mark Battaglini, J.D., Senior Vice President and Head of Global Government Affairs, Alliance Development & Public Policy, bluebird bio

2:30 – 3 p.m. Break

3 – 5 p.m. (CDT) Policy in the pipeline: Spotlight on sickle cell disease (SCD)

  • John Tisdale, M.D., Senior Investigator, Cellular and Molecular Therapeutics Branch, NHLBI
    An introduction to genetic approaches to SCD: Scientific researcher perspective
  • Beverley Francis-Gibson, President & Chief Executive Officer, Sickle Cell Disease Association of America
    Sickle cell disease: Patient perspectives and ethical considerations in gene therapy
  • Samarth Kulkarni, Ph.D., Chief Executive Officer, CRISPR Therapeutics
    Policy implications of  gene editing for SCD: Developer viewpoint
  • Nick Leschly, President & Chief Executive Officer, bluebird bio
    Policy implications of gene therapy for SCD: Developer viewpoint
  • Panel discussion moderated by Beverley Francis-Gibson

Friday, September 25

Access to testing and treatment

8:30 – 8:35 a.m (CDT) Welcome remarks: The Honorable Tim Scott, United States Senator, South Carolina

8:35 – 9 a.m. (CDT) Keynote address: Joe Grogan, former Director of the United States Domestic Policy Council

9 – 10:30 a.m. (CDT) Gene and cell therapy access: Real world experiences

  • 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
  • Andrew Walker, Ph.D., Director, Salus Alba
    Addressing cost effectiveness for gene therapies in the pipeline in the EU
  • Nicola Heffron, Head of Europe, bluebird bio
    Sharing the risks and the rewards - Lessons from taking a partnership approach to launch a novel gene therapy in the EU
  • Panel discussion moderated by Francesca Cook, Senior Director of Pricing and Market Access, REGENXBIO

10:30 – 11 a.m. Break

11 a.m. – 12 p.m. (CDT) Newborn 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 moderated by Christine Harrison, Vice President, Corporate Affairs, Orchard Therapeutics

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 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.

2020

ASGCT Policy Summit

September 23-25, 2020