2020 Policy Summit Agenda

(Subject to change - all times are CDT)

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, Universitá 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

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