Agenda

10:00AM – Welcoming Remarks

10:05AM – Opening Keynote Conversation

• Moderator: Laurie McGinley, fmr. Washington Post Reporter
• Namandjé Bumpus, Principal Deputy Commissioner, U.S. FDA
• Peter Marks, Director, CBER, U.S. FDA

10:30AM – Session 1: Shaping the Future of Cell and Gene Therapies: Challenges & Opportunities

To date, the U.S. Food and Drug Administration (FDA) has approved seven autologous cell-based immunotherapies that have demonstrated remarkable efficacy in a limited set of cancer types. However, to expand their use to a wider range of cancers, the next generation of engineered cellular therapies will require innovative scientific approaches that build upon learnings from existing products to further enhance their safety and efficacy. To advance these novel therapies, several scientific and operational challenges need to be addressed, including overcoming biological constraints, and reducing both the cost and duration of manufacturing and clinical development.

10:30 AM – The Evolution of Cell and Gene Therapies: Current Advances and Challenges (Presentation)

• Carl June, University of Pennsylvania

11:00 AM – Opportunities to Enhance Adoptive T Cell Therapies using CRISPR Gene Editing (Presentation)

•  Alex Marson, Gladstone Institutes

11:20 AM – Panel Discussion and Q&A

• Moderator: Toni Ribas, University of California Los Angeles
• Panelists:
  • Carl June, University of Pennsylvania
  • Alex Marson, Gladstone Institutes
  • Nicole Verdun, CBER, U.S. FDA
  • Desiree Walker, Patient Advocate
  • Tom Whitehead, Patient Advocate
  • Iwen Wu, CBER, U.S. FDA

CRISPR screens, a type of gene-editing test, have identified hundreds of promising gene candidates for tackling critical challenges in treating solid tumors with autologous T cell products. However, nonclinical models fall short in accurately prioritizing gene modifications for human testing. There is an urgent need to explore ways to simultaneously test multiple gene modifications in human clinical trials, while ensuring patient safety. Given the complexity of these novel investigational products, it is crucial to reevaluate and enhance appropriate safety measures.

• John Connolly, Parker Institute for Cancer Immunotherapy
• Ute Dugan, Parker Institute for Cancer Immunotherapy