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Unlocking Complex Cell-based Gene Therapies

Unlocking Complex Cell-based Gene Therapies

Unlocking Complex Cell-based Gene Therapies

Monday, May 6th, 2024
10:00AM – 2:15PM ET
The Ritz Carlton
1150 22nd St NW
Washington, DC 20037

Friends of Cancer Research (Friends) and the Parker Institute for Cancer Immunotherapy (PICI) are proud to announce our upcoming joint public meeting, Unlocking Complex Cell-based Gene Therapies. Discussions among key stakeholders at the meeting will enable the next generation of proposals in the rapidly changing cell and gene therapy space, reimagining trials and treatments for patients. Register above and scroll through below to read more about the meeting.


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
12:05PM – Lunch


12:50PM – Session 2: Discovering & Developing the Next Generation of Cell-based Gene Therapies: Considerations for First-in-Human Trials

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.

12:50 PM – Leveraging Traceable Heterogeneity in CAR-T Investigational Products in Human Trials (Presentation)

  • Marcela Maus, Massachusetts General Hospital

1:20 PM – Panel Discussion and Q&A

  • Moderator: Crystal Mackall, Stanford University
  • Panelists:
    • Rick Bangs, Patient Advocate
    • Asha Das, CBER, U.S. FDA
    • Phil Greenberg, Fred Hutchinson Cancer Center
    • Anna Kwilas, CBER, U.S. FDA
    • Marcela Maus, Massachusetts General Hospital
    • Chris White, Patient Advocate
2:05 PM – The Path Forward: Key Takeaways 
  • John Connolly, Parker Institute for Cancer Immunotherapy
  • Ute Dugan, Parker Institute for Cancer Immunotherapy

2:15 PM – Meeting Adjourns