Regulatory Guidance And Enabling Compliance

1. Collaborating with the FDA: Dr. Peter Marks and Dr. Nicole Verdun Share Advice For Small And Emerging Biotechs 1/30/2024

   In this segment, Dr. Peter Marks and Dr. Nicole Verdun share their advice and best practices for small and emerging biotechs when working with the FDA.

2. How Past Gene Therapy Approvals Are Shaping the Future with Dr. Peter Marks 1/30/2024

   In this segment of Cell & Gene Live, Dr. Peter Marks shared what he and his team have learned from current gene therapy approvals to accelerate future approvals.

3. Race To The Regulatory Finish Line: Dr. Nicole Verdun Talks Efficiencies In Platform Technologies 1/30/2024

   Could a platform approach help certain therapies make their way over the regulatory finish line more efficiently?

4. Challenge #3: Lack Of Animal Models 1/30/2023

   Many cell and gene therapies are intended for diseases and conditions that primarily impact pediatric patients, requiring the prospect of direct benefit to individual subjects enrolled in the trial.  In the absence of good animal models, there may be challenges demonstrating the prospect of direct benefit.  During this segment of Cell & Gene Live, Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO discusses novel approaches to consider going forward.

5. 2025 CGT Regulatory Outlook | Audience Q&A 4/8/2025

   Kimberly Benton, Ph.D., Master Principal and Head of Regulatory at Dark Horse Consulting and Bambi Grilley, RPh, RAC, CIP, CCRC, CCRP, Director, Clinical Research and Early Product Development Center for Cell and Gene Therapy, Professor of Pediatrics Baylor College of Medicine, and Chief Regulatory Officer of International Society for Cell & Gene Therapy (ISCT) answer questions from the audience. They covered key regulatory topics, including the FDA’s process for finalizing draft guidances, the status of INTERACT meetings, and the use of the term “critical component” in manufacturing regulations.

6. Dr. Peter Marks on the Adoption of Novel Precision Analytics 1/30/2024

   Hear advice for developers who are worried that the FDA will not accept changes in their process that include a new analytical method.

7. Key Regulatory Challenges, Innovations, And Opportunities For CGT In 2025 4/8/2025

   Kimberly Benton, Ph.D., Master Principal and Head of Regulatory at Dark Horse Consulting and Bambi Grilley, RPh, RAC, CIP, CCRC, CCRP, Director, Clinical Research and Early Product Development Center for Cell and Gene Therapy, Professor of Pediatrics Baylor College of Medicine, and Chief Regulatory Officer of International Society for Cell & Gene Therapy (ISCT) detail how the regulatory landscape for cell and gene therapy in 2025 will continue to be shaped by advancements in science and manufacturing and evolving regulatory guidance.

8. How The FDA's 2024 Draft Guidances Will Influence CGT Regulation In 2025 4/8/2025

   Kimberly Benton, Ph.D., Master Principal and Head of Regulatory at Dark Horse Consulting and Bambi Grilley, RPh, RAC, CIP, CCRC, CCRP, Director, Clinical Research and Early Product Development Center for Cell and Gene Therapy, Professor of Pediatrics Baylor College of Medicine, and Chief Regulatory Officer of International Society for Cell & Gene Therapy (ISCT) share why the FDA’s 2024 draft guidance documents are expected to shape regulatory standards for cell and gene therapy in 2025, with topics ranging from safety testing of allogeneic cells to manufacturing practices and the use of human and animal-derived materials.

9. Major Trends Impacting Allogeneic Cell Therapies For Oncology 5/6/2024

   We kicked off this Cell & Gene Live, Unlocking the Future of Allogeneic Cell Therapy for Oncology by discussing the major trends impacting allogeneic cell therapies for oncology.

10. Patient Data's Impact On CMC Robustness 1/30/2024

    When CGT products demonstrate dramatic improvements with minimal safety concerns in a Phase 2 study, sponsors may want to modify the protocol mid-study to increase patient enrollment.