Regulatory Guidance And Enabling Compliance

  1. 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.

  2. Challenge #2: Rare Or Ultra-Rare Study Design Considerations And Use Of Existing Data 1/30/2023

    There are more than 7,000 unique, rare diseases recognized in the United States, and 300 million people worldwide living with a rare disease; patients often experience a long journey to receive their diagnosis. Our expert panelists, Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO and Adora Ndu, PharmD, JD, Chief Regulatory Affairs Officer at BridgeBio, examine the regulatory requirements for safety and efficacy demonstration and identify the gaps in regulatory oversight that require clearer guidance.

  3. 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.

  4. 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.

  5. How The New Administration Could Shape FDA Oversight, Compliance, And Guidance 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 new administration may influence the regulatory landscape for cell and gene therapy, particularly regarding enforcement actions against unproven stem cell products and challenges to FDA authority at the state level.

  6. 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.

  7. Innovative Contamination Control: Enabling Integrity And Efficiency 10/15/2024

    An integrated approach can enhance contamination control, boost process efficiency, and ensure the production of high-quality cell therapy products.

  8. Other CGT Regulatory Challenges 1/30/2023

    During this segment of Cell & Gene Live, Tackling Cell and Gene Therapies’ Top 3 Regulatory Impediments, Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO and Adora Ndu, PharmD, JD, Chief Regulatory Affairs Officer at BridgeBio detail their own experiences with roadblocks with regulators and how they and their teams worked to overcome them.

  9. Congruence And Divergence In Global Regulatory Approvals 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) explain how regulatory standards for cell and gene therapy approvals are largely consistent across regions, with North America typically leading in approvals, followed by Europe and then Asia.

  10. 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.