Regulations and Compliance

  1. What's New for 2024: Dr. Nicole Verdun Offers a Look Inside The Office Of Therapeutic Products 1/30/2024

    What is the Office of Therapeutic Products, where did it originate, and what's on its regulatory agenda for 2024?

  2. Making USP Guidance Actionable For CGT Materials 4/1/2026

    In this segment of Cell & Gene Live, Anthony Blaszczyk, Ph.D., Senior Scientist at the U.S. Pharmacopeia, and Basak Clements, Ph.D., Founder and Senior Advisor at biomatria, were joined by Cell & Gene Chief Editor Erin Harris to discuss USP’s Cell & Gene Therapy Expert Committee, which is working to make existing guidance, such as USP 1043, more practical and actionable, while developing updated frameworks that clarify roles across the supply chain and strengthen risk-based control of raw materials.

  3. Dr. Peter Marks Talks a Standardized Playbook for Gene Therapy Manufacturing 1/30/2024

    In this segment, Dr. Peter Marks shares developments in CMC for advanced therapies in the near future.

  4. Master The 2-Point Calibration Process To Maintain Data Integrity 5/19/2025

    Ensure accurate sensor readings with an innovative software by mastering the 2-point calibration process. Learn more about how and why regular calibration supports data integrity and compliance.

  5. Lower Variability For Viral Titer Determination 1/11/2023

    Learn about the benefits of ddPCR for ensuring precise and reproducible viral titers in the development of effective adeno-associated virus AAV-based gene therapies.

  6. 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.

  7. Bridging Analytical Gaps To Ensure CGT Material Quality 4/1/2026

    In this segment of Cell & Gene Live, "Materials Matter: Ensuring Quality of CGT Materials," Cell & Gene Chief Editor Erin Harris was joined by Anthony Blaszczyk, Ph.D., Senior Scientist at the U.S. Pharmacopeia, and Basak Clements, Ph.D., Founder and Senior Advisor at biomatria, to discuss why analytical gaps remain a major challenge in cell and gene therapy.

  8. 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.

  9. 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.

  10. Navigating Global Regulations, Equity Challenges, And The Future Of Patient Access 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) continue the discussion to explore the complexities of expanded access programs (EAPs) in CGT, highlighting global regulatory differences, ethical concerns, and accessibility challenges.