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. Why Clinical Requirements Change: Dr. Peter Marks on Pre-IND Meeting Pathways 1/30/2024

    Learn how clinical requirements change after the FDA and a company agree on a pathway during a pre-IND meeting and how the company might recover as a result.

  3. mRNA For Cancer Immunotherapy 2024 Outlook | Audience Q&A 1/29/2024

    During our Cell & Gene Live, mRNA For Cancer Immunotherapy 2024 Outlook, our expert panelists answered audience questions regarding patient safety, foreseeable challenges in mRNA cancer immunotherapy for solid tumors, and much more.

  4. Leveraging Rapid Sterility Testing To Advance Cell Therapy Production 6/23/2025

    Explore the crucial role of rapid sterility testing in cell therapy manufacturing. This presentation delves into the benefits of swift, accurate detection for product quality and patient safety.

  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. CMC Comparability: Transitioning to Commercial Manufacturing with Dr. Nicole Verdun 1/30/2024

    In this segment, Dr. Nicole Verdun shares some of the challenges demonstrating CMC comparability during late-stage product development (i.e., pivotal or Phase 3 clinical trial) that occur while transitioning to commercial manufacturing.

  7. 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.

  8. Validation Of A qPCR Assay For Host Cell DNA Quantitation 6/9/2025

    Here, we share the development and validation of a new, highly sensitive and accurate integrated solution for detection and quantitation of host cell DNA to help meet regulatory requirements.

  9. Microbial Identification Via DNA-Seq 6/9/2025

    Learn how DNA sequencing-based microbial identification supports regulatory compliance and enhances contamination control in critical environments.

  10. 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.