Regulatory Guidance And Enabling Compliance

1. 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.

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. Challenge #1: The Changing Regulatory Landscape 1/30/2023

   By 2030, 60 CGTs are expected to reach regulatory approval. One of the biopharmaceutical industry's most closely watched regulatory developments is the FDA’s reauthorization of the Prescription Drug User Fee Act (PDUFA) or PDUFA VII, as it provides resources for the FDA and CBER to have the capacity to provide feedback on development and review submitted applications, among other critical activities. During our 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 break down how PDUFA VII, Fiscal Year 2023 Omnibus Appropriations Bill, and more will impact the cell and gene therapy field in the next few years.

4. 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.

5. 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.

6. 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.

7. An All-In-One Solution For Residual DNA Quantitation 5/22/2025

   Explore an all-in-one solution for residual DNA quantitation with a 3D lab tour with virtual demos, videos, and interactive instrument guides to experience the full workflow.

8. CAR-T Innovation: Balancing Allogeneic And Autologous Cell Therapy 6/30/2025

   Join experts as they discuss strategies for optimizing allogeneic and autologous therapies, aiming for balanced development and wider patient accessibility by 2030.

9. Innovations In Mycoplasma And Sterility Testing For Biopharma 5/22/2025

   In this webinar, our expert speakers will discuss the types of analytical testing that can be effectively employed in the early stages of therapeutic development and subsequently scaled to meet production challenges.

10. 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.