Regulations and Compliance

1. Ensuring Consistent Removal Of Residual Impurities 4/15/2025

   In this Cell & Gene Live segment, Pratik Jaluria, Ph.D., SVP Technical Development Strategy at BlueRock Therapeutics, Bruno Marques, Ph.D. VP, Process and Product Development at Century Therapeutics, and Kate Rochlin, Ph.D., COO at IN8bio discuss validating impurity removal in allogeneic cell therapies involves collecting extensive data from early research through GMP manufacturing to support regulatory submissions.

2. Focal Points for 2024: Dr. Verdun and Dr. Marks Talk Regulatory Challenges for Cell-Based Therapies 1/30/2024

   Dr. Verdun and Dr. Marks provide detailed feedback on the regulatory challenges facing cell therapy and explain the Agency’s most important focal points for 2024, patient accessibility, and more.

3. 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.

4. Regulatory Challenges And Future Outlook For iPSC Therapies 5/23/2025

   In this part of our discussion during the Cell & Gene Live, Unlocking the Potential of iPSC Therapies in Regenerative Medicine, Julie Allickson, Ph.D., Chief Technology Officer at Mayo Clinic's Center for Regenerative Biotherapeutics and Erin Kimbrel, Ph.D., Head of Cell & Gene Therapy Research at Astellas discussed key regulatory challenges for iPSC-based therapies, including donor cell sourcing, genetic modification risks, and long-term safety concerns like tumorigenicity.

5. 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.

6. 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.

7. Assessing Residuals In Processing And Fill/Finish For iPSCs 4/15/2025

   In this segment of Cell & Gene Live, Optimizing Storage Solutions for iPSCs, our expert panelists, Pratik Jaluria, Ph.D., SVP Technical Development Strategy at BlueRock Therapeutics, Bruno Marques, Ph.D. VP, Process and Product Development at Century Therapeutics, and Kate Rochlin, Ph.D., COO at IN8bio explain why detecting and quantifying trace levels of cryoprotectants and excipients in cell therapy products involves a risk-based approach, starting with theoretical worst-case calculations based on formulation concentrations and patient exposure limits.

8. CGT's Main Opportunities To Meet Regulatory Requirements 1/30/2023

   Host and Moderator, Erin Harris, Chief Editor of Cell & Gene, began this Cell & Gene Live, Tackling Cell and Gene Therapies’ Top 3 Regulatory Impediments, featuring Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO and Adora Ndu, PharmD, JD, Chief Regulatory Affairs Officer at BridgeBio, by asking the experts to highlight the main opportunities where the cell and gene therapy industry is working to meet regulatory requirements. They cover accelerated and expedited designation of regenerative medicines, navigating the regulatory requirements around the root causes of genetic disease by repairing or replacing a patient’s genes, and much more.

9. Prioritizing Quality, Fit, And Supplier Reliability In CGT Materials 4/1/2026

   In this segment of Cell & Gene Live, "Materials Matter: Ensuring Quality of CGT Materials," 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 why companies should prioritize technical fit, quality, and supply chain reliability when selecting materials in cell and gene therapy.

10. 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.