Regulatory Guidance And Enabling Compliance
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Tackling Cell And Gene Therapies' Top 3 Regulatory Impediments: Audience Q&A
1/30/2023
During each Cell & Gene Live, our audience has the opportunity to submit real-time questions for our expert panelists. Watch and listen as Nina Hunter, Ph.D., VP Corporate Strategy at REGENXBIO and Adora Ndu, PharmD, JD, Chief Regulatory Affairs Officer at BridgeBio provide detailed, insightful responses to our audience questions on everything from regulatory timelines to successful and unsuccessful strategies needed to convince the regulators that current regulation requirements are either inappropriate or alternatively excessively burdensome for cell and gene-based therapies.
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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.
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2024 Regulatory Outlook With Dr. Peter Marks And Dr. Nicole Verdun | Audience Q&A
1/30/2024
Experts on the Cell & Gene Live event, 2024 Regulatory Outlook, answer questions about topics including pediatric development in rare diseases, diversity planning for ultra-rare disease, anticipated approvals by 2025, and more.
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Race To The Regulatory Finish Line: Dr. Nicole Verdun Talks Efficiencies In Platform Technologies
1/30/2024
Could a platform approach help certain therapies make their way over the regulatory finish line more efficiently?
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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.
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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.
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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.
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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.
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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.
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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.