From The Editor | February 27, 2024

Video Clips From Cell & Gene Live: 2024 Regulatory Outlook With FDA's Dr. Peter Marks And Dr. Nicole Verdun


By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1


Back in January, our Cell & Gene Live, 2024 Regulatory Outlook With FDA's Drs. Peter Marks and Nicole Verdun, featured a rich discussion about improving communication with sponsors, increasing speed availability of cell and gene therapies by enabling manufacturing processes and standards development, and much more. Drs. Marks and Verdun shared an immense amount of information during our presentation, and you can view the 45-minute the full-length version on demand.

I know you’re busy though, and so, I’ve broken down the full-length presentation into bite sized video clips that you can watch or listen to at your convenience. Each clip centers on a specific topic, and I think you’ll find each one valuable and informative. Enjoy!

What's New for 2024: Dr. Nicole Verdun Offers a Look Inside the Office Of Therapeutic Products

In this first segment of the Cell & Gene Live, 2024 Regulatory Outlook with Dr. Peter Marks and Dr. Nicole Verdun, Dr. Verdun explains the Office of Therapeutic Products, the new super office, which is the result of a reorganization of CBER’s Office of Tissues and Advanced Therapies. She explains what’s on the Office of Therapeutic Products’ regulatory agenda for 2024.

How Past Gene Therapy Approvals Are Shaping the Future with Dr. Peter Marks

ARM’s recent briefing at 2024 JPM, ARM’s CEO, Tim Hunt stated there are up to 17 possible U.S. and EU approvals this year. In this segment, Dr. Peter Marks shared what he and his team have learned from current gene therapy approvals to accelerate future approvals.

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

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

Race to the Regulatory Finish Line: Dr. Nicole Verdun Talks Efficiencies in Platform Technologies

In this segment, Dr. Nicole Verdun covers why platform approaches may create regulatory efficiencies. She explains whether platform approaches may help put certain therapies over the regulatory finish line more efficiently.

Why Clinical Requirements Change: Dr. Peter Marks on Pre-IND Meeting Pathways

Here, Dr. Peter Marks shares how clinical requirements change after the FDA and a company agree on a pathway during a pre-IND meeting. He covered why might clinical requirements change and how the company might recover as a result.

Dr. Peter Marks Talks a Standardized Playbook for Gene Therapy Manufacturing

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

Dr. Nicole Verdun Shares How Patient Data Impacts CMC Robustness

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 and convert it to a pivotal study. Here, Dr. Verdun explains how the Agency evaluates patient data from a clinical endpoint and CMC robustness perspective from patients enrolled before and after the change.

CMC Comparability: Transitioning to Commercial Manufacturing with Dr. Nicole Verdun

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.

The Need for Speed: Dr. Peter Marks and Dr. Nicole Verdun Explain Meeting Unmet Need for Pediatric Patients

Here, Dr. Peter Marks and Dr. Nicole Verdun share how Sponsor companies can accelerate development in pediatric patients for genetic diseases that affect both children and adults, but where gene therapy holds the greatest promise to address unmet need in pediatric patients.

Dr. Peter Marks on the Adoption of Novel Precision Analytics

The FDA is very committed to improving the quality, consistency, and yields of advanced therapy products through the adoption of novel precision analytics. In this segment, Dr. Peter Marks shares advice for developers who might be worried that the Agency will not accept changes in their process that include a new analytical method.

Understanding AI And Machine Learning's Impact on CGT with Dr. Peter Marks and Dr. Nicole Verdun

During this segment, Dr. Peter Marks and Dr. Nicole Verdun share their take on how AI and machine learning will impact FDA regulation in 2024.

Collaborating with the FDA: Dr. Peter Marks and Dr. Nicole Verdun Share Advice for Small and Emerging Biotechs

In this segment, Drs. Marks and Verdun share their advice and best practices for small and emerging biotechs when working with the FDA.

2024 Regulatory Outlook with Dr. Peter Marks and Dr. Nicole Verdun | Audience Q&A

In this segment, Dr. Peter Marks and Dr. Nicole Verdun spent some time answering questions from the audience. They provided detailed responses on topics including pediatric development in rare disease, diversity planning for ultra rare disease, anticipated approvals by 2025, and more.