In this episode of Science with a Twist, Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research (CBER) at the FDA, joins host Dr. Timothy Miller to dive into the complexities and promise of gene therapy.
They discuss the potential of gene therapy to revolutionize medicine, narrowing in on the scientific progress made and recent advancements in treating patients and families with Rare Diseases.
Dr. Marks shares insights on the FDA's role in ensuring safety and efficacy, emphasizing the importance of collaboration between regulatory bodies, researchers, and developers.
Efforts related to the profround work of the public/private partnerships funding the Accelerating Medicines Partnerships Bespoke Gene Therapy Consotritum are also highlighted.
