Ep. 117 - C-Path's Klaus Romero on moving genetic medicines beyond one patient

The Critical Path Institute’s One to Millions Initiative is using individualized rare disease therapies as the starting point for a development framework that could eventually extend across all genetically defined diseases. The initiative seeks to bring form to FDA’s Plausible Mechanism Framework by standardizing how evidence and methods can be reused across therapies and patients.In conversation with Biopharma Analyst Dr. Tierney Baum on The BioCentury Show, C-Path CEO and CSO Klaus Romero discusses how the principal bottleneck is no longer the ability to design therapies for individual patients but rather the absence of a standardized regulatory and development system that can translate one success into many. C-Path plans to bring companies, academics, patient groups, and regulators together to share data and address recurring development bottlenecks.View full story: https://www.biocentury.com/article/660583#GeneticMedicines #RareDisease #DrugDevelopment #RegulatoryScience #ClinicalTrials00:00 - Introduction01:44 - Current Framework07:01 - Process-based Regulation13:57 - Overcoming Bottlenecks20:28 - Use of AI25:25 - Beyond Rare Diseases