One to Millions will begin with shared ASO safety data, but its ambitions extend far beyond rare disease.
Critical Path Institute’s One to Millions Initiative is using individualized rare disease therapies as the starting point for a development framework that could ultimately 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.
On The BioCentury Show, C-Path CEO and CSO Klaus Romero said the principal bottleneck is no longer the ability to design therapies for individual patients. It is the absence of a standardized regulatory and development system that can translate one success into many.
“We’re not reinventing the wheel. We are trying to maximize what has been done into a joint movement that can meet the vision of what FDA laid out in the guidance,” Romero said.
View the whole episode via BioCentury, here.
