A new nonprofit hopes to make gene therapy development for rare diseases faster by creating treatments that function as “routine procedures,” in contrast to current development and approval pathways, which frame each gene therapy for a rare disease as a new drug.
‘Too Rare to Care’? A New Center for Rare Diseases Hopes to Change That
A new nonprofit wants to streamline gene therapy for diseases often avoided by pharmaceutical companies — making treatment more like a routine procedure than a bespoke drug.
nytimes.com