Great seeing the @U.S. FDA talk more about the Plausible Mechanism Pathway. This kind of clarity and intent are what is needed to move new complex modalities further. Particularly interesting to me how i) they tie approval to underlying biology (not just symptoms), ii) they emphasise the relevance of natural history of untreated disease, and iii) they explicitly note that the same thinking can be applied to modalities beyond gene editing. https://www.nejm.org/doi/full/10.1056/NEJMsb2512695?af…

nejm.org/doi/full/10.10…

FDA’s New Plausible Mechanism Pathway | NEJM

Personalized therapies hold tremendous promise but challenge traditional models of drug and biologic development. The FDA outlines a path to market entry for products where a randomized trial is no...