The US Food and Drug Administration (FDA) has released a final guidance document with immediate effect advising sponsors on chemistry, manufacturing, and controls (CMC) flexibilities for cell and gene ...

CMC hurdles stall Korea drug approvals as KDDF and KCMC mobilize support National push aims to build end‑to‑end CMC capacity ...

FDA’s draft “plausible mechanism” framework gives individualized genetic therapies a clearer path to market, but questions remain, write experts at Morrison Foerster.

The US Food and Drug Administration (FDA) last week finalized guidance to assist sponsors developing human gene therapy (GT) products for neurodegenerative diseases. In the final version, FDA acceded ...

This is part two of a two-part article offering proposals to improve the FDA’s draft guidance to implement a new approval framework for individualized genetic therapies based on a “plausible mechanism ...

FDA feedback centered on CMC documentation and quality/manufacturing controls, a common first-cycle barrier that typically does not necessitate additional efficacy or safety trials. Pediatric ...