FDA releases draft guidance to streamline approval of personalized gene therapies

The FDA announced a draft guidance aimed at expanding access to gene therapies that target different mutations within the same disease.

The proposal outlines an "individualized feature space" that would let manufacturers obtain initial approval for a therapy addressing one mutation using conventional data, then seek additional clearances for other rare mutations based on laboratory data rather than full clinical trials.

This approach is intended to address the challenge of conducting trials for ultra-rare mutations where patient numbers are too small for traditional studies.

The guidance follows earlier FDA steps to accelerate personalized treatments, building on the momentum generated by a high-profile gene-editing case in 2025.