Tag: #geneediting

FDA Issues Draft Guidance on Using Next-Generation Sequencing to Assess Genome-Editing Safety

FDA's draft guidance explains how developers should use next-generation sequencing to detect off-target edits and chromosomal damage before human trials.

#geneediting, #fda, #ngs, #genetherapy

FDA proposes new pathway to approve one-patient gene-editing drugs for ultra-rare diseases

New FDA draft guidance would let individualized CRISPR and RNA therapies win approval with tiny studies, using strong biology and natural-history data.

#fda, #geneediting, #raredisease, #crispr, #drugapproval