FDA to review uniQure’s BLA for Huntington’s gene therapy
The BLA relies on findings from Phase I/II study compared with an external cohort from the Enroll-HD natural history registry.
Cytiva Navigating cell therapy WP 4
FDA calls for stronger potency upkeep in CGT manufacturing
Casgevy gains UK label expansion for younger patient population
Estuary secures $103m to advance kidney disease gene therapies
CAR-T specialist Caribou culls pipeline and seeks exit deal
FDA approves first gene therapy for Sanfilippo syndrome type A
Cellares and GenomeFrontier to assess automated GF-CART01 production
Encoded secures $275m for Dravet syndrome gene therapy
