- Regenxbio hits Duchenne gene therapy milestone, eyes 2027 FDA approvalRegenxbio said Thursday that its Duchenne muscular dystrophy gene therapy met the bar in a pivotal study, and it’s aiming for FDA approval in 2027. The biotech hopes to be the second company to c…Endpoints · 2mo ago↗ source
- BeOne’s next-gen BCL2 inhibitor wins FDA approval, taking aim at VenclextaBeOne Medicines has clinched an accelerated approval for its drug sonrotoclax in mantle cell lymphoma (MCL), marking the start of its bid to challenge AbbVie and Roche's Venclexta across a range of…Endpoints · 2mo ago↗ source
- #ASGCT26: After an apparent cure, a gene editing company struggles to repeat the magicBOSTON — Early last year, startup iECURE’s experimental gene therapy seemingly cured a baby of a life-threatening genetic disease. But new results testing the same therapy in a larger number of b…