Sarepta reported new ELEVIDYS analyses at the World Muscle Society meeting. Twenty-five patients dosed at ages eight to 12 showed a 3.32-point North Star Ambulatory Assessment advantage at two years versus 99 matched natural-history…
SonoThera appointed Adam J. Shaywitz, M.D., Ph.D., chief medical officer. He joins after serving as chief medical officer of BridgeBio Gene Therapy and brings experience across AAV gene therapy, rare disease, enzyme replacement and…
Exegenesis Bio and Modalis Therapeutics entered a research collaboration and license agreement giving Modalis access to EMC181, an engineered muscle-tropic, liver-detargeted adeno-associated virus capsid, for MDL-201. The program combines that capsid with Modalis’s CRISPR-GNDM…
Researchers led by Helmholtz Munich and the Technical University of Munich built more than 100 synthetic transfer vehicles by combining natural RNA-packaging, membrane-binding and budding domains with AI-designed protein assemblies. The lead planar C8…
FDA extended the PDUFA target action date for Capricor’s deramiocel BLA from August 22 to November 22, 2026, after the company submitted a major amendment. The amendment adds 24-month open-label extension data from Phase…
Precision BioSciences disclosed that the first participant received PBGENE-DMD in the Phase 1/2a FUNCTION-DMD trial. The program uses two ARCUS nucleases delivered in one AAV vector to excise DMD exons 45–55 and create an…
FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 that the evidence did not support approval of deramiocel for cardiomyopathy associated with Duchenne muscular dystrophy. The vote is nonbinding, but it materially increases…
FDA briefing materials concluded that Capricor’s HOPE-3 study did not meet its prespecified efficacy endpoints and raised questions about changes in analysis and data interpretation. Capricor disputes the agency’s chronology and interpretation. The event…
Sarepta Therapeutics appointed Michael Severino, M.D., chief executive officer and a director, effective July 28, 2026. He succeeds Doug Ingram, whose retirement plan was disclosed on February 25; Ingram will remain an advisor through…
Dyne launched a $300 million underwritten public offering one day after FDA acceptance and Priority Review of the z-rostudirsen BLA. An additional 30-day underwriter option could increase gross proceeds to $345 million. The timing…
