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…
The FDA accepted Dyne Therapeutics’ biologics license application for zeleciment rostudirsen and granted Priority Review for Duchenne muscular dystrophy amenable to exon 51 skipping. The milestone places Dyne’s antibody-oligonucleotide conjugate at the center of…
SonoThera closed an oversubscribed $125M Series B financing to advance DMD and ADPKD programs toward the clinic, validating its non-viral genetic medicine delivery platform.
