Also known as: AAV9
Solid Biosciences has secured FDA alignment on a registration pathway for its Duchenne muscular dystrophy gene therapy SGT-003, with 36 patients dosed to date showing no drug-induced liver injury, myocarditis, or other serious adverse events reported with competing therapies.
Affinia Therapeutics has received FDA fast track designation and EMA orphan drug designation for AFTX-201, an investigational gene therapy for BAG3-associated dilated cardiomyopathy, a rare genetic heart disease affecting over 70,000 patients.
Benitec Biopharma will present interim clinical study results for BB-301 gene therapy in oculopharyngeal muscular dystrophy patients at the Muscular Dystrophy Association Conference on March 9, 2026, including 12-month and 24-month follow-up data.
The U.S. Food and Drug Administration has approved an Investigational New Drug application for FRF-001, a viral gene therapy for FOXG1 syndrome developed at the University at Buffalo. The first-in-human trial will be independently sponsored by the FOXG1 Research Foundation.
