ATA-100

ATA-100 is a one-time gene replacement therapy developed for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), a rare genetic disorder caused by mutations in the fukutin-related protein (FKRP) gene. The therapy uses an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the human FKRP gene to muscle cells, aiming to restore normal protein function and halt or reverse disease progression. Preclinical and early clinical data have shown that ATA-100 can improve muscle function, increase glycosylation of α-dystroglycan, reduce creatine kinase levels, and enhance quality-of-life measures in treated patients. The drug is administered as a single intravenous infusion and has received Orphan Drug Designation from both the FDA and EMA, as well as Rare Pediatric Disease Designation and Fast Track Designation from the FDA[1][2][4][5][6][7][8][9].

Other names

ATMY-001ATMY001ATMY 001