rAAV9.U7.ACCA

rAAV9.U7.ACCA (also known as AT702) is an investigational gene therapy designed for the treatment of Duchenne muscular dystrophy (DMD) in patients with exon 2 duplications or mutations in exons 1-5 of the dystrophin gene. It consists of a self-complementary adeno-associated virus serotype 9 (scAAV9) vector that delivers four copies of a modified, non-coding U7 small nuclear RNA (U7snRNA) containing antisense sequences targeting the splice donor and splice acceptor sites of DMD exon 2. This vectorized exon-skipping approach promotes the exclusion of the duplicated exon 2, thereby restoring the reading frame and enabling the expression of full-length or highly functional N-truncated dystrophin protein. The therapy was originally developed by researchers at Nationwide Children's Hospital and licensed to Audentes Therapeutics (later acquired by Astellas Gene Therapies). In April 2022, Astellas announced the termination of the AT702 program based on preclinical data, although the investigator-initiated clinical trial (NCT04240314) at Nationwide Children's Hospital continued or completed.

Other names

AAV9.U7-ACCAAAV-9.U7-ACCAAAV 9.U7-ACCAscAAV9.U7-ACCAscAAV-9.U7-ACCAscAAV 9.U7-ACCAscAAV9.U7.ACCAscAAV-9.U7.ACCAscAAV 9.U7.ACCA