scAAV9.U7-ACCA

scAAV9.U7-ACCA is an investigational gene therapy designed for patients with Duchenne muscular dystrophy (DMD) who have a duplication of exon 2 in the DMD gene. The therapy uses a self-complementary adeno-associated virus serotype 9 (scAAV9) vector to deliver four copies of a modified U7 small nuclear RNA (U7snRNA), each containing antisense sequences targeting the splice donor and acceptor sites of DMD exon 2. This approach induces efficient skipping of duplicated exon 2 during mRNA processing, aiming to restore production of full-length or near full-length dystrophin protein in muscle cells. The therapy is administered systemically and has shown promising results in preclinical models and early human trials, including evidence of restored dystrophin expression and improved muscle function[1][2][3][4][5]. The primary developer is Nationwide Children’s Hospital; Astellas Gene Therapies/Audentes Therapeutics are also associated with its development[4][5][6][7].

Other names

non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding RNA