AT751

**AT751** was an investigational, preclinical AAV-based vectorized exon-skipping gene therapy developed for Duchenne muscular dystrophy. It was designed to express an antisense RNA approach that promotes skipping of exon 51 in dystrophin pre-mRNA, aiming to restore the DMD reading frame and enable production of a shorter but potentially functional dystrophin protein in patients with mutations amenable to exon 51 skipping. The program originated at Audentes Therapeutics and was subsequently owned by Astellas following its acquisition of Audentes. Astellas terminated AT751 development in April 2022 following review of preclinical data. ([academic.oup.com](https://academic.oup.com/hmg/article/28/R1/R102/5522633))