In a press release issued today, Audentes Therapeutics provided updates on their AAV-based genetic medicine program for serious rare neuromuscular diseases. This included an update on their gene therapy product candidate AT845 for Pompe disease. They’ve now completed an initial NHP safety study for AT845. They’re also working on GLP toxicology and dose-ranging studies to support a third quarter Investigational New Drug (IND) submission. In their last press release they projected that they’d be in a position for an IND submission in 2019. It’s good to see that it’s advancing as planned.

Positive data was presented at the Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) for their Phase 1/2 study of AT132 for X-Linked Myotubular Myopathy (XLMTM). The programs for Pompe and XLMTM are the two Gene Replacement therapies in their pipeline. Other programs are Vectorized Exon Skipping and Vectorized RNA Knockdown therapies.

Audentes also recently announced expansion of their pipeline to include Duchenne muscular dystrophy. Their initial work will address about 25% of patients with plans to expand that to eventually target as many as 80%.


Tagged: gene therapy, IND, preclinical