Audentes Therapeutics, Inc.

Based in California, US, and founded in 2012, Audentes Therapeutics, Inc. d. b. a.

Astellas Gene Therapies, develops and commercializes treatments for patients with the serious, rare disease through the application of gene therapy technology. In January 2020, Astellas Pharma Inc. completed the acquisition of Audentes Therapeutics, Inc. and operating as a wholly-owned subsidiary.

As of October 2021, Audentes Therapeutics, Inc. has raised a total of USD 137. 5 in funding. The company offers innovative therapies for X-linked Myotubular Myopathy (XLMTM), Pompe Disease, Duchenne muscular dystrophy (DMD), and Myotonic dystrophy type 1 (DM1) diseases.

For X-linked Myotubular Myopathy (XLMTM), the company is developing the novel product candidate AT132, an AAV8 vector containing a functional copy of the MTM1 gene. For Pompe disease, the company is developing AT845, which is a novel gene replacement investigational therapy to address the recognized limitations of ERT (enzyme replacement therapy) by targeting the muscle tissues. For Duchenne muscular dystrophy (DMD), the company is developing AT702, which is an AAV-antisense gene therapy to induce exon 2 skipping for the treatment of DMD patients with duplications in exon 2 and mutations in exons 1 – 5 of the dystrophin gene. For myotonic dystrophy type 1 (DM1), the company is developing an AAV therapy that is being investigated to determine whether it can deliver molecules for RNA knockdown and exon skipping, both designed to prevent toxic RNA from accumulating in affected cells, thereby restoring normal function.

The company plans to build a new 135,000 square foot, state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina, which will be creating over 200 new jobs in 2022.