Audentes Therapeutics focuses on developing gene therapy clinical programs aimed at treating rare diseases. The company serves patients and families affected by conditions such as X-Linked Myotubular Myopathy (XLMTM), Pompe Disease, Duchenne Muscular Dystrophy, and Myotonic Dystrophy Type 1. Operating within the biotechnology and pharmaceutical market, Audentes employs a patient-centric business model, integrating patient experiences and feedback into their development processes. Revenue is generated through the development and potential commercialization of gene therapies, as well as partnerships and collaborations with other biotech firms and research institutions. The company provides educational materials and resources to support the rare disease community, ensuring that patients and their families are well-informed and engaged throughout the clinical trial process.

Keywords: gene therapy, rare diseases, clinical development, patient-centric, biotechnology, pharmaceutical, X-Linked Myotubular Myopathy, Pompe Disease, Duchenne Muscular Dystrophy, Myotonic Dystrophy.