Expanded Access Policy 2017-09-21T08:40:21+00:00

Expanded Access Policy

Audentes is committed to developing and commercializing safe and effective gene therapies for patients living with serious, life-threatening genetic diseases. We appreciate the urgency of bringing these innovative treatments to patients, and this commitment drives our work each day. We believe in the importance of collaborating closely with patients, their families, patient advocacy organizations, physicians, researchers and regulatory authorities to help achieve this goal.

Clinical trials are designed and implemented to gain an understanding of the safety and effectiveness of new therapies in specific populations. Participation in clinical trials accepted by the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA) or other regulatory authorities, is the best way for patients to access investigational treatments for their diseases. It is our hope and intention to quickly enroll eligible patients into clinical studies in order to demonstrate the safety and efficacy of our investigational therapies, and to rapidly obtain the global regulatory approvals needed to make these treatments widely available to patients around the world.

At this time, participation in clinical trials is the only way for patients to gain access to investigational therapies in development by Audentes. We recognize that not all patients will meet the eligibility requirements for these trials, and as more information and clinical data on the safety and efficacy of our investigational therapies become available, this policy may change.

Treating physicians, patients and/or caregivers interested in learning more about Audentes’ investigational gene therapies currently undergoing clinical studies can find more information here. The linked webpages also include links to, which contains additional information about these studies.

For more information, please contact medinfo(at) We anticipate acknowledging receipt of questions or request for more information within five (5) business days of receipt.