Audentes is committed to developing and commercializing safe and effective gene therapy products for patients living with serious, rare neuromuscular diseases with limited or no treatment options. We appreciate the urgency of bringing these innovative products 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.
Our Approach to Clinical Development for Rare Disease
A robust and comprehensive clinical development program – designed with input from experts in the medical community, regulatory authorities and the patient community – is the optimal way to bring an approved therapy to the entire patient community.
Clinical trials are designed and implemented to gain an understanding of the safety and efficacy of investigational new therapies in specific populations. Participation in clinical trials accepted by the U.S. Food and Drug Administration (FDA), U.K. Medicines and Healthcare Products Regulatory Agency (MHRA), or other regulatory authorities, is the best way for patients to access investigational therapies 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 gene therapy products, and to rapidly obtain the global regulatory approvals needed to make these therapies widely available to the patients that need them around the world.
Expanded Access
We understand the importance of the topic of expanded access (also known as “compassionate use” or “early access”). Per regulatory guidelines, expanded access programs must not interfere with ongoing or planned clinical trials designed to enable approval allowing access for the broader patient community.
X-Linked Myotubular Myopathy (XLMTM)
Our priority is to demonstrate the safety and effectiveness of AT132, our gene therapy product for XLMTM, in order to obtain regulatory approval and make it available to appropriate patients as rapidly as possible. We do this by running a thoughtfully designed and robust clinical trial program.
We are currently unable to offer expanded access for AT132 and we believe that participation in one of our clinical trials is the most appropriate way to access our investigational therapies.
Other Programs in Development
At this time, participation in clinical trials is the only way for patients to gain access to investigational therapies that are currently early in development.
More Information
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 www.clinicaltrials.gov, which contains additional information about these studies.
For physicians or healthcare providers interested in more information, please contact [email protected]. We anticipate acknowledging receipt of questions or request for more information within five (5) business days of receipt.
For parents or patients interested in general information, please contact Patient Advocacy at [email protected] and talk with your treating physician.