Audentes Therapeutics is a biotechnology company committed to the development and commercialization of innovative new gene therapy treatments for people with serious rare diseases through the application of adeno-associated virus gene therapy technology.
We have four programs in our pipeline:
- AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM). For more information about XLMTM, please go here.
- AT342 for the treatment of Crigler-Najjar Syndrome (CN).
- AT307 for the treatment of CASQ2-related Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), For more information about CASQ2-related CPVT, please go here.
- AT982 for the treatment of Pompe disease. For more information about Pompe disease, please go here.
Results from preclinical studies to date have been encouraging. We plan to complete preclinical studies with the goal of initiating clinical trials in patients as soon as possible.