Audentes Therapeutics is a biotechnology company committed to the development and commercialization of innovative new gene therapy treatments for people with serious rare diseases. We are a focused, experienced and passionate team driven by the goal of improving the lives of patients.
We currently have four programs in development:
- AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM), an ultra-rare, severe, debilitating disease that affects skeletal muscles in an estimated one in 50,000 newborn males worldwide, and is caused by mutations in the MTM1 gene
- AT342 for the treatment of Crigler-Najjar Syndrome (CN), a rare, congenital autosomal recessive monogenic disease characterized by severely high levels of bilirubin in the blood and risk of irreversible neurological damage and death that affects approximately one in 1,000,000 newborns
- AT307 for the treatment of CASQ2-related Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), an inherited disease characterized by abnormal ventricular heartbeats (arrhythmias) that is estimated to affect more than 6,000 people globally
- AT982 for the treatment of Pompe disease, a rare, inherited disorder characterized by progressive muscle weakness and respiratory impairment that affects one in every 40,000 births