
Encoded Therapeutics is a clinical-stage genetic medicines company developing potentially one-time, disease-modifying therapies for severe neurological disorders. Its proprietary vector engineering technologies combine novel regulatory elements and payloads within AAV vectors to unlock innovative solutions for intractable conditions. The company's lead clinical candidate, ETX101 for Dravet syndrome, is designed to target the underlying cause of the disorder through selective upregulation of SCN1A for potentially long-lasting benefit. Encoded’s pipeline also includes a development-stage vectorized miRNA-based gene therapy designed to restore expression of UBE3A in individuals with Angelman syndrome. In parallel, the company is advancing potentially best-in-class programs for common neurological conditions, including chronic pain and Alzheimer’s disease / tauopathies.