Alcyone Therapeutics

Alcyone Therapeutics is a biotechnology company dedicated to the precision treatment of neurological disorders. The company integrates innovations in neuroscience, precise dosing platforms, and in-house manufacturing capabilities to deliver transformative therapies to patients. Mission-driven, we collaborate with a team of gene therapy and industry leaders to overcome challenges in CNS therapeutic development by applying precise CNS delivery and dosing technologies to novel gene therapy approaches. Alcyone’s pipeline currently includes 12 AAV (adeno-associated virus) gene therapy programs and four gene therapy platform technologies targeting severe central nervous system disorders. Lead programs include ACTX-101, a gene therapy using X reactivation technology to treat Rett syndrome, which is currently in pre-IND enabling studies, and ACTX-401, a gene therapy for spinal muscular atrophy with respiratory distress type 1 (SMARD1), which is currently in the IND stage.