|Alcyone Therapeutics is mission-driven to overcome the challenges in central nervous system (CNS) therapy development through applying precision CNS delivery & dosing technologies to novel gene therapy technologies.|
Alcyone’s pipeline includes 12 AAV (adeno-associated virus) gene therapy programs and four gene therapy platform technologies targeting severe CNS disorders. Lead programs include ACTX-101, a gene therapy for Rett syndrome using X-reactivation technology and currently in pre-IND enabling studies, and ACTX-401, a gene therapy for spinal muscular atrophy with respiratory distress type 1 (SMARD1) currently in IND stage.
Alcyone’s four gene therapy platform technologies include X-reactivation, conventional transgene replacement, vectorized exon skipping and promotor modulation. The X-reactivation platform, which is used in our lead candidate, ACTX-101 for Rett syndrome, has the potential to become the gene therapy industry’s first “pipeline within a product” gene therapy, where the same gene product can be applied to potentially treating other X-related syndromes.
Precise and optimal delivery has a profound impact on both efficacy and safety of gene therapy. Alcyone has developed a transformational CNS delivery technology platform that provides control of biodistribution to the targets of interest. This has the potential to move the frontier of the field by impacting safety, efficacy and potentially the amount of vectors needed for an effective therapy.
Our company headquarters are located in Lowell, MA. The Moscow, ID office focuses on early-stage targeted central nervous system drug delivery research and interacts across the Alcyone Therapeutics’ early pre-clinical to clinical programs.