About
Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company has two clinical-stage programs, ATSN-201 for XLRS and ATSN-101 for LCA1. The company is preparing to initiate The Lighthouse Study, a Phase I/II clinical trial evaluating ATSN-201, which leverages the novel spreading capsid AAV.SPR, in patients with X-linked retinoschisis (XLRS). The company's additional proprietary asset is ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Interim safety and efficacy data from the company’s ongoing Phase I/II clinical trial in patients with GUCY2D-associated Leber congenital amaurosis (LCA1) have demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 6 months post-treatment.
DurhamBiotechnology