Atsena Therapeutics

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. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise.