Atsena Therapeutics Reports Positive Data for AAV Gene Therapy in X-linked Retinoschisis Study

DURHAM, NC – May 19, 2025 – Atsena Therapeutics announced positive clinical data from Part A of its Phase I/II LIGHTHOUSE study evaluating ATSN-201, an AAV gene therapy, for X-linked retinoschisis (XLRS). The AAV therapy utilizes the company’s novel AAV.SPR capsid for targeted gene delivery to the central retina without surgical foveal detachment.

Part A of the study, involving nine adult XLRS patients, demonstrated a favorable safety profile with no treatment-related serious adverse events across three dose levels of the AAV gene therapy. Notably, 7 of 9 treated eyes showed closure of foveal schisis, validating the AAV.SPR capsid’s spread and efficacy. The AAV therapy also showed preliminary evidence of efficacy at all doses, with improvements in Microperimetry and statistically significant improvements in visual acuity.

Part B of the study is currently enrolling additional adult and pediatric patients to further evaluate the safety and efficacy of this AAV gene therapy.

Atsena Therapeutics CEO Patrick Ritschel highlighted the significance of these data, underscoring the promise of their novel AAV capsid for treating XLRS, a disease with no approved therapies.

XLRS, affecting approximately 30,000 males in the U.S. and EU, is caused by RS1 gene mutations. The LIGHTHOUSE study (NCT05878860) is ongoing. Atsena’s AAV.SPR capsid has shown superior retinal spread compared to traditional AAV vectors in preclinical studies.