Atsena Therapeutics reported 18-month results from the phase 1/2 study of the LIGHTHOUSE trial showing sustained structural and functional changes following treatment with ATSN-201, an investigational gene therapy for X-linked retinoschisis (XLRS). The findings were presented by Laura Pardon, OD, PhD, at the American Academy of Optometry 2026 Annual Meeting in Anaheim, California. Nine adults received 1 of 3 doses of ATSN-201 by subretinal injection in 1 eye.

At 18 months, foveal schisis closure was maintained in 7 of 9 treated eyes and was not observed in untreated eyes. Six treated eyes achieved an improvement of at least 7 dB on microperimetry, compared with none of the untreated eyes. Seven treated eyes had gains of at least 10 letters in either best-corrected or low-luminance visual acuity, compared with 1 untreated eye. No drug-related serious adverse events, dose-limiting toxicities, or treatment discontinuations were reported.

ATSN-201 uses the AAV.SPR capsid, designed to spread laterally from the subretinal injection site and target central photoreceptors without requiring foveal detachment. Enrollment is continuing in the 76-patient pivotal Part C cohort. Atsena expects enrollment to finish in the first quarter of 2027, with top-line results anticipated in the first half of 2028.