Atsena reports preliminary ATSN-201 gene therapy results in X-linked retinoschisis
Published in Drugs development & discovery.
October 7, 2026 — Atsena Therapeutics announced preliminary findings from Parts A and B of LIGHTHOUSE, its study of investigational retinal gene therapy ATSN-201 for X-linked retinoschisis.
Across 18 treated patients, the company reported that 13 responded on at least one visual-function measure. Nine were microperimetry responders and nine were visual-acuity responders; 12 showed an OCT structural response. Among three untreated control patients, none responded on OCT or microperimetry, while one responded on visual acuity.
Atsena also reported sustained structural and functional improvements at three years in the earliest-treated patient. These small, preliminary cohorts and individual follow-up observations do not establish the magnitude or durability of benefit for the broader patient population.
The pivotal Part C is a randomized controlled study of 76 patients aged five and older. Enrollment completion is expected in the first quarter of 2027, with topline results anticipated in the first half of 2028. ATSN-201 remains investigational.
Source: Atsena announcement, October 7, 2026.