Atsena Therapeutics presented updated clinical data on 2 investigational inherited retinal disease gene therapies during the 2026 Association for Research in Vision and Ophthalmology (ARVO) meeting in Denver.

The company reported 12-month results from part A of the phase 1/2/3 LIGHTHOUSE trial evaluating ATSN-201 for X-linked retinoschisis (XLRS). According to data presented by Lesley Everett, MD, PhD, of the Casey Eye Institute at Oregon Health & Science University in Portland, Oregon, the therapy demonstrated a favorable safety profile in 9 adult patients, with no drug-related serious adverse events, dose-limiting toxicities, or discontinuations. Foveal schisis closure persisted in 7 of 9 treated eyes at 12 months, and investigators reported statistically significant improvements in microperimetry, best-corrected visual acuity, and low-luminance visual acuity.

Atsena said enrollment is ongoing for the pivotal phase 3 portion of the LIGHTHOUSE study, with a biologics license application planned for 2028.

Separately, Artur Cideciyan, PhD, of the Scheie Eye Institute at the University of Pennsylvania in Philadelphia, presented 36-month data from a phase 1/2 study of ATSN-101 in GUCY2D-associated Leber congenital amaurosis type 1. Patients receiving the high dose maintained an approximately 20 dB improvement in dark-adapted full-field stimulus testing through 3 years after treatment. No drug-related serious adverse events or discontinuations were reported.

The company also presented data on a modified multi-luminance mobility test intended to better detect functional vision changes in patients with retained rod function. Atsena plans to incorporate the modified endpoint into the planned phase 3 trial of ATSN-101, expected to begin in the second half of 2026.