UF startup Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, presented interim six-month results from Part B of the Phase 1/2/3 LIGHTHOUSE Trial evaluating ATSN-201 in patients with X-linked retinoschisis (XLRS) at the Foundation Fighting Blindness Retinal Therapeutics Innovation Summit on May 1, 2026, in Denver, CO.
“Part B of our LIGHTHOUSE study is delivering exactly as we expected — a favorable safety profile, schisis resolution, and functional improvements at six months that closely replicate what we observed at the same time point in Part A of the study. Foveal schisis closure was observed in four of six treated adults, and there was no structural change observed in untreated control subjects or untreated contralateral eyes. The kinetics of microperimetry response across Cohort 4 mirror what we saw in Part A of the study. No serious adverse events were observed in any Part B cohort, including in our pediatric patients,” said Shannon Boye, PhD, Co-Founder and Chief Scientific Officer of Atsena.
