Atsena Therapeutics Reports Promising Phase 1/2 Results for XLRS Gene Therapy

Atsena Therapeutics, a gene therapy company focused on ocular diseases, has unveiled encouraging early-stage data for its X-linked retinoschisis (XLRS) treatment, ATSN-201. The results, presented at the American Society of Cell & Gene Therapy annual meeting in New Orleans, demonstrate both safety and efficacy in the first part of the phase 1/2 Lighthouse trial.

Safety Profile and Structural Improvements

The study, which included nine patients with XLRS, showed no serious treatment-related adverse events across all three dose cohorts. Most reported side effects were mild and associated with the subretinal injection used to administer the therapy. Notably, seven out of nine patients experienced closure of foveal schisis, indicating significant structural improvement in the treated eye.

Dr. Kenji Fujita, Chief Medical Officer at Atsena, emphasized the importance of these findings: "We're particularly pleased that the foveal schisis closure seen in seven of nine patients validates AAV.SPR's ability to spread laterally beyond the subretinal injection blebs and enable safe gene delivery to the central retina."

Functional Benefits and Visual Acuity

In addition to structural improvements, the trial data revealed statistically significant enhancements in visual acuity among treated patients. This functional benefit, coupled with the observed safety profile, suggests that ATSN-201 could potentially address a critical unmet need in XLRS treatment, for which no approved therapies currently exist.

Strategic Positioning and Future Directions

The positive results come on the heels of Atsena's recent $150 million Series C funding round, which will support the continued clinical development of ATSN-201. The therapy has already received fast track, rare pediatric disease, and orphan drug designations from the FDA, highlighting its potential impact on XLRS patients.

As Atsena moves forward with the second phase of the Lighthouse trial, which will include both adult and pediatric patients, the company is positioned at the forefront of gene therapy development in a challenging market landscape. With approximately 31% of biotech layoffs in Q1 2025 affecting cell and gene therapy companies, Atsena's progress and recent funding success stand out as a beacon of promise in the field.