Notable enhancements were observed in Microperimetry (MP), Best Corrected Visual Acuity (BCVA), and Low Luminance Visual Acuity (LLVA) across all dose levels.

Kenji Fujita, M.D., Atsena's Chief Medical Officer, highlighted the therapy's safety profile and its ability to effectively deliver gene therapy across the retina.

Importantly, no serious adverse events were directly linked to the therapy, with most reported adverse events being mild and associated with the injection process.

Atsena Therapeutics has announced promising early-stage data from Part A of the LIGHTHOUSE study, a Phase I/II trial evaluating its gene therapy ATSN-201 for X-linked retinoschisis (XLRS).

This study marks the first XLRS trial to demonstrate both efficacy and safety, with most patients achieving significant improvements in visual function and structural measurements.

Atsena's recent funding will support ongoing clinical development, including work on gene therapy for Leber congenital amaurosis type 1 and a preclinical program targeting MYO7A-associated Usher syndrome.

Atsena's innovative approach with the AAV.SPR capsid allows for lateral spread beyond injection sites, minimizing surgical risks and enhancing treatment effectiveness.

Specifically, seven out of nine patients experienced closure of foveal schisis post-treatment, a condition not observed in untreated eyes, highlighting the therapy's effectiveness.

One patient did experience a serious fever unrelated to the treatment, and no patients discontinued the trial or exhibited dose-limiting toxicities.

The trial involved nine adult patients divided into three cohorts, each receiving the therapy in one eye as part of the phase 1/2 Lighthouse trial.

The second phase of the trial is currently underway, aiming to enroll nine additional adult patients and three children, with FDA designations for fast track, rare pediatric disease, and orphan drug status already secured.

Despite the positive outcomes for Atsena, the broader biotech sector is facing challenges, with significant layoffs occurring in early 2025, particularly within cell and gene therapy companies.