ATSN-201 is currently being studied in the phase 1/2 LIGHTHOUSE trial, which is evaluating the safety and tolerability of the therapy in male patients aged six and older with XLRS caused by RS1 mutations.

In addition to ATSN-201, Atsena Therapeutics is advancing its other gene therapy candidate, ATSN-101, for Leber congenital amaurosis type 1, having completed a Phase I/II trial with positive outcomes and is preparing for a global pivotal trial.

Currently, there are no approved treatments for XLRS, highlighting the urgent need for effective therapies like ATSN-201.

Enrollment for the ongoing LIGHTHOUSE study is actively taking place, focusing on male patients diagnosed with XLRS due to mutations in the RS1 gene.

Approximately 30,000 males in the U.S. and Europe are affected by XLRS, a condition characterized by abnormal splitting of retinal layers that can lead to impaired vision and potential blindness.

XLRS primarily affects males due to its X-linked inheritance pattern and is caused by mutations in the RS1 gene, often diagnosed in early childhood.

The AAV.SPR capsid allows for effective transduction of retinal cells beyond the injection site without the need for surgical detachment, offering a favorable safety profile compared to traditional AAV vectors.

The FDA has granted Atsena Therapeutics' gene therapy candidate, ATSN-201, the regenerative medicine advanced therapy (RMAT) designation for treating X-linked retinoschisis (XLRS), a rare genetic disorder with no approved treatments.

Atsena recently closed an oversubscribed $150 million Series C financing, which will support the development of ATSN-201.

ATSN-201 utilizes a novel spreading capsid, AAV.SPR, which has shown in preclinical studies to effectively deliver therapeutic gene expression to retinal photoreceptors while minimizing surgical risks.

This designation marks the fourth FDA recognition for ATSN-201, which has also received fast track, rare pediatric disease, and orphan drug designations.

The RMAT designation is part of the 21st Century Cures Act, aimed at expediting the development and review process for promising therapies that address serious conditions with unmet medical needs.