Atsena Therapeutics Completes Dosing in Phase I/II Trial for ATSN-201 Gene Therapy for X-linked Retinoschisis

Atsena Therapeutics, a company at the forefront of gene therapy, has recently reached a significant milestone in its efforts to combat X-linked retinoschisis (XLRS), a genetic condition leading to blindness. The company has completed dosing in Part A of the LIGHTHOUSE study, a Phase I/II clinical trial. This trial is focused on evaluating the safety and efficacy of ATSN-201, a promising gene therapy candidate, administered via subretinal injection. ATSN-201 utilizes Atsena's innovative AAV.SPR spreading capsid technology, which facilitates therapeutic gene expression in the central retina's photoreceptors, minimizing the surgical risks typically associated with foveal detachment.

In this initial phase of the study, nine adult participants received ATSN-201 at varying dose levels. According to Dr. Kenji Fujita, Atsena’s Chief Medical Officer, the trial has shown encouraging results, delivering both structural and functional improvements in patients across all tested doses. Importantly, no serious adverse events related to the treatment have been observed. The data collected from this phase is pivotal as it not only validates the effectiveness of Atsena's novel capsid in treating inherited retinal diseases but also informs the safest and most effective strategies for advancing to Part B of the trial.

XLRS is a rare genetic disorder primarily affecting males and is usually diagnosed in early childhood. It is characterized by schisis, or the abnormal splitting of the retinal layers, which results in impaired visual acuity that glasses cannot correct, often leading to blindness. Unfortunately, there are currently no approved treatments for this condition, which impacts approximately 30,000 males in the United States and European Union.

Patrick Ritschel, Atsena's CEO, expressed excitement about completing this phase of the trial, emphasizing that it represents a crucial step forward for patients with XLRS. The positive outcomes noted in the trial so far provide hope for a future therapeutic option for individuals with this condition, as well as for those affected by other inherited retinal diseases who might benefit from the company's novel capsid technology. Ritschel underscored Atsena’s commitment to exploring new approaches in ocular gene therapy, aiming to leverage insights from the LIGHTHOUSE study to further their innovative treatments.

The LIGHTHOUSE study is an open-label, dose-escalation, and dose-expansion clinical trial. It aims to evaluate the safety and tolerability of ATSN-201 in male patients aged six and older diagnosed with XLRS due to mutations in the RS1 gene. This gene encodes retinoschisin, a protein secreted by photoreceptors, and the mutations result in the characteristic retinal layer splitting seen in XLRS. The trial's enrollment is ongoing, and ATSN-201 has already received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration, highlighting its potential impact.

Atsena Therapeutics was established by experts in ocular gene therapy and is backed by a team with extensive experience in addressing vision loss. The company is dedicated to developing top-tier treatments to reverse or prevent blindness caused by inherited retinal diseases. Their pipeline is driven by cutting-edge adeno-associated virus (AAV) technology designed to overcome the specific challenges posed by these conditions. In addition to ATSN-201, Atsena is also advancing ATSN-101 for Leber congenital amaurosis type 1 (LCA1), another major cause of childhood blindness. With a successful Phase 1/2 trial completed, ATSN-101 is moving towards a pivotal trial, further exemplifying Atsena's commitment to transforming the landscape of gene therapy for ocular diseases.