Atsena Therapeutics Gets FDA Orphan Drug Status for ATSN-201 Gene Therapy for X-linked Retinoschisis

Atsena Therapeutics, a company at the forefront of gene therapy, has announced that its product ATSN-201 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for treating X-linked retinoschisis (XLRS). This gene therapy candidate, which previously received the Rare Pediatric Disease designation, aims to address an inherited retinal condition that currently lacks approved treatments.

ATSN-201 utilizes Atsena's proprietary spreading capsid, AAV.SPR, to deliver therapeutic levels of gene expression to photoreceptors in the central retina without the surgical risks associated with foveal detachment. This innovative approach could offer a new therapeutic option for those affected by XLRS, a disease primarily diagnosed in males during early childhood.

Patrick Ritschel, CEO of Atsena Therapeutics, expressed gratitude for the FDA's designation, emphasizing its importance in advancing the development of ATSN-201. Ritschel highlighted the commitment of the Atsena team to the XLRS program and their optimism that these regulatory milestones will accelerate the path to providing relief to patients with this rare disease.

Orphan Drug Designation is granted by the FDA to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases affecting fewer than 200,000 people in the United States. This designation offers incentives such as tax credits for clinical trial costs and waivers for prescription drug user fees. Furthermore, products with Orphan Drug Status are entitled to seven years of market exclusivity for the designated condition, apart from any existing intellectual property rights.

XLRS is a monogenic X-linked condition caused by mutations in the RS1 gene, which encodes the protein retinoschisin. This protein, secreted by photoreceptors, is essential for the structural integrity of the retina. The disease is characterized by schisis, or splitting of the retinal layers, leading to impaired visual acuity and progressive vision loss that eventually results in blindness. Approximately 30,000 males in the U.S. and EU are affected by XLRS.

The ongoing LIGHTHOUSE study, a Phase I/II clinical trial, is evaluating the safety and tolerability of ATSN-201. This open-label, dose-escalation and dose-expansion trial is enrolling male patients aged six and older with XLRS caused by RS1 gene mutations. The study aims to determine the appropriate dosage and gather preliminary data on the product's effectiveness.

Atsena's AAV.SPR capsid is a notable advancement in gene therapy delivery. Unlike traditional AAV vectors that remain confined to their original injection site, AAV.SPR spreads laterally, ensuring efficient transduction of the central retina. Preclinical studies have demonstrated that AAV.SPR promotes gene expression well beyond the initial injection area without inducing inflammation or requiring surgical detachment of the retina.

Atsena Therapeutics, founded by leaders in ocular gene therapy, is focused on developing treatments to reverse or prevent blindness caused by inherited retinal diseases. The company's lead program, ATSN-201, is being tested in the ongoing clinical trial for XLRS. Additionally, another Phase I/II trial is evaluating ATSN-101 for Leber congenital amaurosis type 1 (LCA1), a common cause of childhood blindness. Atsena's innovative approach leverages novel adeno-associated virus (AAV) technology to address the specific challenges of inherited retinal conditions.

With a dedicated team and a robust pipeline, Atsena Therapeutics is committed to bringing life-changing therapies to patients suffering from vision loss due to genetic diseases.