Atsena Therapeutics Gains FDA Designation for ATSN-201 to Treat X-linked Retinoschisis

Atsena Therapeutics, a company dedicated to gene therapy research aimed at combating blindness, has announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for its product ATSN-201. This gene therapy is designed to treat X-linked retinoschisis (XLRS), a genetic condition leading to vision impairment and blindness. This marks the second RPD designation the company has received this year, highlighting the potential efficacy of Atsena's gene therapy technology in addressing critical unmet needs for patients with inherited retinal diseases.

Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics, expressed his satisfaction with the FDA’s decision, noting that it underscores the promise of their technology for patients with inherited retinal conditions. He emphasized the company's commitment to advancing ATSN-201 through clinical trials to offer new hope for those affected by XLRS.

XLRS is a genetic disorder that predominantly affects males and is typically diagnosed in early childhood. There are approximately 30,000 affected males in the United States and the European Union. Currently, no approved treatments for XLRS exist. The condition is characterized by a splitting of the retinal layers, leading to impaired visual acuity, progressive vision loss, and ultimately blindness.

The ongoing Phase I/II clinical trial, named LIGHTHOUSE, is assessing the safety and tolerability of ATSN-201. This trial is open-label, dose-escalation, and dose-expansion in nature, focusing on male patients aged six years and older who have a clinical diagnosis of XLRS caused by mutations in the RS1 gene. The study is currently enrolling participants.

The FDA’s Rare Pediatric Disease designation is awarded to treatments intended for serious or life-threatening rare diseases that primarily affect individuals under 18 years of age. This designation makes ATSN-201 eligible for a priority review voucher (PRV) upon approval. This PRV could be used to expedite another internal program or could be sold to another company, providing significant incentives for companies like Atsena to develop therapies for rare pediatric conditions.

Atsena’s ATSN-201 uses AAV.SPR, a novel spreading capsid, to achieve therapeutic levels of gene expression in the photoreceptors of the central retina while avoiding the risks associated with foveal detachment. Preclinical studies in non-human primates have shown that AAV.SPR facilitates transgene expression beyond the subretinal injection site without causing inflammation, unlike traditional AAV vectors which remain localized within the original injection margins. This technology allows for the efficient transduction of foveal cones at clinically relevant doses without surgical intervention.

Atsena Therapeutics, founded by experts in ocular gene therapy, is advancing its gene therapy pipeline to tackle inherited retinal diseases. The company's lead program, ATSN-201, is currently in a Phase I/II trial for XLRS. Another candidate, ATSN-101, is undergoing clinical evaluation for Leber congenital amaurosis type 1 (LCA1), a common cause of childhood blindness. Atsena's innovative adeno-associated virus (AAV) technology is designed to address the specific challenges of treating inherited retinal diseases.

With a strong focus on developing groundbreaking treatments, Atsena Therapeutics aims to make significant strides in reversing or preventing blindness caused by genetic conditions, improving the quality of life for patients and their families.