Atsena's Gene Therapy ATSN-201 Receives FDA RMAT Status for X-linked Retinoschisis

Atsena Therapeutics, a company at the forefront of gene therapy, has announced a significant milestone in its quest to combat inherited retinal diseases. The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-201, Atsena's innovative gene therapy candidate aimed at treating X-linked retinoschisis (XLRS). This recognition adds to a series of FDA designations granted to ATSN-201, including Fast Track, Rare Pediatric Disease, and Orphan Drug designations, underscoring its potential to address critical medical needs in conditions with no current treatments.

Atsena's ATSN-201 utilizes AAV.SPR, a groundbreaking capsid technology designed to achieve therapeutic gene expression in the retinal photoreceptors without the complications associated with foveal detachment surgery. This approach addresses the challenges of treating XLRS, a genetic condition that leads to progressive vision loss and affects approximately 30,000 males in the United States and Europe.

The RMAT program, established under the 21st Century Cures Act, aims to expedite the development and review of promising regenerative therapies, offering enhanced guidance and possible accelerated approval pathways for treatments addressing serious health conditions. The RMAT designation for ATSN-201 emphasizes its potential to meet the urgent, unmet needs of people with XLRS.

XLRS is a hereditary disorder caused by mutations in the RS1 gene, responsible for producing retinoschisin, a critical protein in the retina. This condition is characterized by the splitting of the retinal layers, leading to impaired vision and eventual blindness. Diagnosed typically in early childhood, XLRS predominantly affects males and currently lacks any approved medical treatments.

Atsena's ongoing clinical trial, known as the LIGHTHOUSE study, is a Phase I/II trial evaluating the safety and tolerability of ATSN-201 in male patients with a confirmed XLRS diagnosis. This trial involves a dose-escalation and dose-expansion approach to ascertain the optimal therapeutic effect of the gene therapy. Enrollment is ongoing, indicating a proactive move towards addressing a significant gap in ophthalmic care.

The innovative AAV.SPR capsid technology deployed in ATSN-201 represents a significant leap forward in gene therapy techniques. Unlike traditional methods that remain localized to the initial injection site, AAV.SPR spreads beyond these boundaries, promoting extensive transgene expression across the central retina. This is particularly advantageous for treating XLRS, where schisis cavities predominantly affect the retina's central region. Preclinical studies in non-human primates have demonstrated that AAV.SPR effectively transduces retinal cells, offering a safer alternative to surgical interventions.

Atsena Therapeutics, founded by leaders in ocular gene therapy, is dedicated to reversing or preventing blindness caused by inherited retinal diseases. The company's commitment is evident in its strategic collaborations and continued development of cutting-edge treatments. In addition to ATSN-201, Atsena is advancing ATSN-101, a promising gene therapy for Leber congenital amaurosis type 1 (LCA1), which has shown positive outcomes in preliminary trials.

With a focus on leveraging novel adeno-associated virus (AAV) technologies, Atsena is at the forefront of developing therapies that could transform the lives of individuals affected by genetic retinal diseases. The company's dedication to innovation and patient care continues to drive its efforts in bringing life-changing treatments to those suffering from vision loss.