​Atsena Therapeutics, founded in 2020 and based in Durham, North Carolina, is a clinical-stage gene therapy company dedicated to developing treatments for inherited retinal diseases (IRDs) that lead to blindness. The company's lead candidate, ATSN-201, is designed to treat X-linked retinoschisis (XLRS), a genetic disorder causing splitting of the retinal layers, leading to vision impairment and potential blindness in males. ATSN-201 utilizes AAV.SPR, Atsena's proprietary spreading capsid, to achieve therapeutic gene expression in central retinal photoreceptors without the need for surgical foveal detachment. ​

In March 2025, the U.S. FDA granted Fast Track designation to ATSN-201, facilitating its development and review process. This recognition complements previous Orphan Drug and Rare Pediatric Disease designations, underscoring the therapy's potential to address the unmet medical needs of approximately 30,000 males affected by XLRS in the U.S. and EU. ​

To support the advancement of its ocular gene therapy programs, Atsena closed an oversubscribed $150 million Series C financing round in April 2025. The funding aims to propel ATSN-201 through clinical development and regulatory approval processes. The ongoing LIGHTHOUSE Phase I/II clinical trial is evaluating the safety and tolerability of ATSN-201 in male patients aged six and older with confirmed RS1 gene mutations. Enrollment for this study is active, reflecting the company's commitment to delivering transformative therapies for individuals with inherited retinal diseases.