Atsena Therapeutics Receives EMA Orphan Designation For Two Gene Therapy Candidates

Atsena Therapeutics, a clinical-stage gene therapy company focused on developing genetic medicines for inherited eye diseases that can cause blindness, has announced that the European Medicines Agency (EMA) has granted orphan designation to its two lead clinical-stage gene therapy candidates. The designations were awarded to ATSN-101, which is being developed for the treatment of Leber congenital amaurosis type 1 (LCA1), and ATSN-201, which is being developed for X-linked retinoschisis (XLRS).

The EMA's decision recognizes the potential of both investigational therapies to address rare inherited retinal diseases for which there are currently limited or no approved treatment options. Orphan designation is intended to encourage the development of therapies for rare, serious, or chronically debilitating diseases by providing regulatory and financial incentives to companies advancing these treatments.

Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics, said the orphan designations highlight the significant unmet medical needs faced by patients living with LCA1 and XLRS. He noted that the Company continues to make progress across both clinical programs, with enrollment advancing rapidly in the pivotal Phase 3 trial of ATSN-201 and preparations underway to begin the global pivotal Phase 3 trial of ATSN-101 later this year. Ritschel added that these milestones bring the Company closer to delivering potential gene therapy treatments for patients in Europe, the United States, and other international markets.

The European Medicines Agency grants orphan designation to medicines intended for the treatment, prevention, or diagnosis of rare diseases that are life-threatening or chronically debilitating. The designation offers several development incentives, including reduced regulatory fees, scientific and clinical protocol assistance, access to research grants, and up to 10 years of market exclusivity within the European Union if the therapy receives marketing approval.

In the United States, both ATSN-101 and ATSN-201 have already received several important regulatory designations from the U.S. Food and Drug Administration (FDA). These include Orphan Drug Designation, Rare Pediatric Disease Designation, Fast Track Designation, and Regenerative Medicine Advanced Therapy (RMAT) Designation, reflecting the therapies' potential to address serious unmet medical needs and supporting their clinical development.

Atsena Therapeutics also provided updates on the progress of its clinical programs. In June 2026, the Company dosed the first patient in the pivotal Phase 3 cohort of the LIGHTHOUSE trial evaluating ATSN-201 for X-linked retinoschisis, with patient enrollment continuing to progress. Later this year, Atsena plans to initiate a global pivotal Phase 3 clinical trial of ATSN-101 for Leber congenital amaurosis type 1. The development of ATSN-101 is being carried out in collaboration with Nippon Shinyaku Co., Ltd.

Through these regulatory milestones and ongoing clinical studies, Atsena Therapeutics continues to advance its gene therapy pipeline with the goal of developing potential treatments that could help preserve or restore vision in patients affected by rare inherited retinal diseases.