Atsena Therapeutics Receives Orphan Designation from the European Medicines Agency (EMA) for ATSN-101 and ATSN-201
Orphan designation provides market exclusivity and development benefits in the European Union as both programs advance in global pivotal clinical trials
DURHAM, N.C., July 23, 2026 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, today announced that the European Medicines Agency (EMA) has granted orphan designation to its two clinical-stage gene therapy product candidates being developed for inherited retinal diseases: ATSN-101 for Leber congenital amaurosis 1 (LCA1), and ATSN-201 for X-linked retinoschisis (XLRS).
"Receiving orphan designation from the EMA for both ATSN-101 and ATSN-201 underscores that these programs address significant unmet needs for patients who currently have no treatment options," said Patrick Ritschel, Chief Executive Officer of Atsena. "With our pivotal trial for ATSN-201 enrolling rapidly and our global pivotal trial for ATSN-101 on track to begin later this year, we are closer than ever to delivering gene therapies that can change the trajectory of these inherited retinal diseases for patients in the U.S., Europe and beyond."
The EMA grants orphan designation to drugs and biologics intended for the treatment, diagnosis, or prevention of rare, life-threatening, or chronically debilitating diseases or conditions. Orphan designation provides companies with certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants, and up to 10 years of market exclusivity in the EU.
In the United States, the U.S. Food and Drug Administration (FDA) has previously granted Orphan Drug, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations to both ATSN-101 and ATSN-201.
Atsena dosed the first patient in the pivotal Phase 3 cohort of the LIGHTHOUSE trial with ATSN-201 in June 2026 and enrollment is progressing rapidly. Later this year, Atsena expects to initiate a global, pivotal Phase 3 clinical trial evaluating ATSN-101, which is being developed in collaboration with Nippon Shinyaku Co., Ltd.
About X-linked Retinoschisis (XLRS)
XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene, which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss and ultimately blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in North America and Europe have XLRS, for which there are currently no approved, disease-specific treatments.
About ATSN-201
ATSN-201 is Atsena's investigational gene therapy leveraging AAV.SPR, a novel, laterally spreading capsid designed to efficiently target photoreceptors in the central retina while avoiding the surgical risks of foveal detachment. It is being evaluated in the LIGHTHOUSE trial, which consists of three parts (A, B and C) across six cohorts. Parts A and B comprised the Phase 1/2 portion of the study, testing different dose concentrations and injection volumes, including in pediatric patients. Part C is the pivotal Phase 3 portion of the study, a randomized, controlled trial of approximately 76 patients ages 6 and older, with microperimetry as the primary endpoint and a 52-week primary readout. The pivotal cohort began dosing in June 2026 and is enrolling ahead of expectations at sites in the U.S., with additional sites in Europe and the United Kingdom (U.K.) expected to open later this year. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860).
In the completed Phase 1/2 portion of the study, ATSN-201 was the first XLRS gene therapy to demonstrate evidence of efficacy and safety, with the majority of patients showing improvements in retinal structure (foveal schisis closure) and meaningful improvements in retinal and visual function as assessed by microperimetry, best-corrected visual acuity and low-luminance visual acuity. ATSN-201 has demonstrated a favorable safety profile and has been well-tolerated, with data now extending out to two years in some patients. ATSN-201 has received Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration and Orphan Designation from the European Medicines Agency.
About AAV.SPR
AAV.SPR, one of Atsena's novel capsids, spreads laterally beyond the subretinal injection site to enable safe and efficient transduction of the central retina when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins, in contrast to benchmark AAV vectors, which remain confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and has a favorable safety profile relative to benchmark capsids. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/.
About GUCY2D-associated Leber Congenital Amaurosis (LCA1)
LCA1 is a monogenic eye disease that disrupts retinal function. It is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. LCA1, one of the most common forms of LCA, has no approved treatments.
About ATSN-101
ATSN-101 is an investigational gene therapy that has completed a Phase 1/2 clinical trial for LCA1. At 36 months post-treatment with the high dose, patients demonstrated durable, clinically significant improvements in vision, and ATSN-101 was well tolerated with no drug-related serious adverse events reported. Atsena expects to initiate a global pivotal Phase 3 clinical trial for ATSN-101 later this year, with sites planned across the U.S., Europe and Japan. ATSN-101 is being developed as part of a strategic collaboration with Nippon Shinyaku Co., Ltd., which holds exclusive rights in the United States and Japan; Atsena retains rights for the rest of the world. ATSN-101 has received Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration and Orphan Designation from the European Medicines Agency.
About Atsena Therapeutics
Atsena is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company's lead program is evaluating ATSN-201 in a pivotal clinical trial for X-linked retinoschisis (XLRS), a genetic condition typically diagnosed in childhood that leads to blindness later in life. Atsena is also developing ATSN-101, an investigational gene therapy for Leber congenital amaurosis type 1 (LCA1), as part of its exclusive strategic collaboration with Nippon Shinyaku Co., Ltd.; ATSN-101 has completed a Phase 1/2 trial with positive results (https://doi.org/10.1016/s0140-6736(24)01447-8), and Atsena expects to initiate a global pivotal Phase 3 clinical trial evaluating ATSN-101 in the second half of 2026. The company's proprietary pipeline also includes gene therapies in development for Usher syndrome type 1B and Stargardt disease. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit https://atsenatx.com/.
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