DURHAM, N.C., April 20, 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 independent Data Monitoring Committee (DMC) for the Phase 1/2/3 LIGHTHOUSE Trial evaluating ATSN-201 for the treatment of X-linked retinoschisis (XLRS) has completed its review of accumulated data from Parts A and B of the study and has recommended that the Company proceed with enrollment of the pivotal Part C cohort. Atsena will initiate Part C screening this month.
"We are excited that the DMC has recommended that we proceed with the pivotal Part C cohort," said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics. "This recommendation reflects the strength of the safety and efficacy data we have accumulated to date and brings us one step closer to delivering what we believe will be the first approved therapy for patients with XLRS."
Part C Enrollment Details
Part C of the LIGHTHOUSE Trial will serve as the Phase 3 pivotal portion of the study, enrolling a total of 76 patients with XLRS across leading clinical sites in North America and Europe. Patients will be separated into two groups: treatment and control. Patients in the treatment arm will receive bilateral or unilateral treatment with ATSN-201, while patients in the control arm will be observed for 12 months and then have the option to receive treatment with ATSN-201. Patient screening is expected to begin in April 2026, with enrollment expected to be completed by the end of the first quarter of 2027. Data from the Phase 3 cohort are expected to support a Biologics License Application (BLA) filing in 2028.
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 the U.S. and EU have XLRS, for which there are currently no approved 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 currently being evaluated in the Phase 1/2/3 LIGHTHOUSE Trial, which consists of three parts (A, B and C) and six cohorts. The Phase 1/2 portion includes cohorts 1-3 (Part A) and cohorts 4-5 (Part B), while cohort 6 (Part C) will serve as the Phase 3 portion of the study. ATSN-201 is the first XLRS gene therapy to demonstrate preliminary evidence of efficacy and safety in a Phase 1/2 trial, with the majority of patients demonstrating 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 for at least one year post-treatment. This best-in-class gene therapy product candidate 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. Enrollment for this study is ongoing. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860).
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. This is 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 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 that is typically diagnosed in childhood and leads to blindness later in life. The company’s proprietary pipeline also includes gene therapies in development for Usher Syndrome Type 1B and for Stargardt Disease. Atsena is also developing ATSN-101, a first-in-class, 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.
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/.
Investor and Media Contact: Argot Partners atsena@argotpartners.com
Business Contact: info@atsenatx.com
