ATSN-201 / Atsena Therap - LARVOL DELTA

Atsena Therapeutics Receives Orphan Designation from the European Medicines Agency (EMA) for ATSN-101 and ATSN-201 (GlobeNewswire) - "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."

New P3 trial • Orphan drug • Ophthalmology • Retinal Disorders

Atsena Therapeutics recently announced that the first patient has been dosed in the phase 3 LIGHTHOUSE trial evaluating ATSN-201 for the treatment of x-linked retinoschisis (XLRS). (Eyes On Eyecare) - The company anticipates completing enrollment by the end of Q1 2027, with topline results anticipated in H1 2028. Pending positive data: A Biologics License Application (BLA) filing is targeted for H2 2028.

FDA filing • P3 data: top line • Trial status • Genetic Disorders

Safety and efficacy of ATSN-201 in patients with X-linked retinoschisis: 12-month dose finding results of the LIGHTHOUSE study (ASGCT 2026) - "All three dose levels demonstrate efficacy signals in the majority of patients. The dose expansion phase of the study will further explore safety and efficacy of the low dose in adult and pediatric patients."

Clinical • Age-related Macular Degeneration • Gene Therapies • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

Safety and efficacy of ATSN-201 in patients with X-linked retinoschisis: 12-month dose finding results of the LIGHTHOUSE study (ARVO 2026) - "Conclusions ATSN-201 is well-tolerated, with treated eyes demonstrating improvements in both structure and function that are durable through 12 months post-treatment. The dose expansion phase of the study will further explore safety and efficacy of the low dose in adult and pediatric patients."

Clinical • Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

LIGHTHOUSE: ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis (clinicaltrials.gov) - P3 | N=97 | Recruiting | Sponsor: Atsena Therapeutics Inc. | Phase classification: P1/2 ➔ P3 | N=21 ➔ 97 | Trial completion date: Oct 2029 ➔ Apr 2033 | Trial primary completion date: Oct 2025 ➔ Apr 2028

Enrollment change • Phase classification • Trial completion date • Trial primary completion date • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Laterally spreading AAV.SPR enables safe and efficient RS1 delivery to the macula after peripheral subretinal injection. (PubMed, Mol Ther) - P1/2 | "Despite being a secreted protein, biodistribution of RS1 remains confined to the area of AAV-RS1 transduction. Having established feasibility for the approach, we performed preclinical proof of concept, safety and efficacy studies in support of 'ATSN-201' (NCT05878860)...A GLP Toxicology study in NHP established safety at the highest dose evaluated. The enhanced transduction and lateral spreading ability of AAV.SPR make it an attractive option for treating inherited retinal diseases including, but not limited to, XLRS."

Journal • Age-related Macular Degeneration • Inflammation • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

Atsena Therapeutics Announces Alignment with FDA on Regulatory Pathway to Approval for ATSN-201 in X-Linked Retinoschisis (XLRS) (Atsena Press Release) - "Atsena Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has agreed to the expansion of the company’s ongoing Phase 1 / 2 LIGHTHOUSE study of ATSN-201 into a continuous Phase 1 / 2 / 3 trial, enabling it to serve as a pivotal trial to support a Biologics License Application (BLA) submission for the treatment of X-linked retinoschisis (XLRS). The BLA submission is anticipated in early 2028...The pivotal cohort is anticipated to begin enrolling in the first quarter of 2026 with a pivotal data readout expected in the second half of 2027."

FDA filing • Trial status • Genetic Disorders • Ophthalmology

Safey and Efficacy of ATSN-201 Dose Escalation in Patients with X-linked Retinoschisis (XLRS) (ASGCT 2025) - "The presence of schisis cavities in the central retina poses a challenge to safe and effective delivery of AAV-RS1 to XLRS patients. In conclusion, ATSN-201 is a promising treatment approach for patients with XLRS, a disease for which no treatments currently exist. Disease Focus of Abstract:Ophthalmic Diseases"

Clinical • Age-related Macular Degeneration • Gene Therapies • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

Atsena Therapeutics Granted U.S. FDA Regenerative Medicine Advanced Therapy Designation for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis (GlobeNewswire) - "Atsena Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for ATSN-201 for the treatment of X-linked retinoschisis (XLRS)."

FDA event • Ophthalmology

Interim safety and efficacy data in XLRS patients validates the use of a novel, laterally spreading capsid for the treatment of retinal disease (ARVO 2025) - "The goal of this phase 1/2 dose escalation study is to evaluate the safety and preliminary efficacy at 3 dose levels of ATSN-201, a laterally spreading subretinal gene therapy, in patients with XLRS...No untreated eyes showed foveal schisis closure. Treated eyes with structural improvements also generally demonstrated improvements in MP average threshold."

Clinical • Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders

Atsena Therapeutics Granted U.S. FDA Fast Track Designation for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis (GlobeNewswire) - "Atsena Therapeutics...today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment...The safety and tolerability of ATSN-201 is currently being evaluated in the LIGHTHOUSE study, a Phase I/II, dose-escalation and dose-expansion clinical trial in male patients ages six and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene."

Fast track • Genetic Disorders • Ophthalmology

Atsena Therapeutics to Present ATSN-201 Safety and Efficacy Data at The Macula Society’s 48th Annual Meeting (GlobeNewswire) - "Atsena Therapeutics...announced that safety and efficacy data on ATSN-201 for the treatment of X-linked retinoschisis (XLRS) will be presented during a panel discussion at The Macula Society’s 48th Annual Meeting taking place February 12-15, 2025....The LIGHTHOUSE study is a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial evaluating the safety and tolerability of ATSN-201 in male patients ages six and older with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1."

P1/2 data • Genetic Disorders • Ophthalmology

Atsena Therapeutics Initiates Part B of Phase I/II Clinical Trial Evaluating Gene Therapy ATSN-201 to Treat X-linked Retinoschisis (GlobeNewswire) - "Atsena Therapeutics...today announced the initiation of Part B of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS)....Part A of the study evaluated the safety and tolerability of three different doses of ATSN-201 administered through subretinal injection."

Trial status • Genetic Disorders • Ophthalmology

Interim Safety and Efficacy of ATSN-201 Dose Escalation Study in Patients With X-linked Retinoschisis (XLRS) (AAO 2024) - No abstract available

Clinical • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Atsena Therapeutics Receives Orphan Drug Designation from the U.S. FDA for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis (GlobeNewswire) - "Atsena Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment....The safety and tolerability of ATSN-201 is currently being evaluated in the LIGHTHOUSE study...Enrollment for this study is ongoing."

Enrollment status • Orphan drug • Genetic Disorders • Ophthalmology

Atsena Therapeutics Receives Rare Pediatric Disease Designation from the U.S. FDA for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis (GlobeNewswire) - "Atsena Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation (RPD) for ATSN-201 for the treatment of X-linked retinoschisis (XLRS)....The safety and tolerability of ATSN-201 is currently being evaluated in the LIGHTHOUSE study, a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial...Enrollment for this study is ongoing."

Enrollment status • FDA event • Genetic Disorders • Ophthalmology

IND‐enabling studies to support the clinical development of ATSN‐201, a subretinally delivered, laterally spreading gene replacement therapy for X‐Linked Retinoschisis (XLRS) (ESGCT 2023) - "A single, subretinal injection of ATSN-201 significantly improved retinal structure and function, supporting the clinical use of ATSN-201 for the treatment of XLRS. The ability of the novel AAV.SPR capsid to effectively deliver the RS1 to the central retina of primates following extrafoveal SRI represents a safer and potentially more effective treatment option for XLRS patients."

Clinical • Age-related Macular Degeneration • CNS Disorders • Inflammation • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders • SPR

LIGHTHOUSE: ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: Atsena Therapeutics Inc.

Gene therapy • Trial completion date • Trial primary completion date • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

LIGHTHOUSE: ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: Atsena Therapeutics Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

LIGHTHOUSE: ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis (clinicaltrials.gov) - P1/2 | N=18 | Not yet recruiting | Sponsor: Atsena Therapeutics Inc.

Gene therapy • New P1/2 trial • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders