Atsena Therapeutics

Bringing patients into focus

with ocular gene therapy

ATSENA THERAPEUTICS is a clinical-stage gene therapy company developing first- and best-in-class treatments for inherited retinal diseases. Our lead program is evaluating ATSN-201 in a pivotal Phase 3 trial for X-linked retinoschisis (XLRS), a condition with no approved treatments today. Beyond XLRS, our pipeline includes ATSN-101 for LCA1, with a pivotal trial planned for later this year, earlier-stage gene therapies for Usher Syndrome 1B and Stargardt disease, and multiple undisclosed discovery programs.

Learn more about Our Programs.

Our Technology

Our proprietary AAV technology platforms, including our novel laterally spreading capsid AAV.SPR and dual vector technology, are engineered to overcome the unique biological challenges of delivering gene therapies to the retina.

Learn about Our Approach.

News

June 22, 2026

Atsena Therapeutics Doses First Patient and Reports Rapid Early Enrollment in Pivotal Trial of ATSN-201 for X-Linked Retinoschisis

May 7, 2026

Atsena Presents Positive Clinical Data from Its XLRS and LCA1 Gene Therapy Programs at ARVO 2026

More News

X-linked Retinoschisis

ATSN-201 | Phase 3 | AAV.SPR
ATSN-201 is the first gene therapy to demonstrate clinical efficacy in XLRS, a disease with no approved treatments, and is now enrolling in a pivotal Phase 3 trial.

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Leber Congenital Amaurosis 1

ATSN-101 | Phase 3 Planned H2 2026
Twelve-month Phase 1/2 data for ATSN-101 were published in The Lancet, and the program is advancing toward a global pivotal trial in partnership with Nippon Shinyaku Co., Ltd.

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Usher Syndrome 1B

ATSN-301 | IND-Enabling | AAV.SPR + Dual Vector
Powered by our novel AAV.SPR capsid, which enables peripheral subretinal delivery to protect fragile foveal cones in USH1B patients, and our dual vector technology to accommodate the large MYO7A gene, the program is IND-ready and poised to enter the clinic.

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Stargardt Disease

ATSN-401 | IND-Enabling | AAV.SPR + Dual Vector
Stargardt disease is a juvenile form of macular degeneration caused by mutations in the ABCA4 gene, affecting approximately 60,000 patients in the US and EU. AAV.SPR leads our STGD approach, addressing the disease’s unique delivery challenges, while dual vector technology, refined through our USH1B program, solves for the outsized ABCA4 payload.

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