Atsena Therapeutics – Company Overview

Focused on bringing the life-changing power of genetic medicine – to reverse or prevent blindness

ATSENA THERAPEUTICS is a clinical-stage gene therapy company developing first- and best-in-class treatments for inherited retinal diseases — conditions that cause progressive, irreversible vision loss and for which few or no approved therapies exist.

Our lead program is evaluating ATSN-201 in a pivotal Phase 3 trial for X-linked retinoschisis (XLRS), a genetic condition typically diagnosed in childhood that causes progressive vision loss in boys and men. ATSN-201 is the first XLRS gene therapy to demonstrate reversal of structural damage to the retina and improvements in visual function in a clinical trial. Our second clinical program evaluating ATSN-101 for Leber congenital amaurosis type 1 (LCA1) has completed a Phase 1/2 trial with positive results published in The Lancet, and is advancing toward a global pivotal trial in partnership with Nippon Shinyaku Co., Ltd.

Our pipeline is powered by proprietary AAV technology platforms, including our novel laterally spreading AAV.SPR capsid and dual vector technology, engineered to overcome the unique biological challenges of delivering gene therapy to the retina. Beyond our two clinical programs, we are advancing earlier-stage candidates for Usher Syndrome 1B and Stargardt disease and multiple undisclosed discovery programs.

Core Values

Patients & Families: We are dedicated to improving the quality of life of patients with vision loss.

People: We are committed to employing an inclusive, diverse, talented workforce, regardless of location, and treating all people with respect and compassion. We actively optimize virtual collaboration and communication practices and strive for an efficient physical footprint, which allows for an effective balance of in-person and virtual teamwork.

Science: We pride ourselves on being at the cutting edge of ocular gene therapeutics and on bringing them to the clinic with excellence, integrity, and urgency.

Quality & Stewardship: We are committed to responsible stewardship of the resources entrusted to us, as well as 100% compliance to ICH principles and transparency and accountability in all aspects of our business.

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.

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.

Focused on bringing the life-changing power of genetic medicine – to reverse or prevent blindness

Mission

Vision

Core Values

X-linked Retinoschisis

Leber Congenital Amaurosis 1

Usher Syndrome 1B

Stargardt Disease