About Atsena
Company Overview
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 best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases.

Our lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a progressive genetic condition affecting boys and men that is typically diagnosed in childhood. Our other ongoing Phase I/II clinical trial is evaluating ATSN-101 for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children.

Our pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, we are led by an experienced team dedicated to addressing the needs of patients with vision loss.


Our mission is to improve the quality of life of patients with inherited retinal disease through the discovery and development of gene therapies that prevent or reverse blindness.


We strive to create technologies that enable targeted solutions for ocular diseases.

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.

X-linked Retinoschisis

Atsena is leveraging one of its novel spreading capsids, AAV.SPR, in a clinical program for X-linked retinoschisis (XLR), a monogenic disease that causes progressive vision loss.


Leber Congenital Amaurosis 1

Atsena has an ongoing Phase I/II clinical trial evaluating a gene therapy for patients with GUCY2D-associated Leber congenital amaurosis (LCA1), a monogenic eye disease that affects the retina.



Usher Syndrome 1B

Atsena is making rapid advancements in evaluating a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B), an inherited disease that affects the retina and the inner ear.


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