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 focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness.

The company has an ongoing Phase I/II clinical trial evaluating a potential therapy for one of the most common causes of blindness in children. Its pipeline of clinical and preclinical assets is powered by adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal disease, and its approach is guided by the specific needs of each patient condition.

Founded by pioneers in ocular gene therapy, Atsena has a licensing, research, and manufacturing collaboration with the University of Florida and is headquartered in North Carolina’s Research Triangle, an environment rich in gene therapy expertise.

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 genetic eye disease that affects the retina.

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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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