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.

Atsena’s pipeline of clinical and preclinical assets is powered by adeno-associated virus (AAV) technologies tailored to overcome the hurdles presented by inherited retinal disease, and our approach for each program is guided by the specific needs of each patient condition. Our lead candidate, ATSN-101, is being evaluated in an ongoing Phase I/II clinical trial for one of the most common causes of blindness in children. We are also advancing programs in X-linked retinoschisis and MYO7A-associated Usher syndrome.

Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is led by a team with deep expertise in AAV gene therapy, ophthalmology, and CMC. The company is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise.

Mission

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.

Vision

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.

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.

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X-linked Retinoschisis

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

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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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