Bringing patients into focus
with ocular gene therapy

ATSENA THERAPEUTICS is a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness. We have two ongoing Phase I/II clinical trials evaluating potential therapies for X-linked retinoschisis (XLRS) and GUCY2D-associated Leber congenital amaurosis (LCA1). Learn more about Our Programs.

Our Technology

Our pipeline of clinical and preclinical assets is powered by an adeno-associated virus (AAV) technology platform tailored to overcome the hurdles presented by inherited retinal disease, and our approach is guided by the specific needs of each patient condition.

Learn about Our Approach.

X-linked Retinoschisis

Atsena is leveraging one of its novel spreading capsids, AAV.SPR, in a clinical program for X-linked retinoschisis (XLRS), a monogenic eye 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.


accessibility optionsAccessibility
Text Size