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. The second cohort in the trial is expected to be dosed in the second half of 2020. This rapidly advancing program is based on more than 15 years of research and preclinical evidence from the founders’ laboratory at University of Florida.
LCA is the most common cause of blindness in children, impacting two to three children per 100,000. LCA1 is caused by mutations in the GUCY2D gene and results is early and severe vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this debilitating inherited retinal disease.