ATSENA THERAPEUTICS is evaluating ATSN-101 in an ongoing Phase I/II clinical trial (ClinicalTrials.gov Identifier: NCT03920007) in patients with GUCY2D-associated Leber congenital amaurosis (LCA1), a monogenic eye disease that disrupts the function of the retina. 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 a group of monogenic, autosomal recessive diseases that are the leading cause of blindness in children. LCA1 is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA, affecting approximately 20 percent of patients who live with this debilitating inherited retinal disease. LCA1 is two times more prevalent than LCA2. There are currently no approved treatments for LCA1.
Atsena has received orphan drug designation from the U.S. Food and Drug Administration for its investigational gene therapy product for the treatment of GUCY2D-associated LCA1.
Atsena presented positive results from the Phase I/II clinical trial of ATSN-101 for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1) at the American Academy of Ophthalmology 2022 Annual Meeting.
View the presentation here.
Additional findings from our trial have been reported in the journal iScience. Adults with LCA1 experienced striking recoveries of night vision within days of receiving ATSN-101.
View the paper here.