ATSENA THERAPEUTICS is evaluating ATSN-101 in an ongoing Phase I/II clinical trial (ClinicalTrials.gov Identifier: NCT03920007) in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). This program, which has the potential to be a major advance in reversing pediatric blindness, is based on more than 15 years of research and preclinical evidence from the founders’ laboratory at University of Florida. Atsena is exploring partnering and out-licensing options to advance ATSN-101 into a pivotal clinical trial.
LCA1 is a monogenic eye disease that disrupts the function of the retina. It 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 group of inherited retinal diseases. There are currently no approved treatments for LCA1.
Atsena has received Rare Pediatric Disease (RPD) designation, Regenerative Medicine Advanced Therapy (RMAT) designation, and 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 12-month results from the Phase I/II clinical trial of ATSN-101 for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1) at the 2024 Macula Society 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.
