ATSENA is evaluating ATSN-101, our investigational gene therapy for Leber congenital amaurosis type 1 (LCA1), in collaboration with Nippon Shinyaku Co., Ltd. Nippon Shinyaku holds rights to ATSN-101 in the United States and Japan. ATSN-101 has completed a Phase 1/2 clinical trial (ClinicalTrials.gov Identifier: NCT03920007) with positive results, and Atsena is advancing the program toward a global pivotal trial later this year. This program is based on more than 20 years of research and preclinical evidence from the founders’ laboratory at the University of Florida.
Disease Background
LCA1 is a monogenic eye disease caused by biallelic mutations in the GUCY2D gene, which disrupts retinal function and results in early and severe vision impairment or blindness. GUCY2D-associated LCA1 is one of the most common forms of Leber congenital amaurosis. There are currently no approved treatments for LCA1.
Clinical Development
The Phase 1/2 trial of ATSN-101 demonstrated a favorable safety profile and durable improvements in vision across 15 patients with LCA1. Patients treated with the high dose demonstrated clinically meaningful improvements in dark-adapted full-field stimulus testing (FST), with a mean improvement of approximately 20 decibels — a 100-fold gain in light sensitivity (up to 10,000 fold gain in some patients) — that has been sustained through at least three years post-treatment. Adults with LCA1 experienced striking recovery of night vision within days of receiving treatment. ATSN-101 was well-tolerated with no drug-related serious adverse events and no patient discontinuations. Twelve-month results were published in The Lancet, and additional findings were reported in the journal iScience. Thirty-six month results were presented in May 2026 at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting.
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.
Thirty-six month results were presented in May 2026 at the Association for Research in Vision and Ophthalmology (ARVO) 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.
FDA Designations
Atsena has received three FDA designations for ATSN-101:
- Regenerative Medicine Advanced Therapy (RMAT)
- Rare Pediatric Disease (RPD), and
- Orphan Drug Designation (ODD).
