Gene Therapy Breakthrough Restores Vision to Blind Patients

Researchers at the University of Florida have developed a groundbreaking gene therapy for Leber congenital amaurosis type I (LCA1), a rare genetic blindness affecting about 3,000 people in the U.S. and Europe, SciTech Daily reported on Friday.

In a small trial, patients experienced life-changing improvements in vision, with some seeing stars or snowflakes for the first time. Those receiving the highest dose saw up to a 10,000-fold increase in light sensitivity and improved navigation abilities.

The therapy demonstrated a clean safety profile, with minor side effects, and is poised for a phase 3 clinical trial pending FDA approval. Shannon Boye, who has worked on this therapy for over 20 years, co-founded Atsena Therapeutics to bring these treatments to market. The study enrolled 15 subjects, with results published in The Lancet. Researchers expect the therapy to provide lasting benefits with just a single treatment per eye, potentially transforming the lives of many with LCA1.