Gene Therapy Restores Vision
A new gene therapy ATSN-101 has restored vision in patients with Leber congenital amaurosis type I (LCA1), a rare genetic condition causing blindness. In a small trial, those receiving the highest dose saw up to a 10,000-fold improvement in light sensitivity and significant gains in reading and navigation abilities.
The therapy, developed by researchers, uses a virus-based system to deliver a functioning gene into the retina’s light-sensitive cells. The results show promise for expanding this treatment, with further trials planned to confirm safety and efficacy.
Key Facts:
Ø Gene therapy for LCA1 improved light sensitivity by up to 10,000-fold.
Ø Patients saw significant improvements in reading and navigating with the therapy.
Ø The treatment is expected to require just one dose per eye and last indefinitely.
After the treatment, one patient saw her first star. Another saw snowflakes for the first time. Other patients were newly able to navigate outside of the home or to read the labels on their child’s Halloween candy.
The cause of these seemingly miraculous improvements? A gene therapy developed by University of Florida scientists, which restored useful vision to most patients with the rare, inherited blindness known as Leber congenital amaurosis type I, or LCA1, in a small trial.
Those who received the highest dose of the gene therapy saw up to a 10,000-fold improvement in their light sensitivity, were able to read more lines on an eye chart, and improved in their ability to navigate a standardized maze. For many patients, it was akin to finally turning on dim lights after trying to navigate their homes in the pitch black for years, the researchers said.
The trial also tested the safety profile of the treatment. Side effects were largely limited to minor surgical complications. The gene therapy itself caused mild inflammation that was treated with steroids.
“This is the first time that anyone with LCA1 has ever been treated, and we showed a very clean safety profile, and we also showed efficacy. These results pave the way for advancing the therapy in a phase 3 clinical trial and eventually commercializing it,” said Shannon Boye, Ph.D., chief of the Division of Cellular and Molecular Therapy at UF, co-author of the study and co-founder of Atsena Therapeutics, the UF spinoff that developed the gene therapy and funded the study.
