Gene Therapy Trial for Vision
Scientists have developed a groundbreaking gene therapy that dramatically improves vision in patients with a genetic disorder causing early vision loss. The therapy targets the specific gene responsible for the condition, leading to significant improvements—up to 100 times better vision for some patients, with the highest dose resulting in a remarkable 10,000-fold enhancement. For instance, a person who could previously only see in bright light might now be able to see clearly on a moonlit night.
This therapy, tested on 15 individuals (including three children) with Leber congenital amaurosis (LCA1), tackles a rare genetic mutation affecting around 100,000 people worldwide. LCA1 causes severe vision loss from infancy, and even with corrective lenses, patients typically couldn’t achieve better than 20/80 vision (meaning they need to be 20 feet away to see what others can see from 80 feet). In the trial, a gene therapy called ATSN-101 was injected directly into the eye. Patients experienced noticeable improvements within a month, with benefits lasting at least a year. Those receiving the highest doses saw the most dramatic results, including better mobility in low-light environments.
This trial, funded by Atsena Therapeutics, is part of a growing wave of gene therapies restoring vision in patients with inherited disorders. Earlier this year, another breakthrough using CRISPR gene editing also led to vision improvements in patients with a different mutation linked to LCA.