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New Gene Therapy Offers Hope for Restoring Vision in Rare Congenital Eye Disorder

NextEdge Admin

08 Sep 2024

2 min 13 sec

Leber congenital amaurosis,Atsena Therapeutics

A recent study has shown promising results for gene therapy as a treatment for Leber congenital amaurosis (LCA1), a rare genetic disorder that causes severe vision loss. LCA1, caused by mutations in the GUCY2D gene, often manifests in early childhood, leading to rapid vision deterioration. Affecting fewer than 100,000 people globally, effective treatments for this condition have been limited.

Researchers from the University of Pennsylvania conducted a Phase 2 clinical trial of ATSN-101, a gene therapy developed by Atsena Therapeutics. The therapy involves injecting a corrective gene directly into the retina. The trial included 15 participants—12 adults and three children—who had severe visual impairment. Patients received one of three doses: low, medium, or high. Six participants, including the children, were treated with the highest dose.

The results were remarkable, with some patients experiencing up to a 10,000-fold improvement in their vision. Improvements were seen within a month of treatment and lasted at least a year. The therapy significantly enhanced patients' ability to navigate in low-light conditions and improved their visual acuity.

While minor side effects related to the procedure, such as eye hemorrhages and temporary inflammation, were reported, they were manageable with standard treatments. This breakthrough offers renewed hope for treating inherited retinal disorders like LCA1. Future larger trials will further assess ATSN-101’s efficacy and safety as it moves toward potential FDA approval.