AIPL1 Gene Therapy Restores vision in small clinical trial

Tuesday, July 1 2025 | 10 h 30 min | Vision Science

Four children with Leber congenital amaurosis 4 (LCA4) have experienced meaningful improvements in their vision after receiving a gene therapy treatment.

The treatment was a gene replacement therapy for individuals with mutations in the AIPL1 gene, which leads to blindness from birth. The new treatment delivers healthy copies of the gene into the retina, helping retinal cells survive and work more effectively.

The treatment was developed by UCL Institute of Ophthalmology and Moorfields Eye Hospital, with support from MeiraGTx. To ensure safety, only one eye was treated. Over the next three to four years, vision in the treated eye improved significantly, while vision in untreated eye continued to decline.

The results of this study were published in The Lancet and highlight the potential of early gene therapy to restore sight in children with severe vision loss caused by and inherited retinal disease.

Researchers are now working to make this treatment more widely available.

About Fighting Blindness Canada

Fighting Blindness Canada (FBC) is the largest charitable funder of vision research in Canada.

Over our 49-year history, FBC has contributed critical funding for the development of sight-saving treatments and cures for blinding eye diseases. By raising and stewarding funds, FBC is helping drive forward research that supports our goal of understanding why vision loss occurs, how it can be slowed and how sight can be restored.

We are an invaluable resource for individuals and families impacted by blindness, providing accurate eye health information through our website and educational events, as well as engaging with government and other stakeholders to advance better vision health policies.

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