In February 2022, ProQR announced a disappointing outcome for their phase 2/3 trial of sepofarsen for Leber congenital amaurosis type 10 (LCA10).
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With all the interest in exciting gene and cell therapies for inherited sight loss, it’s easy to forget that more traditional drugs can also hold promise.
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ProQR has published the top-line results from its Phase 2/3 “Illuminate” trial of the RNA therapy sepofarsen for Leber congenital amaurosis type 10 caused by a specific mutation in the CEP290 gene.
You may have heard that a cell-based treatment approach (sometimes referred to as a “stem cell treatment”), developed by a company called ReNeuron, is being tested in a clinical trial at Oxford Eye Hospital and other centres in the US and Europe.
The Retina UK community is sometimes targeted by adverts online or in social media, offering invasive treatments for inherited sight loss.
In recent years there has, understandably, been a lot of excitement around cutting-edge therapies that target the specific genetic faults underlying inherited sight loss.