Gene therapy shows promise in tackling X-linked RP
Early results from clinical testing of a gene therapy to treat X-linked retinitis pigmentosa (XLRP) have shown partial reversal of sight loss in some patients.
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Early results from clinical testing of a gene therapy to treat X-linked retinitis pigmentosa (XLRP) have shown partial reversal of sight loss in some patients.
Sepul Bio begins clinical trial of ultevursen for USH2A-associated RP
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A discovery made as part of research funded by Retina UK has led to a prestigious award of $2.5 million, enabling scientists to undertake more in-depth investigations and work towards a treatment for a particular type of retinitis pigmentosa.
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.
The FDA has given Ocugen permission to start their phase 3 gene therapy clinical trial for retinitis pigmentosa (RP).
Since the publication of this newsletter our founder and honorary president Lynda Cantor MBE has passed away. We are all deeply saddened by this news.
A huge thank you to everyone who completed our 2025 sight loss survey. This year we received over 600 responses. It follows similar surveys in 2019 and 2022 which led directly to the introduction of our Unlock Genetics and Discover Wellbeing resources, as well as shaping our work with the professional community who support people with inherited sight loss.
The cover of this edition celebrates the success of our amazing London Marathon team. Together they have raised more than £43,000.
We’re all still buzzing from our recent conferences. We do hope you enjoyed them as much as we did. If you weren’t able to join us, then you can watch or listen to the recordings on our website.