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A recently published study has described numerous disease mechanisms that appear to be common across different types of inherited sight loss, suggesting that there is significant potential for drug treatments that could work regardless of the underlying causative gene.
As Luxturna reaches the clinic and other gene-specific therapies for inherited retinal disease get closer to the end of the development pipeline, it is becoming ever more important that affected families can access a genetic diagnosis, potentially opening up choices around treatment and clinical trial participation.
A new Retina UK podcast that introduces our new chair of trustees, Kathy Evans.
Biotechnology company ProQR has announced that its clinical trials of sepofarsen for Leber congenital amaurosis type 10 and ultevursen for USH2A-mediated retinitis pigmentosa will be wound down with immediate effect.
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We are proud to work collaboratively with a number of corporate partners to enable our community to live fulfilled lives today as well as supporting the pharmaceutical industry in their mission to develop potential new treatments.
Retina UK is delighted to have awarded a new three-year research grant to Prof Majlinda Lako at Newcastle University for a project entitled: To establish AAV.PRPF31 gene augmentation in PRPF31-deficient RPE and photoreceptor cells and assess its efficacy in restoring RPE and photoreceptor function.
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 Retina UK community is sometimes targeted by adverts online or in social media, offering invasive treatments for inherited sight loss.
jCyte have released additional preclinical data regarding jCell
