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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
It was previously believed that female carriers of X-linked inherited retinal diseases (IRDs) like X-linked retinitis pigmentosa (RP and Choroideremia) remained unaffected by sight loss.
The Royal National Institute of Blind People (RNIB) have awarded Retina UK the Visibly Better Employer quality standard.
On a typical week in the stem cell lab there are many different experiments going on. Different people work on their individual projects, but we often collaborate to share ideas and help each other.
