Therapy development updates
There are many ongoing clinical and laboratory studies around the world, exploring innovative approaches to treating inherited sight loss.
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There are many ongoing clinical and laboratory studies around the world, exploring innovative approaches to treating inherited sight loss.
Funding from Retina UK has allowed to team to explore the viability of gene therapy as a treatment for RP and related conditions, and build a pipeline of clinical trials.
CRISPR gene editing leads to improvements in vision for people with inherited blindness, clinical trial shows.
Inherited progressive sight loss is caused by a range of rare genetic conditions. We specialise in those which affect the retina.
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Dan found out he had retinitis pigmentosa in February 2022, but he feels it has been “on cards for years”. He said he had “an inkling something was up but never did anything about it: a typical guy I guess!”
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.
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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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.