Meet our community: Mark Baxter
My name is Mark Baxter and from an early age I was diagnosed with a deteriorating retinal eye condition called retinitis pigmentosa or RP for short, which will slowly lead to blindness.
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Why genes matter
Genes exist inside all the cells that make up your body. They contain genetic code (DNA) and provide instructions for constructing proteins, which are the building blocks of our bodies and perform a huge variety of roles.
Meet our volunteers: Joe Rizzo-Naudi
Our amazing volunteers are diverse in age, background and ethnicity. The one thing they have in common is that they are all living with, or directly affected by, an inherited sight loss condition.
Meet our volunteers: Cindy Peacock
“Being part of something and feeling that you are not alone,” are just two of the positive benefits Cindy Peacock says she experiences as a volunteer with Retina UK.
Light sensing small molecule given green light
Biotechnology company Kiora Pharmaceuticals has been given the go-ahead to start in-human testing of a novel “small molecule” treatment, known as KIO-301, for advanced vision loss in retinitis pigmentosa (RP). The phase 1b clinical trial will take place in Adelaide, Australia, and will begin enrolling participants towards the end of 2022.
Meira GTx announces positive data from phase clinical trial
Biotechnology company MeiraGTx has announced encouraging news from its phase 1/2 clinical trial of botaretigene sparoparvovec (previously known as AAV-RPGR), a gene therapy aimed at X-linked retinitis pigmentosa caused by faults in the RPGR gene.
Disappointing news from ProQR clinical trial for LCA10
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.
jCyte update
In spring 2021, the US company jCyte announced detailed results from their successful phase 2b clinical trial of jCell, a stem cell-based treatment for RP.
Encouraging results from ProQR USH2A clinical trial
Biotechnology company ProQR has announced encouraging results from its phase 1/2 clinical trial of an innovative treatment for sight loss caused by faults in a specific section of the USH2A gene.
ProQR clinical trial for Leber’s Congenital Amourosis (LCA) 10
Biopharmaceutical company ProQR is conducting a Phase 2/3 clinical trial (Illuminate) to measure the safety and effectiveness of RNA therapy, Sepofarsen which aims to treat Leber's Congenital Amourosis (LCA) 10.