Search results
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.
Luxturna, a gene therapy, is only for the treatment of Leber congenital amaurosis type 2 (LCA2) and severe early-onset RP caused by mutations in a specific gene called RPE65.
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.
“All the promising research makes you feel like there is light at the end of that very dark tunnel after all.”
Retina UK aims not only to progress research along established threads, but to stimulate new thinking, encourage innovative approaches and nurture original ideas.
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.
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.
Inherited progressive sight loss is caused by a range of rare genetic conditions. We specialise in those which affect the retina.
