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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.
ProQR announces agreement with Laboratoires Théa for inherited retinal disease programmes
Biotechnology company ProQR has announced that two of its RNA therapy development programmes for inherited sight loss are being acquired by Laboratoires Théa, a company specialising in eye care products.
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.
ProQR makes progress with potential treatment for LCA
Despite the challenges of the pandemic, researchers and pharmaceutical companies are still making progress towards delivering new treatments for inherited sight loss.
First clinical trial participant receives CRISPR-based therapy
An American living with Leber congenital amaurosis 10 (LCA10) has become the first clinical trial participant in the world to receive a CRISPR gene editing treatment in vivo (inside the body).
Therapy development updates
There are many ongoing clinical and laboratory studies around the world, exploring innovative approaches to treating inherited sight loss.
Trusts and Foundations
Inherited retinal dystrophies (IRDs) are the leading cause of blindness in working-age people in the UK, and children as young as eighteen-months are regularly diagnosed.
FAQs: Luxturna (voretigene neparvovec)
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.
Look Forward – Summer 2023 – Issue 180
Did you join either of our Conferences this year? You can read about them inside this edition of Look Forward. We’ve had some fantastic feedback from our delegates so if you weren’t able to attend, you can watch or listen to the recordings from the day on our website.