New stem cell treatment gains approval to enter US clinical trials
New stem cell treatment gains approval to enter US clinical trials
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Phase 3 clinical trial has been given approval by FDA
The FDA has given Ocugen permission to start their phase 3 gene therapy clinical trial for retinitis pigmentosa (RP).
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.
e-Newsletter – May 2023
Our monthly e-Newsletter featuring the latest updates from Retina UK. Subscribe now to receive these updates directly to your email.
Research brings hope for the future
“All the promising research makes you feel like there is light at the end of that very dark tunnel after all.”
Does gene therapy always have to be gene-specific?
Early 2020 marked an important milestone for the Retina UK community, when the first person with an inherited retinal condition received NHS treatment to potentially slow or even stop the progression of their sight loss.
Taking on The Thames Path Challenge
Next month, Anisha and Sheena will take on The Thames Path Challenge. Together, they explain why they decided to join #TeamRetinaUK.
Research update – ProQR
In February 2022, ProQR announced a disappointing outcome for their phase 2/3 trial of sepofarsen for Leber congenital amaurosis type 10 (LCA10).
Therapy development updates
There are many ongoing clinical and laboratory studies around the world, exploring innovative approaches to treating inherited sight loss.
First patient receives ground-breaking treatment
In January 23-year-old Jake Ternent became the first person with an inherited sight loss condition to be treated in the UK with Luxturna (voretigene neparvovec) for Leber congenital amaurosis (LCA).