AAVantgarde Completes Enrolment in Phase 1/2 Clinical Trial for Usher Syndrome Type B
The latest research news piece from Retina UK.
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SB-007 update: dose-expansion portion of SpliceBio’s phase 1/2 trial underway with first participant dosed at Oxford
SpliceBio has begun the dose-expansion portion of their phase 1/2 clinical trial of SB-007, a dual adeno-associated viral vector gene therapy for Stargardt disease.
Look Forward – Winter 2022 – Issue 178
The cover of this edition celebrates the success of our amazing London Marathon team. Together they have raised more than £43,000.
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).
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.
Antabuse moves into clinical trial for people with RP
Disulfiram (Antabuse), FDA-approved for deterring alcohol, is in a phase 1 trial at the University of Washington to improve vision in retinitis pigmentosa.
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).
Therapy development updates
There are many ongoing clinical and laboratory studies around the world, exploring innovative approaches to treating inherited sight loss.
Significantly worse vision found in female carriers of X-linked RP and Choroideremia
It was previously believed that female carriers of X-linked inherited retinal diseases (IRDs) like X-linked retinitis pigmentosa (RP and Choroideremia) remained unaffected by sight loss.