Exciting developments for new treatment research
In recent years there has, understandably, been a lot of excitement around cutting-edge therapies that target the specific genetic faults underlying inherited sight loss.
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In recent years there has, understandably, been a lot of excitement around cutting-edge therapies that target the specific genetic faults underlying inherited sight loss.
“All the promising research makes you feel like there is light at the end of that very dark tunnel after all.”
Biotechnology company ProQR has announced encouraging results from its early analysis of the phase 1/2 trial of QR-421a, an innovative approach to treating sight loss caused by mutations in a particular section of the USH2A gene.
James Birtley is a Retina UK supporter and volunteer who is living with retinitis pigmentosa. He is also a scientist with a particular interest in the structure of proteins, the complex molecules that form the building blocks of our bodies.
Retina UK is delighted that the Scottish Medicines Consortium (SMC) has accepted the gene therapy Luxturna (voretigene neparvovec) for ongoing use by the NHS in Scotland.
Researchers who received funding from Retina UK have carried out experimental gene therapy that is reported to have led to ‘life changing improvements’ to sight for four children with inherited sight loss.
During our 50th anniversary year, Retina UK was the focus of a BBC Lifeline Appeal on Sunday 16 November 2025.
This edition marks the start of the charity's 50th anniversary year. It includes lots of ways to get involved over the next 12 months along with details of our Annual and Professionals' Conferences in September.