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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.
In February 2022, ProQR announced a disappointing outcome for their phase 2/3 trial of sepofarsen for Leber congenital amaurosis type 10 (LCA10).
As part of our aim to nurture young scientists, we are very pleased to introduce one of our new PhD students as part of a co-funding agreement with the Macular Society.
There are many ongoing clinical and laboratory studies around the world, exploring innovative approaches to treating inherited sight loss.
Thursday 19 September, 11.00am - 1.00pm - In-person - Come and join us at the Princess Alexandra Eye Pavilion for a special focus group with Dr. Roly Megaw.
As Luxturna reaches the clinic and other gene-specific therapies for inherited retinal disease get closer to the end of the development pipeline, it is becoming ever more important that affected families can access a genetic diagnosis, potentially opening up choices around treatment and clinical trial participation.
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.
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.