NightstaRx Ltd (“Nightstar”), a biopharmaceutical company specialising in developing gene therapies for inherited retinal conditions, have announced the publication from the University of Oxford of promising clinical results and long-term benefits of choroideremia gene therapy in this week’s issue of the New England Journal of Medicine (NEJM). Nightstar is now developing this gene therapy approach and the viral vector known as adeno-associated virus 2 (AAV2) to deliver a copy of the gene encoding Rab-escort protein 1 (REP-1) gene, the mutation of which leads to damage of retinal cells of patients with choroideremia, an inherited form of progressive blindness.
The study enrolled six patients with choroideremia at the University of Oxford’s Nuffield Laboratory of Ophthalmology under principal investigator Professor Robert MacLaren, who spoke at the Fighting Blindness Retina Conference in 2014. As published in the NEJM, the clinical research findings demonstrated that five patients who received the full dose of AAV2-REP1 treatment maintained or improved vision in their treated eye for the extent of post-treatment observation (up to 3.5 years). In two patients, a significant improvement in vision of greater than three lines on an eye chart was sustained to the last time point reached. In the untreated eyes, vision declined in five out of the six patients at 3.5 years.
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