From PackGene: Eleven children who were legally blind at birth all gained visual acuity after receiving MeiraGTx’s investigational gene therapy, data that are prompting the biotech to seek accelerated approval in AIPL1-associated severe retinal dystrophy.
Results from four of the 11 children were published in The Lancet Feb. 21. Visual acuity—measured with a child-friendly, game-like touchscreen test—in treated eyes improved after four or more weeks, while the acuity in untreated eyes became unmeasurable, according to the published results.