Stage 3: excellent clinical results, gene therapy for "blindness" may be launched

Today, spark therapeutics, a Philadelphia based innovative biotechnology company dedicated to gene therapy, released new data on the continuation of phase 3 clinical trials of its leading candidate product voretigene naparvovec (formerly known as spk-rpe65) Previously, voretigene neparvovec has received FDA's breakthrough therapy and orphan drug qualification certification, and is being developed as a potential treatment scheme for hereditary retinopathy (IRD) caused by RPE65 gene mutation IRD is a rare genetic disease causing blindness, which is caused by the loss of function of RPE65 gene The retinal photoreceptor cells (pigment cells) of RPE65 gene mutation patients will gradually lose function and necrosis, eventually leading to complete loss of visual function Spark therapeutics was founded in 2013 Its advanced gene therapy platform is being used in a series of preclinical and clinical research projects related to the treatment of serious genetic diseases, including the above genetic retinal diseases, as well as blood system diseases and neurodegenerative diseases Spark researchers use adeno-associated virus (AAV) or non pathogenic cold virus as the vector, encode RPE65 sequence into the virus vector, and then inject it into the retina of patients to express the protein, which is expected to promote the survival and function of retinal photoreceptor cells, effectively convert light signals into electrical signals, and finally the eyes of patients can restore visual sensitivity In this critical multicenter, randomized, controlled phase 3 clinical trial, 93% (27 / 29) of the patients improved their functional vision one year after treatment with voretigene neparvovec, and the effect of gene therapy lasted for two years At the same time, voretigene neparvovec has been proved safe without serious adverse events Dr Katherine high, President and chief scientific officer of spark therapeutics, said: "we have now collected more data on voretigene neparvovec after administration, and are pleased to see that up to 93% of the subjects have significantly improved functional vision We will continue to analyze clinical trial data in order to further clarify the potential long-term benefits of voretigene neparvovec in patients with RPE65 mediated hereditary retinopathy " Reference: [1] spark therapeutics announcements new positive data from CO identification of phase 3 trial of voretigene neutral [2] spark therapeutics (once) visits long term positive phase III gene therapy data [3] official website of spark therapeutics [4] quick delivery of hemophilia gene therapy recognized by FDA breakthrough therapy - Wuxi apptec