Gene therapy of choroideremia
Nightstar knows how to cure this rare genetic disease that leads to blindness
The British "Nightstar Therapeutics" (Nightstar Therapeutics) has planned to enter the stock market through an initial public offering on the NASDAQ exchange in the amount of $ 86 million. The funds are needed to launch in the first half of 2018 the STAR phase III reference clinical trials of experimental gene therapy for choroideremia (CHM) on 140 patients, as well as to continue the development of two more candidate drugs aimed at other rare ophthalmic diseases.
Choroideremia is a rare (one case per 50 thousand people), degenerative, X–linked genetic disease that causes progressive vision loss with total blindness. The pathology is caused by a mutation of the CHM gene responsible for the synthesis of Rab-satellite protein 1 (REP-1). The latter serves intracellular protein traffic and elimination of metabolic products from retinal cells. The absence of a working REP-1 protein leads to the death of retinal pigment epithelial cells that provide supporting biological functions for photoreceptors and the underlying choroid (vascular membrane of the eye) – they begin to slowly atrophy. Currently, there is no effective therapy for choroideremia.
NSR-REP1 gene therapy involves virus-vector delivery of recombinant human complementary DNA developed for the synthesis of functioning REP-1. The injection of the drug is performed in the subretinal space.
Phase I/II clinical studies have demonstrated the long-term effectiveness of NSR-REP1 gene therapy: in the vast majority of patients, there was a clear improvement in visual acuity compared with a drop in that in the control eye that did not receive an injection of the drug.
Nightstar is also engaged in other orphan diseases: clinical trials of the XIRIUS phase I/II candidate drug NSR-RPGR for the treatment of X-linked retinitis pigmentosa (XLRP) are continuing and a study of preclinical NSR-BEST1 against Best's vitelliform macular dystrophy (Best's disease) is being conducted.
Nightstar Therapeutics Drug Pipeline
As for direct competitors, the gene therapy of choroideremia is carried out by Spark Therapeutics, which is preparing its SPK-7001 for phase I/II clinical trials.
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07.09.2017