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British gene therapy startup gets $35M from NEA, Sycona – MedCity News

By November 11, 2015News
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British biopharma startup NightstaRx, which develops gene therapy for inherited retinal dystrophies, just closed out a $35 million Series B led by New Enterprise Associates. The funding will help NightstaRx advance several gene therapies into human trials, as well as expand its existing clinical program for the treatment of choroideremia.

The company’s approach to gene therapy  involves harnessing an adeno-associated viral vector to deliver a corrective gene into patients with inherited progressive blindness. Specifically, to treat choroideremia, NightstaRx delivers a wild-type copy of the Rab-escort protein 1 (REP-1) gene (AAV2-REP1) straight into the retinal cells.

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