Nightstar Expands Pipeline with Novel Gene Therapy for the Treatment of Stargardt Disease
NIGHTSTAR THERAPEUTICS PLC AMERICAN DEPOSITARY SHA (NITE)
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Source: GlobeNewswire
Fourth Gene Therapy Program Targets the Most Common Inherited Juvenile Macular Dystrophy LEXINGTON, Mass. and LONDON, Nov. 08, 2017 (GLOBE NEWSWIRE) -- Nightstar Therapeutics (NASDAQ:NITE), a clinical-stage gene therapy company developing treatments for rare inherited retinal diseases, today announced an expansion of its pipeline with an exclusive global license for a gene therapy program for the treatment of Stargardt disease from Oxford University Innovation. The gene therapy program utilizes a novel technology developed by the University of Oxford. Preclinical proof-of-concept studies in the Abca4-/- murine model of Stargardt disease have demonstrated the expression, localization and function of the ABCA4 protein. Stargardt disease is the most common form of inherited juvenile macular dystrophy with a prevalence of one in 10,000. This autosomal recessive disease is linked to mutations in the ABCA4 gene that are inherited from both parents of an affected individual. The progres
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