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Biotech Focuses on Gene Therapy for Huntington’s, Heart Disease, Hemophilia B

Nov 17, 2016 10:35 am | Charles Moore

 

gene therapyGene therapy biotech uniQure is prioritizing its drug pipeline to develop treatments for Huntington’s disease, hemophilia B, and its partnered gene therapy programs associated with uniQure’s collaboration with Bristol-Myers Squibb (BMS) in cardiovascular disease. The company will restructure its research and development organization in the Netherlands and consolidate manufacturing in the United States to reduce operating expenses […]

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Messenger RNA Seen as Potential New Treatment Target in Huntington’s

Nov 15, 2016 12:01 pm | Joana Fernandes, PhD

 

mRNA and Huntington'sResearchers identified a new molecular mechanism related to mutant huntingtin (HTT) — messenger RNA — that may play a role in the development of Huntington’s disease. This finding may help define new therapeutic approaches to tackle the disease. The study, “Targeting CAG Repeat RNAs Reduces Huntington’s Disease Phenotype Independently Of Huntingtin Levels”, was published in the […]

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