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Voyager Therapeutics has a new candidate for the treatment of Huntington’s disease. The selected gene therapy is called VY-HTT01 and aims to prevent the production of protein from the mutant huntingtin gene (HTT). The compound is now undergoing preclinical studies of drug characteristics and toxicology as the company plans to file…

A team of researchers has discovered new insights into cell processes that have gone awry in Huntingon’s disease (HD) patients. This discovery could point scientists toward a potential method of interfering with those processes. The study, “KEAP1-modifying small molecule reveals muted NRF2 signaling responses in neural stem cells from Huntington’s…

Researchers have discovered why Huntington’s disease (HD) patients have defects in the blood-brain barrier that contribute to the symptoms of this neurological disorder. Their study, “Huntington’s Disease iPSC-Derived Brain Microvascular Endothelial Cells Reveal WNT-Mediated Angiogenic and Blood-Brain Barrier Deficits,” was published in Cell Reports. “Now we know there are…