Columns

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…

Researchers at the University of California and the Salk Institute for Biological Studies have identified the mechanisms that make the brain’s immune cells different from all other immune cells A finding with implications for Huntington’s disease was that genes associated with neurological illnesses have higher lessons of expression in microglia,…

Abnormal proteins found in neurodegenerative diseases such as Alzheimer’s, Parkinson’s, and Huntington’s seem to rely on the same mechanism to spread within the brain and disrupt its normal functions, according to a study published in the journal Acta Neuropathologica. The cellular process described in the study, “Endocytic vesicle rupture…

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 at the Sussex Drug Discovery Centre in Brighton, England, have received a £3 million grant from Britain’s non-profit Wellcome Trust to develop Huntington’s disease treatments. The team will team up with other researchers to target a protein that plays a role in cognitive impairment, one of the hallmarks of the neurological…

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…

WAVE Life Sciences’ products WVE-120101 (SNP-1) and WVE-120102 (SNP-2) will enter clinical trials in mid-2017 as part of the effort to develop new treatments for patients with Huntington’s disease. “2017 will be an important year for WAVE as we transition our two lead candidates in Huntington’s disease … into clinical…

Taube Philanthropies has donated $3 million to fund a research team using gene editing and stem cell techniques to develop treatments for Huntington’s disease. The team is composed of Dr. Matthew Porteus and Dr. Frank Longo of Stanford Medicine, and Dr. Steve Finkbeiner of the Gladstone Institutes, said a Stanford press…

Disruption in the trafficking of molecules in and out of the cell’s nucleus may contribute to the pathology of Huntington’s disease, according to results of recent research. The study “Mutant Huntingtin Disrupts The Nuclear Pore Complex” was published in the journal Neuron. Huntington’s disease is caused by mutations in…

Stealth BioTherapeutics has started a Phase 1/2 clinical trial to investigate the safety and effectiveness of SBT-20 in patients with early-stage Huntington’s disease in the Netherlands. SBT-20 was designed to restore the proper activity of mitochondria, the cell’s powerhouses, in Huntington’s patients. Mitochondria organelles become faulty as the disease progresses.