A Genetic Lottery
— Rebecca Field

Wave Life Sciences will start two Phase 1b/2a clinical trials to investigate the safety and tolerability of its products WVE-120101 and WVE-120102 in Huntington’s disease patients. The trials will also look at the therapies’ effectiveness. “Wave’s PRECISION-HD program…

Huntington’s patients who are taking Xenazine for the twitching condition known as chorea can switch without concern to the newly approved Austedo, according to Austedo’s maker, Teva Pharmaceutical Industries. A Phase 3 clinical trial showed that the switch was safe and…

A sluggish metabolism in the cells of the striatum, a brain region essential for motor function control, is associated with disease severity in Huntington’s patients, while a higher metabolism in other brain regions seems to be associated with more…

Mutations of the MSH3 gene may be associated with the progression of Huntington’s disease, according to a British study. The gene could become a biomarker for the disease, and scientists may be able to develop Huntington’s therapies around it,…

Mitoconix Bio has secured $20 million in financing to continue developing MTC-1203 as a treatment for mitochondrial impairment in Huntington’s and Parkinson’s diseases. Several pharmaceutical companies and medical-related venture-capital funds participated in the financing. They include Aril Bioscience,…

Ionis Pharmaceuticals has completed enrollment of the Phase 1/2a study evaluating the safety, tolerability, properties and movement within the body of Ionis-HTTRx in patients with Huntington’s disease (HD). Patients who complete the randomized, placebo-controlled, dose escalation…

Researchers have identified a variant in the gene MSH3 that is linked with Huntington’s disease (HD) progression. The study, “Identification of genetic variants associated with Huntington’s disease progression: a genome-wide association study,” was published in the journal…

Inhibiting the activity of an enzyme preserved the movement capabilities of mice with Huntington’s, slowed the progression of the disease and extended the animals’ lives, according to a study. The dSETDB1/ESET enzyme can disrupt a mechanism that controls normal…

Low levels of a protein called Pyk2 may be associated with the development of Huntington’s disease, according to a new study. Researchers found that normalizing its levels in the hippocampus, the brain region responsible for learning and memory, rescues…

A factor called neurofilament light (NfL) may act as a biomarker of progression in Huntington’s disease, as blood levels of the molecule increase with more severe neurodegeneration. However, the factor also might offer a better means to predict disease…

A mutated form of the huntingtin (mHTT) protein has been acknowledged as the cause of Huntington’s disease, but researchers have not been fully clear about how it disrupts the function of brain cells. A study, “Mutant Huntingtin Disrupts…

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…