Patricia Inácio, PhD, science writer —

The biopharmaceutical UCB and Aitia have joined forces to accelerate the development of a novel Huntington’s disease treatment. The collaboration will harness Aitia’s artificial intelligence (AI) technology to identify new targets and therapies for Huntington’s that will be further validated using UCB’s expertise in treatment research and…

Reduced levels of oxytocin — a natural hormone produced in the brain’s hypothalamus that also works as a chemical messenger — in the cerebrospinal fluid (CSF) were found in Huntington’s disease patients with cognitive and social impairments, a new study shows. The findings support oxytocin levels as a biomarker of impaired…

Researchers have developed a new therapy that reduced the toxic buildup of the mutant huntingtin (HTT) protein — the hallmark of Huntington’s disease — in lab-grown neurons from Huntington’s patients and in a mouse model of the disease. The CRISPR-based therapy was designed to specifically target the HTT gene’s messenger…

Abnormal red blood cells, known as acanthocytosis, were identified in four unrelated Chinese adults with Huntington’s disease — undermining researchers’ attempts to differentiate the neurodegenerative disorder from another similar genetic disease. Researchers had expected that the presence or absence of such abnormally shaped red blood cells would help…

ProQR Therapeutics’ experimental therapy QRX-704 significantly reduced levels of the toxic huntingtin (HTT) protein clumps that fuel Huntington’s disease in a mouse model of the disease, a study showed. The therapy, an antisense oligonucleotide (ASO) designed to promote the generation of a shorter, but working version of…

A recent report describes the case of a Chinese woman with a family history of Huntington’s disease (HD) who presented with symptoms but was not diagnosed until 10 years after symptom onset. The case study, “Report of a family with Huntington’s disease,” was published in the…

Bit.Bio has launched a new human cell model to advance research and accelerate therapeutic development for Huntington’s disease. The cells, called ioGlutamatergic Neurons HTT-50CAG/T, are engineered to carry a specific mutation in the HTT gene that causes Huntington’s. By carrying the same genetic…

A Phase 2 trial evaluating PTC518, an investigational treatment for Huntington’s disease, has started in the U.S., PTC Therapeutics announced. The trial, called PIVOT-HD, will run across clinical sites worldwide and is divided into two parts. First, participants will be randomly assigned to a placebo or PTC518…

Annexon’s investigational therapy ANX005 is showing tolerability and a potential to treat people in the earlier stages of Huntington’s disease or at genetic risk of the disorder, according to interim data from an ongoing Phase 2 trial. ANX005, an antibody designed to inhibit the activation of the complement cascade —…

A Phase 1/2 clinical trial testing uniQure’s potential gene therapy AMT-130 in people with Huntington’s disease can continue enrollment, according to a positive review by the study’s independent data safety monitoring board (DSMB). The DSMB reviewed safety data from four patients given the higher dose of the therapy, which…