News

Asklepios Biopharmaceutical (AskBio) has launched a first-in-human clinical trial testing AB-1001 — an experimental gene therapy designed to modulate cholesterol metabolism in the brain — in people with Huntington’s disease. The trial is currently recruiting participants at a site in Paris. It is open to adults, ages 18…

Subtle changes in speech are present before obvious symptoms of Huntington’s disease appear, and could potentially be a quantitative biomarker for the neurodegenerative disorder, a small study found. The changes in speaking identified among Huntington’s patients were linked to age and an individual’s number of disease-causing CAG repeats —…

A Phase 1/2a clinical trial testing VO659, Vico Therapeutics’ experimental therapy, in people with Huntington’s disease and other hereditary neurological disorders caused by a similar type of mutation has dosed the first patient. “We are encouraged by the continued progress of our development program and very pleased to…

The last patient has completed the final visit in the Phase 3 PROOF-HD trial testing oral pridopidine for Huntington’s disease. That means that the study’s main placebo-controlled part is done, according to Prilenia Therapeutics, pridopidine’s developer, which sponsored the nearly 18-month-long trial. Now, the first data are…

Higher levels of healthy and mutant huntingtin protein in saliva, but not in blood, are significantly associated with worse motor symptoms of Huntington’s disease, a study showed. According to the investigators, these findings support the use of noninvasive saliva tests to monitor Huntington’s progression and predict clinical outcomes —…

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…

Using machine learning, a form of artificial intelligence, researchers were able to cluster people with Huntington’s disease into three groups based on the speed at which the disease progresses, a study showed. “Machine learning approaches such as this could be considered for application in real-world clinical practice to support…

Enrollment is underway in a Phase 2 clinical trial testing Roche’s therapeutic candidate tominersen in people with early manifest Huntington’s disease and those in the prodromal phase of the disease, when hallmark disease symptoms are not yet present. The study, dubbed GENERATION HD2 (NCT05686551), aims…

The European Medicines Agency (EMA) has granted orphan drug status to SAGE-718 as a potential therapy for Huntington’s disease. The experimental therapy is being developed by Sage Therapeutics for treating cognitive impairment associated with Huntington’s and other neurological disorders. Orphan drug status is given to treatments with the potential to improve…

Austedo XR, a new extended-release formulation of Austedo (deutetrabenazine) has been approved by the U.S. Food and Drug Administration (FDA) to treat adults with chorea — involuntary muscle contractions — associated with Huntington’s disease. Austedo XR contains the same active agent as the original therapy, but is taken just once a day, with…