News

AAN 2023: Phase 1/2 trial to test AskBio’s gene therapy in patients

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…

High huntingtin protein levels in saliva linked to worse disease

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 —…

Aitia and UCB team up to develop novel Huntington’s treatment

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…

Machine learning may help in predicting likely rate of progression

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…

SAGE-718 named orphan drug in EU for Huntington’s disease

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…