Researchers have developed a protein-like polymer — a large, string-like molecule composed of small repeating building blocks — that was able to slow the onset of Huntington’s disease in a mouse model. In the mice, the protein-like polymer prevented the clumping of misfolded huntingtin protein that drives nerve cell…

Unlike its healthy counterpart, the mutated version of the huntingtin protein that drives Huntington’s disease may keep nerve cells from repairing their damaged DNA, causing them to die, a study showed. Specifically, the mutated protein failed to stimulate the production of poly ADP-ribose, known as PAR — a molecule…

A first clinical trial of SKY-0515, an experimental treatment for Huntington’s disease, is starting to give multiple ascending doses to healthy adults randomized to the therapy rather than a placebo, Skyhawk Therapeutics, its developer, announced. Initiation of this part of the Phase 1 trial (ACTRN12623001161617), taking place in…

Note: This story was updated Jan. 24, 2024, to correct that Takeda is not supporting WVE-003’s development. Wave Sciences is the treatment’s sole developer; Takeda has a codevelopment and co-commercialization option. New data from a Phase 1/2 trial of WVE-003, a therapy targeting the mutant huntingtin protein in people…

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 —…

A single injection of Wave Life Science‘s experimental therapy WVE-003 was found, at either of the two lowest tested doses, to lead to reductions in the mutant huntingtin (mHTT) protein in people with Huntington’s disease — while leaving the healthy version of the protein unaffected. That’s according to…

AMT-130, uniQure’s investigational gene therapy for Huntington’s disease, was generally well-tolerated and led to reductions in the levels of mutant huntingtin protein a year after treatment, according to an update from an ongoing Phase 1/2 trial. Moreover, levels of neurofilament light (NfL), a biomarker of nerve damage —…

Certain people with Huntington’s disease — specifically, younger adults with milder disease — may have benefited from treatment with tominersen in the Phase 3 GENERATION HD1 clinical trial, according to a new analysis of study data. Based on the results, its developer Roche is planning to launch a Phase 2…

Sarah Lynn Mays learned she wouldn’t be returning to Northwestern University for her scheduled intrathecal injection of tominersen when her younger sister called to give the news — Roche was stopping dosing for its Phase 3 clinical trial of the Huntington’s disease treatment.

Researchers have found a protein, called UBR5, that may reduce neurodegeneration in Huntington’s disease (HD). This protein prevents the toxic aggregation (clustering) of mutant huntingtin protein, which is the underlying mechanism that triggers nerve cells’ death in Huntington’s patients. The study, “The ubiquitin ligase UBR5 suppresses proteostasis collapse in pluripotent…