Margarida Maia, PhD,  science writer—

Both DNA repair and non-repair genes influence how and when Huntington’s disease develops, according to a study based on genetic data from more than 16,000 people with the disorder that also provides new potential targets for treatment. In addition, these findings reveal complex mechanisms behind CAG repeat expansions, the…

Introducing small interruptions into the abnormally long CAG repeat expansion that causes Huntington’s disease can stabilize or even reduce the length of that DNA stretch, which would be expected to slow disease progression. These are the findings of a study that tested base editing, a technique that changes a…

The U.S. Food and Drug Administration (FDA) has designated uniQure’s gene therapy candidate AMT-130 a breakthrough therapy for its potential to slow the progression of Huntington’s disease, according to the developer. This status is intended to speed the development and review of medications for serious or life-threatening illnesses…

Pediatric-onset Huntington’s disease is more severe than its adult-onset form, with faster and more extensive shrinkage of brain regions involved in movement, an imaging study has found. Atrophy, or a loss in brain volume, “was remarkable and more severe” in patients with pediatric-onset disease compared with adult-onset Huntington’s, the…

The U.S. Food and Drug Administration (FDA) is supporting financially a study into how well wearable sensors measure two early signs of Huntington’s disease, which could be of use to clinical trials of potential treatments. The funding comes from the agency’s Digital Health Technologies for Drug Development…

Loqus23 Therapeutics has raised 35 million pounds (about $45 million) in financing to advance its lead oral small molecule candidate for Huntington’s disease through preclinical development and early clinical studies, which are anticipated in 2026. The series A financing round was led by Forbion, a life sciences…

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…

Vico Therapeutics’ investigational therapy VO659 was found to lower levels of the disease-causing mutant huntingtin (HTT) protein — known as mHTT — in the cerebrospinal fluid (CSF), the liquid that surrounds the brain and spinal cord, of people with Huntington’s disease. Additionally, treatment was not associated with an…

Researchers in the U.S. have created the first brain-like organoids, or mini-brains, that incorporate a working blood-brain barrier (BBB), which is the membrane that regulates which circulating substances can reach the brain and spinal cord, and that typically challenges the entry of medications. Called a BBB assembloid, the human model…

uniQure expects to begin planned discussions with the U.S. Food and Drug Administration (FDA) in the coming months to outline the future steps for testing its gene therapy candidate AMT-130 for early Huntington’s disease, and seeking regulatory approval of the treatment. In its interactions with the FDA, expected to…