The first Huntington’s disease patient has been dosed in a Phase 2/3 clinical trial testing SKY-0515, Skyhawk Therapeutics‘ oral disease-modifying therapy, in adults with the neurodegenerative condition. The study, dubbed FALCON-HD (NCT06873334), is expected to enroll up to…
Researchers have developed a modified and harmless adeno-associated virus (AAV), called AAV-DB-3, that can efficiently deliver low doses of gene therapies to deep regions of the brain impacted by neurodegenerative diseases such as Huntington’s disease. AAV-DB-3 was more than 200 times more potent at delivering its genetic cargo to…
Two signaling enzymes, GSK3-beta and ERK1, play opposing roles in the development of Huntington’s disease and may represent new therapeutic targets, a study showed. “We propose that ERK1 may protect neurons in the face of Huntington’s disease, while [GSK3-beta] may exacerbate Huntington’s disease,” Shermali Gunawardena, PhD, the study’s senior…
Most adults with the genetic defect associated with Huntington’s disease were completely able to perform daily activities and tasks at a first assessment, and maintained such skills over time, according to data from a natural history study of the neurodegenerative condition. That study assessed participants using the total functioning…
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…
Blood levels of neurofilament light protein (NfL) in people who carry Huntington’s disease-causing mutations, but aren’t showing symptoms, can identify those who will develop the disease in the next decade. These are the findings of a 14-year study that also showed that greater increases in blood NfL levels, a…
Examining the amounts of certain microbes in the gut and levels of specific metabolites in the blood may help distinguish people with and without Huntington’s disease with 100% accuracy, a study suggested. “This study determined crucial functional gut microbiota and potential biomarkers associated with [Huntington’s development], providing new insights…
A high level of education, low to moderate alcohol intake, not smoking, and controlling one’s weight may slow Huntington’s disease progression among those who’ve yet to have symptoms, a study shows. “Reducing modifiable risk factors for [Huntington’s] is one way to support the presymptomatic population,” which is made up…
An investigational oral therapy from PTC Therapeutics called PTC518 safely and effectively reduces levels of the mutant huntingtin protein (mHTT) in Huntington’s disease patients, while being linked to trends of slower disease progression. That’s according to one-year interim data from the ongoing Phase 2a PIVOT-HD clinical trial…
Scientists at the Broad Institute of MIT and Harvard have engineered a harmless adeno-associated virus (AAV) that can efficiently reach the brain, potentially improving the efficacy of brain-targeted gene therapies for neurological conditions such as Huntington’s disease. Current AAVs that deliver gene therapies to cells in the body via…
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