Lindsey Shapiro, PhD,  science writer—

uniQure plans to submit a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) early next year seeking approval of its gene therapy candidate AMT-130 for Huntington’s disease. Top-line, three-year data from the ongoing clinical trials that will support that application are due in the…

Daily treatment with Ingrezza (valbenazine) eases the jerky, involuntary movements known as chorea across different body regions in people with Huntington’s disease, according to new data analyses from the KINECT-HD trial. The primary analyses from the Phase 3 KINECT-HD study (NCT04102579) showed that the oral therapy…

One-year results from the Phase 2 PIVOT-HD clinical trial testing PTC Therapeutics’ Huntington’s disease treatment PTC518 are expected in the second quarter. That’s according to an update from PTC, which also noted that in a December meeting with the U.S. Food and Drug Administration (FDA), the agency…

While the vast majority of Huntington’s disease patients in North America have the uncontrolled and jerky movements known as chorea at clinically relevant levels, about a third of these people are getting medications specifically for this motor symptom. Among treated patients, about half are prescribed VMAT2 inhibitors, the…

Atalanta Therapeutics has raised $97 million to support planned Phase 1 clinical trials of its experimental RNA interference (RNAi)-based therapies for Huntington’s disease and a genetic form of epilepsy. Each treatment aim to silence the activity of genes that underlie these diseases. Atalanta plans to submit investigational…

Subtle biomarkers of neurodegeneration are evident decades before the onset of overt Huntington’s disease symptoms in people with disease-causing mutations, a study found. Somatic CAG repeat expansions — a phenomenon in which Huntington’s-causing mutations grow, or expand, during a person’s lifetime — were associated with these neurodegenerative biomarkers.

Sage Therapeutics is discontinuing the development of dalzanemdor after the investigational therapy failed to improve cognitive abilities relative to a placebo among people with Huntington’s disease enrolled in a Phase 2 clinical trial. Statistically significant gains in cognition with treatment was the main goal of the…

Alnylam Pharmaceuticals has launched a Phase 1 clinical trial to test the safety and pharmacological properties of its treatment candidate ALN-HTT02 in adults with Huntington’s disease, the company announced as part of a financial update. “We [have] made great strides with our pipeline, … initiating a Phase 1…

Higher levels of an enzyme called GST02 in the most affected neurons in Huntington’s disease could underlie early increases in the brain-signaling chemical dopamine that are believed to drive Huntington’s motor symptoms, according to a mouse study. Scientists had previously uncovered that a deficiency in a signaling pathway called…

Diminished production in the brain of a protein known as CHCHD2 may contribute to early neurodevelopmental changes that go on to drive Huntington’s disease, a study reports, with scientists poising the gene responsible for its production as a possible therapeutic target. CHCHD2 is involved in maintaining the workings of…