News

Spark, Neurochase Collaborate on Brain Delivery Technology

Spark Therapeutics and Neurochase have joined forces to develop Neurochase’s proprietary delivery technology for gene therapies that target neurodegenerative diseases, such as Huntington’s disease. The technology, called Convection Enhanced Delivery (CED), allows for a controlled delivery of gene and other therapies directly into specific brain regions with the…

Effects on Different Neurons May Explain How Symptoms Evolve

In Huntington’s disease, neurodegeneration in a brain region called the striatum differs not only across different types of neurons, but also across distinct striatal compartments, according to a study of brain samples from a Huntington’s patient and mouse models. The striosomes, the striatal compartment involved in regulating mood, was more…

Potential Chorea Treatment Valbenazine Under Review in US

The U.S. Food and Drug Administration (FDA) has agreed to review Neurocrine Biosciences’ supplemental new drug application (sNDA) for valbenazine to treat chorea caused by Huntington’s disease. “This sNDA filing advances our effort to bring a potential new treatment option to the many thousands of people experiencing chorea associated with…

New RNA-targeting Therapy Reduces Toxic HTT Protein Buildup

Researchers have developed a new therapy that reduced the toxic buildup of the mutant huntingtin (HTT) protein — the hallmark of Huntington’s disease — in lab-grown neurons from Huntington’s patients and in a mouse model of the disease. The CRISPR-based therapy was designed to specifically target the HTT gene’s messenger…

Study Finds How Branaplam May Work to Help Treat Huntington’s

Branaplam (LMI070), an experimental oral therapy for Huntington’s disease that’s currently in early clinical trials, reduces levels of the mutant huntingtin protein by interfering with a process called mRNA splicing, a new study reveals. “Here, we delineated the mechanism of action of Branaplam,” researchers wrote. The study, “…

Abnormal Red Blood Cells Found in 4 Chinese Huntington’s Patients

Abnormal red blood cells, known as acanthocytosis, were identified in four unrelated Chinese adults with Huntington’s disease — undermining researchers’ attempts to differentiate the neurodegenerative disorder from another similar genetic disease. Researchers had expected that the presence or absence of such abnormally shaped red blood cells would help…

Enrollment Will Resume In Trial Of AMT-130 Gene Therapy

A data safety monitoring committee has recommended that enrollment of the highest dosing group resume in the European Phase 1/2 trial of AMT-130 in people with Huntington’s disease, but with additional safety measures in place. UniQure, the investigational treatment’s developer, announced in August that enrollment in…