Margarida Maia, PhD,  science writer—

The first two patients in Europe have been dosed in uniQure’s Phase 1/2 clinical trial investigating AMT-130, a one-time investigational gene therapy for Huntington’s disease. The trial is underway in Poland, where it is still open for enrollment, and is expected to expand to other sites in the…

The Huntington Study Group is seeking 30 patients to join KINECT-HD2, an open-label study of oral valbenazine capsules for chorea, or jerky involuntary movements, in Huntington’s disease, a press release states. KINECT-HD2 is an extension study of KINECT-HD (NCT04102579), a Phase 3 clinical trial that tested…

NeuExcell Therapeutics has entered into a research deal with Spark Therapeutics to advance the development of a safe and effective neuroregenerative gene therapy for Huntington’s disease. Under the terms of the agreement, Spark, a member of the Roche Group, will have access to NeuExcell’s gene therapy platform…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Vico Therapeutics’ VO659, an investigational antisense oligonucleotide gene-silencing therapy for Huntington’s disease. “We are delighted that FDA has granted this orphan-drug designation,” Rupert Sandbrink, MD, PhD, chief medical officer of VICO, said in a…

Prilenia Therapeutics has enrolled more than 120 patients in PROOF-HD, a Phase 3 clinical trial investigating the efficacy and safety of oral pridopidine in early stage Huntington’s disease. That amounts to 25% of the target enrollment of up to 480 patients, the company announced. The trial is…