Ana de Barros, PhD, managing science editor —

Dutch pharmaceutical company uniQure says it plans to conduct preclinical studies leading to clinical testing of a gene therapy in patients with Huntington’s disease. The company is developing AMT-130, an experimental therapy that aims to block production of the mutated and aggregation-prone huntingtin protein in patients. Composed of a small…

Ionis Pharmaceuticals has completed enrollment of the Phase 1/2a study evaluating the safety, tolerability, properties and movement within the body of Ionis-HTTRx in patients with Huntington’s disease (HD). Patients who complete the randomized, placebo-controlled, dose escalation study (NCT02519036) will be eligible to enroll in an open-label…

Taube Philanthropies has donated $3 million to fund a research team using gene editing and stem cell techniques to develop treatments for Huntington’s disease. The team is composed of Dr. Matthew Porteus and Dr. Frank Longo of Stanford Medicine, and Dr. Steve Finkbeiner of the Gladstone Institutes, said a Stanford press…

Pope Francis will meet May 18 in the Vatican with patients and others who are fighting Huntington’s disease in what organizers are billing as the world’s largest gathering ever of the Huntington’s community. The event is part of Huntington’s advocates’ HDennomore global initiative. The phrase, which means Hidden No…

Teva Pharmaceutical Industries‘ Austedo tablets have received U.S. approval for treating chorea, or severe movement problems, associated with Huntington’s disease (HD). Austedo (deutetrabenazine, SD-809) is the first deuterated drug the U.S. Food and Drug Administration (FDA) has authorized, and only the second therapy it has approved for Huntington’s. The FDA had…

NUI Galway researcher and Professor Robert Lahue has been awarded a newly launched grant to investigate potential treatments for Huntington’s disease. The grant is a joint initiative of the Biotechnology and Biological Sciences Research Council UK (BBSRC) and the Science Foundation Ireland (SFI). The grant will provide nearly 700,000…

The European Union is inviting researchers in 20 countries to work together on projects focusing on Huntington’s disease (HD) and other degenerative diseases. The agency sponsoring the effort, the Joint Programme — Neurodegenerative Disease Research (JPND), said in a press release that neurodegenerative conditions have become a global health epidemic, with heavy economic…

A patent application for Vybion’s potential gene therapy for Huntington’s disease (HD) and other neurodegenerative conditions has been granted Track One status by the U.S. Patent and Trademark Office (USPTO). Patent applications are ordinarily reviewed in the order of their filing date, with the process taking about three…

The University of North Carolina at Chapel Hill recently launched an initiative intended to better equip scientists working to treat rare and orphan diseases, such as Huntington’s disease. The UNC Catalyst initiative, funded with a $2 million grant from the Eshelman Institute for Innovation, will provide patient groups and disease organizations…

Critical Path Institute (C-Path) and CHDI Foundation have joined forces to create a consortium geared toward clearly defining regulatory pathways for faster approval of new Huntington’s disease (HD) treatments. The new HD Regulatory Science Consortium (HD-RSC) will gather participants from the Huntington’s disease community to expedite regulatory endorsement and to facilitate…