News

GENERATION HD1 Trial Fails to Show Benefits of Tominersen

Treatment with tominersen failed to show evidence of clinical benefit in adults with Huntington’s disease during the Phase 3 clinical trial GENERATION HD1. Roche, the company developing tominersen, decided to pause dosing in the clinical trial about a month ago, after a pre-planned review of trial data…

‘Let’s Talk About’ Huntington’s Disease Awareness in May

Activities are underway worldwide to mark Huntington’s Disease Awareness Month, set aside each year to call attention to the neurodegenerative disorder that is thought to affect three to seven per every 100,000 people of European ancestry. May 15 is International Huntington’s Disease Awareness Day. Patients, caregivers, family, and friends…

FDA Gives Go-ahead for Phase 1/2 Trial Testing VY-HTT01 Gene Therapy

The U.S. Food and Drug Administration (FDA) has granted Voyager Therapeutics permission to start a clinical trial testing VY-HTT01, its investigational gene therapy for Huntington’s disease. Voyager is planning to launch the Phase 1/2 clinical trial, called VYTAL, later this year. VYTAL will be a dose-escalation study, meaning that…

Protein With ‘Switch’ Sensitive to Clumping May Aid in Treating Disease

A chaperone protein called DnaJB8 has an internal molecular “switch” that likely regulates how cells dispose of protein aggregates, like those that cause Huntington’s disease. These findings were detailed in the study “Regulatory inter-domain interactions influence Hsp70 recruitment to the DnaJB8 chaperone,” published in Nature Communications. Huntington’s is…

Consortium Will Promote Stem Cell Therapies

A new international consortium, Stem Cells for Huntington’s Disease (SC4HD), has been formed to help facilitate the development of stem cell-based therapies to treat the disorder. The consortium was presented in the Journal of Huntington’s Disease, in the paper, “Stem Cells for Huntington’s Disease (SC4HD): An International…

Anima Biotech, Takeda Team Up to Develop Small Molecule Treatments

Anima Biotech has partnered with Takeda Pharmaceuticals to develop small molecules that control mRNA translation — the process by which the information encoded in a person’s DNA is “translated” into a protein — as potential therapies for neurological disorders with a genetic cause, including Huntington’s disease. Huntington’s…