Author Archives: Marisa Wexler MS

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…

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…