After Scientific Advisory Board review, HDSA is excited to award the Donald A. King Summer Research Fellowship, to two undergraduate researchers. This program, named for former HDSA board chair and tireless advocate Donald King, who passed away in 2004, aims to prepare the next generation of Huntington’s disease (HD) scientists. Ratnesh Kesineni, from the University of Central Florida, will be mentored by former HDSA Human Biology Fellow, Amber Southwell, PhD, to explore a possible link between biological age and aggregation of misfolded mutant huntingtin (mHTT) for potential therapeutic targeting. Russell Wells, from Whitworth University, will work with Michael Sardinia, PhD, DVM, to study the effects of dihexa, a small molecule that has previously shown positive effects in Alzheimer’s and Parkinson’s disease models, for the treatment of Huntington’s disease phenotypes in mice models. We’re delighted to welcome these young researchers to the HDSA family.
Movement Disorders Foundation Virtual Symposium: “Connecting Science to Hope”
Join the Movement Disorders Foundation on May 15th for a free, virtual symposium, “Connecting Science to Hope.” This one-day event will bring together leading scientists and clinicians to discuss the latest advances in movement disorder research and clinical trials. Participants will have the opportunity to attend breakout sessions on Parkinson’s disease, Huntington’s disease, Dystonia, or Essential Tremor. To register and learn more, visit www.movementdisordersfoundation.org/events
Voyager Therapeutic to proceed with Gene Therapy Phase 1/2 Clinical trial
In a recent press release, Voyager Therapeutics announced that the FDA has removed a clinical hold on their investigational new drug, VY-HTT01, a potential disease-modifying treatment for Huntington’s disease (HD). After a thorough review of chemistry, manufacturing, and controls, the FDA confirmed that Voyager may proceed with a phase 1/2 clinical trial of the gene therapy candidate that is intended to begin later this year. VY-HTT01 uses a harmless virus called an AAV to transport a genetic drug into brain tissue. Once in the brain, it interferes with the huntingtin recipe so that less harmful protein is made. Promising pre-clinical data collected from non-human primates shows lowering of HTT-mRNA and HTT protein in the brain, and good drug distribution through areas of the brain affected by HD. The upcoming phase 1/2 study will evaluate the safety and tolerability of VY-HTT01 in individuals in the early manifest stage of HD. Participants will receive a one-time dose of VY-HTT01 via MRI-guided neurosurgery.
Roche/Genentech Community Update on Generation HD-1
In case you missed it, last week HDSA was joined by Drs. Lauren Boak (Roche), Diana Slowiejko (Genentech), and Vicki Wheelock (Director of the HDSA Center of Excellence at UC Davis) to discuss the results of Generation HD-1 and to answer questions from the community on what we can deduce from the data thus far. The webinar recording is available on HDSA’s YouTube channel here.