This week the 2020 European Huntington’s Disease Network (EHDN) hosted a virtual plenary meeting to discuss the latest in HD research and clinical trials. You can use these links to access the entire program and all of the recorded sessions. Here are some highlights: 

Dr. Sarah Tabrizi presented an update on the Roche/Genentech trial of their huntingtin-lowering ASO, tominersen, which is delivered into the spinal cord either 3 or 6 times yearly for 2 years. There are about 800 participants worldwide and we expect data by 2022.  

Wave Life Sciences is also testing two spinally-delivered ASO drugs, both of which are designed to lower mutant huntingtin while leaving healthy huntingtin intact. Their latest update was presented by Dr. Anna Heinzmann. She focused on the reasoning behind Wave’s approach and shared that more data will be available in the first quarter of 2021.   

uniQure is conducting a gene therapy study to test the safety of a drug called AMT-130 which is delivered one time via a brain surgery. Dr. Ralf Reilman presented on behalf of the AMT-130 study team. The trial has begun, with 9 sites in the USA, and now has an official name: HDGeneTRX1.    

The SHIELD-HD study, being conducted by Triplet Therapeutics, will follow 60 HD gene carriers for two years, collecting medical history, blood donations, and neurological test data. The study will help inform Triplet’s future drug trials to stop the expansion of CAG repeats, which continues during an HD patient’s lifetime. Dr. Anne Rosser provided an update. This study is currently recruiting in the USA and Europe and you can find more information at and at   

Additional talks in the clinical trials update session focused on  

·        A new Phase 3 trial of Pridopidine (PROOF-HD) revisiting this drug in a larger group of people. Previous trials were not successful but because it showed some exploratory promise   

·        A trial of stem cell implants in HD patients conducted between 2001 and 2013. It was unfortunately not successful, but there were many important lessons learned, and many technological developments in the stem cell field since the trial began 20 years ago.  

·        Hugh Rickards from the University of Birmingham spoke about the challenges for HD researchers, companies, health systems, and families in ensuring equal access to the HD genetic drugs we anticipate coming to market in the future.   

NYA Virtual Youth Retreats 

HDSA’s National Youth Alliance is hosting a series of Virtual Retreats this fall. Just like their in-person retreats, Virtual NYA Retreats are open to folks 12-29 who are in any way impacted by Huntington’s disease. As always, NYA retreat sessions will include a discussion of the latest in HD research: not only talking about the science, but also creating a space to process the complex emotions that can arise around research news and decisions about participation. You can sign up to participate for free using this link. Sessions will be capped at 20 and are first-come, first-served.  

HDSA Founder’s Day of Giving 

Help support HDSA on Friday September 18th, by joining us for the HDSA Founder’s Day of Giving.  

An anonymous passionate family is generously matching all donations TOMORROW up to $10,000, meaning your gift will have double the impact for families living with HD.  

Your dollars go to support all of HDSA’s programs, including our research initiatives.