uniQure shared a press release this week stating that their safety trial of an HD gene therapy drug, AMT-130, is on track and even ahead of schedule. This is a small study (26 participants) that requires a surgery to deliver the drug directly to the part of the brain most affected by HD. Over the course of nearly a year, uniQure has carefully recruited the first group (cohort) of 10 participants in the USA, and surgeries have been spaced every few months to ensure safety. The treatment involves delivery of an HD-fighting virus deep into the brain that has the potential to permanently lower levels of huntingtin protein. Six participants so far have received AMT-130 and four received placebo. In the next cohort of 16, ten will receive drug and six placebo. Furthermore, uniQure announced that another AMT-130 trial will begin in Europe in the second half of 2021. In this Phase 1b/2 study, 15 participants will receive the drug, and there will be no placebo group – known as an open-label trial. Overall we are delighted to hear that studies of AMT-130 are on track and no dangerous safety concerns have arisen. You can learn more about AMT-130 and uniQure’s work in HD on their website and the nine recruiting US sites for this study are listed at www.hdtrialfinder.org.
16 Years of Gene Veritas
HD Advocate and gene carrier, Gene Veritas, recently celebrated his 300th blog entry under the alias adopted after his positive HD diagnosis. Born of HD-induced isolation for fear of genetic discrimination, the pseudonym has served as a platform for the 61-year-old history professor and family man to chronicle his journey to understand “the truth in his genes.” Since the blog’s inception 16 years ago, Gene’s readers have borne witness to information and research on Huntington’s Disease, reflections on his mother’s disease progression (expanded upon in another recent post), his own fears of HD onset, and coping strategies. In this reflective post, Gene commemorates the identity of his 16-year-old pseudonym, the reveal of his true identity, Kenneth Serbin, 9 years ago, and other milestones of his advocacy, including meeting Pope Francis in 2017 in his special audience with the HD community, “HDdennomore.” His other latest entries have focused on Gene’s reflections on recent challenging news for the HD community, the halting of Roche’s Generation HD-1 and Wave’s Precision-HD studies.
Research Spotlight: Berman-Topper Fellow Lauren Byrne
In a recent interview, we sat down virtually with HDSA Berman-Topper Fellow, Dr. Lauren Byrne, to learn more about her path to HD research, passion for the HD community, and goals for the future. To learn more about Lauren and her work, check out her Researcher Spotlight article here.
Lauren also spoke with us about her work as a board member and research consultant for the Huntington’s Disease Youth Organization (HDYO). This week, she participated in a discussion about the recent news from Roche and Wave, optimism for the future of ASO’s and other disease-modifying therapies in the pipeline, and what we can take from these trials moving forward. Check out the recording here.
