“Executive function” describes how people attend to information, problem solve, and stay on task. Bruce’s team studies different aspects of executive function and how it becomes impaired in HD. #HDCongress2025
October 13, 2025 at 7:14 PM
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Nevertheless, scientists working on safely targeting HD genetic modifiers have shown encouraging results in mice when they lower the DNA repair genes MSH3 and PMS1. #HDCongress2025
October 13, 2025 at 4:21 PM
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Dr. Blair Leavitt from Incisive Genetics will present on the company's gene therapy, which is allele-selective meaning it only targets the expanded HTT gene. Their technology uses CRISPR to make cuts that lead to lower levels of HTT. #HDCongress2025
October 13, 2025 at 8:49 PM
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As a neurologist, Jang-Ho reminds the crowd that medicine in neurology is driven by 3 rules - “location, location, location”. In other words, for a gene therapy to work, it has to hit the right part of the brain and it has to be distributed in an efficient way. #HDCongress2025
October 13, 2025 at 9:26 PM
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We’re back! Kicking off the #HDCongress2025 afternoon programming is Dr. Jeff Carroll, HDBuzz Editor Emeritus and HD researcher, who will be talking about 2 recently accepted papers on the benefit of targeting the toxic HTT1a fragment that we learned about before the break.
October 13, 2025 at 6:33 PM
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This type of data will help answer questions around the contribution that HTT1a has to HD pathology. While researchers still can’t measure levels of the HTT1a fragment in people while they’re alive, this is something they’re working toward. #HDCongress2025
October 13, 2025 at 4:48 PM
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David ends by sharing his perspective that lowering HTT1a and full length expanded HTT are desirable paths toward treatment, but we still don’t have conclusive evidence. He hopes that the future of therapy could involve some combination of addressing mHTT and somatic instability. #HDCongress2025
October 13, 2025 at 4:56 PM
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About 10 years ago, an emergent technology allowed researchers like Dirk to study genetic profiles within brains at the single cell level. This massive library of information allows researchers to build intricate maps of the human brain to understand how it’s built and how it works. #HDCongress2025
October 13, 2025 at 3:16 PM
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Just a reminder that this data is not new, and that moving forward Novartis is planning a Phase 3 trial that will test votoplam in a larger group of people with early HD. If positive results hold, they hope the drug could help people across a range of HD stages. #HDCongress2025
October 12, 2025 at 4:20 PM
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He details the journey of how the HD field developed accurate ways to detect huntingtin protein in the spinal fluid, a convergence of many ideas and resources between academic and industry researchers over the course of a decade. #HDCongress2025
October 12, 2025 at 7:32 PM
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A recent paper examines the financial landscape in ALS and a common Catch-22: it’s hard to get funding for a clinical trial without demonstrating robust human data. The platform trial model is just one way to tackle the problem of helping companies gain momentum with good data. #HDCongress2025
October 12, 2025 at 3:03 PM
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He is reviewing what we know about the developmental ages at which different brain regions, features, and networks mature to drive different functions, some of which don’t come online until after the age of 25. #HDCongress2025
October 13, 2025 at 7:11 PM
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Jeff notes that he “can’t infer causation here” and we would really need a controlled clinical trial to understand if ADMs accelerate HD progression. So while there seems to be an association between ADM use and worsening of some HD symptoms, the results aren’t conclusive. #HDCongress2025
October 12, 2025 at 9:14 PM
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Blair reminds us that CRISPR is a tool involving a CAS9 enzyme, think of this like the molecular scissors that can cut DNA, alongside a guide RNA that targets the gene of interest (in this case, HTT). #HDCongress2025
October 13, 2025 at 8:51 PM
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The first talk in this session is from Hilary Wilkinson at CHDI who is diving into a new biomarker lots of researchers are trying to establish - how might we measure somatic instability, the changes in CAG number over time, and the challenges of tracking this possible driver of HD. #HDCongress2025
October 12, 2025 at 6:57 PM
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Next is Dr. Christopher Mezias from the Critical Path Institute, who will discuss frameworks for regulatory science and biomarker validation. Standardizing biomarker assays and benchmarks is key to accelerating approval of HD therapies. #HDCongress2025
October 13, 2025 at 9:05 PM
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The speakers stress that the research team, from coordinators to clinicians, is there to help folks navigate all the steps of the trial, as well as to support family members and study partners. #HDCongress2025
October 11, 2025 at 6:14 PM
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In a 3rd, “Part C” of the Phase 1 study, SKY-0515 is being tested in HD patients, first for 3 months at 2 doses, then participants have the opportunity to enter an open label extension for a year where everyone gets the drug. They are looking at safety, PD/PK, and some symptoms. #HDCongress2025
October 12, 2025 at 4:50 PM
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Welcome to Day 2 of #HDCongress2025! Daniel Claassen from HSG and Cristina Sampaio from CHDI kick off the meeting with an overview of what we’ll hear about today in HD clinical research and discovery.
October 12, 2025 at 2:10 PM
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Dr. Erin Furr Stimming from UTHealth Houston Neurosciences is now introducing the next session focused on young people and HD. This will be a vital discussion on youth, development, and inclusion. #HDCongress2025
October 13, 2025 at 7:01 PM
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Up now is Dr. Sam Frank, a clinician from Harvard. His talk will detail how the HD integrated staging system (HD-ISS) - developed solely for research - might someday guide patient care, from early detection to clinical decisions. #HDCongress2025
October 13, 2025 at 2:14 PM
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All of the panelists have reiterated that a clinical trial is a major commitment, and having a good understanding of exactly what you’ll be going through is an important part of making choices about participation! #HDCongress2025
October 11, 2025 at 3:27 PM
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This toxic HTT1a fragment is created through a biological process called “splicing” - you can think of this as similar to how movie reels can be cut and spliced together to alter scenes, ultimately piecing together the final product. #HDCongress2025
October 13, 2025 at 4:31 PM
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It also means companies can begin to target people at earlier disease stages with this granular understanding of HD. #HDCongress2025
October 13, 2025 at 2:27 PM
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A Phase 1 trial is about safety, Phase 2 side effects and how the drug works inside the body, and Phase 3 is a larger study to look at effectiveness in a larger population. #HDCongress2025
October 11, 2025 at 3:44 PM
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