HD Clinical Research Congress 2025 | Presentations
The first Huntington’s Disease Clinical Research Congress was held October 12-13, 2025, in Nashville, Tennessee. The program, developed jointly by the Huntington Study Group and CHDI, features long-form platform presentations, new data on HD biomarkers and assessments, and forward-looking conversations on clinical trial design.
Video Index
Clinical research day (Sunday October 12, 2025)
Keynote - Progress in clinical developments
Merit Cudkowicz, MD, MSC, Mass General Brigham Neuroscience Institute & Harvard Medical School | Keynote: Clinical trial design
Presentation not made available by presenter.
Huntington’s disease clinical trial updates
Victor Sung, MD, University of Alabama at Birmingham | Topline results from pivotal phase I/II study of uniQure’s AMT-130, an investigational huntingtin lowering gene therapy candidate
Beth Borowsky, PhD, Novartis Pharmaceuticals | Votoplam in HD: Results from the PIVOT-HD phase 2 study and plans for a phase 3 study
Peter McColgan, MD, PhD, Roche | Toward disease-modifying treatments in HD: An update from Roche
Meghan Miller, PhD, Skyhawk Therapeutics | SKY-0515: A small molecule targeting the cause of Huntington’s disease - clinical program update
Presentation not made available by presenter.
Progress in biomarker research
Hilary Wilkinson, PhD, CHDI | The Moving Target: Challenges in measuring CAG-repeat instability for Huntington’s disease therapies
David Hawellek, PhD, Roche | When research assays become decision tools - (m)HTT and NfL
Kilian Hett, PhD, Vanderbilt University Medical Center | Cerebrospinal fluid circuit in Huntington’s disease
Jamie Adams, MD, University of Rochester | Digital measures in Huntington’s disease
Clinical research insights
Jeff Long, PhD, University of Iowa | Part B: Antidopaminergics and worsening of clinical severity
Stan Lazic, PhD, Prioris.ai | Part C: Contribution of psychological diagnosis to HD-ISS staging
Clinical practice day (Monday October 13, 2025)
Translational issues in Huntington’s disease
Sam Frank, MD, Beth Israel Deaconess Medical Center & Harvard Medical School | Part A: Can the HD-ISS be used in clinic?
C Dirk Keene, MD, PhD, University of Washington | Part B: Neuropathology needs in HD
Joel Braunstein, MD, MBA, C2N Diagnostics | Part C: Trials & triumphs on the journey to developing the first highly accurate blood test for Alzheimer’s disease diagnosis
Presentation not made available by presenter.
Science for clinicians: Hot topics that are important to communicate in clinic
Davina Hensman-Moss, MA(Oxon), MBBS, MRCP, PhD, University College London Institute of Neurology | Part A: Somatic instability: Why it is now a therapeutic target
David Howland, PhD, CHDI | Part B: The emergence and potential importance to target and lower mutant HTT1a/exon1 in HD
Young people and Huntington’s disease
Bruce E Compas, PhD, Vanderbilt University | Part A: Cognition in young people: A developmental perspective on HD
Cristina Sampaio, MD, PhD, CHDI | Part B: Rationale for inclusion/exclusion criteria for clinical trials
Martha Nance, MD, Hennepin HealthCare HD Center of Excellence & Struthers Parkinson’s Center | Part C: Reflections on treating juvenile HD
Blair Leavitt, MDCM, FRCP(C), Incisive Genetics | Targeted allele-selective excisions in the HTT gene: Incisive Genetics’ path to the clinic for Huntington’s disease
Christopher Mezias, PhD, Critical Path Institute | A regulatory science framework for developing biomarkers and pursuing regulatory review in Huntington’s disease using volumetric MRI as a case study
Presentation not made available by presenter.
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