HD Clinical Research Congress 2025 | Presentations
The first HD Clinical Research Congress 2025 was held October 12-13 in Nashville, Tennessee, and the program developed jointly by the Huntington Study Group and CHDI. This year’s program, developed by the Huntington Study Group, CHDI Foundation, and the European Huntington’s Disease Network, features long-form platform presentations, new data on HD biomarkers and assessments, and forward-looking conversations on clinical trial design. Recorded presentations from the Enroll-HD component of this meeting are displayed below.
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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