Biomarker & Intervention Discovery
Huntington's Disease
Pharmacologically actionable gene targets, therapeutic agents ranked by evidence tier, and active clinical trials.
NIH funding$39MFY2025 · RCDC estimate
Funding levelWell-Funded
Spontaneous remissionInevitable progressive neurodegeneration in all carriers of HTT CAG expansion >36 repeats; no spontaneous remission…
Best-intervention remissionTetrabenazine/deutetrabenazine (VMAT2 inhibitors): 25-30% reduction in chorea score (FIRST-HD); no disease-modifying…
Gap sizeVery large — no approved disease-modifying treatment; symptomatic treatment only
Primary barrierbiological
Full remission profile →
Remission & chronicity
Spontaneous remission
Inevitable progressive neurodegeneration in all carriers of HTT CAG expansion >36 repeats; no spontaneous remission; motor, cognitive, psychiatric decline universally progressive
Best-intervention remission
Tetrabenazine/deutetrabenazine (VMAT2 inhibitors): 25-30% reduction in chorea score (FIRST-HD); no disease-modifying treatment proven; genetic CSF biomarker (mutant huntingtin, mHTT) reduction achieved with tominersen ASO — but Phase 3 halted 2021 (higher dose harmful); lower-dose tominersen now in Phase 2/3 after exploratory biomarker signal
Gap size
Very large — no approved disease-modifying treatment; symptomatic treatment only
Primary barrier
biological
HD is unique in having a perfectly predictive genetic test allowing pre-symptomatic intervention — the challenge is that trials of mHTT-lowering agents (tominersen, WVE-003) have faced setbacks; the therapeutic window (pre-manifest vs manifest disease) and appropriate dose remain unclear; genetic counselling for presymptomatic testing is complex
Biomarker Targets & Therapeutic Agents
Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC.
Agents ranked: ● Clinical >
● Mechanistic >
● Correlative.
Pipeline results not yet available. Run: python -m biomarker_pipeline.run_for_diseases --skip-llm
Clinical Trials
Recruiting and recently completed trials from ClinicalTrials.gov.
Data retrieved 2026-07-06.
No recent clinical trials found on ClinicalTrials.gov for this condition.
Agents Found By Disease-Level Search
Searched directly from “Huntington's Disease” via ClinicalTrials.gov interventions and Open Targets'
disease→drug data — independent of the 0-gene target panel above.
This surfaces agents whose mechanism doesn't route through a curated gene (combination
therapies, standard-of-care drugs, targets outside the panel).
Not yet run. python -m biomarker_pipeline.run_disease_agent_discovery --disease "..."