Where Huntington's disease treatment is heading
Huntington's disease · Updated Nov 14, 2026
Huntington's disease is an inherited brain disease caused by an expanded CAG repeat in the huntingtin gene; about 41,000 Americans have symptoms and many more carry the gene, with a few thousand diagnosed each year. It causes involuntary movements, psychiatric problems and dementia, usually starting in midlife. Current drugs only ease movement symptoms. The field is now focused on lowering the harmful huntingtin protein, with a one-time gene therapy under FDA review and several pills in phase 3.
- 64studies open or running
- 7phase 3 trials
- 1,056papers published this year
- 40NIH-funded projects
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Now
Nov 2026
Gene therapy slows the diseaseHappened 75% slower progression
AMT-130 showed about 75% slower progression than matched patients over 3 years.
75%: slowing of disease progression (cUHDRS) vs matched controls.
An FDA filing after a reversalHappened
The FDA first asked for a sham-controlled trial, then allowed an accelerated-approval filing; uniQure submitted in 2026.
Other approaches fell shortHappened -0.11 difference (not significant)
Pridopidine (difference -0.11) and laquinimod did not beat placebo in their main analyses.
-0.11: difference in cUHDRS vs placebo (not significant).
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6 months
by May 2027
Tominersen resultsPossible
Roche's GENERATION HD2 (301 people), testing lower doses of an antisense drug in younger, earlier patients, finished in 2026.
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12 months
by Nov 2027
An FDA decision on gene therapyPossible 75% slower progression
With priority review, a decision on AMT-130 could come in 2027.
75%: slowing of disease progression (cUHDRS) vs matched controls.
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3 years
by late 2029
Huntingtin-lowering pillsPossible
Skyhawk's SKY-0515 FALCON-HD (400 people) is due December 2028.
Pridopidine in the right subgroupLong shot
PRECISE-HD (400 people) tests pridopidine in people not taking antidopaminergic drugs; due June 2028.
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5 years
by late 2031
A large pill trialPossible
Novartis' INVEST-HD (770 people) tests votoplam, an oral splicing modifier that lowers huntingtin; due April 2030.
More gene therapiesLong shot
Roche's SPK-10001 gene therapy and stem-cell approaches are in early trials.
The research paths
Gene therapy
75% slower progressionDetails and 1 trialsHide
One-time brain injections that lower huntingtin.
WatchFDA decision on AMT-130.
Huntingtin-lowering pills and antisense
Details and 4 trialsHide
Oral splicing modifiers and antisense drugs that reduce huntingtin production.
WatchINVEST-HD; FALCON-HD; GENERATION HD2.
- Votoplam, a huntingtin-lowering pill (INVEST-HD)Novartis · 770 people · enrolling Apr 2030
- SKY-0515, a huntingtin-lowering pill (FALCON-HD)Skyhawk Therapeutics · 400 people · enrolling Dec 2028
- SKY-0515 early studySkyhawk Therapeutics · 120 people · fully enrolled May 2027
- Tominersen, an antisense drug (GENERATION HD2)Roche · 301 people · fully enrolled May 2026
Neuroprotection
-0.11 difference (not significant)Details and 3 trialsHide
Drugs protecting nerve cells without lowering huntingtin; results so far disappointing.
WatchPRECISE-HD; NAD-HD.
Borrowed from other conditions
From Spinal muscular atrophy
Splicing modifiers (votoplam)
Phase 3
The same type of RNA-splicing pill as risdiplam, approved for SMA.
From Parkinson disease research
Nicotinamide riboside
Phase 2
A vitamin B3 form tested to support brain cell energy.
Open to join now
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Early Huntington disease
Votoplam
Locations and eligibility → -
Early Huntington disease
Pridopidine
Locations and eligibility → -
Huntington disease
SKY-0515
Locations and eligibility → -
Early Huntington disease
SPK-10001 gene therapy
Locations and eligibility →
See every recruiting Huntington's trial →
When the trials finish
Completion dates the trials registered. Results usually follow 3 to 12 months later.
- Heating up
- Maturing
- Early
- Cooling
- Fully enrolled, waiting on results
- Still enrolling
What just happened
FDA allows accelerated-approval filing for AMT-130
After an earlier reversal.
Pridopidine misses main goal
PROOF-HD, Nature Medicine.
Standard treatment today
- Gene carrier, no symptomsGenetic counseling and monitoring; prevention trials are being planned.
- Early symptomsTetrabenazine, deutetrabenazine or valbenazine for chorea; clinical trials of disease-slowing drugs.
- Moderate diseaseSymptom treatment, psychiatric care, physical, occupational and speech therapy.
- Advanced diseaseSupportive and palliative care.
How to read this page, and sourcesHow to read this page, and sources
- The gene therapy result compares patients with matched external data rather than a placebo group; regulators are weighing how much that evidence shows.
- Forecasts are editorial judgments by Find a Trial, not predictions from sponsors or regulators.
See Huntington's trials recruiting now →