AMT-130
Early / Mid-stage
active
active, not recruiting
CT.gov grounded
One of the most strategically important Huntington's programs because it represents a direct gene-therapy path rather than a small-molecule or ASO variant.
Program overview
- Mechanism
- HTT-lowering gene therapy
- Modality
- AAV gene therapy
- Phase
- Early / Mid-stage
- Status
- active
- Recruitment
- active, not recruiting
- Confidence
- medium
- Watch priority
- 1
- Last verified
- 20 Apr 2026
Sponsor and trial identifiers
- Sponsor
- uniQure
- Trial IDs
- NCT05243017, NCT04120493
ClinicalTrials.gov exact matches: NCT05243017, NCT04120493
Regions and recruitment
Poland
United Kingdom
United States
Indication tags
Huntington's disease
gene therapy
Milestones and catalysts
- Last milestone
- A major gene-therapy attempt in Huntington's disease that deserves a top-tier watch position.
- Next expected catalyst
- Longer-run safety and functional read-throughs from treated cohorts.
Related pages
Disease hub
Huntington's disease hub
Disease-specific views with programs, mechanisms, and related context.
Related programs
Each card explains why it is here so you can read the overlap at a glance.
Mid-stage
active
active, not recruiting
Huntingtin-lowering antisense program with unusually high field significance.
- Mechanism
- huntingtin-lowering antisense
- Modality
- ASO
- Next catalyst
- Updated data from the redesigned development path.
- Last verified
- 20 Apr 2026
Early / Mid-stage
active
recruiting
A repeat-targeting oligonucleotide program that broadens the Huntington's pipeline beyond the biggest legacy names.
- Mechanism
- RNA modulation of expanded repeats
- Modality
- antisense oligonucleotide
- Next catalyst
- Safety, pharmacokinetic, and early pharmacodynamic read-throughs.
- Last verified
- 20 Apr 2026
Late-stage
completed
completed
A major Huntington's program that deserves inclusion as part of the real clinical history of the field, not just the surviving winners.
- Mechanism
- sigma-1 receptor agonism
- Modality
- small molecule
- Next catalyst
- Mainly interpretation and strategic follow-through rather than a clean new expansion path.
- Last verified
- 20 Apr 2026
Early / Mid-stage
completed
completed
Small-molecule Huntington's program with mechanism-level significance if it continues to progress.
- Mechanism
- huntingtin mRNA splicing modulation
- Modality
- small molecule
- Next catalyst
- Further safety and target-engagement updates.
- Last verified
- 20 Apr 2026