Dna (Recombinant Replication-Deficient Adeno-Associated Virus 5 Vector Amt-130 Chicken .Beta.-Actin Promoter Plus Human Huntingtin Mrna-Targeting Microrna Mihtt-Specifying)
Huntington Disease
Explore 25 assets in Huntington Disease →Floatz Rating
CC45/100
Confidence
Indicative
Sponsor
UniQure Biopharma B.V.
Modality
gene_therapy
Development Phase
Phase 1/2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
33Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05243017CT.gov Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease | — | Active Not Recruiting | — | — | — |
NCT04120493CT.gov Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease | — | Active Not Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
81Indicative
Detailed axis rationale is planned and will be published soon.
Same indication · Huntington Disease
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Dna (Recombinant Replication-Deficient Adeno-Associated Virus 5 Vector Amt-130 Chicken .Beta.-Actin Promoter Plus Human Huntingtin Mrna-Targeting Microrna Mihtt-Specifying) (this asset) | UniQure Biopharma B.V. | P1/2 | CC · 45 |
| Deutetrabenazine | Teva Branded Pharmaceutical Products R&D LLC | P4 | BBB |
| Tetrabenazine | William Ondo, MD | P4 | BBB |
| Tominersen | Hoffmann-La Roche | P3 | BBB |
| Latrepirdine | Medivation, Inc. | P3 | BB |
| Pridopidine | Prilenia | P3 | BB |
| MT-5199 | Neurocrine Biosciences | P3 | BB |
| Memantine | University of British Columbia | P4 | BB |
| Dalzanemdor | Supernus Pharmaceuticals, Inc. | P3 | BB |
+42 more in the Huntington Disease cohort
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Related assets
Citation
Floatz Terminal. Dna (Recombinant Replication-Deficient Adeno-Associated Virus 5 Vector Amt-130 Chicken .Beta.-Actin Promoter Plus Human Huntingtin Mrna-Targeting Microrna Mihtt-Specifying) in Huntington Disease. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/dna-recombinant-replication-deficient-adeno-associated-virus-5-vector-amt-130-chicken-beta-actin-promoter-plus-human-huntingtin-mrna-targeting-microrna-mihtt-specifying-huntington-disease
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