Dna (Recombinant Replication-Defective Adeno-Associated Virus Serotype 9 Vector Ux701 Murine Transthyretin Enhancer Plus Human Transthyretin Promoter Plus Simian Virus 40 Intron Plus Human Adenosine Triphosphatase Copper-Transporting ? Metal-Binding Domain 4/5/6 Transgene Atp7B-Mbd456 Plus Simian Virus 40 Polyadenylation Signal Plus Adeno-Associated Virus 2 Inverted Terminal Repeat Flanks Plus Adeno-Associated Virus 2 Rep Region Plus Adeno-Associated Virus 9 Cap Region-Specifying)
Wilson Disease
Explore 11 assets in Wilson Disease →Floatz Rating
CC47/100
Confidence
Indicative
Sponsor
Ultragenyx Pharmaceutical Inc
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.
33Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT04884815CT.gov A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease | — | Active Not Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
89Indicative
Detailed axis rationale is planned and will be published soon.
Same indication · Wilson Disease
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Dna (Recombinant Replication-Defective Adeno-Associated Virus Serotype 9 Vector Ux701 Murine Transthyretin Enhancer Plus Human Transthyretin Promoter Plus Simian Virus 40 Intron Plus Human Adenosine Triphosphatase Copper-Transporting ? Metal-Binding Domain 4/5/6 Transgene Atp7B-Mbd456 Plus Simian Virus 40 Polyadenylation Signal Plus Adeno-Associated Virus 2 Inverted Terminal Repeat Flanks Plus Adeno-Associated Virus 2 Rep Region Plus Adeno-Associated Virus 9 Cap Region-Specifying) (this asset) | Ultragenyx Pharmaceutical Inc | P1/2 | CC · 47 |
| Zinc Acetate | Nobelpharma | P4 | BBB |
| Teta 4Hcl | Orphalan | P3 | BBB |
| Zinc D-Gluconate (1:2) | Jun Li | P4 | BBB |
| Trientine | Excelsior | P3 | BB |
| Penicillamine | Orphalan | P3 | BB |
| Alxn1840 | Alexion Pharmaceuticals, Inc. | P3 | BB |
| Gandouling | Jun Li | P4 | BB |
| Tetrathiomolybdate | National Center for Research Resources (NCRR) | P3 | B |
+11 more in the Wilson 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-Defective Adeno-Associated Virus Serotype 9 Vector Ux701 Murine Transthyretin Enhancer Plus Human Transthyretin Promoter Plus Simian Virus 40 Intron Plus Human Adenosine Triphosphatase Copper-Transporting ? Metal-Binding Domain 4/5/6 Transgene Atp7B-Mbd456 Plus Simian Virus 40 Polyadenylation Signal Plus Adeno-Associated Virus 2 Inverted Terminal Repeat Flanks Plus Adeno-Associated Virus 2 Rep Region Plus Adeno-Associated Virus 9 Cap Region-Specifying) in Wilson 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-defective-adeno-associated-virus-serotype-9-vector-ux701-murine-transthyretin-enhancer-plus-human-transthyretin-promoter-plus-simian-virus-40-intron-plus-human-adenosine-triphosphatase-copper-transporting-metal-binding-domain-456-transgene-atp7b-mbd456-plus-simian-virus-40-polyadenylation-signal-plus-adeno-associated-virus-2-inverted-terminal-repeat-flanks-plus-adeno-associated-virus-2-rep-region-plus-adeno-associated-virus-9-cap-region-specifying-wilson-disease
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