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
Ultragenyx Pharmaceutical Incgene_therapyPhase 1/2Active
CC
47/ 100
Provisional rating · Methodology v0.2
Rating breakdown
- Clinical Evidence
- 33/ 100
- Competitive Position
- 89/ 100
Competitive context
11 Active assets in the Wilson Disease cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
ZINC ACETATE Nobelpharma | Phase 4 | BBB |
Teta 4Hcl Orphalan | Phase 3 | BBB |
ZINC D-GLUCONATE (1:2) Jun Li | Phase 4 | BBB |
Trientine Excelsior | Phase 3 | BB |
Penicillamine Orphalan | Phase 3 | BB |
View all 21 →
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