ISOFAGOMINE
Type 1 Gaucher Disease
Amicus TherapeuticsSmall moleculePhase 2Dormant
BB
67/ 100
Provisional rating · Methodology v0.2
Rating breakdown
- Clinical Evidence
- 52/ 100
- Competitive Position
- 95/ 100
Competitive context
0 Active assets in the Type 1 Gaucher Disease cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
Elelyso Pfizer | Phase 4 | BB |
at2101 Amicus Therapeutics | Phase 1 | CC |
AUTOLOGOUS CD34+ CELLS ENCODING .BETA.A-T87Q-GLOBIN GENE AVROBIO | D |
Same molecule, other indications
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