Floatz Rating
CC43/100
Confidence
Indicative
Sponsor
University of California, San Francisco
Modality
protein_peptide
Development Phase
Phase 1
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
28Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT04532047CT.gov PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) | — | Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Wolman Disease
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Iduronidase, .Alpha.-L-(8-Histidine) (Human) (this asset) | University of California, San Francisco | P1 | CC · 43 |
| Cyclophosphamide | Masonic Cancer Center, University of Minnesota | P2/3 | BB |
| Busulfanum | Masonic Cancer Center, University of Minnesota | P2/3 | BB |
| Anti-Human T-Lymphocyte Immunoglobulin, Rabbit | Masonic Cancer Center, University of Minnesota | P2/3 | B |
| Sebelipase Alfa | Alexion Pharmaceuticals, Inc. | P2/3 | B |
| Hydroxyurea | Masonic Cancer Center, University of Minnesota | P2 | B |
| Clofarabine | Masonic Cancer Center, University of Minnesota | P2 | B |
| Melphalan | Masonic Cancer Center, University of Minnesota | P2 | B |
| Alemtuzumab | Masonic Cancer Center, University of Minnesota | P2 | B |
+2 more in the Wolman Disease cohort
Other indications for Iduronidase, .Alpha.-L-(8-Histidine) (Human)
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Mucopolysaccharidosis Type 1 | — | P4 | BBB · 72 |
| Hurler Syndrome | — | P4 | BB · 68 |
| Hurler-Scheie Syndrome | — | P4 | BB · 65 |
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
- CyclophosphamideMasonic Cancer Center, University of MinnesotaBB
- BusulfanumMasonic Cancer Center, University of MinnesotaBB
- Anti-Human T-Lymphocyte Immunoglobulin, RabbitMasonic Cancer Center, University of MinnesotaB
- Sebelipase AlfaAlexion Pharmaceuticals, Inc.B
- HydroxyureaMasonic Cancer Center, University of MinnesotaB
Citation
Floatz Terminal. Iduronidase, .Alpha.-L-(8-Histidine) (Human) in Wolman 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/iduronidase-alpha-l-8-histidine-human-wolman-disease
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