Iduronidase, .Alpha.-L-(8-Histidine) (Human)
Gaucher Disease Type III
Explore 7 assets in Gaucher Disease Type III →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 · Gaucher Disease Type III
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Iduronidase, .Alpha.-L-(8-Histidine) (Human) (this asset) | University of California, San Francisco | P1 | CC · 43 |
| Elelyso | Ari Zimran | P4 | BB |
| .Alpha.-D-Glucosyl-N-Acylsphingosine Glucohydrolase | Sanofi | P3 | BB |
| Eliglustat | Sanofi | P3 | BB |
| Venglustat | Sanofi | P3 | B |
| Velaglucerase Alfa | Shire | P1/2 | CCC |
| Arimoclomol | ZevraDenmark | P2 | CCC |
| Low-Dose Can103 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | P1/2 | CCC |
| High-Dose Can103 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | P1/2 | CCC |
+1 more in the Gaucher Disease Type III 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
Citation
Floatz Terminal. Iduronidase, .Alpha.-L-(8-Histidine) (Human) in Gaucher Disease Type III. 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-gaucher-disease-type-iii
Are you the sponsor?
Submit your data room for a Verified Rating. The public-data rating remains visible alongside.
Contact →