Start with Gaucher disease, then reuse UniProt P04062 as the stable protein anchor across project notes and exports.
CHEMBL2179 Target Snapshot
Lysosomal acid glucosylceramidase · SINGLE PROTEIN · Homo sapiens
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As of 2026-09-14Lysosomal acid glucosylceramidase shows clinical signal disease relevance led by Gaucher disease. 1 linked drug keeps the current disease frame grounded. Protein identity stays anchored to UniProt P04062. Start with Gaucher disease, then reuse UniProt P04062 as the stable protein anchor across project notes and exports.
Lysosomal acid glucosylceramidase shows clinical signal disease relevance led by Gaucher disease. 1 linked drug remains visible in the same block.
Start review with Gaucher disease because it currently carries clinical signal support. Linked drugs include AFEGOSTAT TARTRATE. This disease block keeps the Open Targets-ready contract explicit while current evidence comes from ChEMBL mechanism and indication mappings.
Disease source · ChEMBL drug mechanism + indication proxyLysosomal acid glucosylceramidase
Review this target as Lysosomal acid glucosylceramidase, mapped to UniProt P04062. Sequence length is 536 aa.
Protein source · UniProt accession via ChEMBL component mappingCross-source identity
Keep the review anchor, source IDs, and context contract aligned before reusing this target elsewhere.
This review treats Lysosomal acid glucosylceramidase as ChEMBL target CHEMBL2179, mapped to UniProt P04062. Disease framing currently uses the Open Targets-ready proxy contract.
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Start here when your first question is whether Lysosomal acid glucosylceramidase is the right protein anchor across sources, using UniProt P04062 as the stable mapping.
Jump to Protein ContextUse this block first when you need to confirm why Gaucher disease is currently framed as clinical signal support. It currently carries 1 linked drug in the same disease frame.
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Open Review-ready CardBasic Information
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| Preferred Name | Lysosomal acid glucosylceramidase |
| Target Type | SINGLE PROTEIN |
| Organism | Homo sapiens |
| UniProt | P04062 |
Next Actions
UniProt Target Context
Reviewable protein naming and mapping context for this target snapshot.
| Protein Label | Lysosomal acid glucosylceramidase |
| UniProt Accession | P04062 |
| Component Type | Protein |
| Sequence Length | 536 aa |
Lysosomal acid glucosylceramidase
How to read this target
Review this target as Lysosomal acid glucosylceramidase, mapped to UniProt P04062. Sequence length is 536 aa.
Disease Relevance & Evidence Level
Open Targets-ready disease context using the current target-indication evidence contract.
Lysosomal acid glucosylceramidase shows clinical signal disease relevance led by Gaucher disease.
How to read disease relevance
Start review with Gaucher disease because it currently carries clinical signal support. Linked drugs include AFEGOSTAT TARTRATE. This disease block keeps the Open Targets-ready contract explicit while current evidence comes from ChEMBL mechanism and indication mappings.
Clinical Phase Distribution
Activity Type Distribution
Top Inhibitors (by pChEMBL)
| ChEMBL ID | pChEMBL | Type | Value (nM) | Phase |
|---|---|---|---|---|
| CHEMBL2029773 | 9.4 | Ki | 0.4 | Preclinical |
| CHEMBL5192083 | 9.22 | IC50 | 0.6 | Preclinical |
| CHEMBL3684925 | 9.05 | Ki | 0.9 | Preclinical |
| CHEMBL3692692 | 9.02 | Ki | 0.95 | Preclinical |
| CHEMBL2164231 | 9.0 | Ki | 1.0 | Preclinical |
| CHEMBL3289676 | 9.0 | Ki | 1.0 | Preclinical |
| CHEMBL4080589 | 9.0 | Ki | 1.0 | Preclinical |
| CHEMBL3350092 | 8.92 | Ki | 1.2 | Preclinical |
| CHEMBL3692694 | 8.81 | Ki | 1.56 | Preclinical |
| CHEMBL206468 | 8.77 | Ki | 1.7 | Clinical |
pChEMBL Value Distribution
Assay Landscape
Binding — 374 assays, 759 compounds
| BAO Format | Assays | Compounds | Avg pChEMBL |
|---|---|---|---|
| single protein format | 234 | 603 | 6.2 |
| cell-based format | 70 | 86 | 6.3 |
| assay format | 56 | 41 | 5.0 |
| tissue-based format | 13 | 25 | 5.7 |
| organism-based format | 1 | 4 | 7.2 |
Functional — 33 assays, 4 compounds
| BAO Format | Assays | Compounds | Avg pChEMBL |
|---|---|---|---|
| assay format | 28 | 0 | — |
| tissue-based format | 4 | 0 | — |
| cell-based format | 1 | 4 | 5.4 |