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Target Snapshot

CHEMBL2179 Target Snapshot

Lysosomal acid glucosylceramidase · SINGLE PROTEIN · Homo sapiens

12,554Compounds
407Assays
4Approved Drugs
1,619.0Activities
Free Research Context

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Broader Open DB context

Frame the target before identity details

Structured disease, protein, and project context should read before the lower-level identity rail.

As of 2026-09-14

Lysosomal 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.

Review focus
Review-ready target frame

Start with Gaucher disease, then reuse UniProt P04062 as the stable protein anchor across project notes and exports.

ChEMBL component mapping + Open Targets-ready proxy + ChEMBL 36 via Core Engine 2026-09-14 1 linked drug
Open source guide
Disease program
Gaucher disease

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 proxy
Clinical signal Open Targets-ready proxy 1 linked drug
Open disease block
Identity Layer

Cross-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.

Review anchor
Lysosomal acid glucosylceramidase
SINGLE PROTEIN · Homo sapiens
As of 2026-09-14
ChEMBL target ID
CHEMBL2179
Primary anchor for compounds, assays, approvals, and exports
ChEMBL 36 via Core Engine As of 2026-09-14
www.ebi.ac.uk
UniProt accession
P04062
Lysosomal acid glucosylceramidase
UniProt accession via ChEMBL component mapping As of 2026-09-14
www.uniprot.org
Disease evidence contract
Open Targets-ready proxy
ChEMBL drug mechanism + indication proxy
ChEMBL drug mechanism + indication proxy As of 2026-09-14
www.ebi.ac.uk

Reuse the same identifiers in notes, watch rules, exports, and review packs so provenance stays stable across surfaces.

Source Guidance

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UniProt Target Context
UniProt accession via ChEMBL component mapping

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 Context
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Disease Relevance & Evidence Level
ChEMBL drug mechanism + indication proxy

Use 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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Evidence Card
Review-ready rationale with source-aware context

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Overview

Basic Information

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UniProt Context

UniProt Target Context

Reviewable protein naming and mapping context for this target snapshot.

Protein LabelLysosomal acid glucosylceramidase
UniProt AccessionP04062
Component TypeProtein
Sequence Length536 aa

Lysosomal acid glucosylceramidase

Source · UniProt accession via ChEMBL component mapping
Review Cue

How to read this target

Review this target as Lysosomal acid glucosylceramidase, mapped to UniProt P04062. Sequence length is 536 aa.

Mapped IDP04062
Review nameLysosomal acid glucosylceramidase
OrganismHomo sapiens
Disease Relevance

Disease Relevance & Evidence Level

Open Targets-ready disease context using the current target-indication evidence contract.

Clinical signal Open Targets-ready proxy

Lysosomal acid glucosylceramidase shows clinical signal disease relevance led by Gaucher disease.

Clinical signal Phase II
Gaucher disease
1 linked drug · 1 direct · 1 efficacy
Linked drugAFEGOSTAT TARTRATE
Source · ChEMBL drug mechanism + indication proxy
Review Cue

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.

Lead diseaseGaucher disease
Strongest levelClinical signal
Block modeOpen Targets-ready proxy
Clinical Profile

Clinical Phase Distribution

4
Approved
2
Phase III
6
Phase II
Assay Mix

Activity Type Distribution

IC501,035.0
KI292.0
EC50271.0
KD21.0
Lead Set

Top Inhibitors (by pChEMBL)

ChEMBL IDpChEMBLTypeValue (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
Distribution

pChEMBL Value Distribution

175
5.0
184
5.5
155
6.0
131
6.5
93
7.0
106
7.5
94
8.0
21
8.5
8
9.0
0
9.5
pChEMBL
Assay Landscape

Assay Landscape

407
Total Assays
759
Tested Compounds
2
Assay Types
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

Confidence Score Distribution

Source · ChEMBL 36 via Core Engine