MCOLN1

Mucolipin-1

Score: 0.586 Price: $0.59 Low Druggability Status: active Wiki: MCOLN1

🧠 Neurodegeneration

HYPOTHESES

PAPERS

KG EDGES

250

DEBATES

3D Protein Structure

🧬 MCOLN1 — PDB 5W3S Click to expand

Experimental structure from RCSB PDB | Powered by Mol*

Druggability & Clinical Context

Druggability

Low

Score: 0.41

Clinical Stage

Phase I

Target Class

Ion Channel

Safety

0.30

Druggability Analysis

Drug Development0.15

Structural Tractability0.85

Target Class0.85

Safety Profile0.30

Key Metrics

PDB Structures:

Known Drugs:

Approved:

In Clinical Trials:

Drug Pipeline (1 compounds)

1 Preclinical

Therapeutic Areas:

Mucolipidosis type IV Lysosomal storage disorders Neurodegeneration Autophagy-related diseases Lysosomal dysfunction disorders

Druggability Rationale: MCOLN1 presents moderate druggability (0.60) with strong structural support from 38 PDB structures at 2.1 Å resolution and cryo-EM data, enabling rational design of small molecule modulators. The existence of ML-SA1 as a validated research tool demonstrates feasibility, though ion channels typically present challenges with shallow binding pockets and potential selectivity issues across TRP channel family members.

Mechanism: Small molecule agonists or modulators of lysosomal calcium channel activity

Drug Pipeline (1 compounds)

1 Preclinical

Known Drugs:

ML-SA1 (research_tool) — Lysosomal storage disorders

Structural Data:

PDB (38) ✓AlphaFold ✓Cryo-EM ✓

5TJA 5TJB 5TJC 5WJ5 5WJ9+33 more

UniProt: M0QXD0

Binding Pocket Analysis:

Structural data suggests potential allosteric modulation sites distinct from the ion conduction pore, as evidenced by ML-SA1's agonist mechanism; high-resolution structures indicate multiple druggable pockets suitable for small molecule engagement, though the exact binding mode of current agonists remains under investigation.

🧬 3D Protein Structure

🧬 MCOLN1 — PDB 5W3S Click to expand interactive 3D viewer

Experimental structure from RCSB PDB | Powered by Mol* | Rotate: click+drag | Zoom: scroll

Selectivity & Safety Considerations

Selectivity is a key challenge given MCOLN1's structural homology to other TRP channel family members (TRPML2, TRPML3); achieving selectivity will require targeting unique structural features or allosteric sites specific to MCOLN1. Off-target effects on related ion channels could cause unwanted lysosomal dysfunction in peripheral tissues.