Drug intelligence / Profile preview

ML-SA1

Development stage
Preclinical
Lead developer
Merck
Modality
Small Molecules
01

Overview

ML-SA1 (Mucolipin synthetic agonist 1) is a cell-permeable, synthetic small molecule agonist that activates the transient receptor potential mucolipin (TRPML) channels, specifically TRPML1, TRPML2, and TRPML3. TRPML1 is a calcium-permeable cation channel localized primarily on late endosomes and lysosomes, playing a critical role in lysosomal calcium release, lysosomal trafficking, and autophagy. By activating TRPML1, ML-SA1 induces lysosomal calcium efflux, which promotes lysosomal acidification, enhances autophagic flux, and facilitates the clearance of accumulated cellular waste. ML-SA1 is widely utilized as a chemical biology tool in preclinical research to study lysosomal storage disorders (such as Niemann-Pick disease), neurodegenerative diseases (including Alzheimer's and Parkinson's diseases), and viral infections (such as Dengue and Zika viruses) where lysosomal function is compromised.

Other names
2-[2-(3,4-dihydro-2,2,4-trimethyl-1(2H)-quinolinyl)-2-oxoethyl]-1H-isoindole-1,3(2H)-dioneML SA1Mucolipin synthetic agonist 1
02

Targets

MCOLN1 (Transient receptor potential cation channel mucolipin subfamily member 1)

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