Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
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.
Beyond the preview
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Follow clinical development from study design and recruitment through results.
Explore development by indication, patient population, and geography.
Trace asset ownership, licensing agreements, and commercial partnerships.
Explore the patent landscape and regulatory exclusivity around an asset.
Compare development programs by target, modality, and indication.
Connect source evidence and development news to your research questions.
See how Gosset can support your research on ML-SA1.