2839908-40-4
Chemical Structure
MZ-101
Synonym(s): GYS1-IN-2
- CAS No.: 2839908-40-4
- Formula:C25H28FN5O2
- Molecular Weight:449.52
InChIKey: VIXMWFGMNPKXHI-SFLYRZDNSA-N
SMILES: O=C([C@H]1N(C[C@@H](C1)F)C(CC2=CN=NN2)=O)N[C@@H](C3=CC=CC=C3)C4=CC=C(C=C4)C(C)C
Biological Activity: MZ-101 (GYS1-IN-2) is an orally active, potent and selective small-molecule glycogen synthase 1 (GYS1) inhibitor with with an IC50 value of 0.041 µM. MZ-101 reduces glycogen concentrations in cells and in mice. MZ-101 can used to study GYS1 -mediated Pompe disease and other glycogen storage diseases[1][2][3].
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MZ-101 | 99.42% | MZ-101 (GYS1-IN-2) is an orally active, potent and selective small-molecule glycogen synthase 1 (GYS1) inhibitor with with an IC50 value of 0.041 µM. MZ-101 reduces glycogen concentrations in cells and in mice. MZ-101 can used to study GYS1 -mediated Pompe disease and other glycogen storage diseases. | ||||||||||||||||||||
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- [1]. David John Morgans, et al. Inhibitors of glycogen synthase 1 (gys1) and methods of use thereof. WO2022198196. 2022-09-22.
- [2]. Ullman JC, et al. Small-molecule inhibition of glycogen synthase 1 for the treatment of Pompe disease and other glycogen storage disorders. Sci Transl Med. 2024 Jan 17;16(730):eadf1691. [Content Brief]
- [3]. Gaspar RC, et al. Small molecule inhibition of glycogen synthase I reduces muscle glycogen content and improves biomarkers in a mouse model of Pompe disease. Am J Physiol Endocrinol Metab. 2024 Oct 1;327(4):E524-E532. [Content Brief]
Keywords