2406205-61-4
Chemical Structure
GSK716
Synonym(s): A1AT modulator 1
- CAS No.: 2406205-61-4
- Formula:C21H23FN2O3
- Molecular Weight:370.42
IUPAC Name: N-((1S,2R)-1-(3-fluoro-2-methylphenyl)-1-hydroxypentan-2-yl)-2-oxoindoline-4-carboxamide
InChIKey: MSZBLPYPBQYDNI-QUCCMNQESA-N
SMILES: O=C(C1=CC=CC2=C1CC(N2)=O)N[C@@H]([C@H](C3=CC=CC(F)=C3C)O)CCC
Biological Activity: GSK716 (A1AT modulator 1) is an orally active Z α1-antitrypsin (Z-A1AT) inhibitor with a pIC50 of 8.3. GSK716 binds a cryptic Z α1-antitrypsin pocket, stabilizes monomeric folding intermediates, blocks polymerization, and increases monomer secretion. GSK716 acts on select sensitive α1-antitrypsin variants but fails to affect resistant variants. GSK716 can be used for the research of α1-antitrypsin deficiency and α1-antitrypsin deficiency-associated liver disease[1][2][3].
| Cat. No. | Product Name | Purity | Description | Pricing | |||||||||||||||||||
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GSK716 | 99.77% | GSK716 (A1AT modulator 1) is an orally active Z α1-antitrypsin (Z-A1AT) inhibitor with a pIC50 of 8.3. GSK716 binds a cryptic Z α1-antitrypsin pocket, stabilizes monomeric folding intermediates, blocks polymerization, and increases monomer secretion. GSK716 acts on select sensitive α1-antitrypsin variants but fails to affect resistant variants. GSK716 can be used for the research of α1-antitrypsin deficiency and α1-antitrypsin deficiency-associated liver disease. | ||||||||||||||||||||
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References
- [1]. Lomas DA, et al. Development of a small molecule that corrects misfolding and increases secretion of Z α1 -antitrypsin. EMBO Mol Med. 2021;13(3):e13167. [Content Brief]
- [2]. Ronzoni R, et al. Susceptibility of alpha-1 antitrypsin deficiency variants to polymer-blocking therapy. JCI Insight. 2025;10(15):e194354. Published 2025 Jul 8. [Content Brief]
- [3]. Kate Smith, et al. Novel compounds. WO2019243841A1.
