COMPUTED DESCRIPTORS
| Molecular Weight | 199.63 g/mol |
|---|---|
| Hydrogen Bond Donor Count | 6 |
| Hydrogen Bond Acceptor Count | 5 |
| Rotatable Bond Count | 1 |
| Exact Mass | 199.0611356 g/mol |
| Monoisotopic Mass | 199.0611356 g/mol |
| Topological Polar Surface Area | 93 Ų |
| Heavy Atom Count | 12 |
| Formal Charge | 0 |
| Complexity | 132 |
| Isotope Atom Count | 0 |
| Defined Atom Stereocenter Count | 4 |
| Undefined Atom Stereocenter Count | 0 |
| Defined Bond Stereocenter Count | 0 |
| Undefined Bond Stereocenter Count | 0 |
| Covalently-Bonded Unit Count | 2 |
| Compound Is Canonicalized | Yes |
PRODUCT INTRODUCTION
description
AT2220 is an experimental, oral therapy for the treatment of Pompe disease and belongs to a class of molecules known as pharmacological chaperones. It is a small molecule designed to act as a pharmacological chaperone that specifically binds, stabilizes, and facilitates the proper folding and trafficking of α-glucosidase (GAA). GAA to the lysosome, where it can perform its normal function. AT2220 has been shown to increase GAA activity in cell lines derived from Pompe patients and in transfected cells expressing misfolded forms of GAA.