| Bioactivity | mHTT-IN-2 (compound 27) is a potent inhibitor (EC50=0.066 μM) of mutant huntingtin (mHTT). mHTT-IN-2 reduces canonical splicing of HTT RNA exons [49-50] and is a splicing regulator of the huntingtin (HTT) gene. mHTT-IN-2 exhibits inhibitory activity in vitro and in vivo in human HD stem cells and mouse BACHD models. mHTT-IN-2 may be used in the study of branaplam-related peripheral neuropathy[1]. |
| Target | EC50: 0.066 μM (mHTT protein) |
| Name | mHTT-IN-2 |
| CAS | 2743336-70-9 |
| Formula | C20H22FN7O |
| Molar Mass | 395.43 |
| Transport | Room temperature in continental US; may vary elsewhere. |
| Storage | Please store the product under the recommended conditions in the Certificate of Analysis. |
| Reference | [1]. Liu L, et al. Identification and Optimization of RNA-Splicing Modulators as Huntingtin Protein-Lowering Agents for the Treatment of Huntington's Disease. J Med Chem. 2023 Sep 15.. . |