2743336-70-9

2743336-70-9 structure

Name: mHTT-IN-2
Chemical Name: mHTT-IN-2
CAS Number: 2743336-70-9
Molecular Formula: C₂₀H₂₂FN₇O
Molecular Weight: 395.43
Catalog: Research Areas Neurological Disease
Create Date: 2023-11-04 19:14:21
Modify Date: 2025-08-26 08:51:56
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].

Name	mHTT-IN-2

Description	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].
Related Catalog	Research Areas >> Neurological Disease
Target	EC50: 0.066 μM (mHTT protein)[1]
References	[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.. .

Molecular Formula	C₂₀H₂₂FN₇O
Molecular Weight	395.43