MHTT-IN-2

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].

Product Specifications

CAS Number

[2743336-70-9]

UNSPSC

12352005

Target

Huntingtin

Type

Reference compound

Related Pathways

Neuronal Signaling

Applications

Neuroscience-Neuromodulation

Field of Research

Neurological Disease

Assay Protocol

https://www.medchemexpress.com/mhtt-in-2.html

Purity

99.35

Solubility

DMSO : 100 mg/mL (ultrasonic)

Smiles

FC1=CC(NC(C2=NC=C(N3C[C@@H](CC3)NC4CC4)N=C2)=O)=CN5C1=NC(C)=C5

Molecular Formula

C20H22FN7O

Molecular Weight

395.43

References & Citations

[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.. .