mHTT-IN-2

• CAS Number:

  2743336-70-9

• UNSPSC 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].

• Target Antigen:

  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

• 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 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.. .

• Shipping Conditions:

  Room Temperature

• Clinical Information:

  No Development Reported