Branaplam hydrochloride

SKU: orb1908994

Description

Branaplam hydrochloride (LMI070) is an orally active, selective SMN2 splicing modulator with an EC50 of 20 nM for SMN. It also inhibits hERG channels (IC50 = 6.3 µM). In vivo, it elevates full-length SMN protein and improves survival in severe SMA mouse models, supporting its research in spinal muscular atrophy.

Research Area

Cell Biology, Molecular Biology, Pharmacology & Drug Discovery

Key Properties

• CAS Number: 1562338-39-9
• MW: 429.95
• Purity: 98.45% (May vary between batches)
• Formula: C₂₂H₂₈ClN₅O₂
• SMILES: Cl.OC1=CC(=CC=C1C=2N=NC(OC3CC(NC(C)(C)C3)(C)C)=CC2)C=4C=NNC4
• Target: Others,Potassium Channel,DNA/RNA Synthesis
• Solubility: H2O:＜1mg/mL (Insoluble);DMSO:200 mg/mL (465.17 mM)

Bioactivity

• Target IC50:
  SMN:20 nM (EC50)|ERG (human):6.3 μM
• In Vivo:
  After oral administration of Branaplam hydrochloride (3, 10, 30 mg/kg), the SMN2-FL transcript and SMN protein levels in the brain and spinal cord of mice showed a dose-dependent increase .
  Read more...
• In Vitro:
  In Homo sapiens fibroblasts, Branaplam hydrochloride caused changes in the expression levels of 175 genes.

Storage & Handling

• Storage: -20°C
• Expiration Date: 12 months from date of receipt.
• Disclaimer: For research use only

Alternative Names

LMI 070 hydrochloride, LMI070 hydrochloride, NVS-SM1 hydrochloride

Compound Identifiers

PubChem CID

135565041