Branaplam

SKU: orb1302690

Description

Branaplam (LMI 070) is an orally bioavailable small molecule that potently and selectively modulates SMN2 splicing. It has demonstrated efficacy in increasing SMN protein levels in both cellular models and in vivo studies, supporting its research application for spinal muscular atrophy.

Research Area

Cell Biology, Molecular Biology, Pharmacology & Drug Discovery

Key Properties

• CAS Number: 1562338-42-4
• MW: 393.48
• Purity: 99.73% (May vary between batches)
• Formula: C₂₂H₂₇N₅O₂
• SMILES: CC1(C)CC(CC(C)(C)N1)Oc1ccc(nn1)-c1ccc(cc1O)-c1cn[nH]c1
• Target: DNA/RNA Synthesis,Potassium Channel
• Solubility: DMSO:3.96 mg/mL (10.06 mM)

Bioactivity

• Target IC50:
  ERG (human):6.3 μM (EC50)|SMN:20 nM (IC50)
• In Vivo:
  To evaluate the efficacy of NVS-SM1, we used the SMNΔ7 mouse model, which displays a severe phenotype. In the specific colony used for this study, death typically occurs before postnatal day 15. We were pleased to observe that, in addition to a dose-dependent elevation of SMN protein in the brain, oral administration of NVS-SM1 improved body weight and extended lifespan, with 50% of the animals in the 1 mg per kg body weight group and 62% of animals in the 3 mg per kg body weight group showing increased survival.

Storage & Handling

• Storage: store at low temperature | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature.
• Expiration Date: 12 months from date of receipt.
• Disclaimer: For research use only

Alternative Names

Potassium Channel, PotassiumChannel, RNA Synthesis, RNASynthesis, SMN2, SMN, SMA, splicing, spinal, survival, Inhibitor, LMI070, LMI-070, LMI 070, KcsA, motor, inhibit, muscular, orally, NVS-SM1, neuron-2, gene, hERG, DNA Synthesis, DNA/RNA Synthesis, DNASynthesis, Branaplam, atrophy

Compound Identifiers

PubChem CID

135565042