LMI-070

SKU: orb1226097

Description

LMI070 (NVS-SM1, Branaplam ) is a potent SMN2 splice modulator (EC50=20 nM) that potential treatment of spinal muscular atrophy (SMA).Spinal Muscular Atrophy (SMA),Phase 2 Clinical.

Key Properties

• CAS Number: 1562338-42-4
• MW: 393.4821
• Purity: >98% (HPLC)
• Formula: C₂₂H₂₇N₅O₂
• SMILES: CC1(CC(CC(N1)(C)C)OC2=NN=C(C=C2)C3=C(C=C(C=C3)C4=CNN=C4)O)C
• Target: SMN2
• Solubility: DMSO: 6.12 mg/mL

Bioactivity

• In Vivo:
  Animal model: C/+ SMA mouse model. Dosage: 3, 10, 30 mg/kg. Administration: Oral. Result: Produced dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord. Animal model: Male Sprague-Dawley rat. Dosage: 1 mg/kg (IV); 3 mg/kg (PO) (Pharmacokinetic Analysis). Administration: IV or PO. Result: Had a CL of 25 mL/min/kg and an AUC of 3.03 μM h.

Storage & Handling

• Storage: Storage temperature: -20°C. Stability: ≥ 2 years
• Expiration Date: 12 months from date of receipt.
• Disclaimer: For research use only

Alternative Names

NVS-SM1 | Branaplam

Quick Database Links

Gene Symbol

SMN2