Risdiplam

SKU: orb1307794

Description

Risdiplam (RG7916) is an orally available small molecule that modifies SMN2 pre-mRNA splicing, elevating survival motor neuron (SMN) protein levels throughout the body. It is widely used in preclinical in vitro and in vivo research for spinal muscular atrophy (SMA), investigating therapeutic mechanisms and efficacy.

Research Area

Cell Biology, Molecular Biology, Pharmacology & Drug Discovery

Key Properties

• CAS Number: 1825352-65-5
• MW: 401.46
• Purity: 99.43% (May vary between batches)
• Formula: C₂₂H₂₃N₇O
• SMILES: Cc1cn2nc(cc(C)c2n1)-c1cc(=O)n2cc(ccc2n1)N1CCNC2(CC2)C1
• Target: DNA/RNA Synthesis
• Solubility: H2O:2.22 mg/mL (5.53 mM);Ethanol:2 mg/mL (4.98 mM);DMSO:< 1 mg/mL (insoluble or slightly soluble)

Bioactivity

• In Vivo:
  METHODS: Wistar rats received either a single oral dose of 14C-risdiplam (RO7034067) (by gastric gavage) or a single intravenous dose of 14C-risdiplam (by tail vein injection). Dose levels for oral and intravenous doses were 5 or 2 mg/kg, respectively. RESULTS Radioactive 14C-risdiplam (RO7034067) was rapidly and extensively distributed in rat QWBA after oral administration, with peak concentrations in most tissues measured at 2 hours. Radioactivity concentrations in plasma and tissues declined during the study period.
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• In Vitro:
  METHODS: LLC-PK1, MDCKII, L-MDR1 (LLC-PK1 cells transfected with human MDR1), L-Mdr1a (LLC-PK1 cells transfected with rodent Mdr1a), M-BCRP (MDCKII cells transfected with human breast cancer resistance protein; BCRP), and M-Bcrp1 (MDCKII cells transfected with rodent Bcrp1) cells were treated with Risdiplam (RG7916) (1 μM) and 10 μM Lucifer Yellow added to the donor compartment to initiate transcellular transport. RESULTS Risdiplam had a mean passive permeability of approximately 350 nm/s in parental LLC-PK1 cells. Similar permeability was observed in L-MDR1, L-Mdra1, M-BCRP, and M-Bcrp cells in the presence of the inhibitor.
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Storage & Handling

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

Alternative Names

DNA Synthesis, DNA/RNA Synthesis, DNASynthesis, Inhibitor, inhibit, SMN2, RG7916, RG-7916, RG 7916, RNA Synthesis, RNASynthesis, Risdiplam, RO 7034067, RO7034067, RO-7034067

Compound Identifiers

PubChem CID

118513932