Description
RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; RG7800 hydrochloride has the potential to treat spinal muscular atrophy.
Product information
Molecular Weight: 416.52
Formula: C24H28N6O
Chemical Name: 2-{4-ethyl-6-methylpyrazolo[1,5-a]pyrazin-2-yl}-9-methyl-7-(1-methylpiperidin-4-yl)-4H-pyrido[1,2-a]pyrimidin-4-one hydrogen
Smiles: CN1CCC(CC1)C1=CN2C(=O)C=C(N=C2C(C)=C1)C1C=C2C(CC)=NC(C)=CN2N=1
InChiKey: GYFRQCMDLBNZSF-UHFFFAOYSA-N
InChi: InChI=1S/C24H28N6O/c1-5-19-22-11-21(27-30(22)13-16(3)25-19)20-12-23(31)29-14-18(10-15(2)24(29)26-20)17-6-8-28(4)9-7-17/h10-14,17H,5-9H2,1-4H3
Technical Data
Appearance: Solid Power
Purity: ≥98% (or refer to the Certificate of Analysis)
Solubility: H2O : ≥ 113.5 mg/mL.
Shipping Condition: Shipped under ambient temperature as non-hazardous chemical or refer to Certificate of Analysis
Storage Condition: Dry, dark and -20 oC for 1 year or refer to the Certificate of Analysis.
Shelf Life: ≥12 months if stored properly.
Stock Solution Storage: 0 - 4 oC for 1 month or refer to the Certificate of Analysis.
Drug Formulation: To be determined
HS Tariff Code: 382200
How to use
In Vitro:
RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; and is the first small molecule SMN2 splicing modifier to enter human clinical trials.
In Vivo:
RG7800 is a favorable agent metabolism and pharmacokinetic (DMPK) profile in the rat and in cynomolgus monkey with good oral bioavailability. RG7800 (1, 3, 10 mg/kg, p.o.) dose-dependently elevates the SMN protein level in the brain and in peripheral tissue of Δ7 mice.
Products are for research use only. Not for human use.
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