Description
RG7800 is a SMN2 splicing modifier. RG7800 has the potential for spinal muscular atrophy treatment.
Product information
CAS Number: 1449598-06-4
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
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: DMSO : 1.4 mg/mL (3.36 mM; Need ultrasonic and warming). H2O : 1.25 mg/mL (3.00 mM; ultrasonic and adjust pH to 3 with HCl).
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 increases the SMN protein level via induction of alternative splicing of the SMN2 mRNA. RG7800 is shown to promote the inclusion of exon 7 in SMN2 mRNA, generating full-length mRNA in vitro using fibroblasts from an SMA type I patient.
In Vivo:
RG7800 shows favorable drug metabolism and pharmacokinetic profile in the rat and in cynomolgus monkey with good oral bioavailability. In SMA mouse model, treatment of RG7800 shows a clear dose dependent increase in SMN protein levels. Mice treated with RG7800 demonstrate a dose dependent increase in survival beginning at the low dose (0.3/1 mg/kg). In the middle and high dose groups (1/3 and 3/10 mg/kg, respectively), approximately 80−90% survive beyond PND50/PND60 with profound body weight gain when the study is terminated. RG7800 dose-dependently corrects SMN2 splicing by including exon 7 to create FL mRNA, suggesting that RG7800 corrects alternative splicing of the human SMN2 gene in the brain of transgenic SMA model mice, leading to an increase of the SMN protein in the brain.
Products are for research use only. Not for human use.
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