SMN-C3 是一种可口服的 SMN2 剪接调节剂，可用于脊椎肌肉萎缩 (SMA) 的研究。
Discovery of a CNS penetrant small molecule SMN2 splicing modulator with improved tolerability for spinal muscular atrophy
PMID 33060681; DOI 10.1038/s41598-020-74346-9; Scientific reports 2020 10; 10(1):17472
IUPAC/Chemical Name: 2-(4,6-Dimethylpyrazolo[1,5-a]pyrazin-2-yl)-7-(1-ethylpiperidin-4-yl)-9-methyl-4H-pyrido[1,2-a]pyrimidin-4-one
InChi Key: ZACWYYKZMLGOTG-UHFFFAOYSA-N
InChi Code: InChI=1S/C24H28N6O/c1-5-28-8-6-18(7-9-28)19-10-15(2)24-26-20(12-23(31)29(24)14-19)21-11-22-17(4)25-16(3)13-30(22)27-21/h10-14,18H,5-9H2,1-4H3
SMILES Code: O=C1C=C(C2=NN3C(C(C)=NC(C)=C3)=C2)N=C4N1C=C(C5CCN(CC)CC5)C=C4C
Appearance: Solid powder
Purity: >98% (or refer to the Certificate of Analysis)
Shipping Condition: Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.
Storage Condition: Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).
Solubility: Soluble in DMSO
Shelf Life: >3 years if stored properly
Drug Formulation: This drug may be formulated in DMSO
Stock Solution Storage: 0 - 4 C for short term (days to weeks), or -20 C for long term (months).
HS Tariff Code: 2934.99.9001
