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Risdiplam
- RG7916
Axon 3093
CAS:
1825352-65-5
Purity:
99%
- Soluble in 0.1N HCl(aq)
- MF: C22H23N7O
- MW: 401.46
Description
Risdiplam is a selective SMN2 pre-mRNA splicing modifier that promotes inclusion of exon 7 and increases production of full-length survival motor neuron (SMN) protein. The compound stabilizes productive interactions between SMN2 RNA and spliceosomal components at the exon 7 splice site, with a reported EC1.5× value of 4 nM in a cellular splicing assay.
Risdiplam is orally active and distributes to both central and peripheral tissues, making it a valuable translational tool for studying RNA-selective small molecules, splice-site recognition and SMN biology. It is widely used in spinal muscular atrophy research to connect correction of SMN2 splicing with motor-neuron survival and neuromuscular function.
Key Features
- Selective SMN2 splicing modifier
- EC1.5×: 4 nM
- Promotes exon 7 inclusion and full-length SMN protein production
- Orally active with central and peripheral distribution
Applications
- SMN2 splicing and RNA-targeted drug research
- Spinal muscular atrophy models
- Motor-neuron survival and neuromuscular studies
- Small-molecule modulation of spliceosome assembly
More Information
| Parent CAS No. | 1825352-65-5 |
|---|---|
| Chemical Name | 2-(2,8-Dimethylimidazo[1,2-b]pyridazin-6-yl)-7-(4,7-diazaspiro[2.5]octan-7-yl)-4H-pyrido[1,2-a]pyrimidin-4-one |
| SMILES | C1=CC2=NC(C3C=C(C)C4=NC(C)=CN4N=3)=CC(=O)N2C=C1N1CCNC2(CC2)C1 |
| MFCD | N.A. |
| InChi | InChI=1S/C22H23N7O/c1-14-9-18(26-29-11-15(2)24-21(14)29)17-10-20(30)28-12-16(3-4-19(28)25-17)27-8-7-23-22(13-27)5-6-22/h3-4,9-12,23H,5-8,13H2,1-2H3 |
| InChiKey | ASKZRYGFUPSJPN-UHFFFAOYSA-N |
| CID | 118513932 |
| Short Description | SMN2 modifier |
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