Risdiplam
Risdiplam is an orally bioavailable small molecule splicing modifier that corrects aberrant splicing of the SMN2 pre-mRNA by binding to and stabilizing the exon 7 splicing enhancer, increasing the production of full-length functional survival motor neuron (SMN) protein. It is approved for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients and is under investigation for other RNA splicing-related conditions.
分子量
401.5000 g/mol
LogP
0.50
TPSA
78.10 Ų
Lipinski 五规则
符合
治疗领域
作用机制
Delivers synthetic messenger RNA encoding a specific protein to host cells, where it is translated by ribosomes to produce the therapeutic protein without integrating into the host genome.
Pharmacokinetics (PK)
Pharmacodynamics (PD)
Delivers synthetic messenger RNA encoding a specific protein to host cells, where it is translated by ribosomes to produce the therapeutic protein without integrating into the host genome.
二维结构
Cite this structure
Embed this structure
SMILES
Cc1cn2nc(-c3cc(=O)n4cc(N5CCNC6(CC6)C5)ccc4n3)cc(C)c2n1
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
Molecular Formula
C22H23N7O
HBD / HBA
1 / 6
可旋转键数
2
重原子数
30
No targets recorded
Target interaction data is not yet available for this drug.
No interactions recorded
Drug interaction data is not yet available for this compound.
No side effects recorded
Side effect data is not yet available for this drug.
常见问题
Risdiplam is an orally bioavailable small molecule splicing modifier that corrects aberrant splicing of the SMN2 pre-mRNA by binding to and stabilizing the exon 7 splicing enhancer, increasing the production of full-length functional survival motor neuron (SMN) protein. It is approved for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients and is under investigation for other RNA splicing-related conditions.
Delivers synthetic messenger RNA encoding a specific protein to host cells, where it is translated by ribosomes to produce the therapeutic protein without integrating into the host genome.
Yes, Risdiplam is an approved drug. It has reached clinical phase 4. It is classified as a Small molecule.
References & Data Sources
- ChEMBL — European Bioinformatics Institute (EBI). CHEMBL4297528. Open-access bioactivity database.
- PubChem — National Center for Biotechnology Information (NCBI). CID 118513932. Chemical information database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-03-28.
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