Phenol, 2-[6-[methyl(2,2,6,6-tetramethyl-4-piperidinyl)amino]-3-pyridazinyl]-5-(1H-pyrazol-4-yl)-
- CAS No.
- 1562333-92-9
- Chemical Name:
- Phenol, 2-[6-[methyl(2,2,6,6-tetramethyl-4-piperidinyl)amino]-3-pyridazinyl]-5-(1H-pyrazol-4-yl)-
- Synonyms
- NVS-SM2;NVS SM2,NVS-SM-2,NVSSM2;Phenol, 2-[6-[methyl(2,2,6,6-tetramethyl-4-piperidinyl)amino]-3-pyridazinyl]-5-(1H-pyrazol-4-yl)-
- CBNumber:
- CB08188221
- Molecular Formula:
- C23H30N6O
- Molecular Weight:
- 406.52
- MDL Number:
- MFCD34469206
- MOL File:
- 1562333-92-9.mol
Phenol, 2-[6-[methyl(2,2,6,6-tetramethyl-4-piperidinyl)amino]-3-pyridazinyl]-5-(1H-pyrazol-4-yl)- Chemical Properties,Uses,Production
Biological Activity
NVS-SM2 is a potent, orally active and brain-penetrant SMN2 splicing enhancer with an EC50 of 2 nM for SMN. NVS-SM2 enhances U1-pre-mRNA association. NVS-SM2 promotes exon 7 inclusion and restores normal survival motor neuron (SMN) protein expression. NVS-SM2 can be used for spinal muscular atrophy (SMA) research[1][2]. For NVS-SM2, the molecular mechanism of action is via stabilization of the transient double-strand RNA structure formed by the SMN2 pre-mRNA and U1 small nuclear ribonucleic protein (snRNP) complex. The binding affinity of U1 snRNP to the 5’ splice site is increased in a sequence-selective manner, discrete from constitutive recognition[1]. NVS-SM2 (0.1-1 mg/kg; s.c.; for 30 days) treatment extends survival in a severe SMA mouse model[2]. Pharmacokinetic analysis demonstrate that NVS-SM2 is readily available in the brain after IV and oral (PO) administration in mouse and rat with Tmax of 3 h after PO with 3 mg/kg in mice, and treatment induced a 1.5-fold increase in SMN protein levels in the mouse brain[1].
References
[1]. James Palacino, et al. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. Nat Chem Biol. 2015 Jul;11(7):511-7. [2]. Anne Rietz, et al. Short-duration splice promoting compound enables a tunable mouse model of spinal muscular atrophy. Life Sci Alliance. 2020 Nov 24;4(1):e202000889.
Phenol, 2-[6-[methyl(2,2,6,6-tetramethyl-4-piperidinyl)amino]-3-pyridazinyl]-5-(1H-pyrazol-4-yl)- Preparation Products And Raw materials
Raw materials
Preparation Products
Phenol, 2-[6-[methyl(2,2,6,6-tetramethyl-4-piperidinyl)amino]-3-pyridazinyl]-5-(1H-pyrazol-4-yl)- Suppliers
Supplier | Tel | Country | ProdList | Advantage | |
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TargetMol Chemicals Inc. | +1-781-999-5354 | support@targetmol.com | United States | 19973 | 58 |
Aladdin Scientific | +1-833-552-7181 | sales@aladdinsci.com | United States | 52927 | 58 |
TargetMol Chemicals Inc. | 4008200310 | marketing@tsbiochem.com | China | 24018 | 58 |
Supplier | Advantage |
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TargetMol Chemicals Inc. | 58 |
Aladdin Scientific | 58 |
TargetMol Chemicals Inc. | 58 |