NVS-SM2

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].

Product Specifications

CAS Number

[1562333-92-9]

UNSPSC

12352005

Target

DNA/RNA Synthesis

Type

Reference compound

Related Pathways

Cell Cycle/DNA Damage

Applications

Neuroscience-Neuromodulation

Field of Research

Neurological Disease

Assay Protocol

https://www.medchemexpress.com/nvs-sm2.html

Purity

99.15

Solubility

DMSO : 75 mg/mL (ultrasonic)

Smiles

OC1=C(C(N=N2)=CC=C2N(C(CC3(C)C)CC(C)(N3)C)C)C=CC(C4=CNN=C4)=C1

Molecular Formula

C23H30N6O

Molecular Weight

406.52

References & Citations

[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.