NVS-SM2

• UNSPSC Description:

  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].

• Target Antigen:

  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 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.

• Shipping Conditions:

  Blue Ice

• Storage Conditions:

  -20°C, 3 years (Powder)

• Clinical Information:

  No Development Reported

• CAS Number:

  1562333-92-9