Description
NVS-SM1, also known as LMI070, is a highly potent, selective small molecule SMN2 splicing modulator with potential for treating spinal muscular atrophy. It works by increasing the amount of functional survival of motor neuron protein produced by the SMN2 gene through modifying its splicing pattern.
NVS-SM1, also known as LMI070, is a highly potent, selective small molecule SMN2 splicing modulator with potential for treating spinal muscular atrophy. It works by increasing the amount of functional survival of motor neuron protein produced by the SMN2 gene through modifying its splicing pattern.
Alternate Name/Synonyms: (6Z)-3-(1H-pyrazol-4-yl)-6-[3-(2,2,6,6-tetramethylpiperidin-4-yl)oxy-1H-pyridazin-6-ylidene]cyclohexa-2,4-dien-1-one; Branaplam; LMI070
Appearance: Crystalline solid
Formulation: N/A
CAS Number: 1562338-42-4
Structure Available?: Yes
Peptide sequence: N/A
Salt Form: N/A
Molecular Formula: C₂₂H₂₇N₅O₂
Molecular Weight: 393.49
Cell-Permeable?: Yes
Purity: ≥98% by HPLC
Solubilities: DMSO
Handling: Protect from air and light
Country of Origin: USA
Tag Line: A highly potent, selective small molecule SMN2 splicing modulator
MDL Number: N/A
PubChem CID: 89971189
SMILES: CC1(CC(CC(N1)(C)C)OC2=NNC(=C3C=CC(=CC3=O)C4=CNN=C4)C=C2)C
InChi: InChI=1S/C22H27N5O2/c1-21(2)10-16(11-22(3,4)27-21)29-20-8-7-18(25-26-20)17-6-5-14(9-19(17)28)15-12-23-24-13-15/h5-9,12-13,16,25,27H,10-11H2,1-4H3,(H,23,24)/b18-17+
InChi Key: YIFFDXMJVNKGBL-ISLYRVAYSA-N
Additional Information
Storage Condition: |
-20°C |
Shipping Condition: |
Gel Pack |
Shelf Life: |
36 months |