NVS-SM1, SMN2 Splicing Modulator

NVS-SM1, also known as Branaplam and LMI-070, is a highly potent, selective and orally active small molecule SMN2 splicing modulator as a potential treatment for SMA Type 1 (Spinal Muscular Atrophy) .NVS-SM1 works by increasing the amount of functional SMN protein produced by the “back-up” gene, SMN2, through modifying its splicing.

Product information

CAS Number: 1562338-42-4

Molecular Weight: 393.48

Formula: C22H27N5O2

Synonym:

Branaplam

LMI-070

Chemical Name: 5-(1H-pyrazol-4-yl)-2-{6-[(2,2,6,6-tetramethylpiperidin-4-yl)oxy]pyridazin-3-yl}phenol

Smiles: CC1(C)CC(CC(C)(C)N1)OC1=CC=C(N=N1)C1=CC=C(C=C1O)C1C=NNC=1

InChiKey: STWTUEAWRAIWJG-UHFFFAOYSA-N

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/h5-9,12-13,16,27-28H,10-11H2,1-4H3,(H,23,24)

Technical Data

Appearance: Solid Power.

Purity: ≥98% (or refer to the Certificate of Analysis)

Solubility: DMSO: 3 mg/mL(7.62 mM). Water: Insoluble.

Shipping Condition: Shipped under ambient temperature as non-hazardous chemical or refer to Certificate of Analysis

Storage Condition: Dry, dark and -20 ^oC for 1 year or refer to the Certificate of Analysis.

Shelf Life: ≥360 days if stored properly.

Stock Solution Storage: 0 - 4 ^oC for 1 month or refer to the Certificate of Analysis.

Drug Formulation: To be determined.

HS Tariff Code: 382200

References:

1. Cheung AK, et al. J Med Chem. 2018, 61(24):11021-11036.

Products are for research use only. Not for human use.