LMI070

LMI070 is an SMN2 splice modulator. It selectively enhances SMN2 splicing by stabilizing the complex formed by the U1 small nuclear ribonucleic protein (snRNP) and SMN2 pre-mRNA. LMI070 (3-30 mg/kg) increases the expression of the full-length SMN2 trancript and, at doses ranging from 0.3 to 30 mg/kg, increases SMN protein levels in the brain and spinal cord in the C/+ mouse model of spinal muscular atrophy (SMA). It also increases survival of SMNDelta7 mice, a model of severe SMA, when administered at doses of 1 and 3 mg/kg.Formal Name: 5-(1H-pyrazol-4-yl)-2-[6-[(2,2,6,6-tetramethyl-4-piperidinyl)oxy]-3-pyridazinyl]-phenol. CAS Number: 1562338-42-4. Synonyms: Branaplam, NVS-SM1. Molecular Formula: C22H27N5O2. Formula Weight: 393.5. Purity: >98%. Formulation: (Request formulation change), A crystalline solid. Solubility: DMF: 16 mg/ml, DMSO: 16 mg/ml, Ethanol: 0.5 mg/ml, PBS (pH 7.2): Slightly soluble. lambdamax: 216, 282 nm. SMILES: CC(NC(C)(C)C1)(C)CC1OC2=CC=C(C3=C(O)C=C(C4=CNN=C4)C=C3)N=N2. InChi Code: 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). InChi Key: STWTUEAWRAIWJG-UHFFFAOYSA-N.