An SMN2 splice modulator
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Product Type

Biochemicals

Research Area

Neuroscience

LMI070

Item No. 26757

Product Insert (PDF)Safety Data Sheet (SDS) (PDF)
Technical Information
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(Learn about Variance in Solubility)
DMF: 16 mg/mlDMSO: 16 mg/mlEthanol: 0.5 mg/mlPBS (pH 7.2): Slightly soluble
λmax
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
Shipping & Storage Information
Storage
-20°C
Shipping
Wet ice in continental US; may vary elsewhere
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    1 mg

    $49

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    5 mg

    $223

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    10 mg

    $400

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    25 mg

    $876

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    Product Description

    LMI070 is an SMN2 splice modulator.1 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).1,2 It also increases survival of SMNΔ7 mice, a model of severe SMA, when administered at doses of 1 and 3 mg/kg.1

    WARNING This product is not for human or veterinary use.

    References & Product Citations
    Product Description References

    1. Palacino, J., Swalley, S.E., Song, C., et al. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. Nat. Chem. Biol. 11(7), 511-517 (2015).

    2. Cheung, A.K., Hurley, B., Kerrigan, R., et al. Discovery of small molecule splicing modulators of survival motor neuron-2 (SMN2) for the treatment of spinal muscular atrophy (SMA). J. Med. Chem. 61(24), 11021-11036 (2018).