WARNING: This product is for research use only, not for human or veterinary use.
MedKoo CAT#: 526734
Description: RG7800, also known as RO6885247 is a potent and selective SMN2 splicing modifier, which is currently under human clinical trials for the treatment of Spinal Muscular Atrophy (SMA). SMA is a genetic disorder caused by the mutation or deletion of the survival of motor neuron (SMN1) gene. It affects one in approximately 10,000 live births and in the most severe forms is assocaited with a high rate of childhood mortality. RG7800 exhibits excellent pharmacokinetic and in vivo efficacy and had a favorable safety profile. RG7800 corrects alternative splicing of the human SMN2 gene in the brain of transgenic SMA model mice, leading to an increase of the SMN protein in the brain.
MedKoo Cat#: 526734
Chemical Formula: C24H28N6O
Exact Mass: 416.2325
Molecular Weight: 416.529
Elemental Analysis: C, 69.21; H, 6.78; N, 20.18; O, 3.84
Synonym: RG7800; RG-7800; RG 7800; RO6885247; RO-6885247; RO 6885247.
IUPAC/Chemical Name: 2-(4-ethyl-6-methylpyrazolo[1,5-a]pyrazin-2-yl)-9-methyl-7-(1-methylpiperidin-4-yl)-4H-pyrido[1,2-a]pyrimidin-4-one
InChi Key: GYFRQCMDLBNZSF-UHFFFAOYSA-N
InChi Code: InChI=1S/C24H28N6O/c1-5-19-22-11-21(27-30(22)13-16(3)25-19)20-12-23(31)29-14-18(10-15(2)24(29)26-20)17-6-8-28(4)9-7-17/h10-14,17H,5-9H2,1-4H3
SMILES Code: O=C1C=C(C2=NN3C(C(CC)=NC(C)=C3)=C2)N=C4N1C=C(C5CCN(C)CC5)C=C4C
Spinal muscular atrophy (SMA) is the leading genetic cause of infant and toddler mortality, and there is currently no approved therapy available. SMA is caused by mutation or deletion of the survival motor neuron 1 (SMN1) gene. These mutations or deletions result in low levels of functional SMN protein. SMN2, a paralogous gene to SMN1, undergoes alternative splicing and exclusion of exon 7, producing an unstable, truncated SMN∆7 protein.
1: Ratni H, Karp GM, Weetall M, Naryshkin NN, Paushkin SV, Chen KS, McCarthy KD,
Qi H, Turpoff A, Woll MG, Zhang X, Zhang N, Yang T, Dakka A, Vazirani P, Zhao X,
Pinard E, Green L, David-Pierson P, Tuerck D, Poirier A, Muster W, Kirchner S,
Mueller L, Gerlach I, Metzger F. Specific correction of alternative survival
motor neuron 2 (SMN2) splicing by small molecules: Discovery of a potential novel
medicine to treat spinal muscular atrophy. J Med Chem. 2016 Jun 14. [Epub ahead
of print] PubMed PMID: 27299419.