RG7800

• CAT Number: I009027
• CAS Number: 1449598-06-4
• Molecular Formula: C24H28N6O
• Molecular Weight: 416.529
• Purity: ≥95%
• Solubility: Soluble in DMSO, not in water
• Storage: 0 - 4 °C for short term, or -20 °C for long term
• InChI: 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
• InChIKey: GYFRQCMDLBNZSF-UHFFFAOYSA-N
• SMILES: O=C1C=C(C2=NN3C(C(CC)=NC(C)=C3)=C2)N=C4N1C=C(C5CCN(C)CC5)C=C4C

RG7800（Cat No.:I009027）, also known as RO6885247, is a potent and selective SMN2 splicing modifier currently undergoing clinical trials for the treatment of Spinal Muscular Atrophy (SMA). SMA is a genetic disorder characterized by the absence or mutation of the survival of the motor neuron (SMN1) gene, leading to motor neuron degeneration. RG7800 has demonstrated promising pharmacokinetic properties, efficacy, and safety in preclinical studies. It corrects the alternative splicing of the SMN2 gene, resulting in increased SMN protein levels in the brain, which is crucial for motor neuron survival.

Reference

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.