RG7800

Description: RG7800, formerly 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.

References: 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 survivalmotor neuron 2 (SMN2) splicing by small molecules: Discovery of a potential novelmedicine to treat spinal muscular atrophy. J Med Chem. 2016 Jun 14. Epub aheadof print PubMed PMID: 27299419.