Catalog No.: PC-42423Not For Human Use, Lab Use Only.
RG7800 (RG-7800, RO6885247) is a potent, orally available, small molecule SMN2 splicing modifier with EC1.5X of 23 nM and 87 nM for SMN2 splicing and SMN2 protein, respectively.
RG7800 (RG-7800, RO6885247) is a potent, orally available, small molecule SMN2 splicing modifier with EC1.5X of 23 nM and 87 nM for SMN2 splicing and SMN2 protein, respectively.
RG7800 specifically modifies the alternative splicing of SMN2 exon 7 in SMA patient-derived cells and in two SMA mouse models.
RG7800 exhibits excellent pharmacokinetic and in vivo efficacy and has a favorable safety profile.
