SMN-C2, an analog of RG-7916, is a selective modulator of SMN2 gene splicing that acts by binding SMN2 pre-mRNA, thereby increasing far upstream element binding protein 1 (FUBP1) and KH-spliced RNA binding Protein affinity regulator protein (KHSRP) to the SMN2 pre-mRNA complex. SMN-C2 can be used in spinal muscular atrophy (SMA) research.
SMN-C2 (20 mg/kg, daily) can replace SMN2 splicing and divert FL mRNA, resulting in increased SMN protein levels in the brain and spinal cord of mice[2].