RO6885247

RO6885247 (also known as RG7800) is an experimental small molecule drug developed as a survival of motor neuron 2 (SMN2) splicing modifier. Its mechanism of action involves stimulating the production of functional SMN protein by modifying the splicing of SMN2 pre-mRNA, which is deficient in patients with spinal muscular atrophy (SMA). The drug was designed for oral administration and was investigated primarily for the treatment of SMA. Clinical development included multicenter, randomized, double-blind, placebo-controlled studies in both adult and pediatric patients with SMA. However, clinical trials were terminated prematurely during Phase 1 due to safety concerns[1][5][6][7].