RP-0172

RP-0172 is an orally bioavailable small molecule mRNA splicing modulator developed by Libo (Shenzhen) Biotechnology for the treatment of Huntington's disease (HD). The compound is designed to selectively modulate the splicing of Huntingtin (HTT) pre-mRNA, promoting the inclusion of a 'pseudo-exon' that contains a premature stop codon. This process triggers nonsense-mediated decay (NMD) of the HTT mRNA, leading to a reduction in the levels of both mutant and wild-type Huntingtin protein. By lowering the production of the toxic mutant Huntingtin (mHTT) protein, RP-0172 aims to address the underlying genetic cause of HD and mitigate neurodegeneration. It is currently in Phase I clinical development, with studies evaluating its safety, tolerability, and pharmacokinetics in healthy volunteers.