votoplam

Votoplam is an orally bioavailable small molecule developed as a gene splicing modulator for the treatment of Huntington's disease. Its primary mechanism involves modulation of RNA splicing, specifically targeting the huntingtin (HTT) gene to reduce production of both mutant and normal HTT protein. By altering pre-mRNA processing, votoplam lowers toxic HTT protein levels—a hallmark of Huntington’s disease pathology—potentially slowing or modifying disease progression. Clinical trials have demonstrated dose-dependent reductions in blood and cerebrospinal fluid HTT protein, with early evidence suggesting improvements in clinical measures and biomarkers such as neurofilament light chain (NfL). Votoplam is administered orally, distinguishing it from other investigational therapies that require more invasive delivery methods. The drug was originally developed by PTC Therapeutics and is now being advanced globally through a collaboration with Novartis[1][5][6][8].

Other names

votoplam [INN]VOTOPLAM [USAN]2407849-89-0D7EZ7B585XD-7EZ7B585XD 7EZ7B585XCHEMBL4850295CHEMBL-4850295CHEMBL 4850295SCHEMBL23702142SCHEMBL-23702142SCHEMBL 23702142D12970D-12970D 12970