RG6496

RG6496 is an investigational therapeutic being developed by Roche for the treatment of Huntington's disease. It is administered via intrathecal injection to target the central nervous system. As of early 2024, the drug is in Phase I clinical development (NCT06206031) to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single-ascending doses in individuals who carry the Huntington's disease gene expansion. While the specific biological target has not been widely publicized in high-level pipeline summaries, its development by Roche in the Huntington's space follows their previous efforts with antisense oligonucleotides (like tominersen), though RG6496 represents a distinct, next-generation program aimed at modulating the progression of this neurodegenerative disorder.

Other names

huntingtin single nucleotide polymorphism antisense oligonucleotideHTT SNP ASO