wt-IDH1 siRNA

wt-IDH1 siRNA is a small interfering RNA (siRNA) therapeutic designed to silence the expression of wild-type isocitrate dehydrogenase 1 (wt-IDH1). Developed through a collaboration involving Alnylam Pharmaceuticals and academic institutions such as Northwestern University, this agent specifically targets wt-IDH1 mRNA in tumor-associated myeloid cells (TAMCs) within the glioblastoma (GBM) tumor microenvironment. By reducing wt-IDH1 protein levels, the siRNA metabolic reprogramming shifts TAMCs from an immunosuppressive state to an inflammatory, tumoricidal phenotype. This transition increases CD8+ T cell infiltration and enhances the efficacy of immune checkpoint therapies like PD-1 blockade. Preclinical studies involving intrathecal delivery have demonstrated that wt-IDH1 inhibition can overcome resistance to immunotherapy and promote long-term anti-tumor immune memory in glioblastoma models.

Other names

siRNA targeted to wt-IDH1wild-type IDH1 siRNA