siGPX4-lactosomes + siFSP1-lactosomes

This therapeutic combination consists of two distinct siRNA-based nanotherapeutics designed to induce ferroptosis in cholangiocarcinoma (CCA). The system utilizes milk-derived nanovesicles, termed lactosomes, which are functionalized with an EpCAM-targeting aptamer to ensure selective uptake by EpCAM-expressing tumor cells. The lactosomes are packaged with small interfering RNAs (siRNAs) targeting two critical ferroptosis defense mechanisms: Glutathione Peroxidase 4 (GPX4) and Ferroptosis Suppressor Protein 1 (FSP1). By simultaneously silencing these targets, the treatment promotes phospholipid peroxidation and triggers iron-catalyzed, caspase-independent cell death. Developed by researchers at the Mayo Clinic, this approach has demonstrated significant tumor suppression and target engagement in orthotopic murine models of CCA without evidence of systemic toxicity.

Other names

EpCAM-coated siGPX4/siFSP1-lactosomesferroptosis-inducing siRNA-based nanotherapeutics