LeuFectC

LeuFectC is a lipopolymer-based delivery platform designed for the therapeutic administration of short interfering RNA (siRNA) in the treatment of leukemia. It is composed of low molecular weight polyethyleneimine (PEI) grafted with specific lipids, a structure optimized to complex with siRNA to form stable nanoparticles that facilitate intracellular delivery and gene silencing. Developed by researchers at the University of Alberta and the British Columbia Cancer Agency, LeuFectC has demonstrated the ability to effectively deliver siRNA targeting oncogenes such as STAT5A and FLT3 in primary acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) patient cells. Preclinical studies indicate that LeuFectC-mediated silencing of these drivers can reduce cell proliferation and clonogenicity of leukemia stem cells while maintaining a favorable safety profile in non-malignant peripheral blood mononuclear cells.