CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has submitted an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for ALN-VSP, an RNAi therapeutic for the treatment of liver cancers, including hepatocellular carcinoma and other solid tumors with liver involvement. ALN-VSP contains two small interfering RNAs (siRNAs), the molecules that mediate RNAi, formulated in a lipid nanoparticle developed by Tekmira Pharmaceuticals Corporation. ALN-VSP is designed to target two genes critical in the growth and development of cancer: kinesin spindle protein, or KSP, required for tumor proliferation; and vascular endothelial growth factor, or VEGF, required for tumor growth. Pre-clinical data in mouse tumor model studies have demonstrated robust efficacy of ALN-VSP, including suppression of these targeted genes, demonstration of an RNAi mechanism of action, tumor reduction, and extension of survival.
