MUSCULOSKELETAL DRUG-DELIVERING PROPERTIES AND PREPARATIONS CONTAINING THESE DRUG-DELIVERING PROPERTIES

Abstract

The invention relates to drug delivery systems that specifically and effectively direct drug carriers to skeletal muscle cells in vivo. The drug delivery systems described herein include targeting ligands (such as compounds with affinity for integrins, including alpha-β-beta-6) and pharmacokinetic / pharmacodynamic modulators (PK / PD), to facilitate the delivery of payloads to cells, including skeletal muscle cells. Suitable payloads for use in the drug delivery systems described herein include RNA agents, which can be bound or conjugated to the drug delivery systems, and when delivered in vivo, provide inhibition of gene expression in skeletal muscle cells. Pharmaceuticals comprising skeletal muscle cell drug delivery systems for the treatment of various diseases and disorders in which delivery of therapeutic payloads to skeletal muscle cells is desired are also described.