Galactose Cluster-Pharmacokinetic Modulator Targeting Moiety for siRNA
AssigneeArrowhead Madison Inc.
InventorsPhilipp Hadwiger, Torsten Hoffmann, Eric A Kitas, Peter Mohr, Ingo Roehl, Linda Valis, David B Rozema, David L Lewis, Darren H Wakefield
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Abstract
The present invention is directed compositions for targeted delivery of RNA interference (RNAi) polynucleotides to cell in vivo. The pharmacokinetic modulator improve in vivo targeting compared to the targeting ligand alone. Targeting ligand-pharmacokinetic modulator targeting moiety targeted RNAi polynucleotides can be administered in vivo alone or together with co-targeted delivery polymers.
Claims20
A composition for delivering an RNA interference polynucleotide to a liver cell in vivo comprising the structure represented by:
- wherein R1 comprises a hydrophobic group having 16 or more carbon atoms, R2 comprises the RNA interference polynucleotide, and R3 comprises a galactose or galactose derivative having affinity for the asialoglycoprotein receptor equal to or greater than that of galactose.
Dependencies are inferred from the claim text.