Publication number: 20140045919
Abstract: The present invention provides iRNA agent including at least one monomer having the structure shown in formula (I?) wherein: A and B are each independently for each occurrence O, N(RN) or S; X is H, a protecting group, a phosphate group, a phosphodiester group, an activated phosphate group, an activated phosphite group, a phosphoramidite, a solid support, —P(Z?)(Z?)O-nucleoside, —P(Z?)(Z?)O-oligonucleotide, a lipid, a PEG, a steroid, a polymer, —P(Z?)(Z?)O-L6-Q?-L7-OP(Z??)(Z??)O-oligonucleotide, a nucleotide, or an oligonucleotide; Y is H, a protecting group, a phosphate group, a phosphodiester group, an activated phosphate group, an activated phosphite group, a phosphoramidite, a solid support, —P(Z?)(Z?)O-nucleoside, —P(Z?)(Z?)O-oligonucleotide, a lipid, a PEG, a steroid, a lipophile, a polymer, —P(Z?)(Z?)O-L6-Q?-L7-OP(Z??)(Z??)O-oligonucleotide, a nucleotide, or an oligonucleotide; R is folate, a folate analog a folate mimic or a folate receptor binding ligand; L6 and L7 are each independently for e
Type:
Application
Filed:
July 8, 2013
Publication date:
February 13, 2014
Applicant:
Alnylam Pharmaceuticals, Inc.
Inventors:
Muthiah Manoharan, Kallanthottathil G. Rajeev, Muthusamy Jayaraman, Jayaprakash Nair