Conjugates and compositions for cellular delivery
AssigneeSirna Therapeutics, Inc.
InventorsChandra Vargeese, Peter Haeberli, Weimin Wang, Tongqian Chen
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Abstract
This invention features conjugates, degradable linkers, compositions, methods of synthesis, and applications thereof, including cholesterol, folate, galactose, galactosamine, N-acetyl galactosamine, PEG, phospholipid, peptide and human serum albumin (HSA) derived conjugates of biologically active compounds, including antibodies, antivirals, chemotherapeutics, peptides, proteins, hormones, nucleosides, nucleotides, non-nucleosides, and nucleic acids including enzymatic nucleic acids, DNAzymes, allozymes, antisense, dsRNA, siNA, siRNA, triplex oligonucleotides, 2,5-A chimeras, decoys and aptamers.
Claims9
A compound having Formula 107:
- wherein X comprises a short interfering nucleic acid (siNA) molecule;
- each W independently comprises a linker molecule or chemical linkage selected from the group consisting of amide, phosphate, phosphate ester, phosphoramidate, or thiophosphate ester linkage, Y comprises a linker molecule that can be present or absent;
- each R1, R2, R3, and R4 independently comprises O, OH, H, alkyl, alkylhalo, O-alkyl, O-alkylcyano, S, S-alkyl, S-alkylcyano, N, amide, alkylamine, aminoacid, polyamine, cyclic amine or heterocyclic amine, and Cholesterol comprises cholesterol.
A compound having the formula below:
- wherein X comprises a short interfering nucleic acid (siNA) molecule;
- each W independently comprises a linker molecule or chemical linkage selected from the group consisting of amide, phosphate, phosphate ester, phosphoramidate, or thiophosphate ester linkage, Y comprises a linker molecule that can be present or absent;
- each R1, R2, R3, and R4 independently comprises O, OH, H, alkyl, alkylhalo, O-alkyl, O-alkylcyano, S, S-alkyl, S-alkylcyano, N, amide, alkylamine, aminoacid, polyamine, cyclic amine or heterocyclic amine;
- and n is independently an integer from about 1 to about 20.
A compound having Formula 111:
- wherein X comprises a short interfering nucleic acid (siNA) molecule capable of mediating RNA interference (RNAi);
- W comprises a linker molecule or chemical linkage that can be present or absent, and when present, forms only one covalent bond to the siNA molecule;
- and n is an integer from about 1 to about 20.
Dependencies are inferred from the claim text.