Endosomal cleavable linkers
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsJayaprakash K. NAIR, Kallanthottathil G. RAJEEV, Scott LENTINI, Christopher S. THEILE, Martin MAIER, Ivan ZLATEV
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Abstract
The present disclosure relates generally to cleavable linkers and uses thereof.
Claims28
A molecule comprising at least two effector molecules, wherein the effector molecules are connected together via an endosomal cleavable linker, wherein:
- the effector molecules are selected independently from the group consisting of siRNA, shRNA, antisense oligonucleotide, microRNA, anti-microRNA or antimir, supermir, antagomir, ribozyme, triplex-forming oligonucleotide, decoy oligonucleotide, splice-switching oligonucleotide, immunostimulatory oligonucleotide, RNA activator, U1 adaptor, and any combinations thereof, and
- the endosomal cleavable linker comprises one or more saccharide units independently selected from:
- wherein m and n are each independently an integer selected from 1 to 12.
A conjugate comprising an effector molecule linked to a ligand via an endosomal cleavable linker or a protease cleavable linker, wherein the linker is a carbohydrate linker and wherein the linker is cleaved at least 1.25 times faster in the cell (or under in vitro conditions selected to mimic intracellular conditions) as compared to blood or serum (or under in vitro conditions selected to mimic extracellular conditions).
A conjugate comprising an endosomal agent linked with a ligand via an endosomal cleavable linker or a protease cleavable linker.
Dependencies are inferred from the claim text.