Invention Grant
- Patent Title: iRNA agents with biocleavable tethers
- Patent Title (中): 具有生物可清除系绳的iRNA试剂
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Application No.: US12724267Application Date: 2010-03-15
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Publication No.: US08426377B2Publication Date: 2013-04-23
- Inventor: Muthiah Manoharan , Kallanthottathil G. Rajeev
- Applicant: Muthiah Manoharan , Kallanthottathil G. Rajeev
- Applicant Address: US MA Cambridge
- Assignee: Alnylam Pharmaceuticals, Inc.
- Current Assignee: Alnylam Pharmaceuticals, Inc.
- Current Assignee Address: US MA Cambridge
- Agency: LeClairRyan
- Agent Jeffrey N. Townes
- Main IPC: A61K31/70
- IPC: A61K31/70 ; C07H21/04 ; C07H5/06

Abstract:
The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group. The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent (e.g., a single stranded or double stranded iRNA agent), coupled by way of the cleavable linking group to a targeting agent—to targets cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent.
Public/Granted literature
- US20100267941A1 IRNA AGENTS WITH BIOCLEAVABLE TETHERS Public/Granted day:2010-10-21
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