Muthiah Manoharan - Cambridge MA, US Takeshi Yamada - Cambridge MA, US David Butler - Cambridge MA, US K. Narayanannair Jayaprakash - Cambridge MA, US Muthusamy Jayraman - Cambridge MA, US Shigeo Matsuda - Cambridge MA, US Rajendra K. Pandey - Cambridge MA, US Chang Geng Peng - Cambridge MA, US
The invention features compounds of formula I or II:In one embodiment, the invention relates compounds and processes for conjugating ligand to oligonucleotide. The invention further relates to methods for treating various disorders and diseases such as viral infections, bacterial infections, parasitic infections, cancers, allergies, autoimmune diseases, immunodeficiencies and immunosuppression.
Muthiah Manoharan - Cambridge MA, US Alexander V. Kelin - Cambridge MA, US Sudhakar Rao Takkellapati - Cambridge MA, US Shigeo Matsuda - Cambridge MA, US
The present invention provides nucleosides of formula (1) and oligonucleotides comprising at least one nucleoside of formula (2): Another aspect of the invention relates to a method of inhibiting the expression of a gene in cell, the method comprising (a) contacting an oligonucleotide of the invention with the cell; and (b) maintaining the cell from step (a) for a time sufficient to obtain degradation of the mRNA of the target gene.
Biodegradable Lipids For The Delivery Of Active Agents
The present invention relates to a cationic lipid having one or more biodegradable groups located in a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
Biodegradable Lipids For The Delivery Of Active Agents
Muthiah MANOHARAN - Cambridge MA, US Martin Maier - Cambridge MA, US Muthusamy Jayaraman - Cambridge MA, US Shigeo Matsuda - Cambridge MA, US Akin Akinc - Cambridge MA, US Thomas A. Baillie - Seattle WA, US
The present invention relates to a cationic lipid having one or more biodegradable groups located in the mid- or distal section of a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
Biodegradable Lipids For The Delivery Of Active Agents
The present invention relates to a cationic lipid having one or more biodegradable groups located in a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
Oligonucleotide-Ligand Conjugates And Process For Their Preparation
- CAMBRIDGE MA, US Jayaprakash K. Nair - Cambridge MA, US Pachamuthu Kandasamy - Cambridge MA, US Shigeo Matsuda - Cambridge MA, US Alexander V. Kelin - Cambridge MA, US Muthusamy Jayaraman - Cambridge MA, US
International Classification:
C12N 15/113 C07H 21/00 C12N 15/11 A61K 47/54
Abstract:
The present invention relates to ligand conjugates of oligonucleotides (e.g., iRNA agents) and methods for their preparation. The ligands are derived primarily from monosaccharides These conjugates are useful for the in vivo delivery of oligonucleotides.
Biodegradable Lipids For The Delivery Of Active Agents
The present invention relates to a cationic lipid having one or more biodegradable groups located in a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
Biodegradable Lipids For The Delivery Of Active Agents
The present invention relates to a cationic lipid having one or more biodegradable groups located in a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
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