WO2007086881A3 - Cationic lipids and formulated molecular compositions containing them - Google Patents

Cationic lipids and formulated molecular compositions containing them Download PDF

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Publication number
WO2007086881A3
WO2007086881A3 PCT/US2006/005127 US2006005127W WO2007086881A3 WO 2007086881 A3 WO2007086881 A3 WO 2007086881A3 US 2006005127 W US2006005127 W US 2006005127W WO 2007086881 A3 WO2007086881 A3 WO 2007086881A3
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WIPO (PCT)
Prior art keywords
molecules
cationic lipids
nanoparticles
rna
microparticles
Prior art date
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PCT/US2006/005127
Other languages
French (fr)
Other versions
WO2007086881A2 (en
Inventor
Tongqian Chen
Chandra Vargeese
Kurt Vagle
Weimin Wang
Ye Zhang
Original Assignee
Sirna Therapeutics Inc
Tongqian Chen
Chandra Vargeese
Kurt Vagle
Weimin Wang
Ye Zhang
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Publication date
Application filed by Sirna Therapeutics Inc, Tongqian Chen, Chandra Vargeese, Kurt Vagle, Weimin Wang, Ye Zhang filed Critical Sirna Therapeutics Inc
Priority to CA002597724A priority Critical patent/CA2597724A1/en
Priority to AU2006336384A priority patent/AU2006336384B2/en
Priority to EP06849713A priority patent/EP1922300A2/en
Priority to JP2007556237A priority patent/JP5042863B2/en
Publication of WO2007086881A2 publication Critical patent/WO2007086881A2/en
Publication of WO2007086881A3 publication Critical patent/WO2007086881A3/en

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    • C—CHEMISTRY; METALLURGY
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    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
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    • C07C217/02—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C217/04—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C217/28—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having one amino group and at least two singly-bound oxygen atoms, with at least one being part of an etherified hydroxy group, bound to the carbon skeleton, e.g. ethers of polyhydroxy amines
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    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy

Abstract

The present invention relates to novel cationic lipids, transfection agents, microparticles, nanoparticles, and short interfering nucleic acid (siNA) molecules. The invention also features compositions, and methods of use for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity in a subject or organism. Specifically, the invention relates to novel cationic lipids, microparticles, nanoparticles and transfection agents that effectively transfect or deliver biologically active molecules, such as antibodies (e.g., monoclonal, chimeric, humanized etc.), cholesterol, hormones, antivirals, peptides, proteins, chemotherapeutics, small molecules, vitamins, co-factors, nucleosides, nucleotides, oligonucleotides, enzymatic nucleic acids, antisense nucleic acids, triplex forming oligonucleotides, 2,5-A chimeras, dsRNA, allozymes, aptamers, decoys and analogs thereof, and small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double- stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules, to relevant cells and/or tissues, such as in a subject or organism. Such novel cationic lipids, microparticles, nanoparticles and transfection agents are useful, for example, in providing compositions to prevent, inhibit, or treat diseases, conditions, or traits in a cell, subject or organism. The compositions described herein are generally referred to as formulated molecular compositions (FMC) or lipid nanoparticles (LNP).
PCT/US2006/005127 2005-02-14 2006-02-14 Cationic lipids and formulated molecular compositions containing them WO2007086881A2 (en)

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AU2006336384A AU2006336384B2 (en) 2005-02-14 2006-02-14 Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
EP06849713A EP1922300A2 (en) 2005-02-14 2006-02-14 Cationic lipids and formulated molecular compositions containing them
JP2007556237A JP5042863B2 (en) 2005-02-14 2006-02-14 Lipid nanoparticle-based compositions and methods for delivering biologically active molecules

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US70394605P 2005-07-29 2005-07-29
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