CN101331231B - 用于rna治疗的肽核糖核酸缩合粒子的复合物及方法 - Google Patents

用于rna治疗的肽核糖核酸缩合粒子的复合物及方法 Download PDF

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Publication number
CN101331231B
CN101331231B CN2006800468655A CN200680046865A CN101331231B CN 101331231 B CN101331231 B CN 101331231B CN 2006800468655 A CN2006800468655 A CN 2006800468655A CN 200680046865 A CN200680046865 A CN 200680046865A CN 101331231 B CN101331231 B CN 101331231B
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China
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peptide
sirna
lys
arg
rna
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Chinese (zh)
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CN101331231A (zh
Inventor
罗杰·C·阿达米
朱恬英
崔坤元
小迈克尔·E·休斯顿
陈力山
陈郁静
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Marina Biotech Inc
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MDRNA Inc
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
    • C07K14/003—Peptide-nucleic acids (PNAs)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A61K47/6455—Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/16—Antivirals for RNA viruses for influenza or rhinoviruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1136—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against growth factors, growth regulators, cytokines, lymphokines or hormones
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10—TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10T—TECHNICAL SUBJECTS COVERED BY FORMER US CLASSIFICATION
    • Y10T428/00—Stock material or miscellaneous articles
    • Y10T428/29—Coated or structually defined flake, particle, cell, strand, strand portion, rod, filament, macroscopic fiber or mass thereof
    • Y10T428/2982—Particulate matter [e.g., sphere, flake, etc.]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Rheumatology (AREA)
  • Endocrinology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pain & Pain Management (AREA)
  • Immunology (AREA)
  • Pulmonology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
CN2006800468655A 2005-10-14 2006-10-13 用于rna治疗的肽核糖核酸缩合粒子的复合物及方法 Expired - Fee Related CN101331231B (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US72721605P 2005-10-14 2005-10-14
US60/727,216 2005-10-14
US73366405P 2005-11-04 2005-11-04
US60/733,664 2005-11-04
US82587806P 2006-09-15 2006-09-15
US60/825,878 2006-09-15
PCT/US2006/040174 WO2007047482A2 (en) 2005-10-14 2006-10-13 Compounds and methods for peptide ribonucleic acid condensate particles for rna therapeutics

Publications (2)

Publication Number Publication Date
CN101331231A CN101331231A (zh) 2008-12-24
CN101331231B true CN101331231B (zh) 2012-11-21

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CN2006800468655A Expired - Fee Related CN101331231B (zh) 2005-10-14 2006-10-13 用于rna治疗的肽核糖核酸缩合粒子的复合物及方法

Country Status (9)

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US (2) US20100129460A1 (enExample)
EP (1) EP1934360A2 (enExample)
JP (3) JP5536334B2 (enExample)
KR (1) KR20080061397A (enExample)
CN (1) CN101331231B (enExample)
AU (1) AU2006304291A1 (enExample)
CA (1) CA2625473A1 (enExample)
MX (1) MX2008004899A (enExample)
WO (1) WO2007047482A2 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AR069704A1 (es) * 2007-12-18 2010-02-10 Alcon Res Ltd Sistema de administracion de rnai de interferencia y usos del mismo
EP2323695B1 (en) 2008-08-19 2018-12-05 Nektar Therapeutics Complexes of small-interfering nucleic acids
WO2010059829A2 (en) * 2008-11-19 2010-05-27 Mdrna, Inc. Compositions and methods for triggered release rna therapeutics
WO2013020986A1 (de) 2011-08-08 2013-02-14 Universität Regensburg Polyanion-nanokomplexe für therapeutische anwendungen
EP2623978A1 (en) 2012-02-03 2013-08-07 Charité - Universitätsmedizin Berlin CD8+ T-cell subsets as markers for prediction of delayed fracture healing
WO2014183017A1 (en) * 2013-05-09 2014-11-13 KIPPERMAN, Richard, M. as Chapter 7 Bankruptcy Trustee for TRAVERSA THERAPEUTICS, INC. Improved delivery of rna interfering agents
KR20180056750A (ko) * 2015-10-05 2018-05-29 신젠타 파티서페이션즈 아게 리보핵산의 생물학적 활성을 보존하는 방법
WO2021015234A1 (ja) * 2019-07-24 2021-01-28 国立大学法人東北大学 キメラ分子、医薬組成物、標的核酸の切断方法、及び、標的核酸切断用又は診断用キット

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GB9218164D0 (en) * 1992-08-26 1992-10-14 Applied Research Systems Virus and substances related thereto
US5679559A (en) * 1996-07-03 1997-10-21 University Of Utah Research Foundation Cationic polymer and lipoprotein-containing system for gene delivery
AU2281201A (en) * 1999-12-29 2001-07-09 A. James Mixson Histidine copolymer and methods for using same
AU2850001A (en) * 2000-02-07 2001-08-20 Transgene S.A. Non-naturally occurring nucleic acid compositions, their use for the preparationof formulations useful for transfecting nucleic acid into cells and applicati ons
WO2001095946A2 (en) * 2000-06-14 2001-12-20 Transgene S.A. Combination product for carrying out a cytotoxic treatment in a mammal
WO2002027031A2 (en) * 2000-09-28 2002-04-04 Cellomics, Inc. Methods and reagents for live-cell gene expression quantification
JP2004035409A (ja) * 2002-05-15 2004-02-05 Geneshuttle Biopharm Inc ベクターとしての使用のための新規融合タンパク質
US20040019008A1 (en) * 2002-05-28 2004-01-29 Lewis David L. Compositions and processes using siRNA, amphipathic compounds and polycations
EP1507874A4 (en) * 2002-05-28 2006-06-28 Mirus Bio Corp COMPOSITIONS AND METHODS FOR INHIBITING GENE EXPRESSION USING POLYNUCLEOTIDES
FR2841137B1 (fr) * 2002-06-20 2004-08-13 Bioalliance Pharma Systeme de vectorisation comprenant des nanoparticules de taille homogene d'au moins un polymere et d'au moins un polysaccharide charge positivement
AU2003279010A1 (en) * 2002-09-28 2004-04-19 Massachusetts Institute Of Technology Compositions and methods for delivery of short interfering rna and short hairpin rna
US8324365B2 (en) * 2003-04-03 2012-12-04 Korea Advanced Institute Of Science And Technology Conjugate for gene transfer comprising oligonucleotide and hydrophilic polymer, polyelectrolyte complex micelles formed from the conjugate, and methods for preparation thereof
US20050136437A1 (en) * 2003-08-25 2005-06-23 Nastech Pharmaceutical Company Inc. Nanoparticles for delivery of nucleic acids and stable double-stranded RNA
ES2411962T3 (es) * 2003-10-24 2013-07-09 Gencia Corporation Métodos y composiciones para suministrar polinucleótidos
US8748403B2 (en) * 2004-01-22 2014-06-10 University Of Massachusetts Modulation of HSV infection
CN101103111A (zh) * 2004-11-05 2008-01-09 因特拉迪格姆公司 用于治疗呼吸道病毒感染的组合物及其用途
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CA2604441A1 (en) * 2005-04-12 2006-10-19 Intradigm Corporation Composition and methods of rnai therapeutics for treatment of cancer and other neovascularization diseases
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Also Published As

Publication number Publication date
MX2008004899A (es) 2008-09-04
EP1934360A2 (en) 2008-06-25
WO2007047482A3 (en) 2007-11-29
US20100129460A1 (en) 2010-05-27
CN101331231A (zh) 2008-12-24
HK1130506A1 (en) 2009-12-31
US20130072424A1 (en) 2013-03-21
JP2014110796A (ja) 2014-06-19
JP2016073290A (ja) 2016-05-12
CA2625473A1 (en) 2007-04-26
KR20080061397A (ko) 2008-07-02
WO2007047482B1 (en) 2008-01-17
JP5536334B2 (ja) 2014-07-02
JP2009511600A (ja) 2009-03-19
AU2006304291A1 (en) 2007-04-26
WO2007047482A2 (en) 2007-04-26

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