CA2487274A1 - Spermine chemically linked to lipids and cell-specific targeting molecules as a transfection agent - Google Patents

Spermine chemically linked to lipids and cell-specific targeting molecules as a transfection agent Download PDF

Info

Publication number
CA2487274A1
CA2487274A1 CA002487274A CA2487274A CA2487274A1 CA 2487274 A1 CA2487274 A1 CA 2487274A1 CA 002487274 A CA002487274 A CA 002487274A CA 2487274 A CA2487274 A CA 2487274A CA 2487274 A1 CA2487274 A1 CA 2487274A1
Authority
CA
Canada
Prior art keywords
composition
nucleic acid
spermine
cell
inclusive
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Abandoned
Application number
CA002487274A
Other languages
English (en)
French (fr)
Inventor
C. Satishchandran
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Alnylam Pharmaceuticals Inc
Original Assignee
Individual
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Individual filed Critical Individual
Publication of CA2487274A1 publication Critical patent/CA2487274A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • 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
    • A61K47/54—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 an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/107—Emulsions ; Emulsion preconcentrates; Micelles
    • A61K9/1075—Microemulsions or submicron emulsions; Preconcentrates or solids thereof; Micelles, e.g. made of phospholipids or block copolymers
    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • 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
    • 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
    • A61P31/04—Antibacterial agents
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • 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
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/40—Vectors comprising a peptide as targeting moiety, e.g. a synthetic peptide, from undefined source
    • C12N2810/405—Vectors comprising RGD peptide

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Dispersion Chemistry (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Endocrinology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
CA002487274A 2002-05-06 2003-05-06 Spermine chemically linked to lipids and cell-specific targeting molecules as a transfection agent Abandoned CA2487274A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US37819102P 2002-05-06 2002-05-06
US60/378,191 2002-05-06
PCT/US2003/014288 WO2003093449A2 (en) 2002-05-06 2003-05-06 Methods for delivery of nucleic acids

Publications (1)

Publication Number Publication Date
CA2487274A1 true CA2487274A1 (en) 2003-11-13

Family

ID=29401591

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002487274A Abandoned CA2487274A1 (en) 2002-05-06 2003-05-06 Spermine chemically linked to lipids and cell-specific targeting molecules as a transfection agent

Country Status (6)

Country Link
US (2) US20060084617A1 (https=)
EP (2) EP1549352A4 (https=)
JP (2) JP4868739B2 (https=)
AU (1) AU2003266014B2 (https=)
CA (1) CA2487274A1 (https=)
WO (1) WO2003093449A2 (https=)

Families Citing this family (76)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2477820A1 (en) * 2002-02-22 2003-08-28 Otsuka Pharmaceutical Co., Ltd. Polynucleotide for target gene
US20050176008A1 (en) * 2002-02-22 2005-08-11 Mikio Suzuki Polynucleotide for target gene
AU2005218674B2 (en) * 2004-03-03 2009-12-17 Phoenix Moon (Holdings) Pty Ltd A therapeutic apparatus and a method of treatment using the apparatus
US7906137B2 (en) * 2004-05-21 2011-03-15 Mediplex Corporation, Korea Delivery agents for enhancing mucosal absorption of therapeutic agents
EP1791567B1 (en) 2004-08-10 2015-07-29 Alnylam Pharmaceuticals Inc. Chemically modified oligonucleotides
CA2577519A1 (en) 2004-08-23 2006-03-30 Nucleonics, Inc. Multiple rna polymerase iii promoter expression constructs
JP2008514202A (ja) 2004-09-24 2008-05-08 ニュークレオニクス・インコーポレイテッド Ｒｎａｉによる一本鎖ウイルスの逆鎖複製中間体のターゲティング
NL1027479C2 (nl) * 2004-10-21 2006-05-01 Synvolux Ip B V Bescherming van biologisch actieve moleculen met behulp van amphifielen.
US20070031512A1 (en) * 2005-08-03 2007-02-08 Amcol International Corporation Virus-interacting layered phyllosilicates and methods of inactivating viruses
US20100272769A1 (en) * 2005-08-03 2010-10-28 Amcol International Virus-, Bacteria-, and Fungi-Interacting Layered Phyllosilicates and Methods of Use
US20080184618A1 (en) * 2005-08-03 2008-08-07 Amcol International Virus-Interacting Layered Phyllosilicates and Methods of Use
EP2799547B1 (en) 2006-11-08 2016-12-21 Veritas Bio, LLC In Vivo Delivery of RNA to a Target Cell
MX2009012317A (es) 2007-05-16 2010-04-01 Mat Malta Advanced Technologie Tratamiento y prevencion de influenza.
US9822364B2 (en) 2008-03-11 2017-11-21 Yale University Compositions and methods for controlled delivery of inhibitory ribonucleic acids
WO2009114614A2 (en) * 2008-03-11 2009-09-17 Yale University Compositions and methods for controlled delivery of inhibitory ribonucleic acids
JP5883381B2 (ja) 2009-05-05 2016-03-15 ミラゲン セラピューティクス，インコーポレイテッド 親油性ポリヌクレオチド接合体
US20130079382A1 (en) 2009-10-12 2013-03-28 Larry J. Smith Methods and Compositions for Modulating Gene Expression Using Oligonucleotide Based Drugs Administered in vivo or in vitro
WO2012061810A1 (en) 2010-11-05 2012-05-10 Miragen Therapeutics Base modified oligonucleotides
EA201370139A1 (ru) 2010-12-15 2013-10-30 Мираген Терапеутикс Ингибиторы микро-рнк, содержащие закрытые нуклеотиды
CA2832899A1 (en) 2011-04-12 2012-10-18 Beth Israel Deaconess Medical Center, Inc. Micro-rna inhibitors and their uses in disease
CA2840965C (en) 2011-07-06 2021-03-02 Novartis Ag Cationic oil-in-water emulsions
SG10201605512WA (en) 2011-07-06 2016-09-29 Novartis Ag Oil-in-water emulsions that contain nucleic acids
EP2755663A4 (en) 2011-09-13 2015-10-07 Ottawa Hospital Res Inst MicroRNA Inhibitors
US9388408B2 (en) 2012-06-21 2016-07-12 MiRagen Therapeutics, Inc. Oligonucleotide-based inhibitors comprising locked nucleic acid motif
WO2014058535A1 (en) * 2012-08-19 2014-04-17 Michigan Technological University Lysosomal targeting probes
CA2902571A1 (en) 2013-03-15 2014-09-18 MiRagen Therapeutics, Inc. Bridged bicyclic nucleosides
EP3068783B1 (en) 2013-11-15 2020-09-23 The Board of Trustees of the Leland Stanford Junior University Agonists of hypocretin receptor 2 for use for treating heart failure
US10772974B2 (en) 2013-11-18 2020-09-15 Beth Israel Deaconess Medical Center, Inc. Compositions and methods for cardiac regeneration
CA3236835A1 (en) 2013-11-22 2015-05-28 Mina Therapeutics Limited C/ebp alpha short activating rna compositions and methods of use
WO2015171516A1 (en) 2014-05-05 2015-11-12 Thetis Pharmaceuticals Llc Compositions and methods relating to ionic salts of peptides
CA2951923A1 (en) 2014-06-18 2015-12-23 Thetis Pharmaceuticals Llc Mineral amino-acid complexes of active agents
US9242008B2 (en) 2014-06-18 2016-01-26 Thetis Pharmaceuticals Llc Mineral amino-acid complexes of fatty acids
WO2016011203A1 (en) 2014-07-15 2016-01-21 Life Technologies Corporation Compositions with lipid aggregates and methods for efficient delivery of molecules to cells
CN108064175A (zh) 2014-08-04 2018-05-22 米拉根医疗股份有限公司 Myh7b的抑制剂及其用途
AU2015315298B2 (en) 2014-09-08 2020-05-14 MiRagen Therapeutics, Inc. miR-29 mimics and uses thereof
US9885042B2 (en) 2015-01-20 2018-02-06 MiRagen Therapeutics, Inc. miR-92 inhibitors and uses thereof
MX368314B (es) 2015-06-05 2019-09-27 Miragen Therapeutics Inc Inhibidores del mir-155 para tratar linfoma cutáneo de células t (ctcl).
WO2017062502A1 (en) * 2015-10-05 2017-04-13 The Regents Of The University Of Colorgo, A Body Corporate Lipoplexes formulated for catalytic delivery
WO2017087486A1 (en) 2015-11-16 2017-05-26 Ohio State Innovation Foundation Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein
ES2827796T3 (es) 2016-06-03 2021-05-24 Thetis Pharmaceuticals Llc Composiciones y procedimientos relativos a sales de mediadores pro-resolutivos especializados de inflamación
EP3472193A4 (en) 2016-06-20 2020-01-08 The Board of Trustees of the Leland Stanford Junior University CIRCULAR RNA AND THEIR USE IN IMMUNOMODULATION
UY37376A (es) 2016-08-26 2018-03-23 Amgen Inc Construcciones de arni para inhibir expresión de asgr1 y métodos para su uso
ES2659845B1 (es) 2016-09-19 2019-01-04 Univ Valencia Modulación de microRNAs contra la distrofia miotónica tipo 1 y antagonistas de microRNAs para ello
WO2019048645A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
CA3075205A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
US10765638B2 (en) 2017-11-03 2020-09-08 Yale University Particle formulation with polycation complex
BR112020018705A2 (pt) 2018-03-14 2021-01-05 Beth Israel Deaconess Medical Center Inibidores de microrna 22
EP3775211B1 (en) 2018-04-12 2023-04-05 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
CN113166761B (zh) 2018-12-10 2025-09-09 美国安进公司 用于抑制pnpla3表达的rnai构建体
EP3894554A1 (en) 2018-12-10 2021-10-20 Amgen Inc. Chemically-modified rnai constructs and uses thereof
EP3953473A1 (en) 2019-04-12 2022-02-16 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
TW202111124A (zh) 2019-05-30 2021-03-16 美商安進公司 用於抑制ｓｃａｐ表現之ｒｎａｉ構建體及其使用方法
WO2021030613A1 (en) 2019-08-13 2021-02-18 Amgen Inc. Rnai constructs for inhibiting slc30a8 expression and methods of use thereof
US20230078200A1 (en) 2019-12-09 2023-03-16 Amgen Inc. RNAi CONSTRUCTS AND METHODS FOR INHIBITING LPA EXPRESSION
WO2021234607A1 (en) 2020-05-20 2021-11-25 St. Jude Children's Research Hospital Detection of alzheimer's disease using specific biomarkers
MX2022015169A (es) 2020-06-01 2023-07-03 Amgen Inc Constructos de arni para inhibir la expresion de hsd17b13 y metodos de uso de los mismos.
EP3929295A1 (en) 2020-06-26 2021-12-29 Universitat Pompeu Fabra Artificial rnas for modulating rna fragments
PE20230993A1 (es) 2020-08-13 2023-06-23 Amgen Inc Construcciones de iarn y metodos para inhibir la expresion de marc1
WO2022098841A1 (en) 2020-11-05 2022-05-12 Amgen Inc. METHODS FOR TREATING ATHEROSCLEROTIC CARDIOVASCULAR DISEASE WITH LPA-TARGETED RNAi CONSTRUCTS
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
EP4314292A1 (en) 2021-03-26 2024-02-07 MiNA Therapeutics Limited Tmem173 sarna compositions and methods of use
WO2023278295A1 (en) 2021-06-29 2023-01-05 The Broad Institute, Inc. Compositions and methods for ameliorating anterodorsal thalamus hyperexcitability
CN118556125A (zh) 2021-10-05 2024-08-27 美国安进公司 用于增强寡核苷酸化合物的基因沉默活性的组合物和方法
CA3235262A1 (en) 2021-10-22 2023-04-27 Amgen Inc. Rnai constructs for inhibiting gpam expression and methods of use thereof
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
IL317825A (en) 2022-07-25 2025-02-01 Amgen Inc RNA interference constructs and methods for inhibiting fam13a expression
EP4353823A1 (en) 2022-10-12 2024-04-17 Resalis Therapeutics S.r.l. Inhibitors of micro-rna 22
WO2024125597A1 (en) 2022-12-14 2024-06-20 Providence Therapeutics Holdings Inc. Compositions and methods for infectious diseases
AR131389A1 (es) 2022-12-16 2025-03-12 Amgen Inc CONSTRUCTOS DE ARNi PARA INHIBIR LA EXPRESIÓN DE TTR Y MÉTODOS DE USO DE LOS MISMOS
WO2024134199A1 (en) 2022-12-22 2024-06-27 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
WO2025260042A1 (en) 2024-06-14 2025-12-18 Amgen Inc. Rnai constructs and methods for inhibiting cnr1 expression
WO2026006275A2 (en) 2024-06-26 2026-01-02 Amgen Inc. Rnai constructs and methods for inhibiting expression of inhbe
WO2026027887A2 (en) 2024-08-02 2026-02-05 Mina Therapeutics Limited Hbg1/2-sarna compositions and methods of use
CN120168501B (zh) * 2025-02-26 2025-11-07 北京炜烨创新科技有限公司 一种核酸纳米微球及其制备方法和应用

Family Cites Families (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4283393A (en) * 1979-03-13 1981-08-11 Merck & Co., Inc. Topical application of interferon inducers
US4897355A (en) * 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5107065A (en) * 1986-03-28 1992-04-21 Calgene, Inc. Anti-sense regulation of gene expression in plant cells
US5922602A (en) * 1988-02-26 1999-07-13 Biosource Technologies, Inc. Cytoplasmic inhibition of gene expression
US5703055A (en) * 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5231020A (en) * 1989-03-30 1993-07-27 Dna Plant Technology Corporation Genetic engineering of novel plant phenotypes
AU5356490A (en) 1989-04-03 1990-11-05 Purdue Research Foundation Method for enhancing transmembrane transport of exogenous molecules
US5108921A (en) 1989-04-03 1992-04-28 Purdue Research Foundation Method for enhanced transmembrane transport of exogenous molecules
US5279833A (en) * 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5264618A (en) * 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US5639595A (en) * 1990-05-01 1997-06-17 Isis Phamaceuticals, Inc. Identification of novel drugs and reagents
US5422241A (en) * 1991-07-03 1995-06-06 Ambion, Inc. Methods for the recovery of nucleic acids from reaction mixtures
US5283185A (en) * 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
CA2130454C (en) * 1992-02-19 2008-04-08 William G. Dougherty Production of viral resistant plants via introduction of untranslatable plus sense viral rna
US5792751A (en) * 1992-04-13 1998-08-11 Baylor College Of Medicine Tranformation of cells associated with fluid spaces
US5593972A (en) * 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
US5981505A (en) * 1993-01-26 1999-11-09 The Trustees Of The University Of Pennsylvania Compositions and methods for delivery of genetic material
US5739118A (en) * 1994-04-01 1998-04-14 Apollon, Inc. Compositions and methods for delivery of genetic material
US5585263A (en) * 1994-05-20 1996-12-17 University Of Alabama At Birmingham Research Foundation Purified retroviral constitutive transport enhancer and its use to facilitate mRNA transport, and to produce recombinant, attenuated HIV
US5837533A (en) 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
US5948767A (en) * 1994-12-09 1999-09-07 Genzyme Corporation Cationic amphiphile/DNA complexes
US5962428A (en) * 1995-03-30 1999-10-05 Apollon, Inc. Compositions and methods for delivery of genetic material
US6051429A (en) * 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
US5932241A (en) * 1995-06-07 1999-08-03 Valentis, Incorporated Cationic lipid DNA complexes for gene targeting
US5668272A (en) * 1995-06-30 1997-09-16 National Research Council Of Canada Method for producing synthetic N-linked glycoconjugates
CA2279673A1 (en) * 1995-12-15 1997-06-16 Enzo Therapeutics, Inc. Property effecting and/or property exhibiting constructs for the expression of non-native nucleic acids for therapeutic and diagnostic uses
US5935936A (en) * 1996-06-03 1999-08-10 Genzyme Corporation Compositions comprising cationic amphiphiles and co-lipids for intracellular delivery of therapeutic molecules
US5849727A (en) * 1996-06-28 1998-12-15 Board Of Regents Of The University Of Nebraska Compositions and methods for altering the biodistribution of biological agents
PT941066E (pt) * 1996-08-26 2004-03-31 Transgene Sa Complexos de lipido cationico - acido nucleico
US6217900B1 (en) 1997-04-30 2001-04-17 American Home Products Corporation Vesicular complexes and methods of making and using the same
EP1027074A1 (en) * 1997-10-28 2000-08-16 American Home Products Corporation Compositions and methods for delivery of genetic material
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US20020065213A1 (en) * 1998-01-16 2002-05-30 Robert J. Debs Methods and compositions for nonviral gene delivery
DE19852928C1 (de) * 1998-11-17 2000-08-03 Steffen Panzner Strukturen in Form von Hohlkugeln
EP2314700A1 (en) 1999-01-28 2011-04-27 Medical College of Georgia Research Institute, Inc Composition and method for in vivo and in vitro attenuation of gene expression using double stranded RNA
WO2000063364A2 (en) 1999-04-21 2000-10-26 American Home Products Corporation Methods and compositions for inhibiting the function of polynucleotide sequences
DE10010264A1 (de) * 2000-03-02 2001-09-13 Novosom Gmbh Stabilisierte Liposomen und Hüllstrukturen
AU4801501A (en) * 2000-05-26 2001-11-29 Transgene S.A. Complex for transferring an anionic substance of interest into a cell
EP1299087A1 (en) * 2000-07-07 2003-04-09 Corixa Corporation Microspheres and adjuvants for dna vaccine delivery
EP1229134A3 (en) * 2001-01-31 2004-01-28 Nucleonics, Inc Use of post-transcriptional gene silencing for identifying nucleic acid sequences that modulate the function of a cell
DE10109897A1 (de) * 2001-02-21 2002-11-07 Novosom Ag Fakultativ kationische Liposomen und Verwendung dieser
DE10127526A1 (de) * 2001-05-31 2002-12-12 Novosom Ag Verfahren zur Herstellung und Auflösung von Nano- und Mikrokapseln
US20060014695A1 (en) * 2002-02-01 2006-01-19 Hamidreza Ghandehari Hpma-polyamine conjugates and uses therefore
DE10207177A1 (de) * 2002-02-19 2003-09-04 Novosom Ag Fakultativ kationische Lipide
DE10207178A1 (de) * 2002-02-19 2003-09-04 Novosom Ag Komponenten für die Herstellung amphoterer Liposomen

Also Published As

Publication number Publication date
JP2006508896A (ja) 2006-03-16
JP4868739B2 (ja) 2012-02-01
US20090163436A1 (en) 2009-06-25
WO2003093449A2 (en) 2003-11-13
US20060084617A1 (en) 2006-04-20
AU2003266014B2 (en) 2009-05-14
WO2003093449A3 (en) 2004-03-11
JP2010235618A (ja) 2010-10-21
EP1549352A2 (en) 2005-07-06
EP1549352A4 (en) 2005-07-27
EP2298358A1 (en) 2011-03-23
AU2003266014A1 (en) 2003-11-17

Similar Documents

Publication Publication Date Title
AU2003266014B2 (en) Methods for delivery of nucleic acids
Li et al. Lipid–oligonucleotide conjugates for bioapplications
JP3907662B2 (ja) 自己構築ポリヌクレオチド送達システム
CN107406396B (zh) 阳离子性脂质
Weissig et al. Cationic bolasomes with delocalized charge centers as mitochondria-specific DNA delivery systems
US8426554B2 (en) In vivo polynucleotide delivery conjugates having enzyme sensitive linkages
US9511024B2 (en) Amino lipids, their synthesis and uses thereof
JP2021050212A (ja) 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム
US20030235916A1 (en) Novel methods for the delivery of polynucleotides to cells
AU2014296278A1 (en) Block copolymers and their conjugates or complexes with oligonucleotides
WO2023029928A1 (zh) 一种氨基脂质及其应用
Ma et al. Targeted delivery of in situ PCR-amplified Sleeping Beauty transposon genes to cancer cells with lipid-based nanoparticle-like protocells
JP7411824B2 (ja) アミノ脂質化合物、その調製方法、およびその用途
Blagbrough et al. Asymmetrical N 4, N 9-diacyl spermines: SAR studies of nonviral lipopolyamine vectors for efficient siRNA delivery with silencing of EGFP reporter gene
WO2022166747A1 (zh) 一种氨基脂质化合物及其制备方法和用途
Qian et al. Synthesis of novel cholesterol-based ionizable lipids for mRNA delivery
Thorsell et al. Lipid mediated gene delivery in the adult rat brain: quantitative analysis of expression
Liang Use of biodegradablepH-sensitive surfactants in liposome mediated oligonucleotide delivery
Mu et al. Aplicación de los Liposomas Como Potenciadores Inmunológicos en la Prevención y el Control de las Enfermedades Tumorales.
CN121909024A (zh) 包封有核酸的配体修饰脂质纳米颗粒的制造方法
CN121135600A (zh) 可电离脂质化合物及其应用
He Synthesis of Peptide Nucleic Acid (PNA) and Entrapment of PNA into Liposomes
Ghosh et al. Gene delivery using cationic micelles and vesicles
Judge et al. 582. Synthetic siRNA Can Activate the Mammalian Innate Immune Response in a Sequence Dependent Manner
EP1380289A1 (en) Delivery system for pharmaceutical agents

Legal Events

Date Code Title Description
EEER Examination request
FZDE Discontinued

Effective date: 20130723