JP2012509258A - 核酸送達系のための分岐カチオン性脂質 - Google Patents

核酸送達系のための分岐カチオン性脂質 Download PDF

Info

Publication number
JP2012509258A
JP2012509258A JP2011536348A JP2011536348A JP2012509258A JP 2012509258 A JP2012509258 A JP 2012509258A JP 2011536348 A JP2011536348 A JP 2011536348A JP 2011536348 A JP2011536348 A JP 2011536348A JP 2012509258 A JP2012509258 A JP 2012509258A
Authority
JP
Japan
Prior art keywords
independently
peg
nanoparticle
substituted
alkyl
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.)
Withdrawn
Application number
JP2011536348A
Other languages
English (en)
Japanese (ja)
Other versions
JP2012509258A5 (enExample
Inventor
ザオ，ホン
ヤン，ウェイリ
シー，リャンジュン
ウー，デシュン
Original Assignee
エンゾン ファーマシューティカルズ，インコーポレーテッド
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 エンゾン ファーマシューティカルズ，インコーポレーテッド filed Critical エンゾン ファーマシューティカルズ，インコーポレーテッド
Publication of JP2012509258A publication Critical patent/JP2012509258A/ja
Publication of JP2012509258A5 publication Critical patent/JP2012509258A5/ja
Withdrawn legal-status Critical Current

Links

Images

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
    • C12N15/87—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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • 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
    • 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
    • A61P35/00—Antineoplastic agents
    • A61P35/04—Antineoplastic agents specific for metastasis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J41/00—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring
    • C07J41/0033—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005
    • C07J41/0055—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005 the 17-beta position being substituted by an uninterrupted chain of at least three carbon atoms which may or may not be branched, e.g. cholane or cholestane derivatives, optionally cyclised, e.g. 17-beta-phenyl or 17-beta-furyl derivatives
    • 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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Dispersion Chemistry (AREA)
  • Oncology (AREA)
  • Pain & Pain Management (AREA)
  • Communicable Diseases (AREA)
  • Rheumatology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Steroid Compounds (AREA)
JP2011536348A 2008-11-17 2009-07-31 核酸送達系のための分岐カチオン性脂質 Withdrawn JP2012509258A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US11530708P 2008-11-17 2008-11-17
US61/115,307 2008-11-17
PCT/US2009/052462 WO2010056403A1 (en) 2008-11-17 2009-07-31 Branched cationic lipids for nucleic acids delivery system

Publications (2)

Publication Number Publication Date
JP2012509258A true JP2012509258A (ja) 2012-04-19
JP2012509258A5 JP2012509258A5 (enExample) 2012-07-19

Family

ID=42170237

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2011536348A Withdrawn JP2012509258A (ja) 2008-11-17 2009-07-31 核酸送達系のための分岐カチオン性脂質

Country Status (7)

Country Link
US (1) US20110305769A1 (enExample)
EP (1) EP2350296A4 (enExample)
JP (1) JP2012509258A (enExample)
CN (1) CN102216462A (enExample)
CA (1) CA2742689A1 (enExample)
TW (1) TW201019969A (enExample)
WO (1) WO2010056403A1 (enExample)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012509272A (ja) * 2008-11-17 2012-04-19 エンゾン ファーマシューティカルズ，インコーポレーテッド 核酸送達系のための放出性カチオン脂質
JP2015520195A (ja) * 2012-06-08 2015-07-16 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 非肺標的細胞へのｍＲＮＡの経肺送達
JP2015530382A (ja) * 2012-08-28 2015-10-15 メディカル リサーチ カウンシル ナノ粒子製剤
JP2019535808A (ja) * 2016-10-17 2019-12-12 南京▲緑▼叶制▲薬▼有限公司 ｂｃｌ−２を抑制するアンチセンスオリゴヌクレオチドの脂質ナノ粒子及びその調製方法

Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3338765T3 (pl) 2009-12-01 2019-06-28 Translate Bio, Inc. Pochodna steroidowa dla dostarczania mrna w ludzkich chorobach genetycznych
EP2527440A1 (en) * 2011-05-27 2012-11-28 Institut Curie Cancer treatment by combining DNA molecules mimicking double strand breaks with hyperthermia
PT2717893T (pt) 2011-06-08 2019-08-20 Translate Bio Inc Composições de nanopartículas lipídicas e métodos para transferência de arnm
WO2013021143A1 (fr) 2011-08-10 2013-02-14 Adocia Solution injectable d'au moins une insuline basale
EP2766482B1 (en) 2011-10-11 2016-12-07 The Brigham and Women's Hospital, Inc. Micrornas in neurodegenerative disorders
US9579338B2 (en) 2011-11-04 2017-02-28 Nitto Denko Corporation Method of producing lipid nanoparticles for drug delivery
CN102516534B (zh) * 2011-11-14 2013-11-20 上海交通大学 可降解为精胺的聚阳离子及其合成方法、纳米颗粒
BR112014016889A8 (pt) 2012-01-09 2017-07-04 Adocia composição sob a forma de uma solução aquosa injetável, sujo ph está compreendido entre 6,0 e 8,0 e, formulação de dose unitária com ph compreendido entre 7 e 7,8
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
US20150314003A2 (en) 2012-08-09 2015-11-05 Adocia Injectable solution at ph 7 comprising at least one basal insulin the isoelectric point of which is between 5.8 and 8.5 and a hydrophobized anionic polymer
US9687448B2 (en) 2012-12-07 2017-06-27 Alnylam Pharmaceuticals, Inc. Nucleic acid lipid particle formulations
ES2981185T3 (es) 2013-03-14 2024-10-07 Translate Bio Inc Métodos para la purificación de ARN mensajero
EP2968586B1 (en) 2013-03-14 2018-07-25 Translate Bio, Inc. Cftr mrna compositions and related methods and uses
MX2016005239A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Tratamiento con acido ribonucleico mensajero para la fenilcetonuria.
US11224642B2 (en) 2013-10-22 2022-01-18 Translate Bio, Inc. MRNA therapy for argininosuccinate synthetase deficiency
WO2015061467A1 (en) * 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Lipid formulations for delivery of messenger rna
JP6571679B2 (ja) 2014-04-25 2019-09-04 トランスレイト バイオ， インコーポレイテッド メッセンジャーｒｎａの精製方法
CN104922068B (zh) * 2015-05-22 2017-10-13 江苏凯基生物技术股份有限公司 一种Decoy核酸阳离子脂质体载体及其制备方法
ES2879434T3 (es) 2015-07-23 2021-11-22 Inst Curie Uso de una combinación de molécula Dbait e inhibidores de PARP para tratamiento del cáncer
CN108271360B (zh) 2015-09-14 2023-01-24 得克萨斯州大学系统董事会 亲脂阳离子树枝状聚合物及其用途
US12318489B2 (en) 2015-10-26 2025-06-03 Translate Bio Ma, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
WO2017083637A1 (en) * 2015-11-12 2017-05-18 The Board Of Trustees Of The Leland Stanford Junior University Cell-penetrating, guanidinium-rich oligophosphotriesters for drug and probe delivery
CA3037046A1 (en) 2016-10-31 2018-05-03 University Of Massachusetts Targeting microrna-101-3p in cancer therapy
AU2018224326B2 (en) 2017-02-27 2024-01-04 Translate Bio, Inc. Novel codon-optimized CFTR mRNA
CA3059321A1 (en) * 2017-04-20 2018-10-25 Synthena Ag Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
MX2019013752A (es) 2017-05-16 2020-07-20 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
EP3662913A4 (en) * 2017-08-04 2021-06-30 Kyowa Kirin Co., Ltd. Nucleic-acid-containing lipid nanoparticles
US10435429B2 (en) 2017-10-03 2019-10-08 Nucorion Pharmaceuticals, Inc. 5-fluorouridine monophosphate cyclic triester compounds
CN111836818B (zh) * 2018-01-10 2023-10-31 纽科利制药公司 氨基磷(膦)酸缩醛和磷(膦)酸缩醛化合物
US11427550B2 (en) 2018-01-19 2022-08-30 Nucorion Pharmaceuticals, Inc. 5-fluorouracil compounds
WO2019183000A1 (en) * 2018-03-19 2019-09-26 University Of Massachusetts Modified guide rnas for crispr genome editing
CN118421617A (zh) 2018-08-24 2024-08-02 川斯勒佰尔公司 用于纯化信使rna的方法
US20210324002A1 (en) * 2018-09-28 2021-10-21 Nutcracker Therapeutics, Inc. Tertiary amino lipidated cationic peptides for nucleic acid delivery
CN113166783B (zh) 2018-10-09 2024-10-11 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
EP3883592A1 (en) 2018-11-21 2021-09-29 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr
EP3999519A4 (en) 2019-07-17 2023-08-16 Nucorion Pharmaceuticals, Inc. CYCLIC DEOXYRIBONUCLEOTIDE COMPOUNDS
WO2021055609A1 (en) 2019-09-20 2021-03-25 Translate Bio, Inc. Mrna encoding engineered cftr
WO2021119268A1 (en) * 2019-12-11 2021-06-17 The General Hospital Corporation Methods for cell imaging
US20230062603A1 (en) * 2020-01-07 2023-03-02 Translate Bio Ma, Inc. Formulations for delivery of oligonucleotides to lung cells
WO2021142059A1 (en) * 2020-01-08 2021-07-15 Microvascular Therapeutics, Llc Alkylated nucleosides, and compositions and methods thereof for nucleic acid delivery
WO2021148511A1 (en) * 2020-01-21 2021-07-29 Etherna Immunotherapies Nv Lipid nanoparticles
EP3865122A1 (en) * 2020-02-11 2021-08-18 Pantherna Therapeutics GmbH Lipid composition and use thereof for delivery of a therapeutically active agent to endothelium
EP4139319A4 (en) 2020-04-21 2024-06-19 Ligand Pharmaceuticals, Inc. BENZYLOXY-PHOSPH(ON)ATE COMPOUNDS
WO2021216427A1 (en) 2020-04-21 2021-10-28 Ligand Pharmaceuticals, Inc. Nucleotide prodrug compounds
CN114315606B (zh) * 2020-12-29 2023-12-26 李斌 一种脂样分子及其应用
JP2024518375A (ja) * 2021-05-06 2024-05-01 アークトゥラス・セラピューティクス・インコーポレイテッド Ｒｎａ送達のためのイオン化可能なカチオン性脂質
WO2023107920A1 (en) * 2021-12-07 2023-06-15 The Trustees Of The University Of Pennsylvania Anisamide-containing lipids and compositions and methods of use thereof
CN114306369B (zh) * 2021-12-23 2023-12-26 北京悦康科创医药科技股份有限公司 一种硫代寡核苷酸注射液及其制备方法

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996001840A1 (en) * 1994-07-08 1996-01-25 Gilead Sciences, Inc. Cationic lipids for delivery of nucleic acids to cells
WO1996040264A1 (en) * 1995-06-07 1996-12-19 The Regents Of The University Of California Separation of active complexes
WO2008043366A2 (en) * 2006-10-13 2008-04-17 Københavns Universitet Three-domain compounds for transmembrane delivery
JP2012509366A (ja) * 2008-11-17 2012-04-19 エンゾン ファーマシューティカルズ，インコーポレーテッド 核酸送達系のための放出可能ポリマー脂質

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5830430A (en) * 1995-02-21 1998-11-03 Imarx Pharmaceutical Corp. Cationic lipids and the use thereof
DE69703878T2 (de) * 1996-03-01 2001-04-26 Centre National De La Recherche Scientifique (C.N.R.S.), Paris Guanidinium-verbindungen, pharmazeutische präparate davon und ihre verwendungen
CA2309766C (en) * 1997-11-20 2008-09-30 Vical Incorporated Treatment of cancer using cytokine-expressing polynucleotides and compositions therefor
US6320017B1 (en) * 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
AU2003295561A1 (en) * 2002-11-14 2004-06-15 Genta Salus Llc Inhibitory oliogonucleotides targeted to bcl-2
US7713738B2 (en) * 2003-02-10 2010-05-11 Enzon Pharmaceuticals, Inc. Oligomeric compounds for the modulation of survivin expression
TWI257924B (en) * 2003-09-22 2006-07-11 Lipotek Inc Cationic lipid for delivery function, and nanoparticle comprising the same
WO2007056861A1 (en) * 2005-11-18 2007-05-24 Protiva Biotherapeutics, Inc. Sirna silencing of influenza virus gene expression
JP2012509272A (ja) * 2008-11-17 2012-04-19 エンゾン ファーマシューティカルズ，インコーポレーテッド 核酸送達系のための放出性カチオン脂質

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996001840A1 (en) * 1994-07-08 1996-01-25 Gilead Sciences, Inc. Cationic lipids for delivery of nucleic acids to cells
WO1996040264A1 (en) * 1995-06-07 1996-12-19 The Regents Of The University Of California Separation of active complexes
WO2008043366A2 (en) * 2006-10-13 2008-04-17 Københavns Universitet Three-domain compounds for transmembrane delivery
JP2012509366A (ja) * 2008-11-17 2012-04-19 エンゾン ファーマシューティカルズ，インコーポレーテッド 核酸送達系のための放出可能ポリマー脂質

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
JPN6014000748; ＬＩＴＭＡＮ，Ｐ．　ｅｔ　ａｌ: Ｂｉｏｃｈｅｍｉｓｔｒｙ Ｖｏｌ．４６, 2007, ｐ．４７１６-４７２４ *
JPN6014000751; ＹＩＮＧＹＯＮＧＮＡＲＯＮＧＫＵＬ，Ｓ．Ｅ．Ｈ．Ｂ．　ｅｔ　ａｌ: Ｃｏｍｂｉｎａｔｏｒｉａｌ　Ｃｈｅｍｉｓｔｒｙ　＆　Ｈｉｇｈ　Ｔｈｒｏｕｇｈｐｕｔ　Ｓｃｒｅｅｎｉｎｇ Ｖｏｌ．７, 2004, ｐ．４２３-４３０ *
JPN6014000753; ＫＩＭ，Ｗ．Ｊ．　ｅｔ　ａｌ: Ｍｏｌｅｃｕｌａｒ　Ｔｈｅｒａｐｙ Ｖｏｌ．１４，　Ｎｏ．３, 2006, ｐ．３４３-３５０ *
JPN6014000756; ＦＵＴＡＫＩ，Ｓ．　ｅｔ　ａｌ: Ｂｉｏｃｏｎｊｕｇａｔｅ　Ｃｈｅｍｉｓｔｒｙ Ｖｏｌ．１２, 2001, ｐ．１００５-１０１１ *

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012509272A (ja) * 2008-11-17 2012-04-19 エンゾン ファーマシューティカルズ，インコーポレーテッド 核酸送達系のための放出性カチオン脂質
JP2015520195A (ja) * 2012-06-08 2015-07-16 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 非肺標的細胞へのｍＲＮＡの経肺送達
JP2015530382A (ja) * 2012-08-28 2015-10-15 メディカル リサーチ カウンシル ナノ粒子製剤
JP2019535808A (ja) * 2016-10-17 2019-12-12 南京▲緑▼叶制▲薬▼有限公司 ｂｃｌ−２を抑制するアンチセンスオリゴヌクレオチドの脂質ナノ粒子及びその調製方法

Also Published As

Publication number Publication date
US20110305769A1 (en) 2011-12-15
CA2742689A1 (en) 2010-05-20
EP2350296A1 (en) 2011-08-03
EP2350296A4 (en) 2013-04-03
CN102216462A (zh) 2011-10-12
WO2010056403A1 (en) 2010-05-20
TW201019969A (en) 2010-06-01

Similar Documents

Publication Publication Date Title
JP2011529912A (ja) 核酸送達システム用のナノ粒子組成物
US20110305769A1 (en) Branched cationic lipids for nucleic acids delivery system
JP2012509272A (ja) 核酸送達系のための放出性カチオン脂質
JP2012509273A (ja) 核酸送達系のための放出可能融合性脂質
US20110305770A1 (en) Releasable polymeric lipids for nucleic acids delivery system
KR101762466B1 (ko) 지질, 지질 조성물 및 이의 사용 방법
CN101291653B (zh) 脂质包封的干扰rna
AU2014236250B2 (en) Process for formulating an anionic agent
JP2018115164A (ja) アミン陽イオン性脂質およびその使用
EP2608785B1 (en) Lipomacrocycles and uses thereof
JP5407862B2 (ja) ｓｉＲＮＡおよび脂質性４，５−二置換２−デオキシストレプタミン環アミノグリコシド誘導体を含む組成物ならびにその用途
JP2025541727A (ja) 新規のポリグリセロールコンジュゲート脂質及びそれを含む脂質ナノ粒子組成物
CN121586703A (zh) 递送治疗剂的脂质化合物及其制备方法与用途
CN117658848A (zh) 递送治疗剂的脂质化合物及其应用
WO2025051994A1 (en) Ionizable lipid nanoparticles
WO2011084721A2 (en) Cationic molecule and method of use
WO2024230827A1 (zh) 聚合物脂质化合物及其组合物、制备和用途
CN121574072A (zh) 一种用于核酸递送的脂质材料及其应用

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20120604

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20120604

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20140114

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20140805

A761 Written withdrawal of application

Free format text: JAPANESE INTERMEDIATE CODE: A761

Effective date: 20140925