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Withdrawn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Withdrawn legal-status Critical Current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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (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)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Oncology (AREA)
  • Dispersion Chemistry (AREA)
  • Rheumatology (AREA)
  • Virology (AREA)
  • Pain & Pain Management (AREA)
  • Communicable Diseases (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (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 (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI2506857T1 (en) 2009-12-01 2018-08-31 Translate Bio, Inc. Delivery of mRNA for the enrichment of proteins and enzymes in human genetic diseases
EP2527440A1 (en) * 2011-05-27 2012-11-28 Institut Curie Cancer treatment by combining DNA molecules mimicking double strand breaks with hyperthermia
KR20220025112A (ko) 2011-06-08 2022-03-03 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
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
CA2853689C (en) * 2011-11-04 2020-06-30 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
US9198971B2 (en) 2012-01-09 2015-12-01 Adocia Injectable solution at pH 7 comprising at least one basal insulin the pI of which is between 5.8 and 8.5 and a substituted co-polyamino acid
EP3536787A1 (en) 2012-06-08 2019-09-11 Translate Bio, 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
AU2013355258A1 (en) 2012-12-07 2015-06-11 Alnylam Pharmaceuticals, Inc. Improved nucleic acid lipid particle formulations
KR102311614B1 (ko) 2013-03-14 2021-10-08 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
EA201591229A1 (ru) 2013-03-14 2016-01-29 Шир Хьюман Дженетик Терапис, Инк. Способы очистки матричной рнк
EP3574923A1 (en) 2013-10-22 2019-12-04 Translate Bio, Inc. Mrna therapy for phenylketonuria
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
ES3032935T3 (en) * 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
EP4729528A3 (en) 2014-04-25 2026-06-24 Translate Bio, Inc. Methods for purification of messenger rna
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
WO2017013237A1 (en) 2015-07-23 2017-01-26 Institut Curie Use of a combination of dbait molecule and parp inhibitors to treat cancer
KR102764250B1 (ko) 2015-09-14 2025-02-07 더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템 지질양이온성 덴드리머 및 이의 용도
EP3368089B1 (en) 2015-10-26 2025-11-05 Translate Bio Ma, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
US10654875B2 (en) 2015-11-12 2020-05-19 The Board Of Trustees Of The Leland Stanford Junior University Cell-penetrating, guanidinium-rich oligophosphotriesters for drug and probe delivery
WO2018081817A2 (en) 2016-10-31 2018-05-03 University Of Massachusetts Targeting microrna-101-3p in cancer therapy
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
CA3059321A1 (en) * 2017-04-20 2018-10-25 Synthena Ag Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
IL270631B2 (en) 2017-05-16 2024-03-01 Translate Bio Inc Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
CA3071968A1 (en) 2017-08-04 2019-02-07 Kyowa Kirin Co., Ltd. Nucleic acid-containing lipid nanoparticle
US10435429B2 (en) 2017-10-03 2019-10-08 Nucorion Pharmaceuticals, Inc. 5-fluorouridine monophosphate cyclic triester compounds
AU2019207626B2 (en) * 2018-01-10 2023-10-05 Nucorion Pharmaceuticals, Inc. Phosphor(n)amidatacetal and phosph(on)atalcetal compounds
US11427550B2 (en) 2018-01-19 2022-08-30 Nucorion Pharmaceuticals, Inc. 5-fluorouracil compounds
US20210363518A1 (en) * 2018-03-19 2021-11-25 University Of Massachusetts Modified guide rnas for crispr genome editing
US11174500B2 (en) 2018-08-24 2021-11-16 Translate Bio, Inc. Methods for purification of messenger RNA
US20210322575A1 (en) * 2018-09-28 2021-10-21 Nutcracker Therapeutics, Inc. Lipid nanoparticle formulations comprising lipidated cationic peptide compounds for nucleic acid delivery
DK3864163T3 (da) 2018-10-09 2024-05-06 Univ British Columbia Sammensætninger og systemer omfattende transfektionskompetente vesikler fri for organiske opløsningsmidler og rengøringsmidler og fremgangsmåder forbundet dermed
AU2019384557B2 (en) 2018-11-21 2025-07-17 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
WO2021011692A1 (en) 2019-07-17 2021-01-21 Nucorion Pharmaceuticals, Inc. Cyclic deoxyribonucleotide compounds
CA3155003A1 (en) 2019-09-20 2021-03-25 Translate Bio, Inc. Mrna encoding engineered cftr
US20230098031A1 (en) * 2019-12-11 2023-03-30 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
AU2021206510A1 (en) * 2020-01-08 2022-08-04 Microvascular Therapeutics, Llc Alkylated nucleosides, and compositions and methods thereof for nucleic acid delivery
KR20230002300A (ko) * 2020-01-21 2023-01-05 이더알엔에이 이뮤노테라피스 엔브이 지질 나노입자
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
WO2021216431A1 (en) 2020-04-21 2021-10-28 Ligand Pharmaceuticals, Inc. Benzyloxy phosph(on)ate compounds
EP4143199B1 (en) 2020-04-21 2025-11-19 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
CN121517316A (zh) * 2021-05-06 2026-02-13 阿克丘勒斯治疗公司 用于rna递送的可电离阳离子脂质
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
WO2026158650A1 (zh) * 2025-01-26 2026-07-30 上海药明生物技术有限公司 非对称分枝型peg修饰的脂质化合物、其制备方法及其用途

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
AU723998B2 (en) * 1996-03-01 2000-09-07 Centre National De La Recherche Scientifique Compounds related to the amidinium family, pharmaceutical compositions containing same, and uses thereof
JP2001523480A (ja) * 1997-11-20 2001-11-27 バイカル インコーポレイテッド サイトカイン発現ポリヌクレオチドおよびそれらの組成物を用いた癌の処置
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
TW201021853A (en) * 2008-11-17 2010-06-16 Enzon Pharmaceuticals Inc Releasable cationic lipids for nucleic acids delivery systems

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

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
CN117658848A (zh) 递送治疗剂的脂质化合物及其应用
CN121586703A (zh) 递送治疗剂的脂质化合物及其制备方法与用途
WO2025051994A1 (en) Ionizable lipid nanoparticles
WO2024230827A1 (zh) 聚合物脂质化合物及其组合物、制备和用途
WO2011084721A2 (en) Cationic molecule and method of use
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