KR20170075742A - B형 간염 바이러스 유전자 발현을 제거하는 조성물 및 방법 - Google Patents

B형 간염 바이러스 유전자 발현을 제거하는 조성물 및 방법 Download PDF

Info

Publication number
KR20170075742A
KR20170075742A KR1020177011725A KR20177011725A KR20170075742A KR 20170075742 A KR20170075742 A KR 20170075742A KR 1020177011725 A KR1020177011725 A KR 1020177011725A KR 20177011725 A KR20177011725 A KR 20177011725A KR 20170075742 A KR20170075742 A KR 20170075742A
Authority
KR
South Korea
Prior art keywords
lipid
seq
nucleic acid
mol
hepatitis
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Withdrawn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
KR1020177011725A
Other languages
English (en)
Korean (ko)
Inventor
제니퍼 엘. 크로스
엠멘 피. 딜런
에이미 씨.에이취. 리
이안 맥라클란
니콜라스 엠. 스니드
에밀리 피. 타이
Original Assignee
프로티바 바이오쎄라퓨틱스, 인코포레이티드
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=54325078&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=KR20170075742(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by 프로티바 바이오쎄라퓨틱스, 인코포레이티드 filed Critical 프로티바 바이오쎄라퓨틱스, 인코포레이티드
Publication of KR20170075742A publication Critical patent/KR20170075742A/ko
Withdrawn legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1131Non-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 viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/24Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
KR1020177011725A 2014-10-02 2015-10-01 B형 간염 바이러스 유전자 발현을 제거하는 조성물 및 방법 Withdrawn KR20170075742A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201462059056P 2014-10-02 2014-10-02
US62/059,056 2014-10-02
US201562120149P 2015-02-24 2015-02-24
US62/120,149 2015-02-24
PCT/US2015/053569 WO2016054421A1 (en) 2014-10-02 2015-10-01 Compositions and methods for silencing hepatitis b virus gene expression

Publications (1)

Publication Number Publication Date
KR20170075742A true KR20170075742A (ko) 2017-07-03

Family

ID=54325078

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020177011725A Withdrawn KR20170075742A (ko) 2014-10-02 2015-10-01 B형 간염 바이러스 유전자 발현을 제거하는 조성물 및 방법

Country Status (17)

Country Link
US (1) US10415037B2 (enExample)
EP (1) EP3201338B1 (enExample)
JP (1) JP2017536092A (enExample)
KR (1) KR20170075742A (enExample)
CN (1) CN107208095B (enExample)
AU (1) AU2015328012A1 (enExample)
BR (1) BR112017006679A2 (enExample)
CA (1) CA2963271A1 (enExample)
CL (1) CL2017000780A1 (enExample)
CO (1) CO2017004290A2 (enExample)
CR (1) CR20170174A (enExample)
IL (1) IL251450A0 (enExample)
PH (1) PH12017500614A1 (enExample)
RU (1) RU2017114964A (enExample)
SG (1) SG11201702662UA (enExample)
TW (2) TW201623614A (enExample)
WO (1) WO2016054421A1 (enExample)

Families Citing this family (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8058069B2 (en) * 2008-04-15 2011-11-15 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
EP2449114B9 (en) 2009-07-01 2017-04-19 Protiva Biotherapeutics Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
EP3403647A1 (en) 2009-12-01 2018-11-21 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
US20140206753A1 (en) 2011-06-08 2014-07-24 Shire Human Genetic Therapies, Inc. Lipid nanoparticle compositions and methods for mrna delivery
EP2859102A4 (en) 2012-06-08 2016-05-11 Shire Human Genetic Therapies NUCLEASE RESISTANT POLYNUCLEOTIDES AND USES THEREOF
MX2014015041A (es) 2012-06-08 2015-06-17 Shire Human Genetic Therapies Administración pulmonar de arnm a células objetivo no pulmonares.
LT2970456T (lt) 2013-03-14 2021-08-10 Translate Bio, Inc. Būdai ir kompozicijos, skirti mrnr koduojamų antikūnų pristatymui
CA2904151C (en) 2013-03-14 2023-09-12 Shire Human Genetic Therapies, Inc. Cftr mrna compositions and related methods and uses
ES2981185T3 (es) 2013-03-14 2024-10-07 Translate Bio Inc Métodos para la purificación de ARN mensajero
PL4332576T3 (pl) 2013-03-15 2025-09-08 Translate Bio, Inc. Synergistyczne wzmocnienie dostarczania kwasów nukleinowych poprzez formulacje zmieszane
US20150110857A1 (en) 2013-10-22 2015-04-23 Shire Human Genetic Therapies, Inc. Cns delivery of mrna and uses thereof
MX2016005238A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Formulaciones de lipidos para la administracion de acido ribonucleico mensajero.
BR112016009014B1 (pt) 2013-10-22 2024-02-06 Translate Bio, Inc USO DE COMPOSIÇÃO COMPREENDENDO mRNA PARA DEFICIÊNCIA DE ARGININOSSUCINATO SINTETASE
WO2015061491A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Mrna therapy for phenylketonuria
MX373952B (es) 2014-04-25 2020-07-13 Shire Human Genetic Therapies Métodos de purificación de arn mensajero.
CN106659731A (zh) 2014-05-30 2017-05-10 夏尔人类遗传性治疗公司 用于递送核酸的可生物降解脂质
UA121863C2 (uk) 2014-06-24 2020-08-10 Транслейт Байо, Інк. Стереохімічно збагачені композиції для доставки нуклеїнових кислот
EP3164112A1 (en) 2014-07-02 2017-05-10 Shire Human Genetic Therapies, Inc. Encapsulation of messenger rna
WO2016090262A1 (en) 2014-12-05 2016-06-09 Shire Human Genetic Therapies, Inc. Messenger rna therapy for treatment of articular disease
CA2979695C (en) 2015-03-19 2025-02-06 Translate Bio Inc RNA THERAPY FOR POMPE DISEASE
US20170137821A1 (en) 2015-07-17 2017-05-18 Arcturus Therapeutics, Inc. Molecules and agents for treating hepatitis b virus
AU2016296592B2 (en) 2015-07-17 2021-08-19 Arcturus Therapeutics, Inc. Compositions and agents against Hepatitis B virus and uses thereof
WO2017019891A2 (en) * 2015-07-29 2017-02-02 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis b virus gene expression
CA3001852A1 (en) 2015-10-14 2017-04-20 Translate Bio, Inc. Modification of rna-related enzymes for enhanced production
JP7150608B6 (ja) 2016-04-08 2022-11-11 トランスレイト バイオ, インコーポレイテッド 多量体コード核酸及びその使用
JP2019522047A (ja) 2016-06-13 2019-08-08 トランスレイト バイオ, インコーポレイテッド オルニチントランスカルバミラーゼ欠損症治療のためのメッセンジャーrna療法
CN116751200A (zh) * 2016-11-07 2023-09-15 爱彼特生物制药公司 含有取代的吡啶酮的三环化合物以及使用其的方法
AU2017379901B2 (en) 2016-12-21 2024-02-15 Arbutus Biopharma Corporation Methods for ameliorating infusion reactions
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
MA49138A (fr) 2017-05-16 2020-03-25 Translate Bio Inc Traitement de la fibrose kystique par administration d'arnm à codons optimisés codant pour la cftr
WO2018232330A1 (en) * 2017-06-16 2018-12-20 Arbutus Biopharma Corporation Therapeutic compositions and methods for treating hepatitis b
CN107291266B (zh) * 2017-06-21 2018-08-31 腾讯科技(深圳)有限公司 图像显示的方法和装置
WO2019051257A2 (en) * 2017-09-11 2019-03-14 Arbutus Biopharma Corporation METHODS OF TREATING HEPATITIS B TYPE INFECTIONS
AU2018350693B2 (en) 2017-10-16 2021-03-04 F. Hoffmann-La Roche Ag Nucleic acid molecule for reduction of PAPD5 and PAPD7 mRNA for treating hepatitis B infection
CA3084061A1 (en) 2017-12-20 2019-06-27 Translate Bio, Inc. Improved composition and methods for treatment of ornithine transcarbamylase deficiency
US11702427B2 (en) 2018-03-12 2023-07-18 Arbutus Biopharma Corporation Substituted 2-pyridone tricyclic compounds, analogues thereof, and methods using same
EP3775208A1 (en) * 2018-04-05 2021-02-17 F. Hoffmann-La Roche AG Use of fubp1 inhibitors for treating hepatitis b virus infection
AU2019252441A1 (en) 2018-04-12 2020-10-22 Arbutus Biopharma Corporation Methods for preparing substituted pyridinone-containing tricyclic compounds
WO2020041793A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
US20210395751A1 (en) * 2018-10-31 2021-12-23 The University Of Sydney Compositions and methods for treating viral infections
EP3877521A4 (en) * 2018-11-09 2022-07-27 Arbutus Biopharma Corporation Lipid nanoparticle formulations
CN113728101A (zh) * 2018-11-09 2021-11-30 阿布特斯生物制药公司 脂质纳米颗粒制剂
AU2019384557B2 (en) 2018-11-21 2025-07-17 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
US12404527B2 (en) 2018-12-24 2025-09-02 Selexs S.A. Characterization and inactivation of endogenous retroviruses in chinese hamster ovary cells
AU2020412891A1 (en) 2019-12-24 2022-06-30 Selexis Sa Targeted integration in mammalian sequences enhancing gene expression
RU2733361C1 (ru) * 2020-07-14 2020-10-01 Федеральное государственное бюджетное учреждение "Государственный научный центр "Институт иммунологии" Федерального медико-биологического агентства России (ФГБУ "ГНЦ Институт иммунологии" ФМБА России) Средство для ингибирования репликации вируса SARS-CoV-2, опосредованного РНК-интерференцией
RU2746362C9 (ru) * 2021-03-11 2021-04-26 Федеральное государственное бюджетное учреждение "Государственный научный центр "Институт иммунологии" Федерального медико-биологического агентства России (ФГБУ "ГНЦ Институт иммунологии" ФМБА России) Комбинированное лекарственное средство, обладающее противовирусным эффектом в отношении нового коронавируса SARS-CoV-2

Family Cites Families (108)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3993754A (en) 1974-10-09 1976-11-23 The United States Of America As Represented By The United States Energy Research And Development Administration Liposome-encapsulated actinomycin for cancer chemotherapy
US4086257A (en) 1976-10-12 1978-04-25 Sears Barry D Phosphatidyl quaternary ammonium compounds
CH624011A5 (enExample) 1977-08-05 1981-07-15 Battelle Memorial Institute
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4522803A (en) 1983-02-04 1985-06-11 The Liposome Company, Inc. Stable plurilamellar vesicles, their preparation and use
US4588578A (en) 1983-08-08 1986-05-13 The Liposome Company, Inc. Lipid vesicles prepared in a monophase
US5208036A (en) 1985-01-07 1993-05-04 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
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US5725871A (en) 1989-08-18 1998-03-10 Danbiosyst Uk Limited Drug delivery compositions comprising lysophosphoglycerolipid
US5286634A (en) 1989-09-28 1994-02-15 Stadler Joan K Synergistic method for host cell transformation
US5707644A (en) 1989-11-04 1998-01-13 Danbiosyst Uk Limited Small particle compositions for intranasal drug delivery
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
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5756353A (en) 1991-12-17 1998-05-26 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol-and liposome-based delivery
DE69312487T2 (de) 1992-05-18 1997-11-27 Minnesota Mining & Mfg Einrichtung zur transmucosalen wirkstoffabgabe
US5792451A (en) 1994-03-02 1998-08-11 Emisphere Technologies, Inc. Oral drug delivery compositions and methods
US5426039A (en) 1993-09-08 1995-06-20 Bio-Rad Laboratories, Inc. Direct molecular cloning of primer extended DNA containing an alkane diol
FR2715847B1 (fr) 1994-02-08 1996-04-12 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
FR2722506B1 (fr) 1994-07-13 1996-08-14 Rhone Poulenc Rorer Sa Composition contenant des acides nucleiques, preparation et utilisations
US5908635A (en) 1994-08-05 1999-06-01 The United States Of America As Represented By The Department Of Health And Human Services Method for the liposomal delivery of nucleic acids
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
DE69527206T2 (de) 1994-09-30 2003-02-27 Inex Pharmaceuticals Corp., Vancouver Mittel zum einbringen polyanionischer materialien in zellen
US5580579A (en) 1995-02-15 1996-12-03 Nano Systems L.L.C. Site-specific adhesion within the GI tract using nanoparticles stabilized by high molecular weight, linear poly (ethylene oxide) polymers
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
DE69634084T2 (de) 1995-06-07 2005-12-08 Inex Pharmaceuticals Corp. Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5756122A (en) 1995-06-07 1998-05-26 Georgetown University Liposomally encapsulated nucleic acids having high entrapment efficiencies, method of manufacturer and use thereof for transfection of targeted cells
US5705385A (en) 1995-06-07 1998-01-06 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
IL115199A (en) 1995-09-07 2005-05-17 Opperbas Holding Bv Composition comprising a polynucleic acid molecule in a liposome and method using said composition
US6620805B1 (en) 1996-03-14 2003-09-16 Yale University Delivery of nucleic acids by porphyrins
AU2911197A (en) 1996-05-24 1998-01-05 Imperial College Of Science, Technology And Medicine Polycationic sterol derivatives as transfection agents
US6251433B1 (en) 1996-08-13 2001-06-26 Chiron Corporation Polycationic polymers
US6126965A (en) 1997-03-21 2000-10-03 Georgetown University School Of Medicine Liposomes containing oligonucleotides
US20030229040A1 (en) 1997-03-21 2003-12-11 Georgetown University Cationic liposomal delivery system and therapeutic use thereof
DE69841002D1 (de) 1997-05-14 2009-09-03 Univ British Columbia Hochwirksame verkapselung von nukleinsäuren in lipidvesikeln
ATE321882T1 (de) 1997-07-01 2006-04-15 Isis Pharmaceuticals Inc Zusammensetzungen und verfahren zur verabreichung von oligonukleotiden über die speiseröhre
US20030073640A1 (en) 1997-07-23 2003-04-17 Ribozyme Pharmaceuticals, Inc. Novel compositions for the delivery of negatively charged molecules
CA2301166A1 (en) 1997-07-24 1999-02-04 Yuan-Peng Zhang Liposomal compositions for the delivery of nucleic acid catalysts
US6110745A (en) 1997-07-24 2000-08-29 Inex Pharmaceuticals Corp. Preparation of lipid-nucleic acid particles using a solvent extraction and direct hydration method
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
US6509323B1 (en) 1998-07-01 2003-01-21 California Institute Of Technology Linear cyclodextrin copolymers
US7091192B1 (en) 1998-07-01 2006-08-15 California Institute Of Technology Linear cyclodextrin copolymers
EP1096921B1 (en) 1998-07-20 2003-04-16 Protiva Biotherapeutics Inc. Liposomal encapsulated nucleic acid-complexes
ES2366100T3 (es) 1999-02-22 2011-10-17 Georgetown University Inmunoliposomas dirigidos mediante un fragmento de anticuerpo para la administración sistémica de genes.
AU783647B2 (en) 1999-04-20 2005-11-17 University Of British Columbia, The Cationic peg-lipids and methods of use
US6852334B1 (en) 1999-04-20 2005-02-08 The University Of British Columbia Cationic peg-lipids and methods of use
US7112337B2 (en) 1999-04-23 2006-09-26 Alza Corporation Liposome composition for delivery of nucleic acid
US7098032B2 (en) 2001-01-02 2006-08-29 Mirus Bio Corporation Compositions and methods for drug delivery using pH sensitive molecules
US6200599B1 (en) 1999-10-07 2001-03-13 The Regents Of The University Of California Ortho ester lipids
US20050037086A1 (en) 1999-11-19 2005-02-17 Zycos Inc., A Delaware Corporation Continuous-flow method for preparing microparticles
CA2402613A1 (en) 2000-03-29 2001-10-04 Igor Gonda Cationic liposomes
US7189705B2 (en) 2000-04-20 2007-03-13 The University Of British Columbia Methods of enhancing SPLP-mediated transfection using endosomal membrane destabilizers
AU6815901A (en) 2000-06-02 2001-12-17 Zycos Inc Delivery systems for bioactive agents
US20030072794A1 (en) 2000-06-09 2003-04-17 Teni Boulikas Encapsulation of plasmid DNA (lipogenes™) and therapeutic agents with nuclear localization signal/fusogenic peptide conjugates into targeted liposome complexes
US7427394B2 (en) 2000-10-10 2008-09-23 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
TW593427B (en) 2000-12-18 2004-06-21 Nektar Therapeutics Al Corp Synthesis of high molecular weight non-peptidic polymer derivatives
US7053150B2 (en) 2000-12-18 2006-05-30 Nektar Therapeutics Al, Corporation Segmented polymers and their conjugates
TWI321054B (en) 2000-12-19 2010-03-01 California Inst Of Techn Compositions containing inclusion complexes
US20020192274A1 (en) 2001-03-26 2002-12-19 Ponnappa Biddanda C. pH sensitive liposomal drug delivery
JP2004525138A (ja) 2001-03-26 2004-08-19 アルザ・コーポレーシヨン 治療剤の改良された細胞内送達のためのリポソーム組成物
US20030026831A1 (en) 2001-04-20 2003-02-06 Aparna Lakkaraju Anionic liposomes for delivery of bioactive agents
WO2002087541A1 (en) 2001-04-30 2002-11-07 Protiva Biotherapeutics Inc. Lipid-based formulations for gene transfer
AU2002256398A2 (en) 2001-04-30 2002-11-11 Targeted Genetics Corporation Lipid-comprising drug delivery complexes and methods for their production
DE10127526A1 (de) 2001-05-31 2002-12-12 Novosom Ag Verfahren zur Herstellung und Auflösung von Nano- und Mikrokapseln
WO2003015698A2 (en) 2001-08-13 2003-02-27 University Of Pittsburgh Application of lipid vehicles and use for drug delivery
US7101995B2 (en) 2001-08-27 2006-09-05 Mirus Bio Corporation Compositions and processes using siRNA, amphipathic compounds and polycations
DE10152145A1 (de) 2001-10-19 2003-05-22 Novosom Ag Stabilisierung von Liposomen und Emulsionen
AU2002368202B2 (en) 2001-11-02 2008-06-05 Insert Therapeutics, Inc Methods and compositions for therapeutic use of RNA interference
DE10157046A1 (de) 2001-11-18 2003-06-12 Novosom Ag Nano- und Mikrokapseln umfassend Reaktivpolymere
AU2002359892A1 (en) 2001-12-31 2003-07-24 Elan Pharmaceuticals, Inc. Efficient nucleic acid encapsulation into medium sized liposomes
AU2003205049B2 (en) 2002-01-09 2009-05-28 Transave, Inc. Efficient nucleic acid encapsulation into medium sized liposomes
US20060051315A1 (en) 2002-02-01 2006-03-09 Scaria Puthupparampil V Polymers for delivering peptides and small molecules in vivo
DE10207177A1 (de) 2002-02-19 2003-09-04 Novosom Ag Fakultativ kationische Lipide
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
AU2003239121A1 (en) 2002-02-22 2003-09-09 Insert Therapeutics, Inc. Carbohydrate-modified polymers, compositions and uses related thereto
US7037520B2 (en) 2002-03-22 2006-05-02 Baylor College Of Medicine Reversible masking of liposomal complexes for targeted delivery
AU2003233464A1 (en) 2002-03-29 2003-10-13 Bristol-Myers Squibb Corporation Lipid mediated screening of drug candidates for identification of active compounds
EP1507561B1 (en) 2002-05-24 2009-07-15 Mirus Bio Corporation Compositions for delivering nucleic acids to cells
ATE489969T1 (de) 2002-06-20 2010-12-15 Cytos Biotechnology Ag Verpackte virusartige partikel in kombination mit cpg zur verwendung als adjuvantien mit allergenen. herstellungsverfahren und verwendung
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
US7682626B2 (en) 2003-02-07 2010-03-23 Roche Madison Inc. Polyvinylethers for delivery of polynucleotides to mammalian cells
SG190613A1 (en) 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
EP1664316B1 (en) 2003-09-15 2012-08-29 Protiva Biotherapeutics Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
JP4764426B2 (ja) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
AU2005252273B2 (en) 2004-06-07 2011-04-28 Arbutus Biopharma Corporation Lipid encapsulated interfering RNA
US7883688B2 (en) 2005-02-03 2011-02-08 Agency For Science, Technology And Research Polycationic polyrotaxanes capable of forming complexes with nucleic acids
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
WO2007086883A2 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
JP5639338B2 (ja) 2005-07-27 2014-12-10 プロチバ バイオセラピューティクス インコーポレイティッド リポソームの製造システムおよび製造方法
CA2927045A1 (en) 2006-10-03 2008-04-10 Muthiah Manoharan Lipid containing formulations
BRPI0811170B8 (pt) 2007-05-22 2021-05-25 Arcturus Therapeutics Inc oligonucleotídeos de rna e complexos de rna substituídos por hidroximetila
US20110117125A1 (en) 2008-01-02 2011-05-19 Tekmira Pharmaceuticals Corporation Compositions and methods for the delivery of nucleic acids
US8058069B2 (en) 2008-04-15 2011-11-15 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
CN101603042B (zh) * 2008-06-13 2013-05-01 厦门大学 可用于乙型肝炎病毒感染治疗的rna干扰靶点
WO2010006282A2 (en) 2008-07-10 2010-01-14 Serina Therapeutics, Inc. Polyoxazolines with inert terminating groups, polyoxazolines prepared from protected initiating groups and related compounds
US9340789B2 (en) 2008-12-03 2016-05-17 Arcturus Therapeutics, Inc. UNA oligomer structures for therapeutic agents
US20110071208A1 (en) 2009-06-05 2011-03-24 Protiva Biotherapeutics, Inc. Lipid encapsulated dicer-substrate interfering rna
EP2449114B9 (en) 2009-07-01 2017-04-19 Protiva Biotherapeutics Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
EP2563922A1 (en) 2010-04-26 2013-03-06 Marina Biotech, Inc. Nucleic acid compounds with conformationally restricted monomers and uses thereof
EP3587574B1 (en) * 2010-08-17 2022-03-16 Sirna Therapeutics, Inc. Rna interference mediated inhibition of hepatitis b virus (hbv) gene expression using short interfering nucleic acid (sina)
CN106434665B (zh) 2011-06-30 2021-06-08 箭头药业股份有限公司 用于抑制乙型肝炎病毒的基因表达的组合物和方法
WO2013159109A1 (en) 2012-04-20 2013-10-24 Isis Pharmaceuticals, Inc. Modulation of hepatitis b virus (hbv) expression

Also Published As

Publication number Publication date
EP3201338B1 (en) 2021-11-03
RU2017114964A (ru) 2018-11-07
PH12017500614A1 (en) 2017-09-25
BR112017006679A2 (pt) 2017-12-26
TW201623614A (zh) 2016-07-01
US20180016583A1 (en) 2018-01-18
IL251450A0 (en) 2017-05-29
JP2017536092A (ja) 2017-12-07
SG11201702662UA (en) 2017-04-27
CO2017004290A2 (es) 2017-09-20
EP3201338A1 (en) 2017-08-09
AU2015328012A1 (en) 2017-05-11
AU2015328012A2 (en) 2018-06-14
CN107208095B (zh) 2021-11-16
WO2016054421A1 (en) 2016-04-07
CA2963271A1 (en) 2016-04-07
CN107208095A (zh) 2017-09-26
CR20170174A (es) 2017-11-07
RU2017114964A3 (enExample) 2018-11-07
CL2017000780A1 (es) 2017-11-03
TW201900876A (zh) 2019-01-01
US10415037B2 (en) 2019-09-17

Similar Documents

Publication Publication Date Title
EP3201338B1 (en) Compositions and methods for silencing hepatitis b virus gene expression
US10626393B2 (en) Delivering CRISPR therapeutics with lipid nanoparticles
US20230212578A1 (en) Compositions and methods for treating hypertriglyceridemia
JP7086870B2 (ja) メッセンジャーrnaを送達するための組成物及び方法
US20180245074A1 (en) Treating hepatitis b virus infection using crispr
US20200297870A1 (en) Compositions and methods for delivering messenger rna
JP5766188B2 (ja) 固形腫瘍に治療剤を送達するための脂質製剤
JP5475753B2 (ja) 核酸送達用の脂質製剤
AU2009236219B8 (en) Silencing of CSN5 gene expression using interfering RNA
WO2017019891A2 (en) Compositions and methods for silencing hepatitis b virus gene expression
WO2016071857A1 (en) Compositions and methods for silencing ebola virus expression
WO2016183366A2 (en) Compositions and methods for silencing expression of hepatitis d virus rna
CA2750561A1 (en) Compositions and methods for silencing apolipoprotein c-iii expression
JP2014530602A (ja) アルデヒドデヒドロゲナーゼをサイレンシングするための組成物および方法
US9035039B2 (en) Compositions and methods for silencing SMAD4
KR20250089561A (ko) B형 간염을 치료하기 위한 치료 조성물 및 방법
US9765333B2 (en) Compositions and methods for silencing marburg virus gene expression
HK1234102B (en) Compositions and methods for silencing hepatitis b virus gene expression

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20170428

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
PN2301 Change of applicant

Patent event date: 20180703

Comment text: Notification of Change of Applicant

Patent event code: PN23011R01D

PC1203 Withdrawal of no request for examination