ES2535419T3 - Silenciamiento de expresión de quinasa tipo polo usando ARN interferente - Google Patents

Silenciamiento de expresión de quinasa tipo polo usando ARN interferente

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Publication number
ES2535419T3
ES2535419T3 ES08867007.0T ES08867007T ES2535419T3 ES 2535419 T3 ES2535419 T3 ES 2535419T3 ES 08867007 T ES08867007 T ES 08867007T ES 2535419 T3 ES2535419 T3 ES 2535419T3
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Spain
Prior art keywords
seq
lipid
mir
plk1424
nucleotides
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ES08867007.0T
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English (en)
Spanish (es)
Inventor
Ian Maclachlan
Adam Judge
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Protiva Biotherapeutics Inc
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Protiva Biotherapeutics Inc
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    • 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
    • C12N15/1137Non-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 enzymes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11021Polo kinase (2.7.11.21)
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
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    • 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
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/335Modified T or U
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    • 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
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    • 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
    • C12N2310/3525MOE, methoxyethoxy
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications

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  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Dispersion Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Dermatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
ES08867007.0T 2007-12-27 2008-12-23 Silenciamiento de expresión de quinasa tipo polo usando ARN interferente Active ES2535419T3 (es)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US17075P 1996-05-01
US45228P 1997-04-29
US1707507P 2007-12-27 2007-12-27
US4522808P 2008-04-15 2008-04-15
US10065308P 2008-09-26 2008-09-26
US100653P 2008-09-26
PCT/CA2008/002285 WO2009082817A1 (en) 2007-12-27 2008-12-23 Silencing of polo-like kinase expression using interfering rna

Publications (1)

Publication Number Publication Date
ES2535419T3 true ES2535419T3 (es) 2015-05-11

Family

ID=40823727

Family Applications (1)

Application Number Title Priority Date Filing Date
ES08867007.0T Active ES2535419T3 (es) 2007-12-27 2008-12-23 Silenciamiento de expresión de quinasa tipo polo usando ARN interferente

Country Status (7)

Country Link
US (2) US9006191B2 (https=)
EP (1) EP2238251B1 (https=)
JP (1) JP5697988B2 (https=)
AU (1) AU2008342535B2 (https=)
CA (1) CA2710713C (https=)
ES (1) ES2535419T3 (https=)
WO (1) WO2009082817A1 (https=)

Families Citing this family (153)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
AU2005222902B2 (en) 2004-03-12 2010-06-10 Alnylam Pharmaceuticals, Inc. iRNA agents targeting VEGF
US7718629B2 (en) 2006-03-31 2010-05-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of Eg5 gene
ES2874149T3 (es) 2006-05-11 2021-11-04 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen PCSK9
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
WO2009082817A1 (en) * 2007-12-27 2009-07-09 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering rna
WO2009086558A1 (en) * 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
MX2010008394A (es) * 2008-01-31 2010-11-12 Alnylam Pharmaceuticals Inc Metodos optimizados para administracion de arndc focalizando el gen pcsk9.
SG188121A1 (en) 2008-03-05 2013-03-28 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of eg5 and vegf genes
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
JP2011530289A (ja) * 2008-08-05 2011-12-22 マリーナ バイオテック,インコーポレイテッド Plk1遺伝子の発現を抑制するための核酸化合物およびその使用
CA2739170A1 (en) 2008-09-25 2010-04-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
EA036772B1 (ru) 2008-10-20 2020-12-18 Элнилэм Фармасьютикалз, Инк. Композиции и способы для ингибирования экспрессии транстиретина
HUE037082T2 (hu) * 2008-11-10 2018-08-28 Arbutus Biopharma Corp Új lipidek és készítmények terápiás hatóanyagok szállítására
EP2373382B1 (en) 2008-12-10 2017-02-15 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
EP3243504A1 (en) * 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
RU2011142620A (ru) * 2009-03-25 2013-04-27 Новартис Аг ФАРМАЦЕВТИЧЕСКАЯ КОМПОЗИЦИЯ, СОДЕРЖАЩАЯ ЛЕКАРСТВЕННОЕ СРЕДСТВО И киРНК
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
NZ597504A (en) * 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
WO2011011447A1 (en) 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression
GB0913442D0 (en) 2009-07-31 2009-09-16 Univ Ramot Cell-targeting nanoparticles comprising polynucleotide agents and uses thereof
US9029338B2 (en) 2009-08-14 2015-05-12 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
WO2011034798A1 (en) 2009-09-15 2011-03-24 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
US9187746B2 (en) 2009-09-22 2015-11-17 Alnylam Pharmaceuticals, Inc. Dual targeting siRNA agents
WO2011038160A2 (en) 2009-09-23 2011-03-31 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes expressed in cancer
US9101643B2 (en) 2009-11-03 2015-08-11 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR)
CA2816925C (en) 2009-11-04 2023-01-10 The University Of British Columbia Nucleic acid-containing lipid particles and related methods
HUE042177T2 (hu) 2009-12-01 2019-06-28 Translate Bio Inc Szteroidszármazék mRNS szállítására humán genetikai betegségekben
EP3000885B1 (en) 2009-12-18 2018-07-25 Arrowhead Pharmaceuticals, Inc. Organic compositions to treat hsf1-related diseases
WO2011076807A2 (en) * 2009-12-23 2011-06-30 Novartis Ag Lipids, lipid compositions, and methods of using them
JP2011155914A (ja) * 2010-02-01 2011-08-18 Osaka Univ 脂質異常症治療薬剤としての化学修飾siRNA
CA2792561C (en) 2010-04-06 2021-10-26 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of cd274/pd-l1 gene
CA3098080A1 (en) 2010-04-23 2011-10-27 Arrowhead Research Corporation Organic compositions to treat beta-enac-related diseases
US20130190383A1 (en) * 2010-04-26 2013-07-25 Marina Biotech, Inc. Nucleic acid compounds with conformationally restricted monomers and uses thereof
WO2011139911A2 (en) * 2010-04-29 2011-11-10 Isis Pharmaceuticals, Inc. Lipid formulated single stranded rna
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
WO2011141705A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
CA2801066C (en) 2010-06-02 2021-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
CN103189511B (zh) 2010-07-12 2016-10-12 国立大学法人鸟取大学 利用siRNA导入的新型hiPSC制作法
US20120039983A1 (en) * 2010-08-11 2012-02-16 Uhrich Kathryn E Amphiphilic macromolecule-lipid complexes
KR101223484B1 (ko) * 2010-10-05 2013-01-17 한국과학기술연구원 사람 혈청 알부민-siRNA 나노입자 전달체
WO2012048265A2 (en) * 2010-10-08 2012-04-12 The Broad Institute Of Mit And Harvard Methods of treating inflammation
AR083445A1 (es) 2010-10-14 2013-02-27 Univ Mie siARN CONTRA LA FIBROSIS
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
EP2648763A4 (en) 2010-12-10 2014-05-14 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR EXPRESSION INHIBITION OF GENES KLF-1 AND BCL11A
EP2649182A4 (en) 2010-12-10 2015-05-06 Alnylam Pharmaceuticals Inc Compositions and methods for increasing erythropoietin (epo) production
KR102365961B1 (ko) 2011-03-29 2022-02-23 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
PL3586861T3 (pl) 2011-06-08 2022-05-23 Translate Bio, Inc. Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna
US20140275211A1 (en) 2011-06-21 2014-09-18 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
ES2923573T3 (es) 2011-06-21 2022-09-28 Alnylam Pharmaceuticals Inc Composiciones de ARNi de proteína 3 de tipo angiopoyetina (ANGPTL3) y métodos de uso de las mismas
EP3366312A1 (en) 2011-06-23 2018-08-29 Alnylam Pharmaceuticals, Inc. Serpina 1 sirnas: compositions of matter and methods of treatment
JP2014526887A (ja) 2011-08-01 2014-10-09 アルナイラム ファーマシューティカルズ, インコーポレイテッド 造血幹細胞移植の成功率を改善する方法
JP2014525749A (ja) * 2011-08-12 2014-10-02 ヴェストファーレン ヴィルヘルム−ウニヴェルジテート ミュンスター 炎症性腸疾患の治療のための新規化合物
US9126966B2 (en) 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
CN103930547A (zh) 2011-09-02 2014-07-16 诺华股份有限公司 用于治疗hsf1相关疾病的有机组合物
WO2013056670A1 (zh) 2011-10-19 2013-04-25 苏州瑞博生物技术有限公司 小干扰RNA及其应用和抑制plk1基因表达的方法
HUE048622T2 (hu) 2011-11-18 2020-08-28 Alnylam Pharmaceuticals Inc RNSi szerek, készítmények és alkalmazási eljárások transztiretin (TTR) asszociált betegségek kezelésére
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
ITRM20110685A1 (it) 2011-12-23 2013-06-24 Internat Ct For Genetic En Gineering And Microrna per la rigenerazione cardiaca attraverso l induzione della proliferazione dei miociti cardiaci
CA2860676A1 (en) 2012-01-09 2013-07-18 Novartis Ag Organic compositions to treat beta-catenin-related diseases
SG11201405157PA (en) 2012-02-24 2014-10-30 Protiva Biotherapeutics Inc Trialkyl cationic lipids and methods of use thereof
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
EP2839015B1 (en) 2012-04-18 2020-04-29 Ramot at Tel-Aviv University Ltd. Lipidated glycosaminoglycan particles for the delivery of nucleic acids
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
EP3272868A1 (en) 2012-05-02 2018-01-24 Arrowhead Pharmaceuticals, Inc. Organic compositions to treat kras-related diseases
US10245229B2 (en) 2012-06-08 2019-04-02 Translate Bio, Inc. Pulmonary delivery of mRNA to non-lung target cells
EP3536787A1 (en) 2012-06-08 2019-09-11 Translate Bio, Inc. Nuclease resistant polynucleotides and uses thereof
US10125369B2 (en) 2012-12-05 2018-11-13 Alnylam Pharmaceuticals, Inc. PCSK9 iRNA compositions and methods of use thereof
WO2014130922A1 (en) 2013-02-25 2014-08-28 Trustees Of Boston University Compositions and methods for treating fungal infections
DE102013003869B4 (de) * 2013-02-27 2016-11-24 Friedrich-Schiller-Universität Jena Verfahren zur gezielten Abtötung von Zellen durch zur mRNA-Anbindung ausgerichtete Nukleotid-Moleküle sowie Nukleotid-Moleküle und Applikationskit für solche Verwendung
SG10201706960TA (en) 2013-02-28 2017-10-30 Arrowhead Res Corp Organic compositions to treat epas1-related diseases
PL3467108T3 (pl) 2013-03-14 2024-09-30 Translate Bio, Inc. Sposoby oczyszczania informacyjnego rna
KR20210122917A (ko) 2013-03-14 2021-10-12 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
DK2970456T3 (da) 2013-03-14 2021-07-05 Translate Bio Inc Fremgangsmåder og sammensætninger til levering af mrna-kodede antistoffer
US10130649B2 (en) 2013-03-15 2018-11-20 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
WO2014182661A2 (en) 2013-05-06 2014-11-13 Alnylam Pharmaceuticals, Inc Dosages and methods for delivering lipid formulated nucleic acid molecules
US10077444B2 (en) 2013-10-02 2018-09-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
TWI768330B (zh) 2013-10-04 2022-06-21 美國西奈山伊坎醫學院 抑制alas1基因表現的組合物及方法
WO2015051135A2 (en) 2013-10-04 2015-04-09 Novartis Ag Organic compositions to treat hepcidin-related diseases
AU2014340149B2 (en) 2013-10-22 2020-12-24 Shire Human Genetic Therapies, Inc. CNS delivery of mRNA and uses thereof
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
MX2016005238A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Formulaciones de lipidos para la administracion de acido ribonucleico mensajero.
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
PT3134506T (pt) 2014-04-25 2019-10-31 Translate Bio Inc Métodos de purificação de rna mensageiro
JP6557722B2 (ja) 2014-05-30 2019-08-07 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 核酸の送達のための生分解性脂質
EP3160959B1 (en) 2014-06-24 2023-08-30 Translate Bio, Inc. Stereochemically enriched compositions for delivery of nucleic acids
US10583084B2 (en) 2014-06-26 2020-03-10 Ramot At Tel-Aviv University Ltd. Liposomal formulations for delivery of nucleic acids
BR112016030852A2 (pt) 2014-07-02 2018-01-16 Shire Human Genetic Therapies encapsulação de rna mensageiro
EP3169309B1 (en) 2014-07-16 2023-05-10 Novartis AG Method of encapsulating a nucleic acid in a lipid nanoparticle host
EP3736334A1 (en) 2014-07-16 2020-11-11 Arrowhead Pharmaceuticals, Inc. Rnai compositions to treat apoc3-related diseases
US9765333B2 (en) 2014-08-21 2017-09-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing marburg virus gene expression
DK3185957T3 (da) 2014-08-29 2022-08-29 Alnylam Pharmaceuticals Inc Patisiran til anvendelse ved behandlingen af transthyretin-medieret amyloidosis
EP3191592A1 (en) 2014-09-11 2017-07-19 Novartis AG Inhibition of prmt5 to treat mtap-deficiency-related diseases
EP3227446A1 (en) 2014-12-01 2017-10-11 Novartis AG Compositions and methods for diagnosis and treatment of prostate cancer
WO2016090262A1 (en) 2014-12-05 2016-06-09 Shire Human Genetic Therapies, Inc. Messenger rna therapy for treatment of articular disease
CA2970795A1 (en) 2014-12-18 2016-06-23 Alnylam Pharmaceuticals, Inc. Reversir compounds
US10172924B2 (en) 2015-03-19 2019-01-08 Translate Bio, Inc. MRNA therapy for pompe disease
GB201506509D0 (en) 2015-04-16 2015-06-03 Univ Wageningen Nuclease-mediated genome editing
EP3302525A2 (en) 2015-06-05 2018-04-11 Novartis AG Methods and compositions for diagnosing, treating, and monitoring treatment of shank3 deficiency associated disorders
US10648020B2 (en) 2015-06-18 2020-05-12 The Broad Institute, Inc. CRISPR enzymes and systems
CN108138182B (zh) 2015-07-31 2022-08-19 阿尔尼拉姆医药品有限公司 甲状腺素运载蛋白(TTR)iRNA组合物及其治疗或预防TTR相关疾病的使用方法
CN114686548A (zh) 2015-10-14 2022-07-01 川斯勒佰尔公司 用于增强生产的rna相关酶的修饰
WO2017177169A1 (en) 2016-04-08 2017-10-12 Rana Therapeutics, Inc. Multimeric coding nucleic acid and uses thereof
EP3445853A1 (en) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
CA3027312A1 (en) 2016-06-13 2017-12-21 Translate Bio, Inc. Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency
CN109563511A (zh) * 2016-06-30 2019-04-02 阿布特斯生物制药公司 用于递送信使rna的组合物和方法
WO2018047148A1 (en) 2016-09-12 2018-03-15 Novartis Ag Compounds for the inhibition of mirna
US20180245065A1 (en) 2016-11-01 2018-08-30 Novartis Ag Methods and compositions for enhancing gene editing
CN108283624B (zh) * 2017-01-10 2021-01-12 北京化工大学 一种索拉非尼纳米脂质体制剂及其制备方法
EA201991747A1 (ru) 2017-02-27 2020-06-04 Транслейт Био, Инк. НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR
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.
WO2018222926A1 (en) 2017-05-31 2018-12-06 Ultragenyx Pharmaceutical Inc. Therapeutics for glycogen storage disease type iii
CN107320484B (zh) * 2017-06-08 2022-05-17 中国人民解放军第四军医大学第一附属医院 mir-452-3p在制备治疗肝癌的药物组合物中的用途
US11530413B2 (en) 2017-07-21 2022-12-20 Novartis Ag Compositions and methods to treat cancer
US20200375912A1 (en) * 2017-08-03 2020-12-03 Rita Elena Serda Liposomal coated nanoparticles for immunotherapy applications
US10597657B2 (en) 2017-09-11 2020-03-24 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3)
KR20250046363A (ko) 2017-09-19 2025-04-02 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증을 치료하기 위한 조성물 및 방법
WO2019094578A1 (en) 2017-11-09 2019-05-16 Alnylam Pharmaceuticals Inc. Assays and methods for determining expression of the lect2 gene
EP3727428A1 (en) 2017-12-20 2020-10-28 Translate Bio, Inc. Improved composition and methods for treatment of ornithine transcarbamylase deficiency
WO2019150309A1 (en) 2018-02-02 2019-08-08 Hammack Scott Modulators of gpr68 and uses thereof for treating and preventing diseases
EP3788138A1 (en) 2018-05-02 2021-03-10 Novartis AG Regulators of human pluripotent stem cells and uses thereof
US11987792B2 (en) 2018-08-16 2024-05-21 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
WO2020068798A1 (en) 2018-09-24 2020-04-02 Guo Jimin Living mammalian cells modified with functional modular nanoparticles
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
KR20210105889A (ko) 2018-11-09 2021-08-27 아뷰터스 바이오파마 코포레이션 지질 나노입자 제제
MX2021005969A (es) 2018-11-21 2021-09-14 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr nebulizado.
JP7445657B2 (ja) 2018-12-06 2024-03-07 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 オルニチントランスカルバミラーゼ欠損症を処置するための組成物および方法
MY206794A (en) 2018-12-20 2025-01-08 Humabs Biomed Sa Combination hbv therapy
SG11202105577TA (en) 2019-01-09 2021-06-29 Arrowhead Pharmaceuticals Inc Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use
US12208164B2 (en) 2019-02-28 2025-01-28 Unm Rainforest Innovations Modular metal-organic polyhedra superassembly compositions
AU2020343255A1 (en) 2019-09-03 2022-03-24 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
EP4038189A1 (en) 2019-10-04 2022-08-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
WO2021087325A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
MX2022009763A (es) 2020-02-10 2022-09-09 Alnylam Pharmaceuticals Inc Composiciones y metodos para silenciar la expresion del factor de crecimiento endotelial vascular a (vegf-a).
KR102444740B1 (ko) * 2020-02-25 2022-09-19 한국과학기술연구원 Ksp 억제제와 유사분열 억제제를 포함하는 암의 예방 또는 치료용 약학 조성물
JP7832114B2 (ja) 2020-03-03 2026-03-17 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 オルニチントランスカルバミラーゼ欠損症の治療のための組成物及び方法
EP4127171A2 (en) 2020-03-30 2023-02-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
EP4133078A1 (en) 2020-04-06 2023-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
CA3179678A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
CA3182458A1 (en) 2020-06-24 2021-12-30 Laura ROSEN Engineered hepatitis b virus neutralizing antibodies and uses thereof
IL303195A (en) 2020-11-25 2023-07-01 Akagera Medicines Inc Lipid nanoparticles for delivery of nucleic acids and related methods of use
CA3227852A1 (en) 2021-08-03 2023-02-09 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
WO2023013719A1 (ja) * 2021-08-06 2023-02-09 東レ株式会社 癌の治療及び/又は予防用医薬組成物
WO2023034837A2 (en) 2021-08-31 2023-03-09 Alnylam Pharmaceuticals, Inc. Cell death-inducing dffa-like effector b (cideb) irna compositions and methods of use thereof
EP4531819A2 (en) 2022-05-25 2025-04-09 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
WO2023229535A1 (en) 2022-05-27 2023-11-30 Engine Biosciences Pte. Ltd. Bet polypeptide therapeutic agents for generating synthetic lethality in tumors
WO2024054950A1 (en) * 2022-09-09 2024-03-14 Cardiff Oncology, Inc. Cancer treatment using mtdp inhibitors and plk1 inhibitors
CN115737557B (zh) * 2022-11-18 2024-04-09 黑龙江中医药大学 一种miR-55制剂及其制备方法和应用
WO2024158825A2 (en) 2023-01-23 2024-08-02 Engine Biosciences Pte. Ltd. Methods to modulate tau for treatment of tauopathies
EP4662311A2 (en) 2023-02-09 2025-12-17 Alnylam Pharmaceuticals, Inc. Reversir molecules and methods of use thereof

Family Cites Families (162)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL154598B (nl) 1970-11-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van laagmoleculire verbindingen en van eiwitten die deze verbindingen specifiek kunnen binden, alsmede testverpakking.
US3901654A (en) 1971-06-21 1975-08-26 Biological Developments Receptor assays of biologically active compounds employing biologically specific receptors
US3817827A (en) 1972-03-30 1974-06-18 Scott Paper Co Soft absorbent fibrous webs containing elastomeric bonding material and formed by creping and embossing
US3935074A (en) 1973-12-17 1976-01-27 Syva Company Antibody steric hindrance immunoassay with two antibodies
US3996345A (en) 1974-08-12 1976-12-07 Syva Company Fluorescence quenching with immunological pairs in immunoassays
US4034074A (en) 1974-09-19 1977-07-05 The Board Of Trustees Of Leland Stanford Junior University Universal reagent 2-site immunoradiometric assay using labelled anti (IgG)
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
US3984533A (en) 1975-11-13 1976-10-05 General Electric Company Electrophoretic method of detecting antigen-antibody reaction
US4086257A (en) 1976-10-12 1978-04-25 Sears Barry D Phosphatidyl quaternary ammonium compounds
US4098876A (en) 1976-10-26 1978-07-04 Corning Glass Works Reverse sandwich immunoassay
CH624011A5 (https=) 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
US4452901A (en) 1980-03-20 1984-06-05 Ciba-Geigy Corporation Electrophoretically transferring electropherograms to nitrocellulose sheets for immuno-assays
US4376110A (en) 1980-08-04 1983-03-08 Hybritech, Incorporated Immunometric assays using monoclonal antibodies
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
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying 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
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
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
US6465188B1 (en) * 1990-06-11 2002-10-15 Gilead Sciences, Inc. Nucleic acid ligand complexes
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US6831166B2 (en) 1992-10-23 2004-12-14 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
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
ES2105262T3 (es) 1992-05-18 1997-10-16 Minnesota Mining & Mfg Dispositivo de suministro de farmaco a traves de las mucosas.
US5792451A (en) 1994-03-02 1998-08-11 Emisphere Technologies, Inc. Oral drug delivery compositions and methods
US5334761A (en) * 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
WO1995002698A1 (en) * 1993-07-12 1995-01-26 Life Technologies, Inc. Composition and methods for transfecting eukaryotic cells
US5426039A (en) 1993-09-08 1995-06-20 Bio-Rad Laboratories, Inc. Direct molecular cloning of primer extended DNA containing an alkane diol
US5674908A (en) * 1993-12-20 1997-10-07 Life Technologies, Inc. Highly packed polycationic ammonium, sulfonium and phosphonium lipids
FR2715847B1 (fr) 1994-02-08 1996-04-12 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
US6075012A (en) * 1994-02-11 2000-06-13 Life Technologies, Inc. Reagents for intracellular delivery of macromolecules
US6989434B1 (en) * 1994-02-11 2006-01-24 Invitrogen Corporation Reagents for intracellular delivery of macromolecules
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
ATE219660T1 (de) 1994-09-30 2002-07-15 Inex Pharmaceuticals Corp Mittel zum einbringen polyanionischer materialien in zellen
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5627159A (en) * 1994-10-27 1997-05-06 Life Technologies, Inc. Enhancement of lipid cationic transfections in the presence of serum
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
US6051429A (en) * 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
US5958901A (en) * 1995-06-07 1999-09-28 Genta Incorporated Phosphonic acid-based cationic lipids
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5736392A (en) * 1995-06-07 1998-04-07 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
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
EP0830368A1 (en) * 1995-06-07 1998-03-25 Genta Incorporated Novel carbamate-based cationic lipids
US20030069173A1 (en) * 1998-03-16 2003-04-10 Life Technologies, Inc. Peptide-enhanced transfections
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
ATE285477T1 (de) 1995-06-07 2005-01-15 Inex Pharmaceutical Corp Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer
US6339173B1 (en) * 1996-07-22 2002-01-15 Promega Biosciences, Inc. Amide-based cationic lipids
EP0869937A4 (en) * 1995-07-21 2004-07-21 Promega Biosciences Inc AMID BASED CATIONIC LIPIDS
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
US6330349B1 (en) * 1995-11-30 2001-12-11 Chromavision Medical Systems, Inc. Automated method for image analysis of residual protein
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6620805B1 (en) 1996-03-14 2003-09-16 Yale University Delivery of nucleic acids by porphyrins
US6756054B1 (en) 1996-05-24 2004-06-29 Ic-Vec Limited Polycationic sterol derivatives as transfection agents
US20050119470A1 (en) 1996-06-06 2005-06-02 Muthiah Manoharan Conjugated oligomeric compounds and their use in gene modulation
JP4320054B2 (ja) 1996-08-13 2009-08-26 ノバルティス バクシンズ アンド ダイアグノスティックス,インコーポレーテッド ポリヌクレオチド送達のための組成物および方法
US6111085A (en) 1996-09-13 2000-08-29 Isis Pharmaceuticals, Inc. Carbamate-derivatized nucleosides and oligonucleosides
US5877220A (en) * 1997-03-06 1999-03-02 Genta, Incorporated Amide-based oligomeric cationic lipids
US6034135A (en) * 1997-03-06 2000-03-07 Promega Biosciences, Inc. Dimeric cationic lipids
US20030229040A1 (en) 1997-03-21 2003-12-11 Georgetown University Cationic liposomal delivery system and therapeutic use thereof
US6126965A (en) 1997-03-21 2000-10-03 Georgetown University School Of Medicine Liposomes containing oligonucleotides
CA2289702C (en) 1997-05-14 2008-02-19 Inex Pharmaceuticals Corp. High efficiency encapsulation of charged therapeutic agents in lipid vesicles
DE69834038D1 (de) 1997-07-01 2006-05-18 Isis Pharmaceutical 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
US7091192B1 (en) 1998-07-01 2006-08-15 California Institute Of Technology Linear cyclodextrin copolymers
US6509323B1 (en) 1998-07-01 2003-01-21 California Institute Of Technology Linear cyclodextrin copolymers
WO2000003683A2 (en) 1998-07-20 2000-01-27 Inex Pharmaceuticals Corporation Liposomal encapsulated nucleic acid-complexes
DK1129064T3 (da) * 1998-11-12 2008-04-28 Invitrogen Corp Transfektionsreagenser
ATE511543T1 (de) 1999-02-22 2011-06-15 Univ Georgetown Immunoliposome mit einem targeting- antikörperfragment zur systemischen genverabreichung
US6852334B1 (en) 1999-04-20 2005-02-08 The University Of British Columbia Cationic peg-lipids and methods of use
WO2000062813A2 (en) 1999-04-20 2000-10-26 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
US6583471B1 (en) * 1999-06-02 2003-06-24 Semiconductor Energy Laboratory Co., Ltd. Semiconductor device having first and second insulating films
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
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
WO2002081628A2 (en) * 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
JP2003528131A (ja) 2000-03-29 2003-09-24 アラダイム コーポレーション カチオン性リポソーム
US7189705B2 (en) 2000-04-20 2007-03-13 The University Of British Columbia Methods of enhancing SPLP-mediated transfection using endosomal membrane destabilizers
US20040142475A1 (en) 2000-06-02 2004-07-22 Barman Shikha P. 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
AU2002232387A1 (en) * 2000-10-27 2002-05-06 Invitrogen Corporation Method for introducing antisense oligonucleotides into eucaryotic cells
US7053150B2 (en) 2000-12-18 2006-05-30 Nektar Therapeutics Al, Corporation Segmented polymers and their conjugates
TW593427B (en) 2000-12-18 2004-06-21 Nektar Therapeutics Al Corp Synthesis of high molecular weight non-peptidic polymer derivatives
TWI321054B (en) 2000-12-19 2010-03-01 California Inst Of Techn Compositions containing inclusion complexes
US7108863B2 (en) 2001-03-26 2006-09-19 Alza Corporation Liposome composition for improved intracellular delivery of a therapeutic agent
WO2002076427A2 (en) 2001-03-26 2002-10-03 Thomas Jefferson University Ph sensitive liposomal drug delivery
US20030026831A1 (en) 2001-04-20 2003-02-06 Aparna Lakkaraju Anionic liposomes for delivery of bioactive agents
US20030077829A1 (en) 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
JP2004535388A (ja) 2001-04-30 2004-11-25 ターゲティッド ジェネティクス コーポレイション 脂質含有薬物送達複合体およびそれらの生成方法
US20050282188A1 (en) * 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US20030130186A1 (en) 2001-07-20 2003-07-10 Chandra Vargeese Conjugates and compositions for cellular delivery
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
DE10127526A1 (de) 2001-05-31 2002-12-12 Novosom Ag Verfahren zur Herstellung und Auflösung von Nano- und Mikrokapseln
US7063860B2 (en) 2001-08-13 2006-06-20 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
EP1575976A4 (en) 2001-11-02 2006-08-23 Insert Therapeutics Inc METHODS AND COMPOSITIONS FOR THE 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
CA2472462A1 (en) 2002-01-09 2003-07-24 Elan Pharmaceuticals, Inc. Efficient nucleic acid encapsulation into medium sized liposomes
EP1480657A4 (en) 2002-02-01 2006-07-05 Intradigm Corp POLYMERS FOR ADMINISTERING PEPTIDES AND SMALL MOLECULES IN VIVO / I
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
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
WO2003070283A2 (en) * 2002-02-22 2003-08-28 Klaus Strebhardt Agent for inhibiting development or progress of proliferative diseases and especially cancer diseases and pharmaceutical composition containing said agent
EP1476492A1 (en) 2002-02-22 2004-11-17 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
WO2003083443A2 (en) 2002-03-29 2003-10-09 University Of Florida Lipid mediated screening of drug candidates for identification of active compounds
ES2328031T3 (es) 2002-05-24 2009-11-06 F. Hoffmann-La Roche Ag Composiciones para distribuir acidos nucleicos a celulas.
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
US6906186B1 (en) 2002-07-30 2005-06-14 Isis Pharmaceuticals, Inc. Antisense modulation of polo-like kinase expression
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US7682626B2 (en) 2003-02-07 2010-03-23 Roche Madison Inc. Polyvinylethers for delivery of polynucleotides to mammalian cells
US20040167090A1 (en) 2003-02-21 2004-08-26 Monahan Sean D. Covalent modification of RNA for in vitro and in vivo delivery
AU2004227414A1 (en) 2003-04-03 2004-10-21 Alnylam Pharmaceuticals iRNA conjugates
DK1620544T3 (en) 2003-04-17 2019-01-14 Alnylam Pharmaceuticals Inc MODIFIED iRNA AGENTS
ES2559828T3 (es) * 2003-07-16 2016-02-16 Protiva Biotherapeutics Inc. ARN de interferencia encapsulado en lípidos
NZ592917A (en) 2003-09-15 2012-12-21 Protiva Biotherapeutics Inc Stable polyethyleneglycol (PEG) dialkyloxypropyl (DAA) lipid conjugates
CN100368423C (zh) * 2003-11-21 2008-02-13 杭州新瑞佳生物医药技术开发有限公司 有效杀伤肿瘤细胞的针对PLK1 mRNA的小干扰核糖核酸分子(SiRNA)、其混合物及用途
US7799565B2 (en) * 2004-06-07 2010-09-21 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
US7745651B2 (en) 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
WO2006002538A1 (en) * 2004-07-02 2006-01-12 Protiva Biotherapeutics, Inc. Immunostimulatory sirna molecules and uses therefor
JPWO2006035515A1 (ja) 2004-09-28 2008-05-15 平 前川 膀胱表在性癌の治療又は予防用医薬組成物、及びその利用
CA2587411A1 (en) * 2004-11-17 2006-05-26 Protiva Biotherapeutics, Inc. Sirna silencing of apolipoprotein b
SG124419A1 (en) 2005-02-03 2006-08-30 Agency Science Tech & Res Polycations 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
JP5042863B2 (ja) 2005-02-14 2012-10-03 サーナ・セラピューティクス・インコーポレイテッド 生物学的に活性な分子をデリバリーするための脂質ナノ粒子系組成物および方法
WO2006102163A2 (en) * 2005-03-17 2006-09-28 Invitrogen Corporation Transfection reagents for non-adherent suspension cells
US9005654B2 (en) 2005-07-27 2015-04-14 Protiva Biotherapeutics, Inc. Systems and methods for manufacturing liposomes
US20070054873A1 (en) * 2005-08-26 2007-03-08 Protiva Biotherapeutics, Inc. Glucocorticoid modulation of nucleic acid-mediated immune stimulation
WO2007048046A2 (en) * 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. Sirna silencing of filovirus gene expression
US8101741B2 (en) * 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
US7915399B2 (en) * 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
US20080311040A1 (en) * 2007-03-06 2008-12-18 Flagship Ventures METHODS AND COMPOSITIONS FOR IMPROVED THERAPEUTIC EFFECTS WITH siRNA
EP2848688A1 (en) * 2007-12-10 2015-03-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor VII gene
WO2009082817A1 (en) * 2007-12-27 2009-07-09 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering rna
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
AU2009238175C1 (en) * 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
CN104119242B (zh) 2008-10-09 2017-07-07 泰米拉制药公司 改善的氨基脂质和递送核酸的方法
ES2613498T3 (es) * 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
US20120136073A1 (en) * 2010-11-15 2012-05-31 Life Technologies Corporation Amine-Containing Transfection Reagents and methods for making and using same

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AU2008342535B2 (en) 2015-02-05
US20090291131A1 (en) 2009-11-26
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