HK1136848A1 - Compositions for enhancing delivery of double-stranded rna to regulate gene expression in mammalian cells rna - Google Patents

Compositions for enhancing delivery of double-stranded rna to regulate gene expression in mammalian cells rna

Info

Publication number
HK1136848A1
HK1136848A1 HK10104471.8A HK10104471A HK1136848A1 HK 1136848 A1 HK1136848 A1 HK 1136848A1 HK 10104471 A HK10104471 A HK 10104471A HK 1136848 A1 HK1136848 A1 HK 1136848A1
Authority
HK
Hong Kong
Prior art keywords
rna
compositions
double
gene expression
mammalian cells
Prior art date
Application number
HK10104471.8A
Other languages
English (en)
Chinese (zh)
Inventor
Mohammad Ahmadian
Kunyuan Cui
Lishan Chen
Shu-Chih Chen Quay
Michael E Houston Jr
Original Assignee
Mdrna Inc
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 Mdrna Inc filed Critical Mdrna Inc
Publication of HK1136848A1 publication Critical patent/HK1136848A1/xx

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/554—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • C12N15/1137—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 enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01023—Beta-galactosidase (3.2.1.23), i.e. exo-(1-->4)-beta-D-galactanase

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Botany (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
HK10104471.8A 2004-04-20 2010-05-07 Compositions for enhancing delivery of double-stranded rna to regulate gene expression in mammalian cells rna HK1136848A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US56454304P 2004-04-20 2004-04-20

Publications (1)

Publication Number Publication Date
HK1136848A1 true HK1136848A1 (en) 2010-07-09

Family

ID=35907831

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Application Number Title Priority Date Filing Date
HK10104471.8A HK1136848A1 (en) 2004-04-20 2010-05-07 Compositions for enhancing delivery of double-stranded rna to regulate gene expression in mammalian cells rna

Country Status (7)

Country Link
US (5) US20060014289A1 (xx)
EP (2) EP1773998A2 (xx)
JP (1) JP4635046B2 (xx)
CA (1) CA2564616C (xx)
HK (1) HK1136848A1 (xx)
MX (1) MXPA06012076A (xx)
WO (1) WO2006019430A2 (xx)

Families Citing this family (75)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2564616C (en) * 2004-04-20 2016-08-30 Nastech Pharmaceutical Company Inc. Methods and compositions for enhancing delivery of double-stranded rna or a double-stranded hybrid nucleic acid to regulate gene expression in mammalian cells
US20060035815A1 (en) * 2004-05-04 2006-02-16 Nastech Pharmaceutical Company Inc. Pharmaceutical compositions for delivery of ribonucleic acid to a cell
US20060040882A1 (en) * 2004-05-04 2006-02-23 Lishan Chen Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
US20070213257A1 (en) * 2005-08-12 2007-09-13 Nastech Pharmaceutical Company Inc. Compositions and methods for complexes of nucleic acids and peptides
EP1986697B1 (en) * 2006-02-17 2016-06-29 GE Healthcare Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
US20070281900A1 (en) * 2006-05-05 2007-12-06 Nastech Pharmaceutical Company Inc. COMPOSITIONS AND METHODS FOR LIPID AND POLYPEPTIDE BASED siRNA INTRACELLULAR DELIVERY
WO2008022046A2 (en) * 2006-08-18 2008-02-21 Nastech Pharmaceutical Company Inc. Dicer substrate rna peptide conjugates and methods for rna therapeutics
NZ575090A (en) 2006-09-06 2012-02-24 Univ California Molecular diagnosis and classification of malignant melanoma using RGS1, ARPC2, FN1, SPP1 and WNT-2 polypeptide markers
JP5352462B2 (ja) * 2006-09-22 2013-11-27 ダーマコン， インコーポレイテッド 二本鎖オリゴヌクレオチド複合体、ｒｎａ干渉による遺伝子サイレンシング方法、および医薬品組成物
DE102007008596B4 (de) 2007-02-15 2010-09-02 Friedrich-Schiller-Universität Jena Biologisch wirksame Moleküle auf Grundlage von PNA und siRNA, Verfahren zu deren zellspezifischen Aktivierung sowie Applikationskit zur Verabreichung
EP2522755B1 (en) 2007-08-13 2014-04-02 Baxter International Inc. IVIG modulation of chemokines for treatment of Multiple Sclerosis, Alzheimer's disease, and Parkinson's disease
EP2212693B1 (en) 2007-10-22 2015-04-22 The Regents of The University of California Biomarkers for prenatal diagnosis of congenital cytomegalovirus
WO2009102427A2 (en) 2008-02-11 2009-08-20 Rxi Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
DE102009043743B4 (de) 2009-03-13 2016-10-13 Friedrich-Schiller-Universität Jena Zellspezifisch wirksame Moleküle auf Grundlage von siRNA sowie Applikationskits zu deren Herstellung und Verwendung
EP2257810B1 (en) 2008-03-05 2013-06-05 The Regents of the University of California Molecular diagnosis and classification of malignant melanoma
KR20100131509A (ko) * 2008-03-31 2010-12-15 내셔날 인스티튜트 오브 어드밴스드 인더스트리얼 사이언스 앤드 테크놀로지 Ｒｎａ 간섭 효과가 높은 2본쇄 지질 수식 ｒｎａ
WO2010008582A2 (en) 2008-07-18 2010-01-21 Rxi Pharmaceuticals Corporation Phagocytic cell drug delivery system
WO2010021718A1 (en) 2008-08-19 2010-02-25 Nektar Therapeutics Complexes of small-interfering nucleic acids
AU2009293658A1 (en) 2008-09-22 2010-03-25 James Cardia Reduced size self-delivering RNAi compounds
EP3495488A1 (en) * 2008-10-27 2019-06-12 Baxalta GmbH Models of thrombotic thrombocytopenic purpura and methods of use thereof
WO2010053548A2 (en) * 2008-11-04 2010-05-14 Anchor Therapeutics, Inc. Pthr1 receptor compounds
EP3238738B1 (en) * 2008-11-10 2020-09-23 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
CA2748520C (en) * 2008-12-26 2013-12-17 Se-Ho Kim Pharmaceutical composition containing an anionic drug, and a production method therefor
WO2010078536A1 (en) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
CN102439151A (zh) 2009-03-19 2012-05-02 默沙东公司 使用短干扰核酸（siNA）的RNA干扰介导的BTB和CNC同系物1，碱性亮氨酸拉链转录因子1（Bach1）基因表达的抑制
EP2408915A2 (en) 2009-03-19 2012-01-25 Merck Sharp&Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GATA BINDING PROTEIN 3 (GATA3) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US20120016011A1 (en) 2009-03-19 2012-01-19 Merck Sharp & Dohme Corp. RNA Interference Mediated Inhibition of Connective Tissue Growth Factor (CTGF) Gene Expression Using Short Interfering Nucleic Acid (siNA)
US20120035247A1 (en) 2009-03-19 2012-02-09 Merck Sharp & Dohme Corp. RNA Interference Mediated Inhibition of Signal Transducer and Activator of Transcription 6 (STAT6) Gene Expression Using Short Interfering Nucleic Acid (siNA)
JP2012521764A (ja) 2009-03-27 2012-09-20 メルク・シャープ・エンド・ドーム・コーポレイション 低分子干渉核酸（ｓｉＮＡ）を用いた胸腺間質性リンパ球新生因子（ＴＳＬＰ）遺伝子発現のＲＮＡ干渉媒介性阻害
WO2010111464A1 (en) 2009-03-27 2010-09-30 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF APOPTOSIS SIGNAL-REGULATING KINASE 1 (ASK1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2010111468A2 (en) 2009-03-27 2010-09-30 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF THE NERVE GROWTH FACTOR BETA CHAIN (NGFß) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
EP2411019A2 (en) 2009-03-27 2012-02-01 Merck Sharp&Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF SIGNAL TRANSDUCER AND ACTIVATOR OF TRANSCRIPTION 1 (STAT1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
SG174581A1 (en) 2009-03-27 2011-10-28 Merck Sharp & Dohme RNA INTERFERENCE MEDIATED INHIBITION OF THE INTERCELLULAR ADHESION MOLECULE 1 (ICAM-1)GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2011035065A1 (en) 2009-09-17 2011-03-24 Nektar Therapeutics Monoconjugated chitosans as delivery agents for small interfering nucleic acids
WO2011094759A2 (en) 2010-02-01 2011-08-04 The Regents Of The University Of California Novel diagnostic and therapeutic targets associated with or regulated by n-cadherin expression and/or epithelial to mesenchymal transition (emt) in prostate cancer and other malignancies
EP2550001B1 (en) 2010-03-24 2019-05-22 Phio Pharmaceuticals Corp. Rna interference in ocular indications
EP3560503B1 (en) 2010-03-24 2021-11-17 Phio Pharmaceuticals Corp. Rna interference in dermal and fibrotic indications
WO2011119852A1 (en) 2010-03-24 2011-09-29 Rxi Pharmaceuticals Corporation Reduced size self-delivering rnai compounds
WO2011120023A1 (en) 2010-03-26 2011-09-29 Marina Biotech, Inc. Nucleic acid compounds for inhibiting survivin gene expression uses thereof
WO2011133584A2 (en) 2010-04-19 2011-10-27 Marina Biotech, Inc. Nucleic acid compounds for inhibiting hras gene expression and uses thereof
WO2011139843A2 (en) 2010-04-28 2011-11-10 Marina Biotech, Inc. Multi-sirna compositions for reducing gene expression
CN103068980B (zh) 2010-08-02 2017-04-05 瑟纳治疗公司 使用短干扰核酸（siNA）的RNA干扰介导的联蛋白（钙粘蛋白关联蛋白质），β1（CTNNB1）基因表达的抑制
KR102072631B1 (ko) 2010-08-17 2020-02-03 시르나 쎄러퓨틱스 인코퍼레이티드 짧은 간섭 핵산 （ｓｉＮＡ）을 사용한 Ｂ형 간염 바이러스 （ＨＢＶ） 유전자 발현의 ＲＮＡ 간섭 매개 억제
EP3372684B1 (en) 2010-08-24 2020-10-07 Sirna Therapeutics, Inc. Single-stranded rnai agents containing an internal, non-nucleic acid spacer
EP2609106A4 (en) 2010-08-26 2014-03-19 Merck Sharp & Dohme RNA INTERFERENCE-MEDIATED INHIBITION OF EXPRESSION OF PHD2 GENE (PROLYL HYDROXYLASE DOMAIN 2) USING SMALL INTERFERING NUCLEIC ACID (PANI)
CA2812300A1 (en) 2010-10-01 2012-04-05 The Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services Manipulation of stem cell function by p53 isoforms
EP3766975A1 (en) 2010-10-29 2021-01-20 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acid (sina)
ES2899043T3 (es) 2011-12-15 2022-03-09 Bioneer Corp Novedosos conjugados de oligonucleótidos y su uso
CN104114704A (zh) * 2012-01-05 2014-10-22 株式会社百奥尼 高效率的纳米颗粒型双螺旋寡rna结构体及其制备方法
US20150299696A1 (en) 2012-05-02 2015-10-22 Sirna Therapeutics, Inc. SHORT INTERFERING NUCLEIC ACID (siNA) COMPOSITIONS
WO2014005596A1 (en) * 2012-07-03 2014-01-09 Aarhus Universitet Modified payload molecules and their interactions and uses
AU2013356383B2 (en) 2012-12-06 2017-08-31 Merck Sharp & Dohme Corp. Disulfide masked prodrug compositions and methods
JP6276390B2 (ja) * 2013-05-21 2018-02-07 成都先導薬物開発有限公司 化合物の細胞膜透過の方法
WO2014187315A1 (zh) * 2013-05-21 2014-11-27 成都先导药物开发有限公司 一种化合物给药前体及药物载体制剂
RU2744194C2 (ru) 2013-12-02 2021-03-03 Фио Фармасьютикалс Корп Иммунотерапия рака
WO2015168108A2 (en) 2014-04-28 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treating cancer using nucleic targeting mdm2 or mycn
KR102506169B1 (ko) 2014-09-05 2023-03-08 피오 파마슈티칼스 코프. Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법
WO2016161388A1 (en) * 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna
EP3995581A3 (en) 2015-04-03 2022-10-26 University of Massachusetts Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
CA2980337A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Oligonucleotide compounds for targeting huntingtin mrna
WO2016196366A1 (en) 2015-05-29 2016-12-08 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Extension of replicative lifespan in diseases of premature aging using p53 isoforms
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
CN108135923B (zh) 2015-07-06 2021-03-02 菲奥医药公司 靶向超氧化物歧化酶1（sod1）的核酸分子
EP3334499A4 (en) 2015-08-14 2019-04-17 University of Massachusetts BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES
EP3365446A4 (en) 2015-10-19 2019-06-26 Phio Pharmaceuticals Corp. SELF ADMINISTRATION-REDUCED SIZE NUCLEIC ACID COMPOUNDS TARGETING LONGS NON-CODING LONGS
JP2019503394A (ja) 2016-01-31 2019-02-07 ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ 分岐オリゴヌクレオチド
US11753638B2 (en) 2016-08-12 2023-09-12 University Of Massachusetts Conjugated oligonucleotides
KR101797167B1 (ko) * 2016-11-09 2017-11-13 주식회사 바이오셀트란 신규의 프로테인 트랜스덕션 도메인 및 이의 용도
BR112021002440A2 (pt) 2018-08-10 2021-05-04 University Of Massachusetts oligonucleotídeos modificados visando snps
JPWO2020209285A1 (xx) * 2019-04-08 2020-10-15
KR102246906B1 (ko) * 2019-04-29 2021-04-30 주식회사 바이오셀트란 피부 또는 세포 투과능이 우수한 피부주름 방지 또는 개선용 화장료 조성물
EP4359534A1 (en) * 2021-06-21 2024-05-01 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating pompe disease
BR112023026862A2 (pt) 2021-06-23 2024-03-05 Beth Israel Deaconess Medical Ct Inc Compostos de oligonucleotídeos anti-flt1 otimizados para tratamento de pré-eclâmpsia e outros distúrbios angiogênicos

Family Cites Families (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX9203290A (es) * 1983-09-19 1992-08-01 Liposome Co Inc Suministro localizado usando conjugados de fibronectina.
US4904582A (en) * 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
US5962219A (en) * 1990-06-11 1999-10-05 Nexstar Pharmaceuticals, Inc. Systematic evolution of ligands by exponential enrichment: chemi-selex
DE4216134A1 (de) * 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US6447796B1 (en) * 1994-05-16 2002-09-10 The United States Of America As Represented By The Secretary Of The Army Sustained release hydrophobic bioactive PLGA microspheres
US6071890A (en) * 1994-12-09 2000-06-06 Genzyme Corporation Organ-specific targeting of cationic amphiphile/DNA complexes for gene therapy
US6331524B1 (en) * 1994-12-09 2001-12-18 Genzyme Corporation Organ-specific targeting of cationic amphiphile / DNA complexes for gene therapy
US5744335A (en) * 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
US5679559A (en) * 1996-07-03 1997-10-21 University Of Utah Research Foundation Cationic polymer and lipoprotein-containing system for gene delivery
US6001991A (en) * 1996-10-04 1999-12-14 Isis Pharmaceuticals Inc. Antisense oligonucleotide modulation of MDR P-glycoprotein gene expression
US6001311A (en) * 1997-02-05 1999-12-14 Protogene Laboratories, Inc. Apparatus for diverse chemical synthesis using two-dimensional array
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US6080580A (en) * 1998-10-05 2000-06-27 Isis Pharmaceuticals Inc. Antisense oligonucleotide modulation of tumor necrosis factor-α (TNF-α) expression
US6228642B1 (en) * 1998-10-05 2001-05-08 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of tumor necrosis factor-(α) (TNF-α) expression
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
EP1305333A4 (en) * 2000-07-31 2006-04-12 Active Motif ADMINISTRATION OF MOLECULES IN CELLS BY PEPTIDE MEDIATION
JP4095895B2 (ja) * 2000-12-01 2008-06-04 マックス−プランク−ゲゼルシャフト ツール フォーデルング デル ヴィッセンシャフテン エー．ヴェー． Ｒｎａ干渉を媒介する短鎖ｒｎａ分子
US20020130430A1 (en) * 2000-12-29 2002-09-19 Castor Trevor Percival Methods for making polymer microspheres/nanospheres and encapsulating therapeutic proteins and other products
US20050153913A1 (en) * 2001-04-10 2005-07-14 Kosak Kenneth M. Nucleic acid carrier compositions and methods for their synthesis
US20030130186A1 (en) * 2001-07-20 2003-07-10 Chandra Vargeese Conjugates and compositions for cellular delivery
US7135453B2 (en) * 2001-08-23 2006-11-14 Arizona Board Of Regents Reagents and methods for smooth muscle therapies
US7101995B2 (en) * 2001-08-27 2006-09-05 Mirus Bio Corporation Compositions and processes using siRNA, amphipathic compounds and polycations
US20040019008A1 (en) * 2002-05-28 2004-01-29 Lewis David L. Compositions and processes using siRNA, amphipathic compounds and polycations
JP4709545B2 (ja) * 2002-07-26 2011-06-22 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 修飾された低分子干渉ｒｎａ分子および使用方法
US20040053289A1 (en) * 2002-09-09 2004-03-18 The Regents Of The University Of California Short interfering nucleic acid hybrids and methods thereof
EP2000160A3 (en) * 2002-10-30 2009-03-11 Gambro Lundia AB Method and apparatuses for determining the efficiency of dialysis
EP1585756B1 (en) * 2002-11-26 2010-04-21 University of Massachusetts Delivery of sirnas
AU2003299864A1 (en) * 2002-12-27 2004-07-29 P. Radhakrishnan Sirna compounds and methods for the downregulation of gene expression
DE10302421A1 (de) * 2003-01-21 2004-07-29 Ribopharma Ag Doppelsträngige Ribonukleinsäure mit verbesserter Wirksamkeit
US20040147027A1 (en) * 2003-01-28 2004-07-29 Troy Carol M. Complex for facilitating delivery of dsRNA into a cell and uses thereof
US20040198640A1 (en) * 2003-04-02 2004-10-07 Dharmacon, Inc. Stabilized polynucleotides for use in RNA interference
EP1620544B1 (en) * 2003-04-17 2018-09-19 Alnylam Pharmaceuticals Inc. MODIFIED iRNA AGENTS
ITMI20030860A1 (it) 2003-04-29 2004-10-30 Univ Bologna Metodo per l'inibizione selettiva del gene n-myc
EP2567693B1 (en) * 2003-07-16 2015-10-21 Protiva Biotherapeutics Inc. Lipid encapsulated interfering RNA
EP2418281B1 (en) * 2003-10-24 2016-06-01 Gencia Corporation Methods and compositions for delivering polynucleotides
CA2564616C (en) * 2004-04-20 2016-08-30 Nastech Pharmaceutical Company Inc. Methods and compositions for enhancing delivery of double-stranded rna or a double-stranded hybrid nucleic acid to regulate gene expression in mammalian cells

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