DE50206993D1 - Verfahren zur hemmung der expression eine zielgens - Google Patents

Verfahren zur hemmung der expression eine zielgens

Info

Publication number
DE50206993D1
DE50206993D1 DE50206993T DE50206993T DE50206993D1 DE 50206993 D1 DE50206993 D1 DE 50206993D1 DE 50206993 T DE50206993 T DE 50206993T DE 50206993 T DE50206993 T DE 50206993T DE 50206993 D1 DE50206993 D1 DE 50206993D1
Authority
DE
Germany
Prior art keywords
dsrna1
inhibiting
expression
target
overhang
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.)
Expired - Lifetime
Application number
DE50206993T
Other languages
English (en)
Inventor
Roland Kreutzer
Stephan Limmer
Sylvia Limmer
Philipp Hadwiger
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Alnylam Europe AG
Original Assignee
Alnylam Europe AG
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=27437921&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=DE50206993(D1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from DE10100586A external-priority patent/DE10100586C1/de
Application filed by Alnylam Europe AG filed Critical Alnylam Europe AG
Priority to DE50206993T priority Critical patent/DE50206993D1/de
Application granted granted Critical
Publication of DE50206993D1 publication Critical patent/DE50206993D1/de
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • A61P33/00—Antiparasitic agents
    • A61P33/02—Antiprotozoals, e.g. for leishmaniasis, trichomoniasis, toxoplasmosis
    • A61P33/06—Antimalarials
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/04—Antineoplastic agents specific for metastasis
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131—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 viruses
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • 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
    • C12N2320/50—Methods for regulating/modulating their activity
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
DE50206993T 2001-01-09 2002-01-09 Verfahren zur hemmung der expression eine zielgens Expired - Lifetime DE50206993D1 (de)

Priority Applications (1)

Application Number Priority Date Filing Date Title
DE50206993T DE50206993D1 (de) 2001-01-09 2002-01-09 Verfahren zur hemmung der expression eine zielgens

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
DE10100586A DE10100586C1 (de) 2001-01-09 2001-01-09 Verfahren zur Hemmung der Expression eines Ziegens
DE10155280 2001-10-26
DE10158411 2001-11-29
DE10160151A DE10160151A1 (de) 2001-01-09 2001-12-07 Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
DE50206993T DE50206993D1 (de) 2001-01-09 2002-01-09 Verfahren zur hemmung der expression eine zielgens
PCT/EP2002/000152 WO2002055693A2 (de) 2001-01-09 2002-01-09 Verfahren zur hemmung der expression eines zielgens

Publications (1)

Publication Number Publication Date
DE50206993D1 true DE50206993D1 (de) 2006-07-06

Family

ID=27437921

Family Applications (2)

Application Number Title Priority Date Filing Date
DE10160151A Withdrawn DE10160151A1 (de) 1999-11-24 2001-12-07 Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
DE50206993T Expired - Lifetime DE50206993D1 (de) 2001-01-09 2002-01-09 Verfahren zur hemmung der expression eine zielgens

Family Applications Before (1)

Application Number Title Priority Date Filing Date
DE10160151A Withdrawn DE10160151A1 (de) 1999-11-24 2001-12-07 Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens

Country Status (8)

Country Link
EP (1) EP1352061B9 (de)
JP (1) JP4210116B2 (de)
CN (1) CN1630724A (de)
AT (1) ATE328075T1 (de)
AU (1) AU2002229701B2 (de)
CA (1) CA2432350A1 (de)
DE (2) DE10160151A1 (de)
WO (1) WO2002055693A2 (de)

Families Citing this family (174)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
US7601494B2 (en) 1999-03-17 2009-10-13 The University Of North Carolina At Chapel Hill Method of screening candidate compounds for susceptibility to biliary excretion
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
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
WO2001075164A2 (en) 2000-03-30 2001-10-11 Whitehead Institute For Biomedical Research Rna sequence-specific mediators of rna interference
TR200401292T3 (tr) 2000-12-01 2004-07-21 Max@Planck@Gesellschaft�Zur�F�Rderung�Der�Wissenschaften RNAÁgirişimineÁyolÁaçanÁküçükÁRNAÁmolekülleri
WO2003035869A1 (de) * 2001-10-26 2003-05-01 Ribopharma Ag Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens
EP1438405A1 (de) * 2001-01-09 2004-07-21 Ribopharma AG Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens
US7767802B2 (en) 2001-01-09 2010-08-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of anti-apoptotic genes
US8546143B2 (en) 2001-01-09 2013-10-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
US20050148530A1 (en) 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
CA2921821A1 (en) 2001-07-12 2003-01-23 University Of Massachusetts In vivo production of small interfering rnas that mediate gene silencing
US10590418B2 (en) 2001-07-23 2020-03-17 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for RNAi mediated inhibition of gene expression in mammals
PT2280070E (pt) 2001-07-23 2015-10-29 Univ Leland Stanford Junior Métodos e composições para inibição mediada por iarn da expressão génica em mamíferos
DE10163098B4 (de) * 2001-10-12 2005-06-02 Alnylam Europe Ag Verfahren zur Hemmung der Replikation von Viren
US7745418B2 (en) 2001-10-12 2010-06-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting viral replication
EP1438409A1 (de) * 2001-10-26 2004-07-21 Ribopharma AG Verwendung einer doppelsträngigen ribonukleinsäure zur behandlung einer infektion mit einem (+)-strang-rna-virus
DE10202419A1 (de) 2002-01-22 2003-08-07 Ribopharma Ag Verfahren zur Hemmung der Expression eines durch eine Chromosomen-Aberration entstandenen Zielgens
EP1432724A4 (de) 2002-02-20 2006-02-01 Sirna Therapeutics Inc Durch rna-interferenz vermittelte inhibierung von map-kinase-genen
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
WO2003099227A2 (en) 2002-05-23 2003-12-04 Ceptyr, Inc. Modulation of ptp1b signal transduction by rna interference
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
US20050042646A1 (en) 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
EP1389637B1 (de) * 2002-08-05 2012-05-30 Silence Therapeutics Aktiengesellschaft Interferierende RNS Moleküle mit glatten Enden
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
AU2003260370B2 (en) 2002-08-05 2008-05-22 Silence Therapeutics Gmbh Further novel forms of interfering RNA molecules
ES2665274T5 (es) * 2002-08-05 2021-06-30 Silence Therapeutics Gmbh Nuevas formas adicionales de moléculas de ARN de interferencia
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
DE60328214D1 (de) 2002-08-12 2009-08-13 New England Biolabs Inc Verfahren und zusammensetzungen in verbindung mit gen-silencing
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
US9879266B2 (en) 2002-11-14 2018-01-30 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US9719094B2 (en) 2002-11-14 2017-08-01 Thermo Fisher Scientific Inc. RNAi targeting SEC61G
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US9719092B2 (en) 2002-11-14 2017-08-01 Thermo Fisher Scientific Inc. RNAi targeting CNTD2
US9771586B2 (en) 2002-11-14 2017-09-26 Thermo Fisher Scientific Inc. RNAi targeting ZNF205
US10011836B2 (en) 2002-11-14 2018-07-03 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US9839649B2 (en) 2002-11-14 2017-12-12 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
DE10254214A1 (de) * 2002-11-20 2004-06-09 Beiersdorf Ag Oligoribonukleotide zur Behandlung von degenerativen Hauterscheinungen durch RNA-Interferenz
JP4526228B2 (ja) * 2002-11-22 2010-08-18 隆 森田 ＲＮＡｉによる新規治療法および治療剤
DE10302421A1 (de) * 2003-01-21 2004-07-29 Ribopharma Ag Doppelsträngige Ribonukleinsäure mit verbesserter Wirksamkeit
US20060178327A1 (en) * 2003-05-30 2006-08-10 Yeung Wah Hin A Inhibition of gene expression by delivery of specially selected double stranded or other forms of small interfering RNA precursors enabling the formation and function of small interfering RNA in vivo and in vitro
JP4505749B2 (ja) 2003-05-30 2010-07-21 日本新薬株式会社 Ｂｃｌ−２の発現抑制をするオリゴ二本鎖ＲＮＡとそれを含有する医薬組成物
ES2864206T3 (es) 2003-06-02 2021-10-13 Univ Massachusetts Métodos y composiciones para mejorar la eficacia y la especificidad del ARNi
EP3502252B1 (de) 2003-06-02 2023-04-05 University of Massachusetts Verfahren und zusammensetzungen zur kontrolle der wirksamkeit von rna-silencing
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
EP1486564A1 (de) * 2003-06-13 2004-12-15 Ribopharma AG SiRNA mit erhöhter Stabilität in Serum
DK3604537T3 (da) * 2003-06-13 2022-02-28 Alnylam Europe Ag Dobbeltstrenget ribonukleinsyre med forøget effektivitet i en organisme
MXPA06002216A (es) 2003-08-28 2006-04-27 Novartis Ag Interferencia de arn doble que tiene extremos romos y modificaciones 3'.
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
WO2005027980A1 (en) 2003-09-12 2005-03-31 University Of Massachusetts Rna interference for the treatment of gain-of-function disorders
US20060134787A1 (en) 2004-12-22 2006-06-22 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA
US7858769B2 (en) 2004-02-10 2010-12-28 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using multifunctional short interfering nucleic acid (multifunctional siNA)
WO2005078848A2 (en) 2004-02-11 2005-08-25 University Of Tennessee Research Foundation Inhibition of tumor growth and invasion by anti-matrix metalloproteinase dnazymes
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
EP1742958B1 (de) 2004-03-15 2017-05-17 City of Hope Verfahren und zusammensetzungen für die spezifische hemmung der genexpression durch doppelstrang-rna
WO2005105157A2 (en) 2004-04-23 2005-11-10 The Trustees Of Columbia University In The City Ofnew York INHIBITION OF HAIRLESS PROTEIN mRNA
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
EP2290071B1 (de) 2004-05-28 2014-12-31 Asuragen, Inc. Verfahren und Zusammensetzungen mit MicroRNA
EP2484780A1 (de) 2004-07-23 2012-08-08 The University of North Carolina At Chapel Hill Verfahren und Materialien zur Bestimmung der Schmerzempfindlichkeit und Vorhersage und Behandlung zugehöriger Störungen
WO2006022689A1 (en) * 2004-08-06 2006-03-02 Applera Corporation Method and compositions for treating diseases targeting cd51
JP4468989B2 (ja) 2004-08-16 2010-05-26 クアーク・ファーマスーティカルス、インコーポレイテッド Ｒｔｐ８０１阻害剤の治療への使用
JP5087924B2 (ja) 2004-08-26 2012-12-05 日本新薬株式会社 ガラクトース誘導体、薬物担体及び医薬組成物
JPWO2006025154A1 (ja) * 2004-08-30 2008-05-08 国立大学法人岐阜大学 修飾オリゴヌクレオチド
NZ553987A (en) 2004-09-28 2011-01-28 Quark Pharmaceuticals Inc Oligoribonucleotides and methods of use thereof for treatment of alopecia, acute renal failure and other diseases
DK2302055T3 (da) 2004-11-12 2014-10-13 Asuragen Inc Fremgangsmåder og sammensætninger involverende miRNA og miRNA-inhibitormolekyler
US8703769B2 (en) 2005-07-15 2014-04-22 The University Of North Carolina At Chapel Hill Use of EGFR inhibitors to prevent or treat obesity
WO2007022470A2 (en) 2005-08-18 2007-02-22 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating neurological disease
KR20080082956A (ko) 2005-09-15 2008-09-12 노보솜 아게 양쪽성 리포솜의 개선 또는 양쪽성 리포솜에 관련된 개선
EP1931789B1 (de) 2005-09-20 2016-05-04 BASF Plant Science GmbH VERFAHREN ZUR STEUERUNG DER GENEXPRESSION MITTELS ta- siRNA
US8093369B2 (en) 2005-10-11 2012-01-10 Ben Gurion University Of The Negev Research And Development Authority Ltd. Compositions for silencing the expression of VDAC1 and uses thereof
NL2000439C2 (nl) 2006-01-20 2009-03-16 Quark Biotech Therapeutische toepassingen van inhibitoren van RTP801.
US7825099B2 (en) 2006-01-20 2010-11-02 Quark Pharmaceuticals, Inc. Treatment or prevention of oto-pathologies by inhibition of pro-apoptotic genes
JP5093095B2 (ja) 2006-03-01 2012-12-05 日本新薬株式会社 ガラクトース誘導体、薬物担体及び医薬組成物
US7910566B2 (en) 2006-03-09 2011-03-22 Quark Pharmaceuticals Inc. Prevention and treatment of acute renal failure and other kidney diseases by inhibition of p53 by siRNA
FR2898908A1 (fr) 2006-03-24 2007-09-28 Agronomique Inst Nat Rech Procede de preparation de cellules aviaires differenciees et genes impliques dans le maintien de la pluripotence
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
EP2026843A4 (de) 2006-06-09 2011-06-22 Quark Pharmaceuticals Inc Therapeutische verwendungen von rtp801l
JP5424885B2 (ja) 2006-10-13 2014-02-26 マリーナ バイオテック，インコーポレイテッド 両性リポソームにおけるまたは両性リポソームに関する改善、両性リポソームを処方する方法および両性リポソームに充填する方法
JP2010507387A (ja) 2006-10-25 2010-03-11 クアーク・ファーマスーティカルス、インコーポレイテッド 新規のｓｉＲＮＡおよびその使用方法
US8455188B2 (en) 2007-01-26 2013-06-04 University Of Louisville Research Foundation, Inc. Modification of exosomal components for use as a vaccine
JP2010518880A (ja) 2007-02-26 2010-06-03 クアーク・ファーマスーティカルス、インコーポレイテッド Ｒｔｐ８０１のインヒビター及びその疾患の治療における使用
WO2008109546A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting tgfbr gene expression and uses thereof
JP5759673B2 (ja) 2007-03-21 2015-08-05 ブルックヘブン サイエンス アソシエイツ，エルエルシー 組み合わされたヘアピン−アンチセンス組成物および発現を調節するための方法
US7812002B2 (en) 2007-03-21 2010-10-12 Quark Pharmaceuticals, Inc. Oligoribonucleotide inhibitors of NRF2 and methods of use thereof for treatment of cancer
DE102007029471A1 (de) 2007-06-20 2008-12-24 Novosom Ag Neue fakultativ kationische Sterole
DK2170403T3 (da) 2007-06-27 2014-06-16 Quark Pharmaceuticals Inc Sammensætninger og fremgangsmåder til hæmning af ekspressionen af proapoptotiske gener
WO2009036332A1 (en) 2007-09-14 2009-03-19 Asuragen, Inc. Micrornas differentially expressed in cervical cancer and uses thereof
JP5646997B2 (ja) 2007-10-03 2014-12-24 クォーク ファーマシューティカルズ インコーポレーティッドＱｕａｒｋ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 新規ｓｉＲＮＡ構造
WO2009070805A2 (en) 2007-12-01 2009-06-04 Asuragen, Inc. Mir-124 regulated genes and pathways as targets for therapeutic intervention
US8614311B2 (en) 2007-12-12 2013-12-24 Quark Pharmaceuticals, Inc. RTP801L siRNA compounds and methods of use thereof
KR100949791B1 (ko) * 2007-12-18 2010-03-30 이동기 오프-타겟 효과를 최소화하고 ＲＮＡｉ 기구를 포화시키지않는 신규한 ｓｉＲＮＡ 구조 및 그 용도
WO2009102427A2 (en) 2008-02-11 2009-08-20 Rxi Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
WO2009137807A2 (en) 2008-05-08 2009-11-12 Asuragen, Inc. Compositions and methods related to mirna modulation of neovascularization or angiogenesis
WO2009147684A2 (en) 2008-06-06 2009-12-10 Quark Pharmaceuticals, Inc. Compositions and methods for treatment of ear disorders
TWI455944B (zh) 2008-07-01 2014-10-11 Daiichi Sankyo Co Ltd 雙股多核苷酸
WO2010008582A2 (en) 2008-07-18 2010-01-21 Rxi Pharmaceuticals Corporation Phagocytic cell drug delivery system
EP2326351B1 (de) 2008-08-19 2017-12-27 Nektar Therapeutics Konjugate von kurzen interferierenden nukleinsäuren
US10138485B2 (en) 2008-09-22 2018-11-27 Rxi Pharmaceuticals Corporation Neutral nanotransporters
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
SG171952A1 (en) 2008-12-04 2011-07-28 Opko Ophthalmics Llc Compositions and methods for selective inhibition of pro-angiogenic vegf isoforms
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
BR112012000255B1 (pt) 2009-07-09 2022-11-29 Marina Biotech, Inc Compostos compreendendo imino lipídios e composição compreendendo os ditos compostos
WO2011035065A1 (en) 2009-09-17 2011-03-24 Nektar Therapeutics Monoconjugated chitosans as delivery agents for small interfering nucleic acids
CA2776568A1 (en) 2009-11-26 2011-06-03 Quark Pharmaceuticals, Inc. Sirna compounds comprising terminal substitutions
US8293718B2 (en) 2009-12-18 2012-10-23 Novartis Ag Organic compositions to treat HSF1-related diseases
WO2011083147A1 (en) 2010-01-08 2011-07-14 Cemm-Forschungsinstitut Für Molekulare Medizin Gmbh Wave1 inhibition in the medical intervention of inflammatory diseases and/or infections caused by a pathogen
MX2012009318A (es) 2010-02-10 2012-09-07 Novartis Ag Metodos y compuestos para el crecimiento muscular.
KR20180044433A (ko) 2010-03-24 2018-05-02 알엑스아이 파마슈티칼스 코포레이션 진피 및 섬유증성 적응증에서의 ｒｎａ 간섭
RU2615143C2 (ru) 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
US9095504B2 (en) 2010-03-24 2015-08-04 Rxi Pharmaceuticals Corporation RNA interference in ocular indications
US20130116304A1 (en) 2010-04-19 2013-05-09 Ernst R. Werner Tmem195 encodes for tetrahydrobiopterin-dependent alkylglycerol monooxygenase activity
EP3372684B1 (de) 2010-08-24 2020-10-07 Sirna Therapeutics, Inc. Einsträngige rnai-mittel mit einem internen nukleinsäurefreien spacer
WO2012028703A1 (en) 2010-09-02 2012-03-08 INSERM (Institut National de la Santé et de la Recherche Médicale) Method for the prognosis of the progression of cancer
EP2433644A1 (de) 2010-09-22 2012-03-28 IMBA-Institut für Molekulare Biotechnologie GmbH Brustkrebstherapeutika
JP5795072B2 (ja) 2010-10-22 2015-10-14 ソンギュングヮン ユニバーシティ ファウンデーション フォー コーポレート コラボレーション Ｒｎａ干渉を誘導する核酸分子及びその用途
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
US9833439B2 (en) 2011-05-25 2017-12-05 Universite Paris Descartes ERK inhibitors for use in treating spinal muscular atrophy
US8951966B2 (en) 2011-07-01 2015-02-10 Ngm Biopharmaceuticals, Inc. Compositions comprising variants and fusions of FGF19 polypeptides, and uses and methods thereof for treatment of metabolic disorders and diseases
WO2013040251A2 (en) 2011-09-13 2013-03-21 Asurgen, Inc. Methods and compositions involving mir-135b for distinguishing pancreatic cancer from benign pancreatic disease
WO2013053919A2 (en) 2011-10-14 2013-04-18 Inserm Biomarkers of renal disorders
US10023862B2 (en) 2012-01-09 2018-07-17 Arrowhead Pharmaceuticals, Inc. Organic compositions to treat beta-catenin-related diseases
WO2013104798A1 (en) 2012-01-12 2013-07-18 Cemm - Center For Molecular Medicine Of The Austrian Academy Of Sciences Modulators of immune responses
CN108148838A (zh) 2012-05-22 2018-06-12 奥利克斯医药有限公司 具有细胞内穿透能力的诱导rna干扰的核酸分子及用途
EP2700949A1 (de) 2012-08-24 2014-02-26 IMG Institut für medizinische Genomforschung Planungsgesellschaft M.B.H. Verwendung von Biliverdinreduktaseproteinen als Krebsmarker
EP2914260A1 (de) 2012-10-31 2015-09-09 INSERM (Institut National de la Santé et de la Recherche Médicale) Verfahren zur vorbeugung des antiphospholipid-syndroms (aps)
EP3798228A1 (de) 2012-11-28 2021-03-31 NGM Biopharmaceuticals, Inc. Zusammensetzungen und verfahren zur behandlung von stoffwechselstörungen und -erkrankungen
US9290557B2 (en) 2012-11-28 2016-03-22 Ngm Biopharmaceuticals, Inc. Compositions comprising variants and fusions of FGF19 polypeptides
CN108888757A (zh) 2012-12-27 2018-11-27 恩格姆生物制药公司 用于调节胆汁酸体内稳态及治疗胆汁酸紊乱和疾病的方法
US9273107B2 (en) 2012-12-27 2016-03-01 Ngm Biopharmaceuticals, Inc. Uses and methods for modulating bile acid homeostasis and treatment of bile acid disorders and diseases
KR102178945B1 (ko) 2013-10-28 2020-11-13 엔지엠 바이오파마슈티컬스, 아이엔씨. 암 모델 및 관련 방법
EP3079707A4 (de) 2013-12-02 2017-10-18 RXi Pharmaceuticals Corporation Immuntherapie für krebs
BR112016017248A8 (pt) 2014-01-24 2018-04-17 Ngm Biopharmaceuticals Inc anticorpo ou fragmento seu, agente de ligação, animal transgênico, hibridoma, vetor, composição farmacêutica, método de indução de sinalização de semelhantes a fgf19 e/ou fgf21, método para ativar um complexo klotho beta/receptor de fgf, método para melhorar o metabolismo da glicose em um indivíduo, método de detecção da presença de klotho beta, uso do anticorpo ou fragmento seu, uso da composição farmacêutica, método de tratamento e método para melhorar parâmetros metabólicos
KR102228828B1 (ko) 2014-03-11 2021-03-16 셀렉티스 동종이형 이식에 양립성인 t-세포들을 만들어내는 방법
EP3137119B1 (de) 2014-04-28 2020-07-01 Phio Pharmaceuticals Corp. Verfahren zur behandlung von krebs mithilfe von einer nukleinsaüre gegen mdm2
US10398758B2 (en) 2014-05-28 2019-09-03 Ngm Biopharmaceuticals, Inc. Compositions comprising variants of FGF19 polypeptides and uses thereof for the treatment of hyperglycemic conditions
CA2951153A1 (en) 2014-06-16 2015-12-23 Ngm Biopharmaceuticals, Inc. Methods and uses for modulating bile acid homeostasis and treatment of bile acid disorders and diseases
CN107073294A (zh) 2014-09-05 2017-08-18 阿克赛医药公司 使用靶向tyr或mmp1的核酸治疗老化和皮肤病症的方法
KR102569907B1 (ko) 2014-10-23 2023-08-24 엔지엠 바이오파마슈티컬스, 아이엔씨. 펩티드 변이체를 포함하는 약제학적 조성물 및 그의 사용 방법
WO2016073855A1 (en) 2014-11-07 2016-05-12 Ngm Biopharmaceuticals, Inc. Methods for treatment of bile acid-related disorders and prediction of clinical sensitivity to treatment of bile acid-related disorders
US10500273B2 (en) 2015-03-02 2019-12-10 180 Therapeutics Lp Method of treating a localized fibrotic disorder using an IL-33 antagonist
WO2017007825A1 (en) 2015-07-06 2017-01-12 Rxi Pharmaceuticals Corporation Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
JP6983752B2 (ja) 2015-07-06 2021-12-17 フィオ ファーマシューティカルズ コーポレーションPhio Pharmaceuticals Corp. スーパーオキシドディスムターゼ１（ｓｏｄ１）を標的とする核酸分子
US10800843B2 (en) 2015-07-29 2020-10-13 Ngm Biopharmaceuticals, Inc. Beta klotho-binding proteins
WO2017029391A1 (en) 2015-08-20 2017-02-23 INSERM (Institut National de la Santé et de la Recherche Médicale) New method for treating cancer
CA3002744A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
EP3377090B1 (de) 2015-11-09 2021-04-07 NGM Biopharmaceuticals, Inc. Verfahren zur behandlung von gallensäurebedingten erkrankungen
KR20180071362A (ko) 2015-11-16 2018-06-27 올릭스 주식회사 MyD88 또는 TLR3을 타겟팅하는 RNA 복합체를 이용한 연령-관련 황반 변성의 치료
CN108779463B (zh) 2016-02-02 2022-05-24 奥利克斯医药有限公司 使用靶向IL4Rα、TRPA1或F2RL1的RNA复合物治疗特应性皮炎和哮喘
WO2017134526A1 (en) 2016-02-02 2017-08-10 Olix Pharmaceuticals, Inc. Treatment of angiogenesis-associated diseases using rna complexes that target angpt2 and pdgfb
EP3423568A4 (de) 2016-03-04 2019-11-13 University Of Louisville Research Foundation, Inc. Verfahren und zusammensetzungen zur ex-vivo-vermehrung von sehr kleinen embryonalen stammzellen (vsels)
RU2759737C2 (ru) 2016-03-31 2021-11-17 Этрис Гмбх Новые минимальные utr-последовательности
MA45469A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques de bêta-caténine et leurs utilisations
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA45349A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques egfr et leurs utilisations
MA45470A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques kras et leurs utilisations
CA3020487C (en) 2016-04-11 2022-05-31 Olix Pharmaceuticals, Inc. Treatment of idiopathic pulmonary fibrosis using rna complexes that target connective tissue growth factor
KR101916652B1 (ko) 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
US20190367930A1 (en) 2016-07-29 2019-12-05 Danmarks Tekniske Universitet Methods for decoupling cell growth from production of biochemicals and recombinant polypeptides
EP3503882A4 (de) 2016-08-26 2020-07-29 NGM Biopharmaceuticals, Inc. Verfahren zur behandlung von fibroblastenwachstumsfaktor-19-vermittelten karzinomen und tumoren
KR20190108167A (ko) 2017-02-10 2019-09-23 성균관대학교산학협력단 Rna 간섭을 위한 긴 이중가닥 rna
US20200199541A1 (en) 2017-06-16 2020-06-25 Imba - Institut Für Molekulare Biotechnologie Gmbh Blood vessel organoid, methods of producing and using said organoids
MX2020005860A (es) 2017-12-06 2020-09-09 Avidity Biosciences Inc Composiciones y metodos de tratamiento de atrofia muscular y distrofia miotonica.
CN108535480B (zh) * 2018-03-05 2020-03-06 南通大学附属医院 EphA8基因在制备抗乳腺癌药物及其诊断试剂盒中的应用
CN108490180B (zh) * 2018-03-12 2020-03-10 南通大学附属医院 EphA8基因在制备胃癌药物及其诊断试剂盒中的应用
JP2023537798A (ja) 2020-03-19 2023-09-06 アビディティー バイオサイエンシーズ，インク． 顔面肩甲上腕型筋ジストロフィーを処置するための組成物および方法
CN116887812A (zh) 2021-02-26 2023-10-13 埃泽瑞斯公司 用于气溶胶形成的制剂以及用于递送核酸的气溶胶
WO2024042236A1 (en) 2022-08-26 2024-02-29 Ethris Gmbh Stable lipid or lipidoid nanoparticle suspensions
EP4327829A1 (de) 2022-08-26 2024-02-28 Ethris GmbH Stabilisierung von lipid- oder lipidoidnanopartikelsuspensionen

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE171210T1 (de) * 1992-07-02 1998-10-15 Hybridon Inc Selbststabilisierte oligonukleotide als therapeutika
DE19631919C2 (de) * 1996-08-07 1998-07-16 Deutsches Krebsforsch Anti-Sinn-RNA mit Sekundärstruktur
US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
AU776150B2 (en) * 1999-01-28 2004-08-26 Medical College Of Georgia Research Institute, Inc. Composition and method for (in vivo) and (in vitro) attenuation of gene expression using double stranded RNA
DE19956568A1 (de) * 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
WO2001075164A2 (en) * 2000-03-30 2001-10-11 Whitehead Institute For Biomedical Research Rna sequence-specific mediators of rna interference
TR200401292T3 (tr) * 2000-12-01 2004-07-21 Max@Planck@Gesellschaft�Zur�F�Rderung�Der�Wissenschaften RNAÁgirişimineÁyolÁaçanÁküçükÁRNAÁmolekülleri
DE10100587C1 (de) * 2001-01-09 2002-11-21 Ribopharma Ag Verfahren zur Hemmung der Expression eines Zielgens
DE10100588A1 (de) * 2001-01-09 2002-07-18 Ribopharma Ag Verfahren zur Hemmung der Expression eines Zielgens

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