WO2002055693A3 - Verfahren zur hemmung der expression eines zielgens - Google Patents

Verfahren zur hemmung der expression eines zielgens Download PDF

Info

Publication number
WO2002055693A3
WO2002055693A3 PCT/EP2002/000152 EP0200152W WO02055693A3 WO 2002055693 A3 WO2002055693 A3 WO 2002055693A3 EP 0200152 W EP0200152 W EP 0200152W WO 02055693 A3 WO02055693 A3 WO 02055693A3
Authority
WO
WIPO (PCT)
Prior art keywords
target gene
expression
dsrna
inhibiting
dual
Prior art date
Application number
PCT/EP2002/000152
Other languages
English (en)
French (fr)
Other versions
WO2002055693A2 (de
Inventor
Roland Kreutzer
Stephan Limmer
Sylvia Rost
Philipp Hadwiger
Original Assignee
Ribopharma 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=WO2002055693(A3) "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
Priority to DE10163098A priority Critical patent/DE10163098B4/de
Priority to JP2002556740A priority patent/JP4210116B2/ja
Priority to DE50206993T priority patent/DE50206993D1/de
Priority to CA002432350A priority patent/CA2432350A1/en
Application filed by Ribopharma Ag filed Critical Ribopharma Ag
Priority to EP02710786A priority patent/EP1352061B9/de
Priority to AU2002229701A priority patent/AU2002229701B2/en
Priority to DE10230997A priority patent/DE10230997A1/de
Priority to DE10230996A priority patent/DE10230996A1/de
Publication of WO2002055693A2 publication Critical patent/WO2002055693A2/de
Priority to PCT/EP2002/011432 priority patent/WO2003033700A1/de
Priority to US10/493,768 priority patent/US20040248835A1/en
Priority to CNA028251636A priority patent/CN1604783A/zh
Priority to JP2003537650A priority patent/JP2005512976A/ja
Priority to PCT/EP2002/011971 priority patent/WO2003035082A1/de
Priority to EP10011813.2A priority patent/EP2264173A3/de
Priority to JP2003538370A priority patent/JP2005506385A/ja
Priority to JP2003538376A priority patent/JP2005506087A/ja
Priority to PCT/EP2002/011968 priority patent/WO2003035868A1/de
Priority to US10/493,686 priority patent/US20050119202A1/en
Priority to EP02785313A priority patent/EP1438409A1/de
Priority to PCT/EP2002/011973 priority patent/WO2003035876A1/de
Priority to EP02779511A priority patent/EP1438405A1/de
Priority to PCT/EP2002/011969 priority patent/WO2003035869A1/de
Priority to CNA028261798A priority patent/CN1608131A/zh
Priority to PCT/EP2002/011972 priority patent/WO2003035083A1/de
Priority to EP02801917A priority patent/EP1438056A1/de
Priority to EP02785312A priority patent/EP1438406A1/de
Priority to PCT/EP2002/011970 priority patent/WO2003035870A1/de
Priority to CNA02826181XA priority patent/CN1608133A/zh
Priority to US10/384,512 priority patent/US7348314B2/en
Priority to US10/384,434 priority patent/US20040121348A1/en
Priority to US10/384,463 priority patent/US7763590B2/en
Priority to US10/384,339 priority patent/US7829693B2/en
Publication of WO2002055693A3 publication Critical patent/WO2002055693A3/de
Priority to US10/382,634 priority patent/US20040038921A1/en
Priority to US10/666,458 priority patent/US20040126791A1/en
Priority to US11/747,549 priority patent/US20080070856A1/en
Priority to US11/959,936 priority patent/US7745418B2/en
Priority to US12/631,689 priority patent/US8273868B2/en
Priority to US12/817,009 priority patent/US7994309B2/en
Priority to US12/894,018 priority patent/US8546143B2/en
Priority to US13/177,316 priority patent/US8273870B2/en
Priority to US13/594,140 priority patent/US20120322858A1/en
Priority to US13/594,150 priority patent/US20130064879A1/en
Priority to US14/012,994 priority patent/US9074213B2/en
Priority to US14/737,304 priority patent/US9587240B2/en

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P33/00Antiparasitic agents
    • A61P33/02Antiprotozoals, e.g. for leishmaniasis, trichomoniasis, toxoplasmosis
    • A61P33/06Antimalarials
    • 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
    • A61P35/00Antineoplastic agents
    • A61P35/04Antineoplastic agents specific for metastasis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • YGENERAL 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
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Abstract

Die Erfindung betrifft ein Verfahren zur Hemmung der Expression eines Zielgens in einer Zelle umfassend die folgenden Schritte: Einführen mindestens einer doppelstängigen Ribonukleinsäure (dsRNA I) in einer zur Hemmung der Expression des Zielgens ausreichenden Menge, wobei die dsRNA I eine doppelsträngige aus höchstens 49 aufeinanderfolgenden Nukleotidpaaren gebildete Struktur aufweist, und wobei ein Strang (as1) oder zumindest ein Abschnitt des einen Strangs (as1) der doppelsträngigen Struktur komplementär zum Sinn-Strang des Zielgens ist, und wobei die dsRNA am einen Ende (E1) der dsRNA I einen aus 1 bis 4 Nukeotiden gebildeten überhang aufweist.
PCT/EP2002/000152 1999-11-24 2002-01-09 Verfahren zur hemmung der expression eines zielgens WO2002055693A2 (de)

Priority Applications (44)

Application Number Priority Date Filing Date Title
DE10163098A DE10163098B4 (de) 2001-10-12 2001-12-20 Verfahren zur Hemmung der Replikation von Viren
AU2002229701A AU2002229701B2 (en) 2001-01-09 2002-01-09 Method for inhibiting the expression of a target gene
EP02710786A EP1352061B9 (de) 2001-01-09 2002-01-09 Verfahren zur hemmung der expression eine zielgens
DE50206993T DE50206993D1 (de) 2001-01-09 2002-01-09 Verfahren zur hemmung der expression eine zielgens
CA002432350A CA2432350A1 (en) 2001-01-09 2002-01-09 Method for inhibiting the expression of a target gene
JP2002556740A JP4210116B2 (ja) 2001-01-09 2002-01-09 標的遺伝子の発現阻害方法
DE10230997A DE10230997A1 (de) 2001-10-26 2002-07-09 Medikament zur Erhöhung der Wirksamkeit eines Rezeptor-vermittelt Apoptose in Tumorzellen auslösenden Arzneimittels
DE10230996A DE10230996A1 (de) 2001-10-26 2002-07-09 Medikament zur Behandlung eines Pankreaskarzinoms
PCT/EP2002/011432 WO2003033700A1 (de) 2001-10-12 2002-10-11 Verfahren zur hemmung der replikation von viren
EP02785312A EP1438406A1 (de) 2001-10-26 2002-10-25 Medikament zur behandlung eines pankreaskarzinoms
CNA02826181XA CN1608133A (zh) 2001-10-26 2002-10-25 双链核糖核酸用于治疗正(+)链rna病毒感染的用途
EP02785313A EP1438409A1 (de) 2001-10-26 2002-10-25 Verwendung einer doppelsträngigen ribonukleinsäure zur behandlung einer infektion mit einem (+)-strang-rna-virus
EP02779511A EP1438405A1 (de) 2001-01-09 2002-10-25 Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens
PCT/EP2002/011970 WO2003035870A1 (de) 2001-10-26 2002-10-25 Medikament zur behandlung eines pankreaskarzinoms
JP2003537650A JP2005512976A (ja) 2001-10-26 2002-10-25 Rna干渉により線維化疾患を処置するための医薬
PCT/EP2002/011971 WO2003035082A1 (de) 2001-10-26 2002-10-25 Medikament zur hemmung der expression eines zielgens
EP10011813.2A EP2264173A3 (de) 2001-01-09 2002-10-25 Verwendung einer doppelsträngigen Ribonukleinsäure zur gezielten Hemmung der Expression eines vorgegebenen Zielgens
JP2003538370A JP2005506385A (ja) 2001-10-26 2002-10-25 膵臓癌を処置するための医薬
JP2003538376A JP2005506087A (ja) 2001-10-26 2002-10-25 プラス鎖rnaウイルスによる感染症を処置するための2本鎖リボ核酸の使用
PCT/EP2002/011968 WO2003035868A1 (de) 2001-10-26 2002-10-25 Medikament zur erhöhung der wirksamkeit eines rezeptor-vermittelt apoptose in tumorzellen auslösenden arzeimittels
US10/493,686 US20050119202A1 (en) 2001-10-26 2002-10-25 Medicament to treat a fibrotic disease
CNA028251636A CN1604783A (zh) 2001-10-26 2002-10-25 通过rna干扰治疗纤维化疾病的药物
PCT/EP2002/011973 WO2003035876A1 (de) 2001-10-26 2002-10-25 Verwendung einer doppelsträngigen ribonukleinsäure zur behandlung einer infektion mit einem (+)-strang-rna-virus
US10/493,768 US20040248835A1 (en) 2001-10-26 2002-10-25 Use of a double-stranded ribonucleic acid for treating an infection with a positivestrand rna-virus
PCT/EP2002/011969 WO2003035869A1 (de) 2001-10-26 2002-10-25 Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens
CNA028261798A CN1608131A (zh) 2001-10-26 2002-10-25 治疗胰腺癌的药物
PCT/EP2002/011972 WO2003035083A1 (de) 2001-10-26 2002-10-25 Medikament zur behandlung einer fibrotischen erkrankung durch rna interferenz
EP02801917A EP1438056A1 (de) 2001-10-26 2002-10-25 Medikament zur behandlung einer fibrotischen erkrankung durch rna interferenz
US10/384,463 US7763590B2 (en) 2001-10-12 2003-03-07 Compositions and methods for inhibiting expression of a mutant gene
US10/384,512 US7348314B2 (en) 2001-10-12 2003-03-07 Compositions and methods for inhibiting viral replication
US10/384,339 US7829693B2 (en) 1999-11-24 2003-03-07 Compositions and methods for inhibiting expression of a target gene
US10/384,434 US20040121348A1 (en) 2001-10-26 2003-03-07 Compositions and methods for treating pancreatic cancer
US10/382,634 US20040038921A1 (en) 2001-10-26 2003-08-11 Composition and method for inhibiting expression of a target gene
US10/666,458 US20040126791A1 (en) 2001-10-26 2003-09-19 Compositions and methods for treating trail-resistant cancer cells
US11/747,549 US20080070856A1 (en) 2001-10-26 2007-05-11 Medicament to treat a fibrotic disease
US11/959,936 US7745418B2 (en) 2001-10-12 2007-12-19 Compositions and methods for inhibiting viral replication
US12/631,689 US8273868B2 (en) 2001-10-12 2009-12-04 Compositions and methods for inhibiting viral replication
US12/817,009 US7994309B2 (en) 2001-10-26 2010-06-16 Compositions and methods for inhibiting expression of a mutant gene
US12/894,018 US8546143B2 (en) 2001-01-09 2010-09-29 Compositions and methods for inhibiting expression of a target gene
US13/177,316 US8273870B2 (en) 2001-10-26 2011-07-06 Compositions and methods for inhibiting expression of a mutant gene
US13/594,150 US20130064879A1 (en) 2001-10-12 2012-08-24 Compositions and Methods for Inhibiting Viral Replication
US13/594,140 US20120322858A1 (en) 2001-10-26 2012-08-24 Compositions and Methods for Inhibiting Expression of a Mutant Gene
US14/012,994 US9074213B2 (en) 2001-01-09 2013-08-28 Compositions and methods for inhibiting expression of a target gene
US14/737,304 US9587240B2 (en) 2001-01-09 2015-06-11 Compositions and methods for inhibiting expression of a target gene

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
DE10100586A DE10100586C1 (de) 2001-01-09 2001-01-09 Verfahren zur Hemmung der Expression eines Ziegens
DE10100586.5 2001-01-09
DE10155280.7 2001-10-26
DE10155280 2001-10-26
DE10158411.3 2001-11-29
DE10158411 2001-11-29
DE10160151.4 2001-12-07
DE10160151A DE10160151A1 (de) 2001-01-09 2001-12-07 Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2002/000151 Continuation WO2002055692A2 (de) 2001-01-09 2002-01-09 Verfahren zur hemmung der expression eines zielgens und medikament zur therapie einer tumorerkrankung

Related Child Applications (2)

Application Number Title Priority Date Filing Date
PCT/EP2002/011969 Continuation WO2003035869A1 (de) 2001-01-09 2002-10-25 Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens
US10/384,339 Continuation-In-Part US7829693B2 (en) 1999-11-24 2003-03-07 Compositions and methods for inhibiting expression of a target gene

Publications (2)

Publication Number Publication Date
WO2002055693A2 WO2002055693A2 (de) 2002-07-18
WO2002055693A3 true WO2002055693A3 (de) 2003-07-17

Family

ID=27437921

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2002/000152 WO2002055693A2 (de) 1999-11-24 2002-01-09 Verfahren zur hemmung der expression eines 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)

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8846894B2 (en) 2002-02-20 2014-09-30 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US8853384B2 (en) 2000-12-01 2014-10-07 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. RNA interference mediating small RNA molecules
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
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
US9290557B2 (en) 2012-11-28 2016-03-22 Ngm Biopharmaceuticals, Inc. Compositions comprising variants and fusions of FGF19 polypeptides
US9580483B2 (en) 2011-07-01 2017-02-28 Ngm Biopharmaceuticals, Inc. Methods of using compositions comprising variants and fusions of FGF19 polypeptides for treatment of diabetes

Families Citing this family (168)

* 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
WO2001075164A2 (en) 2000-03-30 2001-10-11 Whitehead Institute For Biomedical Research Rna sequence-specific mediators of rna interference
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
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
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
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
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

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994001550A1 (en) * 1992-07-02 1994-01-20 Hybridon, Inc. Self-stabilized oligonucleotides as therapeutic agents
WO1998005770A2 (de) * 1996-08-07 1998-02-12 Deutches Krebsforschungszentrum Stiftung Des Öffentlichen Rechts Anti-sinn-rna mit sekundärstruktur
WO1999032619A1 (en) * 1997-12-23 1999-07-01 The Carnegie Institution Of Washington Genetic inhibition by double-stranded rna
WO2000044914A1 (en) * 1999-01-28 2000-08-03 Medical College Of Georgia Research Institute, Inc. Composition and method for in vivo and in vitro attenuation of gene expression using double stranded rna
WO2000044895A1 (de) * 1999-01-30 2000-08-03 Roland Kreutzer Verfahren und medikament zur hemmung der expression eines vorgegebenen gens

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994001550A1 (en) * 1992-07-02 1994-01-20 Hybridon, Inc. Self-stabilized oligonucleotides as therapeutic agents
WO1998005770A2 (de) * 1996-08-07 1998-02-12 Deutches Krebsforschungszentrum Stiftung Des Öffentlichen Rechts Anti-sinn-rna mit sekundärstruktur
WO1999032619A1 (en) * 1997-12-23 1999-07-01 The Carnegie Institution Of Washington Genetic inhibition by double-stranded rna
WO2000044914A1 (en) * 1999-01-28 2000-08-03 Medical College Of Georgia Research Institute, Inc. Composition and method for in vivo and in vitro attenuation of gene expression using double stranded rna
WO2000044895A1 (de) * 1999-01-30 2000-08-03 Roland Kreutzer Verfahren und medikament zur hemmung der expression eines vorgegebenen gens

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
AMBROS VICTOR: "Dicing up RNAs", SCIENCE, AMERICAN ASSOCIATION FOR THE ADVANCEMENT OF SCIENCE,, US, vol. 293, no. 5531, 3 August 2001 (2001-08-03), pages 811 - 813, XP002183122, ISSN: 0036-8075 *
BASS BRENDA L: "Double-stranded RNA as a template for gene silencing", CELL, CELL PRESS, CAMBRIDGE, NA, US, vol. 101, no. 3, 28 April 2000 (2000-04-28), pages 235 - 238, XP002194756, ISSN: 0092-8674 *
ELBASHIR SAYDA M ET AL: "RNA interference is mediated by 21- and 22-nucleotide RNAs", GENES AND DEVELOPMENT, COLD SPRING HARBOR LABORATORY PRESS, NEW YORK, US, vol. 15, no. 2, 15 January 2001 (2001-01-15), pages 188 - 200, XP002204651, ISSN: 0890-9369 *
PARRISH S., FLEENOR J., ET AL.: "Functional Anatomy of a dsRNA trigger: differential requirement for the two trigger strands in RNA interference.", MOL. CELL, vol. 6, November 2000 (2000-11-01), pages 1077 - 187, XP002226361 *
UHLMANN E ET AL: "ANTISENSE OLIGONUCLEOTIDES: A NEW THERAPEUTIC PRINCIPLE", CHEMICAL REVIEWS, AMERICAN CHEMICAL SOCIETY. EASTON, US, vol. 90, no. 4, 1 June 1990 (1990-06-01), pages 543 - 584, XP000141412, ISSN: 0009-2665 *
ZAMORE PHILLIP D ET AL: "RNAi: Double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals", CELL, CELL PRESS, CAMBRIDGE, NA, US, vol. 101, no. 1, 31 March 2000 (2000-03-31), pages 25 - 33, XP002208683, ISSN: 0092-8674 *

Cited By (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8853384B2 (en) 2000-12-01 2014-10-07 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. RNA interference mediating small RNA molecules
US8933044B2 (en) 2000-12-01 2015-01-13 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. RNA interference mediating small RNA molecules
US8993745B2 (en) 2000-12-01 2015-03-31 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. RNA interference mediating small RNA molecules
US8846894B2 (en) 2002-02-20 2014-09-30 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US9580483B2 (en) 2011-07-01 2017-02-28 Ngm Biopharmaceuticals, Inc. Methods of using compositions comprising variants and fusions of FGF19 polypeptides for treatment of diabetes
US9290557B2 (en) 2012-11-28 2016-03-22 Ngm Biopharmaceuticals, Inc. Compositions comprising variants and fusions of FGF19 polypeptides
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

Also Published As

Publication number Publication date
ATE328075T1 (de) 2006-06-15
CN1630724A (zh) 2005-06-22
CA2432350A1 (en) 2002-07-18
DE50206993D1 (de) 2006-07-06
EP1352061B9 (de) 2007-03-07
WO2002055693A2 (de) 2002-07-18
EP1352061A2 (de) 2003-10-15
DE10160151A1 (de) 2003-06-26
JP2004519458A (ja) 2004-07-02
JP4210116B2 (ja) 2009-01-14
EP1352061B1 (de) 2006-05-31
AU2002229701B2 (en) 2006-08-10

Similar Documents

Publication Publication Date Title
WO2002055693A3 (de) Verfahren zur hemmung der expression eines zielgens
CA2513809A1 (en) Lipophilic derivatives of double-stranded ribonucleic acid
WO2005014782A8 (en) Double-stranded ribonucleic acid with increased effectiveness in an organism
HK1160487A1 (zh) 抑制預定基因表現的寡核糖核苷酸
WO2007056326A3 (en) Compositions and methods for inhibiting expression of nav1.8 gene
WO2007051045A3 (en) Compositions and methods for inhibiting expression of huntingtin gene
WO2004015107A3 (en) Further novel forms of interfering rna molecules
WO2007115168A3 (en) Compositions and methods for inhibiting expression of eg5 gene
WO2002055692A3 (de) Verfahren zur hemmung der expression eines zielgens und medikament zur therapie einer tumorerkrankung
WO2006032041A3 (en) Compositions and methods for inhibiting expression of anti-apoptotic genes
WO2003012052A3 (en) Specific inhibition of gene expression by small double stranded rnas
WO2007134161A8 (en) Compositions and methods for inhibiting expression of the pcsk9 gene
WO2007056153A3 (en) Peptide-dicer substrate rna conjugates as delivery vehicles for sirna
WO2005089287A3 (en) Methods and compositions for the specific inhibition of gene expression by double-stranded rna
WO2007127919A3 (en) Compositions and methods for inhibiting expression of a gene from the jc virus
WO2003062423A3 (de) Verfahren zur hemmung der expression eines durch eine chromosomen-aberration entstandenen zielgens
WO2008009477A3 (en) Means for inhibiting the expression of protein kinase 3
WO2010006973A3 (en) Compositions and methods for inhibiting expression of tgf-beta receptor genes
WO2004065600A3 (en) Rna interference by palindromic or modified rna molecules
WO2007137220A3 (en) Compositions and methods for inhibiting expression of ikk-b gene
WO2004001013A3 (en) Inhibition of gene expression in vertebrates using double-stranded rna (rnai)
HK1159988A1 (en) Compositions and methods for inhibiting expression of factor v leiden mutant gene
WO2005017127A3 (en) Rna interference compositions and methods
WO2010034487A3 (en) Means for inhibiting the expression of orc-1
WO2007121946A3 (en) Means for inhibiting the expression of cd31

Legal Events

Date Code Title Description
AK Designated states

Kind code of ref document: A2

Designated state(s): AE AG AL AM AT AU AZ BA BB BG BR BY BZ CA CH CN CO CR CU CZ DE DK DM DZ EC EE ES FI GB GD GE GH GM HR HU ID IL IN IS JP KE KG KP KR KZ LC LK LR LS LT LU LV MA MD MG MK MN MW MX MZ NO NZ OM PH PL PT RO RU SD SE SG SI SK SL TJ TM TN TR TT TZ UA UG US UZ VN YU ZA ZM ZW

AL Designated countries for regional patents

Kind code of ref document: A2

Designated state(s): GH GM KE LS MW MZ SD SL SZ TZ UG ZM ZW AM AZ BY KG KZ MD RU TJ TM AT BE CH CY DE DK ES FI FR GB GR IE IT LU MC NL PT SE TR BF BJ CF CG CI CM GA GN GQ GW ML MR NE SN TD TG

DFPE Request for preliminary examination filed prior to expiration of 19th month from priority date (pct application filed before 20040101)
121 Ep: the epo has been informed by wipo that ep was designated in this application
WWE Wipo information: entry into national phase

Ref document number: 537/MUMNP/2003

Country of ref document: IN

WWE Wipo information: entry into national phase

Ref document number: 2002710786

Country of ref document: EP

WWE Wipo information: entry into national phase

Ref document number: 2003/04127

Country of ref document: ZA

Ref document number: 200304127

Country of ref document: ZA

WWE Wipo information: entry into national phase

Ref document number: 572/MUMNP/2003

Country of ref document: IN

WWE Wipo information: entry into national phase

Ref document number: 2002229701

Country of ref document: AU

WWE Wipo information: entry into national phase

Ref document number: 2432350

Country of ref document: CA

WWE Wipo information: entry into national phase

Ref document number: 2002556740

Country of ref document: JP

WWE Wipo information: entry into national phase

Ref document number: 028035577

Country of ref document: CN

REG Reference to national code

Ref country code: DE

Ref legal event code: 8642

WWP Wipo information: published in national office

Ref document number: 2002710786

Country of ref document: EP

WWG Wipo information: grant in national office

Ref document number: 2002710786

Country of ref document: EP