CA2521464C - Conjugues arni - Google Patents

Conjugues arni Download PDF

Info

Publication number
CA2521464C
CA2521464C CA2521464A CA2521464A CA2521464C CA 2521464 C CA2521464 C CA 2521464C CA 2521464 A CA2521464 A CA 2521464A CA 2521464 A CA2521464 A CA 2521464A CA 2521464 C CA2521464 C CA 2521464C
Authority
CA
Canada
Prior art keywords
irna agent
sequence
target
seq
irna
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
CA2521464A
Other languages
English (en)
Other versions
CA2521464A1 (fr
Inventor
Muthiah Manoharan
Sayda Elbashir
Jens Harborth
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 Pharmaceuticals Inc
Original Assignee
Alnylam Pharmaceuticals 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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of CA2521464A1 publication Critical patent/CA2521464A1/fr
Application granted granted Critical
Publication of CA2521464C publication Critical patent/CA2521464C/fr
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/111—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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

L'invention concerne des agents ARNi thérapeutiques ainsi que des procédés de production et d'utilisation associés.
CA2521464A 2003-04-09 2004-04-09 Conjugues arni Expired - Lifetime CA2521464C (fr)

Applications Claiming Priority (31)

Application Number Priority Date Filing Date Title
US46209703P 2003-04-09 2003-04-09
US60/462,097 2003-04-09
US46191503P 2003-04-10 2003-04-10
US60/461,915 2003-04-10
US46289403P 2003-04-14 2003-04-14
US60/462,894 2003-04-14
US46377203P 2003-04-17 2003-04-17
US60/463,772 2003-04-17
US46580203P 2003-04-25 2003-04-25
US46566503P 2003-04-25 2003-04-25
US60/465,665 2003-04-25
US60/465,802 2003-04-25
US46961203P 2003-05-09 2003-05-09
US60/469,612 2003-05-09
US49398603P 2003-08-08 2003-08-08
US60/493,986 2003-08-08
US49459703P 2003-08-11 2003-08-11
US60/494,597 2003-08-11
US50634103P 2003-09-26 2003-09-26
US60/506,341 2003-09-26
US51024603P 2003-10-09 2003-10-09
US60/510,246 2003-10-09
US51031803P 2003-10-10 2003-10-10
US60/510,318 2003-10-10
US51845303P 2003-11-07 2003-11-07
US60/518,453 2003-11-07
US40707004A 2004-03-08 2004-03-08
USUS04/07070 2004-03-08
US41058604A 2004-04-05 2004-04-05
USUS04/10586 2004-04-05
PCT/US2004/011255 WO2004091515A2 (fr) 2003-04-09 2004-04-09 Conjugues arni

Publications (2)

Publication Number Publication Date
CA2521464A1 CA2521464A1 (fr) 2004-10-28
CA2521464C true CA2521464C (fr) 2013-02-05

Family

ID=39524922

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2521464A Expired - Lifetime CA2521464C (fr) 2003-04-09 2004-04-09 Conjugues arni

Country Status (3)

Country Link
AU (1) AU2004229519B2 (fr)
CA (1) CA2521464C (fr)
WO (1) WO2004091515A2 (fr)

Families Citing this family (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
US8017762B2 (en) 2003-04-17 2011-09-13 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
US8796436B2 (en) 2003-04-17 2014-08-05 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
DK1620544T3 (en) 2003-04-17 2019-01-14 Alnylam Pharmaceuticals Inc MODIFIED iRNA AGENTS
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
US7528118B2 (en) 2004-09-24 2009-05-05 Alnylam Pharmaceuticals, Inc. RNAi modulation of ApoB and uses thereof
CA2587411A1 (fr) * 2004-11-17 2006-05-26 Protiva Biotherapeutics, Inc. Silence arnsi de l'apolipoproteine b
US20060166234A1 (en) 2004-11-22 2006-07-27 Barbara Robertson Apparatus and system having dry control gene silencing compositions
US7923207B2 (en) 2004-11-22 2011-04-12 Dharmacon, Inc. Apparatus and system having dry gene silencing pools
US7935811B2 (en) 2004-11-22 2011-05-03 Dharmacon, Inc. Apparatus and system having dry gene silencing compositions
AU2006291836B2 (en) * 2005-09-15 2012-02-23 Santaris Pharma A/S RNA antagonist compounds for the inhibition of Apo-Bl00 expression
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
JP5076374B2 (ja) * 2006-06-28 2012-11-21 東レ株式会社 医薬組成物
CA2681406A1 (fr) 2007-03-22 2008-09-25 Santaris Pharma A/S Composes arn antagonistes pour l'inhibition de l'expression de l'apo-b100
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
KR101141544B1 (ko) 2009-03-13 2012-05-03 한국과학기술원 에스아이알엔에이 다중 접합체 및 이의 제조방법
ES2804764T3 (es) 2009-06-01 2021-02-09 Halo Bio Rnai Therapeutics Inc Polinucleótidos para la interferencia de ARN multivalente, composiciones y métodos de uso de los mismos
ES2555057T3 (es) 2009-06-12 2015-12-28 Roche Innovation Center Copenhagen A/S Nuevos potentes compuestos antisentido anti-ApoB
US8236943B2 (en) 2009-07-01 2012-08-07 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
DK3037538T3 (da) 2010-07-06 2019-06-24 Dicerna Pharmaceuticals Inc Fremgangsmåder og sammensætninger til specifik inhibering af beta-catenin ved hjælp af dobbeltstrenget rna
CA2860676A1 (fr) 2012-01-09 2013-07-18 Novartis Ag Compositions organiques pour traiter des maladies associees a la beta-catenine
SG11201405157PA (en) 2012-02-24 2014-10-30 Protiva Biotherapeutics Inc Trialkyl cationic lipids and methods of use thereof
US9133461B2 (en) * 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
US10676737B2 (en) 2014-12-17 2020-06-09 Proqr Therapeutics Ii B.V. Targeted RNA editing
IL316159A (en) 2015-06-15 2024-12-01 Mpeg La Llc Defined Oligonucleotide Multimers and Methods for Manufacture Thereof
JP2018525037A (ja) 2015-08-24 2018-09-06 ハロー−バイオ・アールエヌエーアイ・セラピューティックス、インコーポレイテッド 遺伝子発現の調節のためのポリヌクレオチドナノ粒子及びその使用
EP3475424A1 (fr) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Oligonucléotides d'édition d'arn monocaténaire
US10941402B2 (en) 2016-09-01 2021-03-09 Proqr Therapeutics Ii B.V. Chemically modified single-stranded RNA-editing oligonucleotides
EP4035659A1 (fr) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosomes destinés à l'administration d'agents thérapeutiques
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
EP3576752A4 (fr) 2017-02-06 2020-12-16 Mpeg La, LLC Oligonucléotides multimères ayant une clairance rénale diminuée
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
MX2022011805A (es) 2020-03-24 2023-01-11 Generation Bio Co Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix.
AU2021244559A1 (en) 2020-03-24 2022-11-17 Generation Bio Co. Non-viral DNA vectors and uses thereof for expressing Gaucher therapeutics
CN117881786A (zh) 2021-04-27 2024-04-12 世代生物公司 表达治疗性抗体的非病毒dna载体及其用途
EP4329884A1 (fr) 2021-04-27 2024-03-06 Generation Bio Co. Vecteurs d'adn non viraux exprimant des anticorps anti-coronavirus et leurs utilisations
CN120775847A (zh) * 2021-10-20 2025-10-14 厦门甘宝利生物医药有限公司 Agt抑制剂及其用途
WO2023177655A1 (fr) 2022-03-14 2023-09-21 Generation Bio Co. Compositions vaccinales prime-boost hétérologues et méthodes d'utilisation
WO2024040222A1 (fr) 2022-08-19 2024-02-22 Generation Bio Co. Adn à extrémités fermées clivable (adnce) et ses procédés d'utilisation
CN118011012B (zh) * 2024-02-19 2025-01-21 中国科学院昆明动物研究所 LL-37-ApoB-100生物标志物及其应用

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304620D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Compounds
US6133246A (en) * 1997-08-13 2000-10-17 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the modulation of JNK proteins
US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
US20040259247A1 (en) 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
AU2002240889A1 (en) * 2000-12-18 2002-07-01 Hybrigenics Proteins that interact with betatrcp
US7407943B2 (en) * 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
WO2003097662A1 (fr) * 2002-05-15 2003-11-27 Isis Pharmaceuticals, Inc. Modulation antisens de l'expression de l'apolipoproteine b
US9150605B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation

Also Published As

Publication number Publication date
WO2004091515A3 (fr) 2007-07-26
AU2004229519A1 (en) 2004-10-28
CA2521464A1 (fr) 2004-10-28
WO2004091515A2 (fr) 2004-10-28
AU2004229519B2 (en) 2011-07-21

Similar Documents

Publication Publication Date Title
US11530408B2 (en) Therapeutic compositions
CA2521464C (fr) Conjugues arni
CA2522637C (fr) Agents modifies d'arni
US20050153337A1 (en) iRNA conjugates
EP1615611B1 (fr) Conjugues arni
AU2017201200A1 (en) Modified iRNA agents
Class et al. Inventors: Muthiah Manoharan (Cambridge, MA, US) Kallanthottathil G. Rajeev (Cambridge, MA, US) David Bumcrot (Cambridge, MA, US)

Legal Events

Date Code Title Description
EEER Examination request
MKEX Expiry

Effective date: 20240409