CA2521464C - Conjugues arni - Google Patents

Conjugues arni Download PDF

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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
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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
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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

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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
    • 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
US46566503P 2003-04-25 2003-04-25
US46580203P 2003-04-25 2003-04-25
US60/465,802 2003-04-25
US60/465,665 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)

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ES2702942T3 (es) * 2003-04-17 2019-03-06 Alnylam Pharmaceuticals Inc Agentes de ARNi modificados
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
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
EP2377873B1 (fr) 2004-09-24 2014-08-20 Alnylam Pharmaceuticals, Inc. Modulation d'ARNi d'ApoB et utilisations associées
CA2587411A1 (fr) * 2004-11-17 2006-05-26 Protiva Biotherapeutics, Inc. Silence arnsi de l'apolipoproteine b
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
US20060166234A1 (en) 2004-11-22 2006-07-27 Barbara Robertson Apparatus and system having dry control gene silencing compositions
KR20080068019A (ko) * 2005-09-15 2008-07-22 산타리스 팔마 에이/에스 아포지단백질-ｂ100 발현 억제용 ｒｎａ 길항제 화합물
CN101346393B (zh) 2005-11-02 2015-07-22 普洛体维生物治疗公司 修饰的siRNA分子及其应用
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JP5076374B2 (ja) * 2006-06-28 2012-11-21 東レ株式会社 医薬組成物
US8470791B2 (en) 2007-03-22 2013-06-25 Santaris Pharma A/S RNA antagonist compounds for the inhibition of Apo-B100 expression
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
WO2010141511A2 (fr) 2009-06-01 2010-12-09 Halo-Bio Rnai Therapeutics, Inc. Polynucléotides pour interférence arn multivalente, compositions et procédés pour les utiliser
CA2764822A1 (fr) 2009-06-12 2010-12-16 Santaris Pharma A/S Nouveaux composes antisens anti-apob puissants
EP2449106B1 (fr) 2009-07-01 2015-04-08 Protiva Biotherapeutics Inc. Compositions et procédés permettant le silençage de l'apolipoprotéine b
WO2011000107A1 (fr) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Formulations lipidiques inédites permettant l'administration d'agents thérapeutiques en direction de tumeurs solides
AU2011276365B2 (en) 2010-07-06 2017-05-11 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of beta-catenin by double-stranded RNA
EP2802658A2 (fr) 2012-01-09 2014-11-19 Novartis AG Compositions organiques pour traiter des maladies associées à la bêta-caténine
EP2817287B1 (fr) 2012-02-24 2018-10-03 Arbutus Biopharma Corporation Lipides cationiques trialkylés et leurs procédés d'utilisation
US9133461B2 (en) * 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
EP3234134B1 (fr) 2014-12-17 2020-05-27 ProQR Therapeutics II B.V. Édition ciblée d'arn
IL316159A (en) 2015-06-15 2024-12-01 Mpeg La Llc Defined Oligonucleotide Multimers and Methods for Manufacture Thereof
WO2017035278A1 (fr) 2015-08-24 2017-03-02 Halo-Bio Rnai Therapeutics, Inc. Nanoparticules polynucléotidiques pour la modulation de l'expression génique et leurs utilisations
WO2017220751A1 (fr) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Oligonucléotides d'édition d'arn monocaténaire
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
CA3043768A1 (fr) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes destines a l'administration d'agents therapeutiques
EP3571300A1 (fr) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Complexes oligonucléotidiques destinés à être utilisés dans l'édition d'arn
SG11201906728TA (en) 2017-02-06 2019-08-27 Mpeg La Llc Multimeric oligonucleotides having decreased kidney clearance
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
US20230138409A1 (en) 2020-03-24 2023-05-04 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
JP2023520763A (ja) 2020-03-24 2023-05-19 ジェネレーション バイオ カンパニー ゴーシェ治療薬を発現するための非ウイルス性ｄｎａベクター及びその使用
US20240216535A1 (en) 2021-04-27 2024-07-04 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
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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
CN119072330A (zh) 2022-03-14 2024-12-03 世代生物公司 异源初免加强疫苗组合物和使用方法
US20260055426A1 (en) 2022-08-19 2026-02-26 Generation Bio Co. CLEAVABLE CLOSED-ENDED DNA (ceDNA) AND METHODS OF USE THEREOF
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Also Published As

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

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Class et al. Inventors: Muthiah Manoharan (Cambridge, MA, US) Kallanthottathil G. Rajeev (Cambridge, MA, US) David Bumcrot (Cambridge, MA, US)

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Effective date: 20240409