JP2011528910A - センス鎖中の一般塩基またはミスマッチを使用したｓｉＲＮＡサイレンシング活性の亢進 - Google Patents

センス鎖中の一般塩基またはミスマッチを使用したｓｉＲＮＡサイレンシング活性の亢進 Download PDF

Info

Publication number
JP2011528910A
JP2011528910A JP2011520219A JP2011520219A JP2011528910A JP 2011528910 A JP2011528910 A JP 2011528910A JP 2011520219 A JP2011520219 A JP 2011520219A JP 2011520219 A JP2011520219 A JP 2011520219A JP 2011528910 A JP2011528910 A JP 2011528910A
Authority
JP
Japan
Prior art keywords
sirna
irna agent
sense strand
gene
expression
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.)
Pending
Application number
JP2011520219A
Other languages
English (en)
Japanese (ja)
Other versions
JP2011528910A5 (enExample
Inventor
マイアー，マーティン
アッデパリ，ハリプリヤ
マノハラン，ムシアー
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 JP2011528910A publication Critical patent/JP2011528910A/ja
Publication of JP2011528910A5 publication Critical patent/JP2011528910A5/ja
Pending legal-status Critical Current

Links

Images

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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/33—Chemical structure of the base
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/331—Universal or degenerate base
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2011520219A 2008-07-25 2009-07-24 センス鎖中の一般塩基またはミスマッチを使用したｓｉＲＮＡサイレンシング活性の亢進 Pending JP2011528910A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US8376308P 2008-07-25 2008-07-25
US61/083,763 2008-07-25
PCT/US2009/051648 WO2010011895A1 (en) 2008-07-25 2009-07-24 Enhancement of sirna silencing activity using universal bases or mismatches in the sense strand

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2016189465A Division JP6476155B2 (ja) 2008-07-25 2016-09-28 センス鎖中の一般塩基またはミスマッチを使用したｓｉＲＮＡサイレンシング活性の亢進

Publications (2)

Publication Number Publication Date
JP2011528910A true JP2011528910A (ja) 2011-12-01
JP2011528910A5 JP2011528910A5 (enExample) 2012-11-29

Family

ID=41258560

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2011520219A Pending JP2011528910A (ja) 2008-07-25 2009-07-24 センス鎖中の一般塩基またはミスマッチを使用したｓｉＲＮＡサイレンシング活性の亢進
JP2016189465A Active JP6476155B2 (ja) 2008-07-25 2016-09-28 センス鎖中の一般塩基またはミスマッチを使用したｓｉＲＮＡサイレンシング活性の亢進

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2016189465A Active JP6476155B2 (ja) 2008-07-25 2016-09-28 センス鎖中の一般塩基またはミスマッチを使用したｓｉＲＮＡサイレンシング活性の亢進

Country Status (6)

Country Link
US (2) US20110223665A1 (enExample)
EP (1) EP2321414B1 (enExample)
JP (2) JP2011528910A (enExample)
AU (2) AU2009273878A1 (enExample)
CA (1) CA2732229C (enExample)
WO (1) WO2010011895A1 (enExample)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015518704A (ja) * 2012-04-02 2015-07-06 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 膜タンパク質の産生のための修飾ポリヌクレオチド
WO2018143475A1 (ja) * 2017-02-06 2018-08-09 日産化学工業株式会社 一本鎖オリゴヌクレオチド
JP2022538404A (ja) * 2019-06-21 2022-09-02 アルナイラム ファーマシューティカルズ， インコーポレイテッド 構造的に明確なｓｉＲＮＡ－二重可変ドメイン免疫グロブリンコンジュゲート
US11530409B2 (en) 2016-01-26 2022-12-20 Nissan Chemical Corporation Single-stranded oligonucleotide
JP2024515734A (ja) * 2021-04-23 2024-04-10 南▲開▼大学 コンジュゲート、その製造方法、及び使用

Families Citing this family (87)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8796436B2 (en) 2003-04-17 2014-08-05 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
EP2660322A3 (en) 2003-04-17 2013-11-13 Alnylam Pharmaceuticals Inc. Modified iRNA agents
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
SG10201500318SA (en) 2008-12-03 2015-03-30 Arcturus Therapeutics Inc UNA Oligomer Structures For Therapeutic Agents
US20150025122A1 (en) 2009-10-12 2015-01-22 Larry J. Smith Methods and Compositions for Modulating Gene Expression Using Oligonucleotide Based Drugs Administered in vivo or in vitro
WO2011109427A2 (en) 2010-03-01 2011-09-09 Alnylam Pharmaceuticals, Inc. Improving the biological activity of sirna through modulation of its thermodynamic profile
WO2012007477A1 (en) 2010-07-12 2012-01-19 Santaris Pharma A/S Anti hcv oligomers
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US9750819B2 (en) * 2012-05-23 2017-09-05 Ohio State Innovation Foundation Lipid nanoparticle compositions and methods of making and methods of using the same
US10059942B2 (en) 2012-05-31 2018-08-28 Bio-Lab Ltd. Therapeutic oligonucleotides comprising pyrazolotriazine nucleotide analogues
WO2014066142A1 (en) * 2012-10-24 2014-05-01 Novamedica Limited Liability Company Nucleic acid regulation of growth arrest-specific protein 6 (gas6)
WO2014144423A2 (en) * 2013-03-15 2014-09-18 Techulon Inc. Antisense molecules for treatment of staphylococcus aureus infection
IL316808A (en) * 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
US20170369872A1 (en) 2014-12-18 2017-12-28 Alnylam Pharmaceuticals, Inc. Reversir tm compounds
US11045488B2 (en) 2014-12-26 2021-06-29 Nitto Denko Corporation RNA interference agents for GST-π gene modulation
US10264976B2 (en) 2014-12-26 2019-04-23 The University Of Akron Biocompatible flavonoid compounds for organelle and cell imaging
JP6892433B2 (ja) 2015-04-03 2021-06-23 ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ 十分に安定化された非対称ｓｉｒｎａ
US11053495B2 (en) 2015-07-17 2021-07-06 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
CA2995110A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
US11208653B2 (en) 2016-04-27 2021-12-28 Whitehead Institute For Biomedical Research Increasing RNAi efficiency through single nucleotide mismatches
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
TW202313978A (zh) 2016-11-23 2023-04-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
EP3642341A4 (en) 2017-06-23 2021-06-16 University Of Massachusetts TWO-DAY SELF-RELEASING SIRNA AND RELATED PROCEDURES
MX2020011570A (es) 2018-05-07 2020-11-24 Alnylam Pharmaceuticals Inc Administracion extrahepatica.
JP7627042B2 (ja) * 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
AU2019350765A1 (en) 2018-09-28 2021-05-27 Alnylam Pharmaceuticals, Inc. Transthyretin (TTR) iRNA compositions and methods of use thereof for treating or preventing TTR-associated ocular diseases
JP2022523467A (ja) 2019-01-18 2022-04-25 ユニバーシティ・オブ・マサチューセッツ 動的な薬物動態を修飾するアンカー
JP2022532652A (ja) 2019-05-17 2022-07-15 アルニラム ファーマスーティカルズ インコーポレイテッド オリゴヌクレオチドの経口送達
CA3149835A1 (en) 2019-08-09 2021-02-18 University Of Massachusetts Chemically modified oligonucleotides targeting snps
EP4013870A1 (en) 2019-08-13 2022-06-22 Alnylam Pharmaceuticals, Inc. Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
WO2021067747A1 (en) 2019-10-04 2021-04-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
AR120341A1 (es) 2019-11-01 2022-02-09 Alnylam Pharmaceuticals Inc COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
EP4051796A1 (en) 2019-11-01 2022-09-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
US20230016929A1 (en) 2019-11-06 2023-01-19 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
EP4055165A1 (en) 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
WO2021102373A1 (en) 2019-11-22 2021-05-27 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
AU2020402885A1 (en) 2019-12-13 2022-06-16 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (C9orf72) iRNA agent compositions and methods of use thereof
US20230136787A1 (en) 2020-02-10 2023-05-04 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing vegf-a expression
EP4114947A1 (en) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
TW202204615A (zh) 2020-03-26 2022-02-01 美商阿尼拉製藥公司 冠狀病毒ｉＲＮＡ組成物及其使用方法
EP4127171A2 (en) 2020-03-30 2023-02-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
KR20230008729A (ko) 2020-04-06 2023-01-16 알닐람 파마슈티칼스 인코포레이티드 Myoc 발현을 사일런싱하기 위한 조성물 및 방법
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
EP4133079A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
WO2021206917A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
BR112022021813A2 (pt) 2020-04-27 2023-01-17 Alnylam Pharmaceuticals Inc Composições de agente de apolipoproteína e (apoe) irna e métodos de uso das mesmas
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
KR102361479B1 (ko) * 2020-06-18 2022-02-11 고려대학교 산학협력단 ｓｉＲＮＡ의 생성 및 기능 증진 활성을 갖는 ＣＭＴＲ１의 신규용도
WO2022011214A1 (en) 2020-07-10 2022-01-13 Alnylam Pharmaceuticals, Inc. Circular sirnas
WO2022066847A1 (en) 2020-09-24 2022-03-31 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
TW202229552A (zh) 2020-10-05 2022-08-01 美商艾拉倫製藥股份有限公司 Ｇ蛋白－偶合受體７５（ＧＰＲ７５）ｉＲＮＡ組成物及其使用方法
WO2022087329A1 (en) 2020-10-23 2022-04-28 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
JP2024501857A (ja) 2020-12-31 2024-01-16 アルナイラム ファーマシューティカルズ， インコーポレイテッド 環状ジスルフィド修飾リン酸ベースのオリゴヌクレオチドプロドラッグ
EP4271695A2 (en) 2020-12-31 2023-11-08 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
AU2022220704A1 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
WO2022182864A1 (en) 2021-02-25 2022-09-01 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods and methods of use thereof
CN114958839A (zh) * 2021-02-25 2022-08-30 上海市东方医院（同济大学附属东方医院） 抑制肌肉瘤细胞中FOXO1基因表达的siRNA序列及应用
EP4305169A1 (en) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
CN117203336A (zh) 2021-03-29 2023-12-08 阿尔尼拉姆医药品有限公司 亨廷顿蛋白(HTT)iRNA药剂组合物以及其使用方法
EP4341401A1 (en) 2021-05-18 2024-03-27 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
JP2024521907A (ja) 2021-06-04 2024-06-04 アルナイラム ファーマシューティカルズ， インコーポレイテッド ヒト９番染色体オープンリーディングフレーム７２（Ｃ９ＯＲＦ７２）ｉＲＮＡ剤組成物及びその使用方法
WO2022271786A1 (en) 2021-06-23 2022-12-29 University Of Massachusetts Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
US20250270562A1 (en) 2021-07-09 2025-08-28 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
WO2023001894A1 (en) 2021-07-20 2023-01-26 Ags Therapeutics Sas Extracellular vesicles from microalgae, their preparation, and uses
AU2022314619A1 (en) 2021-07-21 2024-01-04 Alnylam Pharmaceuticals, Inc. Metabolic disorder-associated target gene irna compositions and methods of use thereof
CA3230404A1 (en) 2021-08-31 2023-03-09 Alnylam Pharmaceuticals, Inc. Cell death-inducing dffa-like effector b (cideb) irna compositions and methods of use thereof
AU2022364838A1 (en) 2021-10-15 2024-04-11 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
JP2024541974A (ja) 2021-10-29 2024-11-13 アルナイラム ファーマシューティカルズ， インコーポレイテッド ハンチンチン（ＨＴＴ）ｉＲＮＡ剤組成物およびその使用方法
EP4469575A2 (en) 2022-01-24 2024-12-04 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2023144127A1 (en) 2022-01-31 2023-08-03 Ags Therapeutics Sas Extracellular vesicles from microalgae, their biodistribution upon administration, and uses
AU2023234536A1 (en) 2022-03-16 2024-09-26 Empirico Inc. Galnac compositions for improving sirna bioavailability
CN119278265A (zh) 2022-03-28 2025-01-07 艾姆皮瑞克公司 经修饰的寡核苷酸
US20250304957A1 (en) 2022-05-13 2025-10-02 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
WO2023232976A1 (en) 2022-06-03 2023-12-07 Ags Therapeutics Sas Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses
EP4547683A2 (en) 2022-06-30 2025-05-07 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
WO2024073732A1 (en) 2022-09-30 2024-04-04 Alnylam Pharmaceuticals, Inc. Modified double-stranded rna agents
JP2025535944A (ja) 2022-10-24 2025-10-30 エイジーエス・セラピューティクス・ソシエテ・パール・アクシオン・サンプリフィエ 微細藻類由来の細胞外小胞、鼻腔内投与時のそれらの生体内分布、及びそれらの使用
CN121241143A (zh) * 2023-03-23 2025-12-30 阿塔兰塔治疗公司 用于改善的基因沉默的组合物和方法
WO2024216155A1 (en) 2023-04-12 2024-10-17 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery of double-stranded rna agents
WO2024238385A2 (en) 2023-05-12 2024-11-21 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
WO2025064660A2 (en) 2023-09-21 2025-03-27 Alnylam Pharmaceuticals, Inc. Activin a receptor type 1c (acvr1c) irna compositions and methods of use thereof
WO2025176843A1 (en) 2024-02-21 2025-08-28 Ags Therapeutics Sas Microalgae extracellular vesicle based gene therapy vectors (mev-gtvs), their preparation, and uses thereof
WO2025259747A2 (en) 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070054279A1 (en) * 2004-08-04 2007-03-08 Alnylam Pharmaceuticals Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase
WO2007053696A2 (en) * 2005-11-01 2007-05-10 Alnylam Pharmaceuticals, Inc. Rnai inhibition of influenza virus replication

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4897355A (en) * 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
FR2645866B1 (fr) * 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5283185A (en) * 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5691316A (en) * 1994-06-01 1997-11-25 Hybridon, Inc. Cyclodextrin cellular delivery system for oligonucleotides
US6509323B1 (en) * 1998-07-01 2003-01-21 California Institute Of Technology Linear cyclodextrin copolymers
US6066500A (en) * 1999-06-25 2000-05-23 Isis Pharmaceuticals Inc. Antisense modulation of Beta catenin expression
US20070026394A1 (en) * 2000-02-11 2007-02-01 Lawrence Blatt Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies
US6627425B1 (en) * 2000-06-02 2003-09-30 Millennium Pharmaceuticals, Inc. Human glucose-6-phosphatase molecules and uses thereof
TWI321054B (en) * 2000-12-19 2010-03-01 California Inst Of Techn Compositions containing inclusion complexes
US7517864B2 (en) * 2001-05-18 2009-04-14 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)
US7888324B2 (en) * 2001-08-01 2011-02-15 Genzyme Corporation Antisense modulation of apolipoprotein B expression
AU2002343792A1 (en) * 2001-11-28 2003-06-10 Center For Advanced Science And Technology Incubation, Ltd. siRNA EXPRESSION SYSTEM AND PROCESS FOR PRODUCING FUNCTIONAL GENE-KNOCKDOWN CELLS AND THE LIKE USING THE SAME
US20050233342A1 (en) * 2003-03-07 2005-10-20 Muthiah Manoharan Methods of preventing off-target gene silencing
US20040198640A1 (en) * 2003-04-02 2004-10-07 Dharmacon, Inc. Stabilized polynucleotides for use in RNA interference
EP1633890B2 (en) * 2003-06-02 2020-11-18 University of Massachusetts METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF FNAi
EP1666593A4 (en) * 2003-08-18 2009-07-15 Japan Health Science Found IMPROVED SIRNA MOLECULE AND METHOD FOR INHIBITING GENE EXPRESSION USED THEREOF
US20080249039A1 (en) * 2004-01-30 2008-10-09 Santaris Pharma A/S Modified Short Interfering Rna (Modified Sirna)
US20080213891A1 (en) * 2004-07-21 2008-09-04 Alnylam Pharmaceuticals, Inc. RNAi Agents Comprising Universal Nucleobases
AU2005328382C1 (en) * 2004-07-21 2013-01-24 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a modified or non-natural nucleobase
US7592322B2 (en) * 2004-10-22 2009-09-22 Alnylam Pharmaceuticals, Inc. RNAi modulation of RSV, PIV and other respiratory viruses and uses thereof
US20110269814A1 (en) * 2008-03-26 2011-11-03 Alnylam Pharamaceuticals, Inc. 2'-f modified rna interference agents
JP2012147677A (ja) * 2009-05-14 2012-08-09 Nippon Shinyaku Co Ltd 二本鎖修飾ｒｎａ

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070054279A1 (en) * 2004-08-04 2007-03-08 Alnylam Pharmaceuticals Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase
WO2007053696A2 (en) * 2005-11-01 2007-05-10 Alnylam Pharmaceuticals, Inc. Rnai inhibition of influenza virus replication

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
JPN6014004590; ＡＣＳ　ＣＨＥＭＩＣＡＬ　ＢＩＯＬＯＧＹ ｖｏｌ．１　ｎｏ．３, 2006, ｐｐ．１７６-１８３ *
JPN6014004598; ＭＯＬＥＣＵＬＡＲ　ＴＨＥＲＡＰＹ ｖｏｌ．１３　ｎｏ．３, 2006, ｐｐ．４９４-５０５ *
JPN6014004601; ＣＨＥＭＩＳＴＲＹ　＆　ＢＩＯＤＩＶＥＲＳＩＴＹ ｖｏｌ．４, 2007, ｐｐ．８５８-８７３ *
JPN6014004604; Ｊ．　Ｍｅｄ．　Ｃｈｅｍ． ｖｏｌ．４８, 2005, ｐｐ．４２４７-４２５３ *
JPN6014004609; Ｎｕｃｌｅｉｃ　Ａｃｉｄｓ　Ｒｅｓｅａｒｃｈ ｖｏｌ．３５　ｎｏ．１９, 2007, ｐｐ．６４２４-６４３８ *

Cited By (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015518704A (ja) * 2012-04-02 2015-07-06 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 膜タンパク質の産生のための修飾ポリヌクレオチド
US11530409B2 (en) 2016-01-26 2022-12-20 Nissan Chemical Corporation Single-stranded oligonucleotide
WO2018143475A1 (ja) * 2017-02-06 2018-08-09 日産化学工業株式会社 一本鎖オリゴヌクレオチド
JPWO2018143475A1 (ja) * 2017-02-06 2019-12-12 日産化学株式会社 一本鎖オリゴヌクレオチド
JP7188087B2 (ja) 2017-02-06 2022-12-13 日産化学株式会社 一本鎖オリゴヌクレオチド
US11572558B2 (en) 2017-02-06 2023-02-07 Nissan Chemical Corporation Single-stranded oligonucleotide
JP2023025156A (ja) * 2017-02-06 2023-02-21 日産化学株式会社 一本鎖オリゴヌクレオチド
JP7601080B2 (ja) 2017-02-06 2024-12-17 日産化学株式会社 一本鎖オリゴヌクレオチド
JP2022538404A (ja) * 2019-06-21 2022-09-02 アルナイラム ファーマシューティカルズ， インコーポレイテッド 構造的に明確なｓｉＲＮＡ－二重可変ドメイン免疫グロブリンコンジュゲート
JP7785543B2 (ja) 2019-06-21 2025-12-15 アルナイラム ファーマシューティカルズ， インコーポレイテッド 構造的に明確なｓｉＲＮＡ－二重可変ドメイン免疫グロブリンコンジュゲート
JP2024515734A (ja) * 2021-04-23 2024-04-10 南▲開▼大学 コンジュゲート、その製造方法、及び使用
JP7668378B2 (ja) 2021-04-23 2025-04-24 コーズィケム・バイオテクノロジー・（テンシン）・カンパニー・リミテッド コンジュゲート、その製造方法、及び使用

Also Published As

Publication number Publication date
WO2010011895A1 (en) 2010-01-28
US20110223665A1 (en) 2011-09-15
AU2009273878A1 (en) 2010-01-28
JP6476155B2 (ja) 2019-02-27
CA2732229A1 (en) 2010-01-28
US20130196434A1 (en) 2013-08-01
AU2016202344B2 (en) 2017-11-30
JP2017002079A (ja) 2017-01-05
EP2321414B1 (en) 2018-01-10
EP2321414A1 (en) 2011-05-18
AU2016202344A1 (en) 2016-05-05
CA2732229C (en) 2023-10-17

Similar Documents

Publication Publication Date Title
JP6476155B2 (ja) センス鎖中の一般塩基またはミスマッチを使用したｓｉＲＮＡサイレンシング活性の亢進
JP6850827B2 (ja) オリゴヌクレオチドの送達剤としての糖質コンジュゲート
JP2011528910A5 (enExample)
JP5788312B2 (ja) 標的リガンドをエンドソーム分解性成分と組み合わせることによる核酸の部位特異的送達
KR102534909B1 (ko) 변형된 ＲＮＡｉ 제제
ES2702942T3 (es) Agentes de ARNi modificados
JP2021073183A (ja) 修飾二本鎖ｒｎａ剤
US20110269814A1 (en) 2'-f modified rna interference agents
JP4991288B2 (ja) 二本鎖ｉＲＮＡ剤、および二本鎖ｉＲＮＡ剤の対合の安定性を調節する方法。
JP2006522158A (ja) ｉＲＮＡ複合体
WO2011109427A2 (en) Improving the biological activity of sirna through modulation of its thermodynamic profile
AU2019204150A1 (en) Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components

Legal Events

Date Code Title Description
A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20120724

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20121009

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20140204

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20140507

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20140514

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20140602

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20150120

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20160629

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20160928