CN110520531A - 产品和组合物 - Google Patents

产品和组合物 Download PDF

Info

Publication number
CN110520531A
CN110520531A CN201880021538.7A CN201880021538A CN110520531A CN 110520531 A CN110520531 A CN 110520531A CN 201880021538 A CN201880021538 A CN 201880021538A CN 110520531 A CN110520531 A CN 110520531A
Authority
CN
China
Prior art keywords
strand
nucleotides
modified
nucleic acid
positions
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
CN201880021538.7A
Other languages
English (en)
Chinese (zh)
Inventor
J.豪普特曼
D.萨马尔斯基
C.弗劳恩多夫
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.)
Selens Treatment Co Ltd
Original Assignee
Selens Treatment Co Ltd
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=61899311&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CN110520531(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from GBGB1707203.4A external-priority patent/GB201707203D0/en
Priority claimed from GBGB1708397.3A external-priority patent/GB201708397D0/en
Application filed by Selens Treatment Co Ltd filed Critical Selens Treatment Co Ltd
Publication of CN110520531A publication Critical patent/CN110520531A/zh
Pending 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or 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
    • 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
    • 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
    • C07H21/04—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 deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • 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
    • C12N2310/313—Phosphorodithioates
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Compositions Of Macromolecular Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
CN201880021538.7A 2017-04-05 2018-04-05 产品和组合物 Pending CN110520531A (zh)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
EP17165129 2017-04-05
EP17165129.2 2017-04-05
GBGB1707203.4A GB201707203D0 (en) 2017-05-05 2017-05-05 Modified positions
GB1707203.4 2017-05-05
GBGB1708397.3A GB201708397D0 (en) 2017-05-25 2017-05-25 Modified positions
GB1708397.3 2017-05-25
EP17201352 2017-11-13
EP17201352.6 2017-11-13
PCT/EP2018/058766 WO2018185241A1 (en) 2017-04-05 2018-04-05 Products and compositions

Publications (1)

Publication Number Publication Date
CN110520531A true CN110520531A (zh) 2019-11-29

Family

ID=61899311

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201880021538.7A Pending CN110520531A (zh) 2017-04-05 2018-04-05 产品和组合物

Country Status (16)

Country Link
US (3) US11015198B2 (https=)
EP (2) EP3607069B1 (https=)
JP (3) JP7155239B2 (https=)
CN (1) CN110520531A (https=)
AU (1) AU2018247925B2 (https=)
DK (1) DK3607069T3 (https=)
ES (1) ES2932831T3 (https=)
FI (1) FI3607069T3 (https=)
HR (1) HRP20221400T1 (https=)
HU (1) HUE061247T2 (https=)
IL (2) IL321221A (https=)
LT (1) LT3607069T (https=)
PL (1) PL3607069T3 (https=)
RS (1) RS63836B1 (https=)
SI (1) SI3607069T1 (https=)
WO (1) WO2018185241A1 (https=)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116761616A (zh) * 2021-01-22 2023-09-15 阿耐拉姆制药公司 经修饰的双链寡核苷酸
WO2024245151A1 (zh) * 2023-05-26 2024-12-05 维亚臻生物技术（苏州）有限公司 一种用于代谢疾病的双链核苷酸化合物及其应用
WO2025031444A1 (zh) * 2023-08-09 2025-02-13 北京福元医药股份有限公司 抑制DGAT2基因表达的siRNA、siRNA缀合物或其前药和药物组合物及用途
WO2026011779A1 (zh) * 2024-07-10 2026-01-15 凯莱英医药集团（天津）股份有限公司 制备Cemdisiran的方法

Families Citing this family (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
EP3483269A1 (en) * 2017-11-13 2019-05-15 Silence Therapeutics GmbH Products and compositions
EP4029942A1 (en) 2017-04-05 2022-07-20 Silence Therapeutics GmbH Products and compositions
WO2018185240A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions
JP7155239B2 (ja) 2017-04-05 2022-10-18 サイレンス・セラピューティクス・ゲーエムベーハー 産物及び組成物
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
WO2019006455A1 (en) 2017-06-30 2019-01-03 Solstice Biologics, Ltd. AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF
MX2020004897A (es) 2017-11-13 2020-10-05 Silence Therapeutics Gmbh Acidos nucleicos para inhibir la expresion de lpa en una celula.
CA3083968C (en) * 2017-12-01 2024-04-23 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method thereof and use thereof
WO2019143621A1 (en) 2018-01-16 2019-07-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting aldh2 expression
EP3598995A1 (en) * 2018-07-26 2020-01-29 Silence Therapeutics GmbH Products and compositions
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
BR112021009213A2 (pt) 2018-11-13 2021-08-10 Silence Therapeutics Gmbh ácidos nucleicos para inibição da expressão de lpa em uma célula
KR20210116509A (ko) * 2019-01-16 2021-09-27 젠자임 코포레이션 Serpinc1 irna 조성물 및 그의 이용 방법
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
CA3135402A1 (en) * 2019-04-04 2020-10-08 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression in the central nervous system
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
JP2022545118A (ja) * 2019-08-23 2022-10-25 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
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
EP4048801A4 (en) * 2019-10-24 2023-11-22 Genevant Sciences Gmbh Conjugates and methods for treating acromegaly
KR20220163960A (ko) 2020-03-06 2022-12-12 알리고스 테라퓨틱스 인코포레이티드 변경된 짧은 개재성 핵산(siNA) 분자 및 이의 용도
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
JP2023549011A (ja) 2020-09-15 2023-11-22 ヴァーヴ・セラピューティクス，インコーポレーテッド 遺伝子編集のための脂質製剤
AU2021373262A1 (en) 2020-11-04 2023-06-01 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of pros1 in a cell
WO2022184852A1 (en) * 2021-03-03 2022-09-09 Silence Therapeutics Gmbh Conjugated nucleic acids comprising a phosphorodithioate for inhibiting gene expression in a cell
WO2022204430A1 (en) * 2021-03-24 2022-09-29 Atalanta Therapeutics, Inc. Double-stranded sirna having patterned chemical modifications
AU2022299169A1 (en) 2021-06-23 2024-02-08 Beth Israel Deaconess Medical Center, Inc. Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
IL312811A (en) 2021-11-16 2024-07-01 Shanghai Argo Biopharmaceutical Co Ltd Composition and method for inhibiting angiotensinogen (agt) protein expression
EP4493570A2 (en) 2022-03-16 2025-01-22 Empirico Inc. Galnac compositions for improving sirna bioavailability
KR20240163743A (ko) 2022-03-28 2024-11-19 엠피리코 인크. 변형된 올리고뉴클레오티드
EP4687983A1 (en) 2023-03-27 2026-02-11 Silence Therapeutics GmbH Compounds and compositions for use in stem cell transplantation
TW202602463A (zh) 2024-03-22 2026-01-16 日商武田藥品工業股份有限公司 用於抑制細胞色素ｐ４５０家族７次家族ａ成員１（ｃｙｐ７ａ１）表現之組成物及方法

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007112414A2 (en) * 2006-03-27 2007-10-04 Isis Pharmaceuticals, Inc. Conjugated double strand compositions for use in gene modulation
WO2009002944A1 (en) * 2007-06-22 2008-12-31 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
CN104854242A (zh) * 2012-12-05 2015-08-19 阿尔尼拉姆医药品有限公司 PCSK9 iRNA组合物及其使用方法
CN105452463A (zh) * 2013-05-22 2016-03-30 阿尔尼拉姆医药品有限公司 Tmprss6 irna组合物及其使用方法
WO2016085852A1 (en) * 2014-11-24 2016-06-02 Alnylam Pharmaceuticals, Inc. Tmprss6 irna compositions and methods of use thereof
WO2016161388A1 (en) * 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna

Family Cites Families (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
US7667030B2 (en) 2002-02-20 2010-02-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of matrix metalloproteinase 13 (MMP13) gene expression using short interfering nucleic acid (siNA)
US9150605B2 (en) 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
KR20080036650A (ko) 2005-08-17 2008-04-28 시르나 쎄러퓨틱스 인코퍼레이티드 Ｒｎａ 간섭을 매개하는 화학적으로 변형된 짧은 간섭 핵산분자
WO2009082607A2 (en) 2007-12-04 2009-07-02 Alnylam Pharmaceuticals, Inc. Targeting lipids
MX2010008394A (es) 2008-01-31 2010-11-12 Alnylam Pharmaceuticals Inc Metodos optimizados para administracion de arndc focalizando el gen pcsk9.
US10131904B2 (en) 2008-02-11 2018-11-20 Rxi Pharmaceuticals Corporation Modified RNAi polynucleotides and uses thereof
EP2342616A2 (en) 2008-09-23 2011-07-13 Alnylam Pharmaceuticals Inc. Chemical modifications of monomers and oligonucleotides with cycloaddition
US9493774B2 (en) * 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
CA2812046A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
WO2012094115A1 (en) 2010-12-17 2012-07-12 Arrowhead Research Corporation Compositions and methods for inhibiting expression of flt3 genes
CA2834610A1 (en) * 2011-06-06 2012-12-13 Merck Sharp & Dohme Corp. Rna interference mediated inhibition of isocitrate dehydrogenase (idh1) gene expression
HRP20181564T1 (hr) 2011-06-30 2018-11-30 Arrowhead Pharmaceuticals, Inc. Pripravci i postupci za inhibiciju ekspresije gena virusa hepatitisa b
EP2780454A2 (en) * 2011-11-18 2014-09-24 Alnylam Pharmaceuticals, Inc. Modified rnai agents
HUE048622T2 (hu) * 2011-11-18 2020-08-28 Alnylam Pharmaceuticals Inc RNSi szerek, készítmények és alkalmazási eljárások transztiretin (TTR) asszociált betegségek kezelésére
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
DK2991656T3 (da) * 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression
JP6702862B2 (ja) 2013-07-11 2020-06-03 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． オリゴヌクレオチド−リガンドコンジュゲートおよびそれらの調製方法
WO2015188194A1 (en) 2014-06-06 2015-12-10 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
US20160272970A1 (en) 2015-03-17 2016-09-22 Arrowhead Madison Inc. RNA Interference Agents
JP2018509913A (ja) 2015-03-17 2018-04-12 アローヘッド ファーマシューティカルズ インコーポレイテッド 第ｘｉｉ因子の遺伝子発現を阻害するための組成物と方法
SMT202000454T1 (it) 2015-04-03 2020-11-10 Univ Massachusetts Cmposti oligonucleotidici per bersagliare l'mrn della huntingtina
KR20180038465A (ko) 2015-08-07 2018-04-16 애로우헤드 파마슈티컬스 인코포레이티드 Ｂ형 간염 바이러스 감염에 대한 ｒｎａｉ 치료법
US10633653B2 (en) * 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JOP20160211B1 (ar) 2015-10-01 2021-08-17 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
EP3228326A1 (en) * 2016-04-05 2017-10-11 Silence Therapeutics GmbH Nucleic acid linked to a trivalent glycoconjugate
WO2017214112A1 (en) 2016-06-06 2017-12-14 Arrowhead Pharmaceuticals, Inc. 5'-cyclo-phosphonate modified nucleotides
JOP20190015A1 (ar) 2016-08-04 2019-02-04 Arrowhead Pharmaceuticals Inc عوامل (ار ان ايه آي) لعدوى فيروس الالتهاب الكبدي الوبائي ب
BR112019014282A2 (pt) 2017-01-10 2020-03-03 Arrowhead Pharmaceuticals, Inc. Agentes de rnai de antitripsina (aat) alfa-1, composições incluindo agentes de rnai de aat, e métodos de uso
AU2018213379A1 (en) 2017-01-30 2019-07-18 Arrowhead Pharmaceuticals Inc. Compositions and methods for inhibition of factor XII gene expression
WO2018185240A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions
JP7155239B2 (ja) * 2017-04-05 2022-10-18 サイレンス・セラピューティクス・ゲーエムベーハー 産物及び組成物
US10597657B2 (en) 2017-09-11 2020-03-24 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3)
MX2020001912A (es) 2017-09-14 2020-03-24 Arrowhead Pharmaceuticals Inc Agentes de iarn y composiciones para inhibir la expresion de la angiopoyetina tipo 3 (angptl3) y metodos de uso.
MX2020004897A (es) * 2017-11-13 2020-10-05 Silence Therapeutics Gmbh Acidos nucleicos para inhibir la expresion de lpa en una celula.
CA3083968C (en) 2017-12-01 2024-04-23 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method thereof and use thereof
BR112021009213A2 (pt) * 2018-11-13 2021-08-10 Silence Therapeutics Gmbh ácidos nucleicos para inibição da expressão de lpa em uma célula

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007112414A2 (en) * 2006-03-27 2007-10-04 Isis Pharmaceuticals, Inc. Conjugated double strand compositions for use in gene modulation
WO2009002944A1 (en) * 2007-06-22 2008-12-31 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
CN104854242A (zh) * 2012-12-05 2015-08-19 阿尔尼拉姆医药品有限公司 PCSK9 iRNA组合物及其使用方法
US20160017335A1 (en) * 2012-12-05 2016-01-21 Alnylam Pharmaceuticals, Inc. PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
CN105452463A (zh) * 2013-05-22 2016-03-30 阿尔尼拉姆医药品有限公司 Tmprss6 irna组合物及其使用方法
WO2016085852A1 (en) * 2014-11-24 2016-06-02 Alnylam Pharmaceuticals, Inc. Tmprss6 irna compositions and methods of use thereof
WO2016161388A1 (en) * 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116761616A (zh) * 2021-01-22 2023-09-15 阿耐拉姆制药公司 经修饰的双链寡核苷酸
WO2024245151A1 (zh) * 2023-05-26 2024-12-05 维亚臻生物技术（苏州）有限公司 一种用于代谢疾病的双链核苷酸化合物及其应用
WO2025031444A1 (zh) * 2023-08-09 2025-02-13 北京福元医药股份有限公司 抑制DGAT2基因表达的siRNA、siRNA缀合物或其前药和药物组合物及用途
WO2026011779A1 (zh) * 2024-07-10 2026-01-15 凯莱英医药集团（天津）股份有限公司 制备Cemdisiran的方法

Also Published As

Publication number Publication date
ES2932831T3 (es) 2023-01-26
FI3607069T3 (fi) 2023-01-13
IL268828B1 (en) 2025-07-01
EP3607069B1 (en) 2022-11-02
US12084656B2 (en) 2024-09-10
JP7583005B2 (ja) 2024-11-13
EP4219713A3 (en) 2023-08-16
WO2018185241A1 (en) 2018-10-11
IL268828B2 (en) 2025-11-01
DK3607069T3 (da) 2022-11-21
PL3607069T3 (pl) 2023-03-06
SI3607069T1 (sl) 2023-02-28
IL321221A (en) 2025-07-01
AU2018247925A1 (en) 2019-10-03
US20200095580A1 (en) 2020-03-26
RS63836B1 (sr) 2023-01-31
EP4219713A2 (en) 2023-08-02
JP2020516312A (ja) 2020-06-11
JP7155239B2 (ja) 2022-10-18
IL268828A (en) 2019-10-31
HRP20221400T1 (hr) 2023-01-06
US11015198B2 (en) 2021-05-25
JP2024147806A (ja) 2024-10-16
CA3057565A1 (en) 2018-10-11
US20250043288A1 (en) 2025-02-06
US20220090067A1 (en) 2022-03-24
JP7823127B2 (ja) 2026-03-03
EP3607069A1 (en) 2020-02-12
HUE061247T2 (hu) 2023-06-28
AU2018247925B2 (en) 2024-06-06
LT3607069T (lt) 2023-01-10
JP2022173554A (ja) 2022-11-18

Similar Documents

Publication Publication Date Title
JP7823127B2 (ja) 産物及び組成物
JP7495544B2 (ja) 産物及び組成物
JP7461886B2 (ja) 標的遺伝子の発現を抑制するためのジチオリン酸結合を含む核酸
US11987794B2 (en) Products and compositions
EP3550022A1 (en) Products and compositions
CA3057565C (en) Products and compositions
RU2812806C2 (ru) Нуклеиновые кислоты для ингибирования экспрессии гена-мишени, содержащие фосфодитиоатные связи
HK40016476A (en) Products and compositions
HK40020523A (en) Products and compositions
HK40020523B (en) Products and compositions
HK40032259A (en) Nucleic acids for inhibiting expression of a target gene comprising phosphorodithioate linkages

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40016476

Country of ref document: HK