JP2023515664A - 修飾された短い干渉核酸（ｓｉＮＡ）分子およびそれらの使用 - Google Patents

修飾された短い干渉核酸（ｓｉＮＡ）分子およびそれらの使用 Download PDF

Info

Publication number
JP2023515664A
JP2023515664A JP2022552538A JP2022552538A JP2023515664A JP 2023515664 A JP2023515664 A JP 2023515664A JP 2022552538 A JP2022552538 A JP 2022552538A JP 2022552538 A JP2022552538 A JP 2022552538A JP 2023515664 A JP2023515664 A JP 2023515664A
Authority
JP
Japan
Prior art keywords
nucleotides
sina
nucleotide sequence
nucleotide
methyl
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
JP2022552538A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023515664A5 (OSRAM
Inventor
バイゲルマン，レオニード
クマール ラジワンシ，ヴィヴェク
ホスバッハ，マルクス
ケー． パンデイ，ラジェンドラ
ホン，ジン
エルテプ，ラクスマン
マルティネス モンテロ，サウル
コスタ，エヌ．ティラニ エス． デ
Original Assignee
アリゴス セラピューティクス，インコーポレーテッド
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 アリゴス セラピューティクス，インコーポレーテッド filed Critical アリゴス セラピューティクス，インコーポレーテッド
Publication of JP2023515664A publication Critical patent/JP2023515664A/ja
Publication of JP2023515664A5 publication Critical patent/JP2023515664A5/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
    • C12N15/113—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
    • C12N15/1131—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 viruses
    • 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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • 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
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • 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
    • C12N2320/31—Combination therapy

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Cosmetics (AREA)
JP2022552538A 2020-03-06 2021-03-05 修飾された短い干渉核酸（ｓｉＮＡ）分子およびそれらの使用 Pending JP2023515664A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062986150P 2020-03-06 2020-03-06
US62/986,150 2020-03-06
US202063109196P 2020-11-03 2020-11-03
US63/109,196 2020-11-03
PCT/US2021/021199 WO2021178885A1 (en) 2020-03-06 2021-03-05 Modified short interfering nucleic acid (sina) molecules and uses thereof

Publications (2)

Publication Number Publication Date
JP2023515664A true JP2023515664A (ja) 2023-04-13
JP2023515664A5 JP2023515664A5 (OSRAM) 2024-03-14

Family

ID=75278356

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022552538A Pending JP2023515664A (ja) 2020-03-06 2021-03-05 修飾された短い干渉核酸（ｓｉＮＡ）分子およびそれらの使用

Country Status (17)

Country Link
US (4) US20220364096A1 (OSRAM)
EP (1) EP4121533A1 (OSRAM)
JP (1) JP2023515664A (OSRAM)
KR (1) KR20220163960A (OSRAM)
CN (1) CN115485384A (OSRAM)
AU (1) AU2021230601A1 (OSRAM)
BR (1) BR112022017287A2 (OSRAM)
CA (1) CA3170466A1 (OSRAM)
CL (1) CL2022002379A1 (OSRAM)
CO (1) CO2022014258A2 (OSRAM)
GE (2) GEP20257742B (OSRAM)
IL (1) IL296045A (OSRAM)
MX (1) MX2022011012A (OSRAM)
PE (1) PE20221578A1 (OSRAM)
PH (1) PH12022552163A1 (OSRAM)
TW (1) TW202208625A (OSRAM)
WO (1) WO2021178885A1 (OSRAM)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11202002149XA (en) * 2017-09-14 2020-04-29 Janssen Biopharma Inc Galnac derivatives
GEP20257742B (en) * 2020-03-06 2025-03-10 Aligos Therapeutics Inc Modified short interfering nucleic acid (sina) molecules and uses thereof
CA3197470A1 (en) 2020-10-21 2022-04-28 Aligos Therapeutics, Inc. Bicyclic compounds
WO2022184852A1 (en) * 2021-03-03 2022-09-09 Silence Therapeutics Gmbh Conjugated nucleic acids comprising a phosphorodithioate for inhibiting gene expression in a cell
US11957683B2 (en) 2021-06-18 2024-04-16 Aligos Therapeutics, Inc. Bicyclic compounds
WO2023088227A1 (zh) 2021-11-16 2023-05-25 上海舶望制药有限公司 抑制血管紧张素原(agt)蛋白表达的组合物和方法
WO2023122317A2 (en) * 2021-12-23 2023-06-29 Aligos Therapeutics, Inc. SHORT INTERFERING NUCLEIC ACID (SINA) MOLECULES FOR TARGETING β-CATENIN AND USES THEREOF
US20230399645A1 (en) * 2022-05-16 2023-12-14 University Of Massachusetts Optimized sirna scaffolds
EP4541804A1 (en) * 2022-06-14 2025-04-23 Rona Bioscience, Limited Cyclic phosphonate-modified nucleotide
WO2024033374A1 (en) 2022-08-11 2024-02-15 Syngenta Crop Protection Ag Novel arylcarboxamide or arylthioamide compounds
WO2024089216A1 (en) 2022-10-27 2024-05-02 Syngenta Crop Protection Ag Novel sulfur-containing heteroaryl carboxamide compounds
EP4634152A1 (en) 2022-12-15 2025-10-22 Syngenta Crop Protection AG Novel bicyclic-carboxamide compounds useful as pesticides
AR131541A1 (es) 2023-01-07 2025-04-09 Syngenta Crop Protection Ag Compuestos de carboxamida novedosos
AU2024212652A1 (en) * 2023-01-26 2025-07-10 Aligos Therapeutics, Inc. Hepatitis b combination therapies
WO2024173593A1 (en) * 2023-02-14 2024-08-22 Basecure Therapeutics Modified double stranded rna agents
WO2024254091A1 (en) * 2023-06-05 2024-12-12 Empirico Inc. Treatment of fgg related diseases and disorders
WO2025032129A1 (en) 2023-08-08 2025-02-13 Syngenta Crop Protection Ag Novel aminoindane and aminotetraline compounds
CN117024485B (zh) * 2023-08-11 2026-02-13 江苏申基生物科技有限公司 一种2’-OMe腺苷的合成方法
IL327476A (en) * 2023-11-03 2026-05-01 Basecure Therapeutics Llc Modified oligonucleotides and methods for using them
WO2025104032A1 (en) 2023-11-14 2025-05-22 Syngenta Crop Protection Ag Novel carboxamide compounds
WO2025109114A1 (en) 2023-11-24 2025-05-30 Syngenta Crop Protection Ag Novel carboxamide compounds
CN117534717A (zh) * 2024-01-09 2024-02-09 凯莱英生命科学技术(天津)有限公司 5′-(e)-乙烯基磷酸酯的合成方法
PY2501648A (es) 2024-01-12 2025-10-31 Syngenta Crop Protection Ag Nuevos compuestos de carboxamida
WO2025149637A1 (en) 2024-01-12 2025-07-17 Syngenta Crop Protection Ag Novel carboxamide compounds
WO2025201529A1 (en) * 2024-03-29 2025-10-02 Ractigen Therapeutics Compounds for synthesizing modified small activating rna molecules, modified small activating rna molecules and uses thereof
WO2025240506A1 (en) 2024-05-14 2025-11-20 Aligos Therapeutics, Inc. Modified antisense oligonucleotides for treating hepatitis b virus
WO2025242321A1 (en) 2024-05-24 2025-11-27 Glaxosmithkline Intellectual Property (No.3) Limited Novel use
WO2026008750A1 (en) 2024-07-05 2026-01-08 Syngenta Crop Protection Ag Novel carboxamide compounds
CN120796266A (zh) * 2024-07-08 2025-10-17 北京悦康科创医药科技股份有限公司 一种降低hbv基因表达的反义寡核苷酸及其应用

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014527401A (ja) * 2011-06-30 2014-10-16 アローヘッド リサーチ コーポレイション Ｂ型肝炎ウイルスの遺伝子発現を阻害するための組成物および方法
JP2015502931A (ja) * 2011-11-18 2015-01-29 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 修飾ＲＮＡｉ剤
WO2018185241A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions
JP2018536689A (ja) * 2015-12-10 2018-12-13 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． ステロール調節エレメント結合タンパク質（ＳＲＥＢＰ）シャペロン（ＳＣＡＰ）ｉＲＮＡ組成物およびその使用方法
JP2019504112A (ja) * 2015-12-07 2019-02-14 ジェンザイム・コーポレーション Ｓｅｒｐｉｎｃ１関連障害を処置するための方法および組成物
WO2019113393A1 (en) * 2017-12-06 2019-06-13 Avidity Biosciences Llc Compositions and methods of treating muscle atrophy and myotonic dystrophy
WO2019217459A1 (en) * 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009002944A1 (en) * 2007-06-22 2008-12-31 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
EP2437752A2 (en) * 2009-06-03 2012-04-11 Dicerna Pharmaceuticals, Inc. Peptide dicer substrate agents and methods for their specific inhibition of gene expression
EP2601204B1 (en) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
CA2939822C (en) * 2014-02-18 2018-09-25 Osaka University Crosslinked nucleoside and nucleotide
KR20180038465A (ko) * 2015-08-07 2018-04-16 애로우헤드 파마슈티컬스 인코포레이티드 Ｂ형 간염 바이러스 감염에 대한 ｒｎａｉ 치료법
JP6989521B2 (ja) 2016-09-02 2022-01-05 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 標的化リガンド
WO2018045317A1 (en) 2016-09-02 2018-03-08 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
CN110997919B (zh) * 2017-12-01 2024-04-02 苏州瑞博生物技术股份有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
WO2019217397A2 (en) * 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for improving strand biased
MX2021005357A (es) 2018-11-08 2021-06-30 Aligos Therapeutics Inc Polimeros oligonucleotidos que inhiben el transporte de antigeno s y metodos.
GEP20257742B (en) * 2020-03-06 2025-03-10 Aligos Therapeutics Inc Modified short interfering nucleic acid (sina) molecules and uses thereof

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014527401A (ja) * 2011-06-30 2014-10-16 アローヘッド リサーチ コーポレイション Ｂ型肝炎ウイルスの遺伝子発現を阻害するための組成物および方法
JP2015502931A (ja) * 2011-11-18 2015-01-29 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 修飾ＲＮＡｉ剤
JP2019504112A (ja) * 2015-12-07 2019-02-14 ジェンザイム・コーポレーション Ｓｅｒｐｉｎｃ１関連障害を処置するための方法および組成物
JP2018536689A (ja) * 2015-12-10 2018-12-13 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． ステロール調節エレメント結合タンパク質（ＳＲＥＢＰ）シャペロン（ＳＣＡＰ）ｉＲＮＡ組成物およびその使用方法
WO2018185241A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions
WO2019113393A1 (en) * 2017-12-06 2019-06-13 Avidity Biosciences Llc Compositions and methods of treating muscle atrophy and myotonic dystrophy
WO2019217459A1 (en) * 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
NUCLEIC ACIDS RESEARCH, vol. 45, no. 6, JPN6025013423, 2017, pages 3528 - 3536, ISSN: 0005717369 *

Also Published As

Publication number Publication date
GEAP202416053A (en) 2024-10-10
BR112022017287A2 (pt) 2022-11-16
KR20220163960A (ko) 2022-12-12
US12129469B2 (en) 2024-10-29
PE20221578A1 (es) 2022-10-06
AU2021230601A1 (en) 2022-10-06
CO2022014258A2 (es) 2022-12-30
WO2021178885A1 (en) 2021-09-10
PH12022552163A1 (en) 2024-01-29
US20230365970A1 (en) 2023-11-16
AU2021230601A2 (en) 2022-10-20
US20230332153A1 (en) 2023-10-19
US20220177888A1 (en) 2022-06-09
CA3170466A1 (en) 2021-09-10
IL296045A (en) 2022-10-01
CL2022002379A1 (es) 2023-07-28
GEP20257742B (en) 2025-03-10
TW202208625A (zh) 2022-03-01
US11549110B2 (en) 2023-01-10
EP4121533A1 (en) 2023-01-25
CN115485384A (zh) 2022-12-16
MX2022011012A (es) 2022-10-07
US20220364096A1 (en) 2022-11-17

Similar Documents

Publication Publication Date Title
US12129469B2 (en) Modified short interfering nucleic acid (siNA) molecules and uses thereof
US20210189392A1 (en) S-antigen transport inhibiting oligonucleotide polymers and methods
US20250011789A1 (en) Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof
US12454691B2 (en) Modified short interfering nucleic acid (siNA) molecules and uses thereof
KR102533038B1 (ko) 변형된 올리고뉴클레오티드 및 사용 방법
US20250243491A1 (en) Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof
JP2025535539A (ja) 修飾された短い干渉核酸（ｓｉＮＡ）分子及びその使用
AU2023236560A1 (en) Modified gapmer oligomers and methods of use thereof
EA050671B1 (ru) МОЛЕКУЛЫ МОДИФИЦИРОВАННЫХ МАЛЫХ ИНТЕРФЕРИРУЮЩИХ НУКЛЕИНОВЫХ КИСЛОТ (миНК) И ИХ ПРИМЕНЕНИЕ
OA21336A (en) Modified short interfering nucleic acid (siNA) molecules and uses thereof.
TW202604551A (zh) 用於治療ｂ型肝炎病毒之經修飾之反義寡核苷酸
WO2024182446A2 (en) Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240305

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240305

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250401

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250701

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20251028

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260227

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20260408