JP7780428B2 - 肝臓外送達 - Google Patents

肝臓外送達

Info

Publication number
JP7780428B2
JP7780428B2 JP2022525709A JP2022525709A JP7780428B2 JP 7780428 B2 JP7780428 B2 JP 7780428B2 JP 2022525709 A JP2022525709 A JP 2022525709A JP 2022525709 A JP2022525709 A JP 2022525709A JP 7780428 B2 JP7780428 B2 JP 7780428B2
Authority
JP
Japan
Prior art keywords
strand
double
irna agent
antisense strand
nucleotides
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.)
Active
Application number
JP2022525709A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023500681A (ja
JP2023500681A5 (https=
JPWO2021092371A5 (https=
Inventor
ケー ナイル，ジャヤプラカーシュ
エー マイアー，マーティン
シー サリナス，ジュアン
マツダ，シゲオ
ヴィ ケリン，アレクサンダー
ピー レンティーニ，スコット
ハァ，グオ
エイチ ユング，ミシェル
エム ピアソン，ジャスティン
マノハラン，ムシア
シー ギュンター，デイル
ズラテフ，イヴァン
エス タイレ，クリストファー
アール ジャドハヴ，ヴァサント
ミルシテイン，スチュアート
ヤナス，マヤ
ダッタ，ドルバジョーティ
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 JP2023500681A publication Critical patent/JP2023500681A/ja
Publication of JP2023500681A5 publication Critical patent/JP2023500681A5/ja
Publication of JPWO2021092371A5 publication Critical patent/JPWO2021092371A5/ja
Priority to JP2025201618A priority Critical patent/JP2026041795A/ja
Application granted granted Critical
Publication of JP7780428B2 publication Critical patent/JP7780428B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • 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
    • C12N2310/35—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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3535—Nitrogen
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2022525709A 2019-11-06 2020-11-06 肝臓外送達 Active JP7780428B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025201618A JP2026041795A (ja) 2019-11-06 2025-11-21 肝臓外送達

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962931631P 2019-11-06 2019-11-06
US62/931,631 2019-11-06
PCT/US2020/059399 WO2021092371A2 (en) 2019-11-06 2020-11-06 Extrahepatic delivery

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025201618A Division JP2026041795A (ja) 2019-11-06 2025-11-21 肝臓外送達

Publications (4)

Publication Number Publication Date
JP2023500681A JP2023500681A (ja) 2023-01-10
JP2023500681A5 JP2023500681A5 (https=) 2023-09-21
JPWO2021092371A5 JPWO2021092371A5 (https=) 2023-09-21
JP7780428B2 true JP7780428B2 (ja) 2025-12-04

Family

ID=73646550

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022525709A Active JP7780428B2 (ja) 2019-11-06 2020-11-06 肝臓外送達
JP2025201618A Pending JP2026041795A (ja) 2019-11-06 2025-11-21 肝臓外送達

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025201618A Pending JP2026041795A (ja) 2019-11-06 2025-11-21 肝臓外送達

Country Status (10)

Country Link
US (1) US20230016929A1 (https=)
EP (1) EP4055166A2 (https=)
JP (2) JP7780428B2 (https=)
KR (1) KR20220110749A (https=)
CN (2) CN119709746A (https=)
AU (1) AU2020378414A1 (https=)
BR (1) BR112022007795A2 (https=)
CA (1) CA3160329A1 (https=)
MX (1) MX2022005220A (https=)
WO (1) WO2021092371A2 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4013767A4 (en) 2019-08-15 2023-10-25 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds and uses thereof
WO2022174000A2 (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
EP4351541A2 (en) * 2021-06-08 2024-04-17 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
KR20240067943A (ko) 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
CN114891509B (zh) * 2021-12-14 2023-05-05 湖北兴福电子材料股份有限公司 一种高选择性的缓冲氧化物蚀刻液
KR20240163743A (ko) * 2022-03-28 2024-11-19 엠피리코 인크. 변형된 올리고뉴클레오티드
JP2025532985A (ja) 2022-09-30 2025-10-03 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 修飾二本鎖ｒｎａ剤
AU2024254919A1 (en) 2023-04-12 2025-10-30 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery of double-stranded rna agents
CN121358748A (zh) * 2023-06-12 2026-01-16 大睿生物医药科技(上海)有限公司 包含糖的寡核苷酸递送配体
TW202517784A (zh) 2023-07-14 2025-05-01 大陸商云合智藥（蘇州）生物科技有限公司 用於抑制己酮糖激酶表達的ＲＮＡｉ劑及其應用
WO2025029913A2 (en) * 2023-07-31 2025-02-06 Basecure Therapeutics Llc Conjugates of n-acetyl-galactosamine (galnac) and oligonucleotides and uses thereof
WO2025040139A1 (zh) 2023-08-23 2025-02-27 云合智药（苏州）生物科技有限公司 用于抑制二酰甘油-O-酰基转移酶2表达的RNAi剂及其应用
WO2025054459A1 (en) * 2023-09-08 2025-03-13 Dicerna Pharmaceuticals, Inc. Rnai oligonucleotide conjugates
WO2025073871A1 (en) 2023-10-03 2025-04-10 Bicycletx Limited Bicyclic peptide ligand complexes specific for tfr1

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