JP7560616B2 - 膜貫通型セリンプロテアーゼ６（ｔｍｐｒｓｓ６）発現を阻害するための組成物および方法 - Google Patents

膜貫通型セリンプロテアーゼ６（ｔｍｐｒｓｓ６）発現を阻害するための組成物および方法 Download PDF

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JP7560616B2
JP7560616B2 JP2023103614A JP2023103614A JP7560616B2 JP 7560616 B2 JP7560616 B2 JP 7560616B2 JP 2023103614 A JP2023103614 A JP 2023103614A JP 2023103614 A JP2023103614 A JP 2023103614A JP 7560616 B2 JP7560616 B2 JP 7560616B2
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nucleotides
oligonucleotide
seq
antisense strand
oligonucleotides
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JP2024002985A (ja
JP2024002985A5 (enExample
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アンナ・リンダ・ブロワ
クリスティーナ・マリー・プリースト
ジヘ・パク
ヘンリク・デュデク
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Dicerna Pharmaceuticals Inc
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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
    • 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
    • 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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • 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
    • C12N2310/32—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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
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  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023103614A 2022-06-24 2023-06-23 膜貫通型セリンプロテアーゼ６（ｔｍｐｒｓｓ６）発現を阻害するための組成物および方法 Active JP7560616B2 (ja)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202263355210P 2022-06-24 2022-06-24
US63/355,210 2022-06-24
EP22209113 2022-11-23
EP22209113 2022-11-23

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JP2024002985A JP2024002985A (ja) 2024-01-11
JP2024002985A5 JP2024002985A5 (enExample) 2024-03-22
JP7560616B2 true JP7560616B2 (ja) 2024-10-02

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US (2) US11952574B2 (enExample)
EP (1) EP4544049A1 (enExample)
JP (1) JP7560616B2 (enExample)
KR (1) KR102759742B1 (enExample)
CN (1) CN119403927A (enExample)
AR (1) AR129710A1 (enExample)
AU (1) AU2023288906A1 (enExample)
CA (1) CA3257962A1 (enExample)
CO (1) CO2024017422A2 (enExample)
IL (1) IL317012A (enExample)
MA (1) MA71238A (enExample)
MX (1) MX2024015763A (enExample)
TW (1) TWI868755B (enExample)
WO (1) WO2023247738A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI868755B (zh) 2022-06-24 2025-01-01 丹麥商諾佛 儂迪克股份有限公司 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法
TW202527968A (zh) * 2024-01-08 2025-07-16 大陸商上海舶望製藥有限公司 用於抑制跨膜絲氨酸蛋白酶6（tmprss6）表達的組合物和方法

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JP2020512838A (ja) 2017-04-05 2020-04-30 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
JP2020529197A (ja) 2017-06-29 2020-10-08 ディセルナ ファーマシューティカルズ インコーポレイテッド Ｈｍｇｂ１発現を阻害するための組成物及び方法
JP2022517742A (ja) 2018-12-28 2022-03-10 ディセルナ ファーマシューティカルズ インコーポレイテッド Ｈｍｇｂ１発現を阻害するための組成物及び方法

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US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
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TWI868755B (zh) 2022-06-24 2025-01-01 丹麥商諾佛 儂迪克股份有限公司 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法

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JP2016520593A (ja) 2013-05-22 2016-07-14 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． Ｔｍｐｒｓｓ６ ｉｒｎａ組成物及びその使用方法
WO2015164635A2 (en) 2014-04-24 2015-10-29 Alnylam Pharmaceuticals, Inc. Methods and compositions for detecting anti-drug antibodies
JP2020512838A (ja) 2017-04-05 2020-04-30 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
JP2020529197A (ja) 2017-06-29 2020-10-08 ディセルナ ファーマシューティカルズ インコーポレイテッド Ｈｍｇｂ１発現を阻害するための組成物及び方法
JP2022517742A (ja) 2018-12-28 2022-03-10 ディセルナ ファーマシューティカルズ インコーポレイテッド Ｈｍｇｂ１発現を阻害するための組成物及び方法

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KR20240002200A (ko) 2024-01-04
MA71238A (fr) 2025-04-30
CO2024017422A2 (es) 2025-05-08
CN119403927A (zh) 2025-02-07
US11952574B2 (en) 2024-04-09
AR129710A1 (es) 2024-09-18
AU2023288906A1 (en) 2024-12-12
JP2024002985A (ja) 2024-01-11
IL317012A (en) 2025-01-01
WO2023247738A1 (en) 2023-12-28
US12503701B2 (en) 2025-12-23
US20240002858A1 (en) 2024-01-04
MX2024015763A (es) 2025-02-10
US20240309388A1 (en) 2024-09-19
CA3257962A1 (en) 2023-12-28
TWI868755B (zh) 2025-01-01
EP4544049A1 (en) 2025-04-30
TW202400193A (zh) 2024-01-01
KR102759742B1 (ko) 2025-02-04

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