TWI868755B - 抑制跨膜絲胺酸蛋白酶6(tmprss6)表現的組成物及方法 - Google Patents

抑制跨膜絲胺酸蛋白酶6(tmprss6)表現的組成物及方法 Download PDF

Info

Publication number
TWI868755B
TWI868755B TW112123278A TW112123278A TWI868755B TW I868755 B TWI868755 B TW I868755B TW 112123278 A TW112123278 A TW 112123278A TW 112123278 A TW112123278 A TW 112123278A TW I868755 B TWI868755 B TW I868755B
Authority
TW
Taiwan
Prior art keywords
nucleotides
oligonucleotide
seq
antisense strand
tmprss6
Prior art date
Application number
TW112123278A
Other languages
English (en)
Chinese (zh)
Other versions
TW202400193A (zh
Inventor
安娜 琳達 布洛伊斯
克里斯蒂娜 馬利 普萊斯特
朴智惠
亨利克 杜德克
Original Assignee
丹麥商諾佛 儂迪克股份有限公司
美商戴瑟納製藥股份有限公司
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 丹麥商諾佛 儂迪克股份有限公司, 美商戴瑟納製藥股份有限公司 filed Critical 丹麥商諾佛 儂迪克股份有限公司
Publication of TW202400193A publication Critical patent/TW202400193A/zh
Application granted granted Critical
Publication of TWI868755B publication Critical patent/TWI868755B/zh

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (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)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Biochemistry (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)
TW112123278A 2022-06-24 2023-06-21 抑制跨膜絲胺酸蛋白酶6(tmprss6)表現的組成物及方法 TWI868755B (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202263355210P 2022-06-24 2022-06-24
US63/355,210 2022-06-24
EP22209113.4 2022-11-23
EP22209113 2022-11-23

Publications (2)

Publication Number Publication Date
TW202400193A TW202400193A (zh) 2024-01-01
TWI868755B true TWI868755B (zh) 2025-01-01

Family

ID=87036742

Family Applications (1)

Application Number Title Priority Date Filing Date
TW112123278A TWI868755B (zh) 2022-06-24 2023-06-21 抑制跨膜絲胺酸蛋白酶6(tmprss6)表現的組成物及方法

Country Status (14)

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

Families Citing this family (3)

* 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
WO2025148896A1 (en) * 2024-01-08 2025-07-17 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of transmembrane serine protease 6 (tmprss6)
WO2026003163A1 (en) * 2024-06-26 2026-01-02 Silence Therapeutics Gmbh Sirna against tmprss6 for treating polycythemia vera

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW201536916A (zh) * 2013-05-22 2015-10-01 阿尼拉製藥公司 TMPRSS6 iRNA組成物及其使用方法
WO2022031850A2 (en) * 2020-08-04 2022-02-10 Dicerna Pharmaceuticals Inc. Compositions and methods for the treatment of metabolic syndrome

Family Cites Families (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6469158B1 (en) 1992-05-14 2002-10-22 Ribozyme Pharmaceuticals, Incorporated Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5804683A (en) 1992-05-14 1998-09-08 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA with alkylamine
ATE227342T1 (de) 1993-09-02 2002-11-15 Ribozyme Pharm Inc Enzymatische nukleiksaüre die nicht-nukleotide enthaltet
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6111086A (en) 1998-02-27 2000-08-29 Scaringe; Stephen A. Orthoester protecting groups
ES2336887T5 (es) 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
US20050159378A1 (en) 2001-05-18 2005-07-21 Sirna Therapeutics, Inc. RNA interference mediated inhibition of Myc and/or Myb gene expression using short interfering nucleic acid (siNA)
WO2003040395A2 (en) 2001-11-07 2003-05-15 Applera Corporation Universal nucleotides for nucleic acid analysis
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
WO2007030167A1 (en) 2005-09-02 2007-03-15 Nastech Pharmaceutical Company Inc. Modification of double-stranded ribonucleic acid molecules
DK2341943T3 (en) 2008-09-22 2019-02-25 Dicerna Pharmaceuticals Inc COMPOSITIONS AND METHODS OF SPECIFIC INHIBITION OF DSRNA REPRINT WITH MODIFICATIONS
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
KR101728655B1 (ko) 2008-12-18 2017-04-19 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
PH12013501969B1 (en) 2011-03-29 2018-08-31 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of tmprss6 gene
AU2012328680A1 (en) 2011-10-25 2014-05-01 Ionis Pharmaceuticals, Inc. Antisense modulation of GCCR expression
HRP20191883T1 (hr) 2011-11-07 2019-12-27 Ionis Pharmaceuticals, Inc. Modulacija ekspresije tmprss6
EP3134732A2 (en) 2014-04-24 2017-03-01 Alnylam Pharmaceuticals, Inc. Methods and compositions for detecting anti-drug antibodies
WO2016085852A1 (en) 2014-11-24 2016-06-02 Alnylam Pharmaceuticals, Inc. Tmprss6 irna compositions and methods of use thereof
DK3569711T3 (da) * 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
MX2017012426A (es) 2015-04-03 2018-01-26 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de tmprss6.
HUE059718T2 (hu) 2016-09-02 2022-12-28 Dicerna Pharmaceuticals Inc 4'-foszfát analógok és azokat tartalmazó oligonukleotidok
SI3607068T1 (sl) 2017-04-05 2023-02-28 Silence Therapeutics Gmbh Z RNA interferenco posredovano inhibiranje TMPRSS6
IL271680B (en) * 2017-06-29 2022-09-01 Dicerna Pharmaceuticals Inc Compositions and methods for inhibiting hmgb1 expression
EP3598995A1 (en) 2018-07-26 2020-01-29 Silence Therapeutics GmbH Products and compositions
SG11202106857VA (en) 2018-12-28 2021-07-29 Dicerna Pharmaceuticals Inc Compositions and methods for inhibiting hmgb1 expression
WO2022226127A1 (en) 2021-04-20 2022-10-27 Alexion Pharmaceuticals, Inc. Compositions and methods for inhibiting complement component 3 expression
EP4330392A1 (en) 2021-04-26 2024-03-06 Alnylam Pharmaceuticals, Inc. Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof
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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW201536916A (zh) * 2013-05-22 2015-10-01 阿尼拉製藥公司 TMPRSS6 iRNA組成物及其使用方法
WO2022031850A2 (en) * 2020-08-04 2022-02-10 Dicerna Pharmaceuticals Inc. Compositions and methods for the treatment of metabolic syndrome

Also Published As

Publication number Publication date
US20240002858A1 (en) 2024-01-04
IL317012A (en) 2025-01-01
EP4544049A1 (en) 2025-04-30
MA71238A (fr) 2025-04-30
WO2023247738A1 (en) 2023-12-28
KR102759742B1 (ko) 2025-02-04
JP2024002985A (ja) 2024-01-11
CO2024017422A2 (es) 2025-05-08
KR20240002200A (ko) 2024-01-04
US20240309388A1 (en) 2024-09-19
JP7560616B2 (ja) 2024-10-02
MX2024015763A (es) 2025-02-10
AR129710A1 (es) 2024-09-18
US12503701B2 (en) 2025-12-23
TW202400193A (zh) 2024-01-01
US11952574B2 (en) 2024-04-09
AU2023288906A1 (en) 2024-12-12
CA3257962A1 (en) 2023-12-28
CN119403927A (zh) 2025-02-07

Similar Documents

Publication Publication Date Title
JP7805386B2 (ja) Lpa発現を阻害するための組成物及び方法
TWI870563B (zh) 用於抑制angptl3表現之組合物及方法
TWI868755B (zh) 抑制跨膜絲胺酸蛋白酶6(tmprss6)表現的組成物及方法
US20250092403A1 (en) Compositions and methods for inhibiting ketohexokinase (khk)
JP7463621B2 (ja) ミトコンドリアアミドキシム還元成分1(marc1)発現を阻害するための組成物および方法
TWI857290B (zh) 用於調節pnpla3表現之組合物及方法
TW202345866A (zh) 用於抑制補體因子b的組成物及方法
BR112021012516A2 (pt) Composições e métodos para inibir a expressão de hmgb1
JP2025516677A (ja) Maptの発現を阻害するための組成物及び方法
JP2024532019A (ja) アルファ-1アンチトリプシン発現を阻害するための組成物及び方法
JP2025518507A (ja) Sncaの発現を阻害するための組成物及び方法
TW202330920A (zh) 用於調節apoc3表現之組合物及方法
CN117396606A (zh) 用于抑制线粒体偕胺肟还原组分1(marc1)表达的组合物和方法
TW202345873A (zh) 調節scap活性之組合物及方法
CN117280031A (zh) 用于抑制核受体亚家族1组h成员3(nr1h3)表达的组合物和方法