AU2023288906A1 - Compositions and methods for inhibiting transmembrane serine protease 6 (tmprss6) expression - Google Patents

Compositions and methods for inhibiting transmembrane serine protease 6 (tmprss6) expression Download PDF

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Publication number
AU2023288906A1
AU2023288906A1 AU2023288906A AU2023288906A AU2023288906A1 AU 2023288906 A1 AU2023288906 A1 AU 2023288906A1 AU 2023288906 A AU2023288906 A AU 2023288906A AU 2023288906 A AU2023288906 A AU 2023288906A AU 2023288906 A1 AU2023288906 A1 AU 2023288906A1
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Australia
Prior art keywords
nucleotides
oligonucleotide
seq
antisense strand
tmprss6
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Pending
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AU2023288906A
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English (en)
Inventor
Anna Linda BLOIS
Henryk Dudek
JiHye PARK
Christina Marie PRIEST
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Novo Nordisk AS
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Novo Nordisk AS
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Assigned to NOVO NORDISK A/S reassignment NOVO NORDISK A/S Request for Assignment Assignors: DICERNA PHARMACEUTICALS INC., NOVO NORDISK A/S
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    • 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
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    • 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)
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  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Microbiology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2023288906A 2022-06-24 2023-06-23 Compositions and methods for inhibiting transmembrane serine protease 6 (tmprss6) expression Pending AU2023288906A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263355210P 2022-06-24 2022-06-24
US63/355,210 2022-06-24
EP22209113 2022-11-23
EP22209113.4 2022-11-23
PCT/EP2023/067070 WO2023247738A1 (en) 2022-06-24 2023-06-23 Compositions and methods for inhibiting transmembrane serine protease 6 (tmprss6) expression

Publications (1)

Publication Number Publication Date
AU2023288906A1 true AU2023288906A1 (en) 2024-12-12

Family

ID=87036742

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2023288906A Pending AU2023288906A1 (en) 2022-06-24 2023-06-23 Compositions and methods for inhibiting transmembrane serine protease 6 (tmprss6) expression

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

Family Cites Families (35)

* 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
WO1995006731A2 (en) 1993-09-02 1995-03-09 Ribozyme Pharmaceuticals, Inc. Non-nucleotide containing enzymatic nucleic acid
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
PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
US20040259247A1 (en) 2000-12-01 2004-12-23 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)
JP2005508196A (ja) 2001-11-07 2005-03-31 アプレラ コーポレイション 核酸分析の汎用ヌクレオチド
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
JP2012504389A (ja) 2008-09-22 2012-02-23 ダイセルナ ファーマシューティカルズ， インコーポレイテッド 修飾を有するｄｓＲＮＡによる遺伝子発現の特異的な阻害のための組成物および方法
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
AU2009336191B2 (en) 2008-12-18 2017-08-24 Novo Nordisk A/S Extended dicer substrate agents and methods for the specific inhibition of gene expression
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
KR102365961B1 (ko) 2011-03-29 2022-02-23 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
JP2015501155A (ja) 2011-10-25 2015-01-15 アイシス ファーマシューティカルズ， インコーポレーテッド Ｇｃｃｒ発現のアンチセンス調整
EP3650544A1 (en) 2011-11-07 2020-05-13 Ionis Pharmaceuticals, Inc. Modulation of tmprss6 expression
KR102486617B1 (ko) 2013-05-22 2023-01-12 알닐람 파마슈티칼스 인코포레이티드 Ｔｍｐｒｓｓ6 조성물 및 이의 사용 방법
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
JP7105065B2 (ja) 2014-12-15 2022-07-22 ダイセルナ ファーマシューティカルズ， インコーポレイテッド リガンド修飾二本鎖核酸
MX2017012426A (es) 2015-04-03 2018-01-26 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de tmprss6.
KR102350647B1 (ko) 2016-09-02 2022-01-14 다이서나 파마수이티컬, 인크. 4'-포스페이트 유사체 및 이를 포함하는 올리고뉴클레오타이드
WO2018185240A1 (en) 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions
KR20200023427A (ko) * 2017-06-29 2020-03-04 다이서나 파마수이티컬, 인크. Hmgb1 발현을 억제하기 위한 조성물 및 방법
EP3598995A1 (en) 2018-07-26 2020-01-29 Silence Therapeutics GmbH Products and compositions
MX2021007855A (es) 2018-12-28 2021-10-26 Dicerna Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de hmgb1.
TW202221120A (zh) * 2020-08-04 2022-06-01 美商黛瑟納製藥公司 用於治療代謝症候群之組成物及方法
WO2022226127A1 (en) 2021-04-20 2022-10-27 Alexion Pharmaceuticals, Inc. Compositions and methods for inhibiting complement component 3 expression
MX2023012586A (es) 2021-04-26 2023-10-31 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de serina 6, proteasa transmembranaria (tmprss6) y sus metodos de uso.
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

Also Published As

Publication number Publication date
EP4544049A1 (en) 2025-04-30
US12503701B2 (en) 2025-12-23
KR20240002200A (ko) 2024-01-04
MX2024015763A (es) 2025-02-10
US20240309388A1 (en) 2024-09-19
US11952574B2 (en) 2024-04-09
CN119403927A (zh) 2025-02-07
WO2023247738A1 (en) 2023-12-28
US20240002858A1 (en) 2024-01-04
JP2024002985A (ja) 2024-01-11
TW202400193A (zh) 2024-01-01
CA3257962A1 (en) 2023-12-28
CO2024017422A2 (es) 2025-05-08
AR129710A1 (es) 2024-09-18
IL317012A (en) 2025-01-01
MA71238A (fr) 2025-04-30
KR102759742B1 (ko) 2025-02-04
JP7560616B2 (ja) 2024-10-02
TWI868755B (zh) 2025-01-01

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Owner name: NOVO NORDISK A/S

Free format text: FORMER APPLICANT(S): NOVO NORDISK A/S; DICERNA PHARMACEUTICALS INC.