AR129710A1 - Composiciones y métodos para inhibir la expresión de serina proteasa transmembrana 6 (tmprss6) - Google Patents

Composiciones y métodos para inhibir la expresión de serina proteasa transmembrana 6 (tmprss6)

Info

Publication number
AR129710A1
AR129710A1 ARP230101626A ARP230101626A AR129710A1 AR 129710 A1 AR129710 A1 AR 129710A1 AR P230101626 A ARP230101626 A AR P230101626A AR P230101626 A ARP230101626 A AR P230101626A AR 129710 A1 AR129710 A1 AR 129710A1
Authority
AR
Argentina
Prior art keywords
seq
antisense strand
tmprss6
strand comprises
oligonucleotide
Prior art date
Application number
ARP230101626A
Other languages
English (en)
Spanish (es)
Inventor
Anna Linda Blois
Christina Marie Priest
Jihye Park
Henryk Dudek
Original Assignee
Novo Nordisk As
Dicerna 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 Novo Nordisk As, Dicerna Pharmaceuticals Inc filed Critical Novo Nordisk As
Publication of AR129710A1 publication Critical patent/AR129710A1/es

Links

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
    • C12N15/1137—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

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)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
ARP230101626A 2022-06-24 2023-06-23 Composiciones y métodos para inhibir la expresión de serina proteasa transmembrana 6 (tmprss6) AR129710A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263355210P 2022-06-24 2022-06-24
EP22209113 2022-11-23

Publications (1)

Publication Number Publication Date
AR129710A1 true AR129710A1 (es) 2024-09-18

Family

ID=87036742

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP230101626A AR129710A1 (es) 2022-06-24 2023-06-23 Composiciones y métodos para inhibir la expresión de serina proteasa transmembrana 6 (tmprss6)

Country Status (14)

Country Link
US (2) US11952574B2 (cg-RX-API-DMAC7.html)
EP (1) EP4544049A1 (cg-RX-API-DMAC7.html)
JP (1) JP7560616B2 (cg-RX-API-DMAC7.html)
KR (1) KR102759742B1 (cg-RX-API-DMAC7.html)
CN (1) CN119403927A (cg-RX-API-DMAC7.html)
AR (1) AR129710A1 (cg-RX-API-DMAC7.html)
AU (1) AU2023288906A1 (cg-RX-API-DMAC7.html)
CA (1) CA3257962A1 (cg-RX-API-DMAC7.html)
CO (1) CO2024017422A2 (cg-RX-API-DMAC7.html)
IL (1) IL317012A (cg-RX-API-DMAC7.html)
MA (1) MA71238A (cg-RX-API-DMAC7.html)
MX (1) MX2024015763A (cg-RX-API-DMAC7.html)
TW (1) TWI868755B (cg-RX-API-DMAC7.html)
WO (1) WO2023247738A1 (cg-RX-API-DMAC7.html)

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
CN122477285A (zh) * 2024-01-08 2026-07-28 上海舶望制药有限公司 用于抑制跨膜丝氨酸蛋白酶6(tmprss6)表达的组合物和方法
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
US5804683A (en) 1992-05-14 1998-09-08 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA with alkylamine
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
EP1253199A1 (en) 1993-09-02 2002-10-30 Ribozyme Pharmaceuticals, Inc. Abasic moieties containing enzymatic nucleic acids
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
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Rna 간섭의 rna 서열 특이적인 매개체
CN100523215C (zh) 2000-12-01 2009-08-05 马普科技促进协会 介导rna干涉的小rna分子
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
WO2010033225A2 (en) 2008-09-22 2010-03-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for the specific inhibition of gene expression by dsrna possessing modifications
KR20110110776A (ko) 2008-12-18 2011-10-07 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
KR102104401B1 (ko) 2011-03-29 2020-04-27 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
AU2012328680A1 (en) 2011-10-25 2014-05-01 Ionis Pharmaceuticals, Inc. Antisense modulation of GCCR expression
EP2776564B1 (en) 2011-11-07 2019-10-02 Ionis Pharmaceuticals, Inc. Modulation of tmprss6 expression
AU2014268529C1 (en) * 2013-05-22 2020-10-01 Alnylam Pharmaceuticals, Inc. TMPRSS6 iRNA compositions and methods of use thereof
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
EP3865576A1 (en) 2014-12-15 2021-08-18 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
CN107429250B (zh) 2015-04-03 2022-03-01 Ionis制药公司 用于调节tmprss6表达的化合物和方法
WO2018045317A1 (en) 2016-09-02 2018-03-08 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
EP4219714A3 (en) 2017-04-05 2023-08-30 Silence Therapeutics GmbH Rna interference mediated inhibition of tmprss6
US10675295B2 (en) * 2017-06-29 2020-06-09 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting HMGB1 expression
EP3598995A1 (en) 2018-07-26 2020-01-29 Silence Therapeutics GmbH Products and compositions
IL284327B2 (en) * 2018-12-28 2024-11-01 Dicerna Pharmaceuticals Inc Compositions and methods for inhibiting hmgb1 expression
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
IL307721A (en) 2021-04-20 2023-12-01 Astrazeneca Ireland Ltd Compounds and methods for inhibiting expression of component 3
CA3216106A1 (en) 2021-04-26 2022-11-03 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

Also Published As

Publication number Publication date
US20240002858A1 (en) 2024-01-04
TW202400193A (zh) 2024-01-01
CN119403927A (zh) 2025-02-07
EP4544049A1 (en) 2025-04-30
JP2024002985A (ja) 2024-01-11
MX2024015763A (es) 2025-02-10
TWI868755B (zh) 2025-01-01
AU2023288906A1 (en) 2024-12-12
MA71238A (fr) 2025-04-30
CO2024017422A2 (es) 2025-05-08
US12503701B2 (en) 2025-12-23
WO2023247738A1 (en) 2023-12-28
KR102759742B1 (ko) 2025-02-04
CA3257962A1 (en) 2023-12-28
KR20240002200A (ko) 2024-01-04
US20240309388A1 (en) 2024-09-19
US11952574B2 (en) 2024-04-09
IL317012A (en) 2025-01-01
JP7560616B2 (ja) 2024-10-02

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