CA3257962A1 - COMPOSITIONS AND METHODS FOR INHIBITING THE EXPRESSION OF TRANSMEMBRANE SERINE PROTEASE 6 (TMPRSS6) - Google Patents
COMPOSITIONS AND METHODS FOR INHIBITING THE EXPRESSION OF TRANSMEMBRANE SERINE PROTEASE 6 (TMPRSS6)Info
- Publication number
- CA3257962A1 CA3257962A1 CA3257962A CA3257962A CA3257962A1 CA 3257962 A1 CA3257962 A1 CA 3257962A1 CA 3257962 A CA3257962 A CA 3257962A CA 3257962 A CA3257962 A CA 3257962A CA 3257962 A1 CA3257962 A1 CA 3257962A1
- Authority
- CA
- Canada
- Prior art keywords
- nucleotides
- oligonucleotide
- seq
- antisense strand
- tmprss6
- 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.)
- Pending
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)
- 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)
- Public Health (AREA)
- 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)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
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 |
|---|---|
| CA3257962A1 true CA3257962A1 (en) | 2023-12-28 |
Family
ID=87036742
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3257962A Pending CA3257962A1 (en) | 2022-06-24 | 2023-06-23 | COMPOSITIONS AND METHODS FOR INHIBITING THE EXPRESSION OF TRANSMEMBRANE SERINE PROTEASE 6 (TMPRSS6) |
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)
| 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)
| 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 |
-
2023
- 2023-06-21 TW TW112123278A patent/TWI868755B/zh active
- 2023-06-23 AR ARP230101626A patent/AR129710A1/es unknown
- 2023-06-23 EP EP23735271.1A patent/EP4544049A1/en not_active Withdrawn
- 2023-06-23 CN CN202380048101.3A patent/CN119403927A/zh not_active Withdrawn
- 2023-06-23 JP JP2023103614A patent/JP7560616B2/ja active Active
- 2023-06-23 WO PCT/EP2023/067070 patent/WO2023247738A1/en not_active Ceased
- 2023-06-23 AU AU2023288906A patent/AU2023288906A1/en active Pending
- 2023-06-23 MA MA71238A patent/MA71238A/fr unknown
- 2023-06-23 KR KR1020230080886A patent/KR102759742B1/ko active Active
- 2023-06-23 CA CA3257962A patent/CA3257962A1/en active Pending
- 2023-06-23 US US18/213,289 patent/US11952574B2/en active Active
- 2023-06-23 IL IL317012A patent/IL317012A/en unknown
-
2024
- 2024-02-14 US US18/441,001 patent/US12503701B2/en active Active
- 2024-12-17 MX MX2024015763A patent/MX2024015763A/es unknown
- 2024-12-18 CO CONC2024/0017422A patent/CO2024017422A2/es unknown
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 |
| CO2024017422A2 (es) | 2025-05-08 |
| AR129710A1 (es) | 2024-09-18 |
| IL317012A (en) | 2025-01-01 |
| AU2023288906A1 (en) | 2024-12-12 |
| MA71238A (fr) | 2025-04-30 |
| KR102759742B1 (ko) | 2025-02-04 |
| JP7560616B2 (ja) | 2024-10-02 |
| TWI868755B (zh) | 2025-01-01 |
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