CN119403927A - 用于抑制跨膜丝氨酸蛋白酶6（tmprss6）表达的组合物和方法 - Google Patents
用于抑制跨膜丝氨酸蛋白酶6（tmprss6）表达的组合物和方法 Download PDFInfo
- Publication number
- CN119403927A CN119403927A CN202380048101.3A CN202380048101A CN119403927A CN 119403927 A CN119403927 A CN 119403927A CN 202380048101 A CN202380048101 A CN 202380048101A CN 119403927 A CN119403927 A CN 119403927A
- Authority
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- China
- Prior art keywords
- nucleotides
- oligonucleotide
- antisense strand
- seq
- sense strand
- 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.)
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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
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)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263355210P | 2022-06-24 | 2022-06-24 | |
| US63/355210 | 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 |
|---|---|
| CN119403927A true CN119403927A (zh) | 2025-02-07 |
Family
ID=87036742
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202380048101.3A Withdrawn CN119403927A (zh) | 2022-06-24 | 2023-06-23 | 用于抑制跨膜丝氨酸蛋白酶6（tmprss6）表达的组合物和方法 |
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)
| 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)
| 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 |
-
2023
- 2023-06-21 TW TW112123278A patent/TWI868755B/zh active
- 2023-06-23 MA MA71238A patent/MA71238A/fr unknown
- 2023-06-23 CA CA3257962A patent/CA3257962A1/en active Pending
- 2023-06-23 IL IL317012A patent/IL317012A/en unknown
- 2023-06-23 US US18/213,289 patent/US11952574B2/en active Active
- 2023-06-23 AU AU2023288906A patent/AU2023288906A1/en active Pending
- 2023-06-23 KR KR1020230080886A patent/KR102759742B1/ko active Active
- 2023-06-23 JP JP2023103614A patent/JP7560616B2/ja active Active
- 2023-06-23 CN CN202380048101.3A patent/CN119403927A/zh not_active Withdrawn
- 2023-06-23 EP EP23735271.1A patent/EP4544049A1/en not_active Withdrawn
- 2023-06-23 WO PCT/EP2023/067070 patent/WO2023247738A1/en not_active Ceased
- 2023-06-23 AR ARP230101626A patent/AR129710A1/es 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 |
|---|---|
| US20240002858A1 (en) | 2024-01-04 |
| TW202400193A (zh) | 2024-01-01 |
| AR129710A1 (es) | 2024-09-18 |
| 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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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PB01 | Publication | ||
| PB01 | Publication | ||
| TA01 | Transfer of patent application right | ||
| TA01 | Transfer of patent application right |
Effective date of registration: 20250521 Address after: Denmark bagsvaerd Applicant after: NOVO NORDISK A/S Country or region after: Denmark Address before: Denmark bagsvaerd Applicant before: NOVO NORDISK A/S Country or region before: Denmark Applicant before: DICERNA PHARMACEUTICALS, Inc. Country or region before: U.S.A. |
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| SE01 | Entry into force of request for substantive examination | ||
| SE01 | Entry into force of request for substantive examination | ||
| WW01 | Invention patent application withdrawn after publication | ||
| WW01 | Invention patent application withdrawn after publication |
Application publication date: 20250207 |