CN116323943A - 用于抑制lpa表达的组合物和方法 - Google Patents
用于抑制lpa表达的组合物和方法 Download PDFInfo
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- CN116323943A CN116323943A CN202180058046.7A CN202180058046A CN116323943A CN 116323943 A CN116323943 A CN 116323943A CN 202180058046 A CN202180058046 A CN 202180058046A CN 116323943 A CN116323943 A CN 116323943A
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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
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- 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
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- 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
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- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21007—Plasmin (3.4.21.7), i.e. fibrinolysin
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- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/312—Phosphonates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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- 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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- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
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- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Agricultural Chemicals And Associated Chemicals (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063061676P | 2020-08-05 | 2020-08-05 | |
| US63/061676 | 2020-08-05 | ||
| US202063074779P | 2020-09-04 | 2020-09-04 | |
| US63/074779 | 2020-09-04 | ||
| PCT/US2021/071109 WO2022032288A1 (en) | 2020-08-05 | 2021-08-05 | Compositions and methods for inhibiting lpa expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN116323943A true CN116323943A (zh) | 2023-06-23 |
Family
ID=77519885
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202180058046.7A Pending CN116323943A (zh) | 2020-08-05 | 2021-08-05 | 用于抑制lpa表达的组合物和方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US12435336B2 (enExample) |
| EP (1) | EP4192954A1 (enExample) |
| JP (2) | JP7476422B2 (enExample) |
| KR (2) | KR102812602B1 (enExample) |
| CN (1) | CN116323943A (enExample) |
| AU (2) | AU2021320550B2 (enExample) |
| BR (1) | BR112023001957A2 (enExample) |
| CA (1) | CA3185348A1 (enExample) |
| IL (1) | IL300338A (enExample) |
| MX (1) | MX2023001538A (enExample) |
| TW (2) | TW202535432A (enExample) |
| WO (1) | WO2022032288A1 (enExample) |
Cited By (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025082058A1 (zh) * | 2023-10-16 | 2025-04-24 | 润佳(上海)医药技术有限公司 | 一种抑制LPA基因表达的dsRNA及其用途 |
| WO2025087442A1 (zh) * | 2023-10-27 | 2025-05-01 | 北京安龙生物医药有限公司 | 靶向脂蛋白a的寡核苷酸及其用途 |
| WO2025103457A1 (zh) * | 2023-11-17 | 2025-05-22 | 纳肽得(青岛)生物医药有限公司 | 抑制LPA表达的siRNA及其用途 |
| WO2025119212A1 (zh) * | 2023-12-05 | 2025-06-12 | 广东东阳光药业股份有限公司 | 一种新型的双链siRNA、其缀合物及其用途 |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4192954A1 (en) | 2020-08-05 | 2023-06-14 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting lpa expression |
| TW202340465A (zh) * | 2021-12-16 | 2023-10-16 | 大陸商上海拓界生物醫藥科技有限公司 | 靶向lpa的sirna及綴合物 |
| CN114703184B (zh) * | 2022-03-11 | 2024-06-18 | 厦门甘宝利生物医药有限公司 | Lpa抑制剂及其用途 |
| IL315878A (en) * | 2022-04-15 | 2024-11-01 | Dicerna Pharmaceuticals Inc | Compounds and Methods for Modulating SCAP Activity |
| IL318877A (en) * | 2022-08-11 | 2025-04-01 | Sirius Therapeutics Inc | Polynucleic acid molecules for inhibiting LP(A) expression, pharmaceutical compositions and uses thereof |
| CN115851723B (zh) * | 2022-10-24 | 2023-10-03 | 厦门甘宝利生物医药有限公司 | 一种抑制lpa基因表达的rna抑制剂及其应用 |
| AU2023384015A1 (en) | 2022-11-23 | 2025-05-22 | Eli Lilly And Company | Methods of synthesizing a targeting ligand-conjugated nucleotide phosphoramidite |
| KR20250110896A (ko) | 2022-11-23 | 2025-07-21 | 일라이 릴리 앤드 캄파니 | 4'-포스페이트 유사체 뉴클레오티드 포스포르아미다이트의 합성 방법 |
| CN120752338A (zh) | 2022-12-22 | 2025-10-03 | 伊莱利利公司 | 寡核苷酸片段以及使用其制备RNAi剂的方法 |
| KR20250130686A (ko) | 2023-01-13 | 2025-09-02 | 일라이 릴리 앤드 캄파니 | 치료 올리고뉴클레오티드-함유 제약 조성물 및 그를 사용한 투여 요법 |
| WO2024197065A2 (en) * | 2023-03-20 | 2024-09-26 | Verve Therapeutics, Inc. | In vivo nickase-based editing of the lpa gene for treatment of cardiovascular disease |
| CN118685405A (zh) * | 2023-06-20 | 2024-09-24 | 施能康医药科技(苏州)有限公司 | 靶向脂蛋白a的核酸及其用途 |
| US20250215044A1 (en) | 2023-09-21 | 2025-07-03 | Ionis Pharmaceuticals, Inc. | Compounds and Methods for Inhibiting LPA |
| WO2025117709A2 (en) * | 2023-11-29 | 2025-06-05 | Sanegene Bio Usa Inc. | Small interfering rna targeting lp(a) and uses thereof |
| WO2025170868A1 (en) * | 2024-02-05 | 2025-08-14 | Sirius Therapeutics, Inc. | Polynucleic acid molecules for inhibiting expression of lp(a) or apoc3, pharmaceutical compositions, and uses thereof |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
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| 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 |
| DE69431669T2 (de) | 1993-09-02 | 2003-10-23 | Ribozyme Pharmaceuticals, 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 |
| KR20080023768A (ko) | 2000-03-30 | 2008-03-14 | 화이트헤드 인스티튜트 포 바이오메디칼 리서치 | Rna 간섭의 rna 서열 특이적인 매개체 |
| KR100909681B1 (ko) | 2000-12-01 | 2009-07-29 | 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. | Rna 간섭을 매개하는 작은 rna 분자 |
| 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 |
| US20090018097A1 (en) | 2005-09-02 | 2009-01-15 | Mdrna, Inc | Modification of double-stranded ribonucleic acid molecules |
| AU2009293636A1 (en) | 2008-09-22 | 2010-03-25 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for the specific inhibition of gene expression by dsRNA possessing modifications |
| US8691971B2 (en) | 2008-09-23 | 2014-04-08 | Scott G. Petersen | Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating RNA interference |
| 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 |
| WO2010093788A2 (en) | 2009-02-11 | 2010-08-19 | Dicerna Pharmaceuticals, Inc. | Multiplex dicer substrate rna interference molecules having joining sequences |
| 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 |
| KR20140084232A (ko) | 2011-10-25 | 2014-07-04 | 아이시스 파마수티컬즈 인코포레이티드 | Gccr 발현의 안티센스 조절 |
| KR20150090917A (ko) | 2012-12-06 | 2015-08-06 | 머크 샤프 앤드 돔 코포레이션 | 디술피드-차폐 전구약물 조성물 및 방법 |
| US20170114341A1 (en) | 2014-06-06 | 2017-04-27 | Solstice Biologics, Ltd. | Polynucleotide constructs having bioreversible and non-bioreversible groups |
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| JOP20210043A1 (ar) * | 2015-10-01 | 2017-06-16 | Arrowhead Pharmaceuticals Inc | تراكيب وأساليب لتثبيط تعبير جيني للـ lpa |
| MX2019002339A (es) | 2016-09-02 | 2019-05-16 | Dicerna Pharmaceuticals Inc | Analogos de 4'-fosfato y oligonucleotidos que los comprenden. |
| SI3710586T1 (sl) | 2017-11-13 | 2023-03-31 | Silence Therapeutics Gmbh | Nukleinske kisline za inhibiranje izražanja LPA v celici |
| AR113490A1 (es) * | 2017-12-12 | 2020-05-06 | Amgen Inc | CONSTRUCCIONES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3 Y MÉTODOS DE USO DE LAS MISMAS |
| AU2020399636A1 (en) * | 2019-12-09 | 2022-06-02 | Amgen Inc. | RNAi constructs and methods for inhibiting LPA expression |
| EP4192954A1 (en) | 2020-08-05 | 2023-06-14 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting lpa expression |
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2021
- 2021-08-05 EP EP21762318.0A patent/EP4192954A1/en active Pending
- 2021-08-05 TW TW114118397A patent/TW202535432A/zh unknown
- 2021-08-05 KR KR1020237006019A patent/KR102812602B1/ko active Active
- 2021-08-05 CA CA3185348A patent/CA3185348A1/en active Pending
- 2021-08-05 BR BR112023001957A patent/BR112023001957A2/pt unknown
- 2021-08-05 KR KR1020257016689A patent/KR20250077604A/ko active Pending
- 2021-08-05 AU AU2021320550A patent/AU2021320550B2/en active Active
- 2021-08-05 US US18/040,302 patent/US12435336B2/en active Active
- 2021-08-05 JP JP2023507767A patent/JP7476422B2/ja active Active
- 2021-08-05 TW TW110128888A patent/TWI886314B/zh active
- 2021-08-05 MX MX2023001538A patent/MX2023001538A/es unknown
- 2021-08-05 IL IL300338A patent/IL300338A/en unknown
- 2021-08-05 WO PCT/US2021/071109 patent/WO2022032288A1/en not_active Ceased
- 2021-08-05 CN CN202180058046.7A patent/CN116323943A/zh active Pending
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2024
- 2024-03-07 JP JP2024034617A patent/JP2024081668A/ja active Pending
- 2024-08-16 US US18/807,591 patent/US12252691B2/en active Active
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2025
- 2025-10-17 AU AU2025252633A patent/AU2025252633A1/en active Pending
Cited By (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025082058A1 (zh) * | 2023-10-16 | 2025-04-24 | 润佳(上海)医药技术有限公司 | 一种抑制LPA基因表达的dsRNA及其用途 |
| WO2025087442A1 (zh) * | 2023-10-27 | 2025-05-01 | 北京安龙生物医药有限公司 | 靶向脂蛋白a的寡核苷酸及其用途 |
| CN120344664A (zh) * | 2023-10-27 | 2025-07-18 | 北京安龙生物医药有限公司 | 靶向脂蛋白a的寡核苷酸及其用途 |
| WO2025103457A1 (zh) * | 2023-11-17 | 2025-05-22 | 纳肽得(青岛)生物医药有限公司 | 抑制LPA表达的siRNA及其用途 |
| WO2025119212A1 (zh) * | 2023-12-05 | 2025-06-12 | 广东东阳光药业股份有限公司 | 一种新型的双链siRNA、其缀合物及其用途 |
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| US20240401053A1 (en) | 2024-12-05 |
| IL300338A (en) | 2023-04-01 |
| KR102812602B1 (ko) | 2025-05-28 |
| TW202535432A (zh) | 2025-09-16 |
| WO2022032288A1 (en) | 2022-02-10 |
| US12435336B2 (en) | 2025-10-07 |
| JP2023536974A (ja) | 2023-08-30 |
| TW202220673A (zh) | 2022-06-01 |
| TWI886314B (zh) | 2025-06-11 |
| CA3185348A1 (en) | 2022-02-10 |
| EP4192954A1 (en) | 2023-06-14 |
| KR20230043912A (ko) | 2023-03-31 |
| AU2021320550B2 (en) | 2025-11-06 |
| MX2023001538A (es) | 2023-03-08 |
| KR20250077604A (ko) | 2025-05-30 |
| AU2025252633A1 (en) | 2025-11-27 |
| BR112023001957A2 (pt) | 2023-04-25 |
| AU2021320550A1 (en) | 2023-02-16 |
| JP2024081668A (ja) | 2024-06-18 |
| US20240344063A1 (en) | 2024-10-17 |
| US12252691B2 (en) | 2025-03-18 |
| JP7476422B2 (ja) | 2024-04-30 |
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