EA201892285A1 - Нуклеотиды, модифицированные циклофосфонатом по положению 5' - Google Patents
Нуклеотиды, модифицированные циклофосфонатом по положению 5'Info
- Publication number
- EA201892285A1 EA201892285A1 EA201892285A EA201892285A EA201892285A1 EA 201892285 A1 EA201892285 A1 EA 201892285A1 EA 201892285 A EA201892285 A EA 201892285A EA 201892285 A EA201892285 A EA 201892285A EA 201892285 A1 EA201892285 A1 EA 201892285A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- cyclophosphonate
- nucleotides modified
- stranded
- regulation
- oligonucleotides
- Prior art date
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/06—Pyrimidine radicals
- C07H19/10—Pyrimidine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/06—Pyrimidine radicals
- C07H19/067—Pyrimidine radicals with ribosyl as the saccharide radical
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/20—Antivirals for DNA viruses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H1/00—Processes for the preparation of sugar derivatives
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/06—Pyrimidine radicals
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/16—Purine radicals
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/16—Purine radicals
- C07H19/167—Purine radicals with ribosyl as the saccharide radical
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/16—Purine radicals
- C07H19/20—Purine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
- C07H19/207—Purine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids the phosphoric or polyphosphoric acids being esterified by a further hydroxylic compound, e.g. flavine adenine dinucleotide or nicotinamide-adenine dinucleotide
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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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
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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
- 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]
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
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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
- 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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- 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
- C12N2310/323—Chemical structure of the sugar modified ring structure
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
Landscapes
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Communicable Diseases (AREA)
- Gastroenterology & Hepatology (AREA)
- Oncology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662346304P | 2016-06-06 | 2016-06-06 | |
| PCT/US2017/036108 WO2017214112A1 (en) | 2016-06-06 | 2017-06-06 | 5'-cyclo-phosphonate modified nucleotides |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| EA201892285A1 true EA201892285A1 (ru) | 2019-07-31 |
Family
ID=60579042
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EA201892285A EA201892285A1 (ru) | 2016-06-06 | 2017-06-06 | Нуклеотиды, модифицированные циклофосфонатом по положению 5' |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US11078227B2 (enExample) |
| EP (1) | EP3464313A4 (enExample) |
| JP (3) | JP7028865B2 (enExample) |
| KR (2) | KR102639586B1 (enExample) |
| CN (2) | CN109526222B (enExample) |
| AU (3) | AU2017279512B2 (enExample) |
| CA (1) | CA3023764A1 (enExample) |
| EA (1) | EA201892285A1 (enExample) |
| IL (2) | IL290633B2 (enExample) |
| MX (2) | MX2018015109A (enExample) |
| TW (2) | TW202423483A (enExample) |
| WO (1) | WO2017214112A1 (enExample) |
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| TW202423483A (zh) * | 2016-06-06 | 2024-06-16 | 美商愛羅海德製藥公司 | 5’-環-磷酸修飾之核苷酸 |
| CN110520531A (zh) | 2017-04-05 | 2019-11-29 | 赛伦斯治疗有限责任公司 | 产品和组合物 |
| MX2020003596A (es) | 2017-10-04 | 2020-07-22 | Avidity Biosciences Inc | Composiciones de acido nucleico-polipeptido y usos de las mismos. |
| CA3142283A1 (en) * | 2019-06-06 | 2020-12-10 | Avidity Biosciences, Inc. | Nucleic acid-polypeptide compositions and uses thereof |
| WO2022056266A2 (en) | 2020-09-11 | 2022-03-17 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use |
| IL303800A (en) | 2020-12-18 | 2023-08-01 | Ionis Pharmaceuticals Inc | Compounds and methods for modulating factor xii |
| KR20230167082A (ko) | 2021-04-08 | 2023-12-07 | 애로우헤드 파마슈티컬스 인코포레이티드 | 진행성 당화 최종 산물에 대한 수용체의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법 |
| AU2022280863A1 (en) | 2021-05-28 | 2023-11-30 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of mucin 5ac (muc5ac), compositions thereof, and methods of use |
| WO2023014938A1 (en) | 2021-08-05 | 2023-02-09 | Sanegene Bio Usa Inc. | 1'-alkyl modified ribose derivatives and methods of use |
| WO2023049258A1 (en) | 2021-09-22 | 2023-03-30 | Sanegene Bio Usa Inc. | 2'-alkyl or 3'- alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides |
| CN118076621A (zh) | 2021-10-05 | 2024-05-24 | 美国圣因生物股份有限公司 | 多羟基化的环戊烷衍生物及使用方法 |
| AU2023224208A1 (en) | 2022-02-22 | 2024-08-22 | Sanegene Bio Usa Inc. | 5'-modified carbocyclic ribonucleotide derivatives and methods of use |
| WO2023220561A1 (en) | 2022-05-09 | 2023-11-16 | Sanegene Bio Usa Inc. | Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof |
| AU2023295010A1 (en) | 2022-06-14 | 2025-01-09 | Rona Bioscience, Limited | Cyclic phosphonate-modified nucleotide |
| JP2025522433A (ja) | 2022-06-15 | 2025-07-15 | アローヘッド ファーマシューティカルズ インコーポレイテッド | スーパーオキシドジスムターゼ１（ＳＯＤ１）の発現を阻害するためのＲＮＡｉ剤、その組成物、及び使用の方法 |
| AU2023307152A1 (en) | 2022-07-11 | 2025-01-09 | Sanegene Bio Usa Inc. | Optimized 2'- modified ribose derivatives and methods of use |
| WO2024031101A1 (en) | 2022-08-05 | 2024-02-08 | Sanegene Bio Usa Inc. | Double stranded rna targeting angiotensinogen (agt) and methods of use thereof |
| CN119894915A (zh) * | 2022-09-21 | 2025-04-25 | 詹森生物科技公司 | 新型稳定的核苷磷酸及其类似物 |
| EP4602164A2 (en) | 2022-10-14 | 2025-08-20 | Sanegene Bio USA Inc. | Small interfering rna targeting c3 and uses thereof |
| JP2025535539A (ja) * | 2022-11-02 | 2025-10-24 | アリゴス セラピューティクス，インコーポレーテッド | 修飾された短い干渉核酸（ｓｉＮＡ）分子及びその使用 |
| WO2024137590A2 (en) | 2022-12-19 | 2024-06-27 | Sanegene Bio Usa Inc. | Small interfering rna targeting cfb and uses thereof |
| US20240309383A1 (en) | 2023-02-24 | 2024-09-19 | Suzhou Sanegene Bio Inc. | Small interfering rna targeting hbv and uses thereof |
| WO2024186673A2 (en) | 2023-03-03 | 2024-09-12 | Sanegene Bio Usa Inc. | Small interfering rna targeting apoc3 and uses thereof |
| AU2024240837A1 (en) | 2023-03-21 | 2025-09-11 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of complement factor b (cfb) pharmaceutical compositions thereof, and methods of use |
| TW202444908A (zh) | 2023-04-06 | 2024-11-16 | 大陸商上海舶望製藥有限公司 | ５＇－膦酸酯修飾的核苷類似物及其製備的寡核苷酸 |
| WO2024263915A2 (en) | 2023-06-21 | 2024-12-26 | Sanegene Bio Usa Inc. | Double stranded rna targeting proprotein convertase subtilisin kexin 9 (pcsk9) and methods of use thereof |
| US20250064939A1 (en) | 2023-07-24 | 2025-02-27 | Sanegene Bio Usa Inc. | Lipid-based enhancement agent for rna delivery and therapy |
| US12503696B2 (en) | 2023-08-30 | 2025-12-23 | Arrowhead Pharmaceuticals, Inc. | RNAi agents for inhibiting expression of inhibin subunit beta E (INHBE), pharmaceutical compositions thereof, and methods of use |
| WO2025137384A1 (en) | 2023-12-20 | 2025-06-26 | Arrowhead Pharmaceuticals, Inc. | Methods and reagents for improved oligonucleotide synthesis |
| WO2025228441A2 (en) | 2024-04-30 | 2025-11-06 | Sanegene Bio Usa Inc. | Small interfering rna targeting inhbe and uses thereof |
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| TW202423483A (zh) * | 2016-06-06 | 2024-06-16 | 美商愛羅海德製藥公司 | 5’-環-磷酸修飾之核苷酸 |
| KR102856522B1 (ko) | 2016-09-02 | 2025-09-08 | 애로우헤드 파마슈티컬스 인코포레이티드 | 표적화 리간드 |
| TWI826365B (zh) * | 2017-01-10 | 2023-12-21 | 美商愛羅海德製藥公司 | α-1抗胰蛋白酶 (AAT) RNAi 藥劑、包含AAT RNAi 藥劑之組合物及使用方法 |
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2017
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