MX2022000497A - Edicion de acido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actua sobre acido ribonucleico endogeno (adar) utilizando acidos ribonucleicos (arn) modificados geneticamente. - Google Patents
Edicion de acido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actua sobre acido ribonucleico endogeno (adar) utilizando acidos ribonucleicos (arn) modificados geneticamente.Info
- Publication number
- MX2022000497A MX2022000497A MX2022000497A MX2022000497A MX2022000497A MX 2022000497 A MX2022000497 A MX 2022000497A MX 2022000497 A MX2022000497 A MX 2022000497A MX 2022000497 A MX2022000497 A MX 2022000497A MX 2022000497 A MX2022000497 A MX 2022000497A
- Authority
- MX
- Mexico
- Prior art keywords
- ribonucleic acid
- rna
- adar
- endogenous
- edition
- Prior art date
Links
- 229920002477 rna polymer Polymers 0.000 title 5
- RSCRGMCYBCTJGF-UHFFFAOYSA-N 3-amino-5-chloro-4-methylbenzenesulfonic acid Chemical compound CC1=C(N)C=C(S(O)(=O)=O)C=C1Cl RSCRGMCYBCTJGF-UHFFFAOYSA-N 0.000 title 1
- 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 title 1
- 102000055025 Adenosine deaminases Human genes 0.000 title 1
- 230000008901 benefit Effects 0.000 title 1
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 239000002126 C01EB10 - Adenosine Substances 0.000 abstract 1
- 238000010357 RNA editing Methods 0.000 abstract 1
- 230000026279 RNA modification Effects 0.000 abstract 1
- 229960005305 adenosine Drugs 0.000 abstract 1
- 230000009615 deamination Effects 0.000 abstract 1
- 238000006481 deamination reaction Methods 0.000 abstract 1
- 230000014789 establishment of RNA localization Effects 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 230000007115 recruitment Effects 0.000 abstract 1
Classifications
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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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
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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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- 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
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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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- C—CHEMISTRY; METALLURGY
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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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04004—Adenosine deaminase (3.5.4.4)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N2310/3519—Fusion with another nucleic acid
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N2310/532—Closed or circular
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16111—Cytomegalovirus, e.g. human herpesvirus 5
- C12N2710/16141—Use of virus, viral particle or viral elements as a vector
- C12N2710/16145—Special targeting system for viral vectors
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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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Medicinal Chemistry (AREA)
- Crystallography & Structural Chemistry (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Virology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
La presente solicitud proporciona métodos para editar ARN mediante la introducción de un ARN de reclutamiento de desaminasas en una célula hospedera para la desaminación de una adenosina en un ARN objetivo; la presente solicitud proporciona además ARN de reclutamiento de desaminasas usados en los métodos de edición de ARN y composiciones y kits que comprenden los mismos.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN2019095802 | 2019-07-12 | ||
| PCT/CN2020/101246 WO2021008447A1 (en) | 2019-07-12 | 2020-07-10 | Targeted rna editing by leveraging endogenous adar using engineered rnas |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2022000497A true MX2022000497A (es) | 2022-02-03 |
Family
ID=74210191
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2022000497A MX2022000497A (es) | 2019-07-12 | 2020-07-10 | Edicion de acido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actua sobre acido ribonucleico endogeno (adar) utilizando acidos ribonucleicos (arn) modificados geneticamente. |
Country Status (16)
| Country | Link |
|---|---|
| US (3) | US20220243194A1 (es) |
| EP (1) | EP3997229A4 (es) |
| JP (1) | JP7651190B2 (es) |
| KR (1) | KR102866423B1 (es) |
| CN (2) | CN114269919B (es) |
| AU (1) | AU2020313143B2 (es) |
| CA (1) | CA3146771A1 (es) |
| CL (1) | CL2022000080A1 (es) |
| CO (1) | CO2022001359A2 (es) |
| CR (1) | CR20220063A (es) |
| EC (1) | ECSP22010372A (es) |
| IL (1) | IL289700A (es) |
| MX (1) | MX2022000497A (es) |
| PE (1) | PE20220300A1 (es) |
| PH (1) | PH12022550059A1 (es) |
| WO (1) | WO2021008447A1 (es) |
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|---|---|---|---|---|
| EP3475424A1 (en) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Single-stranded rna-editing oligonucleotides |
| DK3507366T3 (da) | 2016-09-01 | 2020-11-02 | Proqr Therapeutics Ii Bv | Kemisk modificeret, enkeltstrengede rna-modificerende oligonukleotider |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| KR102851101B1 (ko) | 2019-04-15 | 2025-08-27 | 에디진 테라퓨틱스(베이징) 인크. | Rna를 편집하기 위한 방법 및 조성물 |
| EP3997229A4 (en) * | 2019-07-12 | 2024-07-03 | Peking University | TARGETED RNA EDITING BY HARNESSING ENDOGENOUS ADAR USING MODIFIED RNA |
| EP4069842A1 (en) | 2019-12-02 | 2022-10-12 | Shape Therapeutics Inc. | Therapeutic editing |
| CA3159799A1 (en) * | 2019-12-02 | 2021-06-10 | Prashant MALI | Engineering circular guide rnas |
| US20230242916A1 (en) * | 2020-04-15 | 2023-08-03 | Edigene Therapeutics (Beijing) Inc. | Method and drug for treating hurler syndrome |
| US20230174977A1 (en) * | 2020-05-26 | 2023-06-08 | Shape Therapeutics Inc. | Engineered circular polynucleotides |
| WO2022150974A1 (en) * | 2021-01-12 | 2022-07-21 | Peking University | Targeted rna editing by leveraging endogenous adar using engineered rnas |
| TW202300650A (zh) * | 2021-03-26 | 2023-01-01 | 美商旗艦先鋒創新有限責任(Vii)公司 | 真核系統中環狀多核糖核苷酸的產生 |
| EP4314289A1 (en) * | 2021-03-26 | 2024-02-07 | Flagship Pioneering Innovations VII, LLC | Production of circular polyribonucleotides in a prokaryotic system |
| CA3221008A1 (en) * | 2021-06-02 | 2022-12-08 | Brian CAFFERTY | Circular guide rnas for crispr/cas editing systems |
| JP7755272B2 (ja) * | 2021-08-18 | 2025-10-16 | 北京大学 | 操作されたadar動員rna及びその使用方法 |
| JP7776176B2 (ja) | 2021-08-27 | 2025-11-26 | 北京大学 | 環状rnaを調製するための構築物及び方法 |
| CN116103284B (zh) * | 2021-11-09 | 2025-09-23 | 中国科学院天津工业生物技术研究所 | 环形gRNA及其相关生物材料与应用 |
| WO2023143538A1 (zh) * | 2022-01-28 | 2023-08-03 | 北京辑因医疗科技有限公司 | 基于leaper技术治疗mpsi的方法和组合物 |
| US20250154504A1 (en) | 2022-02-14 | 2025-05-15 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| WO2023237063A1 (en) * | 2022-06-08 | 2023-12-14 | Huidagene Therapeutics Co., Ltd. | Novel guide nucleic acids for rna base editing systems and uses thereof |
| WO2024013360A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
| JP2025524566A (ja) | 2022-07-15 | 2025-07-30 | プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ | Adar媒介rna編集のためのオリゴヌクレオチドおよびその使用 |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| WO2024110565A1 (en) | 2022-11-24 | 2024-05-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis |
| GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
| EP4630553A1 (en) | 2022-12-09 | 2025-10-15 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for the treatment of cardiovascular disease |
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| EP4669753A1 (en) | 2023-02-20 | 2025-12-31 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease |
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| CN118620958A (zh) * | 2023-03-08 | 2024-09-10 | 广州医科大学 | 一种抗原表位的制备方法 |
| GB202304363D0 (en) | 2023-03-24 | 2023-05-10 | Proqr Therapeutics Ii Bv | Chemically modified antisense oligonucleotides for use in RNA editing |
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| WO2024235283A1 (en) * | 2023-05-16 | 2024-11-21 | Peking University | Method of modulating rna splicing |
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| WO2025051946A1 (en) | 2023-09-07 | 2025-03-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| WO2025056035A1 (en) * | 2023-09-13 | 2025-03-20 | Edigene Therapeutics (Beijing) Inc. | Compositions and methods for treating duchenne muscular dystrophy |
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| KR102818452B1 (ko) * | 2023-12-18 | 2025-06-16 | 주식회사 뉴클릭스바이오 | Rna 라이게이즈를 이용한 신규 원형 rna 합성법 |
| WO2025135995A1 (ko) * | 2023-12-18 | 2025-06-26 | 주식회사 뉴클릭스바이오 | Rna 라이게이즈를 이용한 신규 원형 rna 합성법 |
| WO2025132708A1 (en) | 2023-12-20 | 2025-06-26 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of huntington's disease |
| WO2025170919A1 (en) * | 2024-02-05 | 2025-08-14 | Amber Bio Inc. | Methods and compositions for trans-splicing utilizing small nuclear rnas and small nucleolar rnas |
| GB202404661D0 (en) | 2024-04-02 | 2024-05-15 | Proqr Therapeutics Ii Bv | Antisense oligoncleotides for the treatment of liver disease |
| WO2025224230A1 (en) | 2024-04-25 | 2025-10-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of fatty liver disease |
| GB202410081D0 (en) | 2024-07-11 | 2024-08-28 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of cardiovascular disease |
| WO2026022136A1 (en) | 2024-07-23 | 2026-01-29 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
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-
2020
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- 2020-07-10 JP JP2022502174A patent/JP7651190B2/ja active Active
- 2020-07-10 WO PCT/CN2020/101246 patent/WO2021008447A1/en not_active Ceased
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| AU2020313143A1 (en) | 2022-02-10 |
| CO2022001359A2 (es) | 2022-05-10 |
| PH12022550059A1 (en) | 2022-11-21 |
| PE20220300A1 (es) | 2022-03-09 |
| EP3997229A1 (en) | 2022-05-18 |
| CR20220063A (es) | 2022-07-22 |
| KR20220038706A (ko) | 2022-03-29 |
| JP7651190B2 (ja) | 2025-03-26 |
| CN114269919A (zh) | 2022-04-01 |
| WO2021008447A1 (en) | 2021-01-21 |
| IL289700A (en) | 2022-03-01 |
| CA3146771A1 (en) | 2021-01-21 |
| US20230365962A1 (en) | 2023-11-16 |
| KR102866423B1 (ko) | 2025-09-30 |
| US11661596B2 (en) | 2023-05-30 |
| AU2020313143B2 (en) | 2024-04-04 |
| CL2022000080A1 (es) | 2022-11-18 |
| BR112022000291A2 (pt) | 2022-03-15 |
| EP3997229A4 (en) | 2024-07-03 |
| ECSP22010372A (es) | 2022-03-31 |
| CN117778385A (zh) | 2024-03-29 |
| US20220135963A1 (en) | 2022-05-05 |
| CN114269919B (zh) | 2023-08-18 |
| US20220243194A1 (en) | 2022-08-04 |
| JP2022536546A (ja) | 2022-08-17 |
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