BR112022006205A2 - Oligonucleotídeo; composição farmacêutica; composição de oligonucleotídeo; fosforamidita; método de preparação de um oligonucleotídeo ou composição; método para caracterizar um oligonucleotídeo ou uma composição; método para modificar uma adenosina alvo em um ácido nucleico alvo; método para desaminar uma adenosina alvo em um ácido nucleico alvo; método para prevenção ou tratamento de uma afecção, distúrbio ou doença passível de uma mutação de g para a; e composto, oligonucleotídeo, composição ou método - Google Patents
Oligonucleotídeo; composição farmacêutica; composição de oligonucleotídeo; fosforamidita; método de preparação de um oligonucleotídeo ou composição; método para caracterizar um oligonucleotídeo ou uma composição; método para modificar uma adenosina alvo em um ácido nucleico alvo; método para desaminar uma adenosina alvo em um ácido nucleico alvo; método para prevenção ou tratamento de uma afecção, distúrbio ou doença passível de uma mutação de g para a; e composto, oligonucleotídeo, composição ou métodoInfo
- Publication number
- BR112022006205A2 BR112022006205A2 BR112022006205A BR112022006205A BR112022006205A2 BR 112022006205 A2 BR112022006205 A2 BR 112022006205A2 BR 112022006205 A BR112022006205 A BR 112022006205A BR 112022006205 A BR112022006205 A BR 112022006205A BR 112022006205 A2 BR112022006205 A2 BR 112022006205A2
- Authority
- BR
- Brazil
- Prior art keywords
- oligonucleotide
- composition
- nucleic acid
- target
- disease
- Prior art date
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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
-
- 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
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
- C12N2310/314—Phosphoramidates
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
-
- 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
-
- 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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962911334P | 2019-10-06 | 2019-10-06 | |
| US202062959917P | 2020-01-11 | 2020-01-11 | |
| US202063022559P | 2020-05-10 | 2020-05-10 | |
| US202063069696P | 2020-08-24 | 2020-08-24 | |
| PCT/US2020/054436 WO2021071858A1 (en) | 2019-10-06 | 2020-10-06 | Oligonucleotide compositions and methods of use thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022006205A2 true BR112022006205A2 (pt) | 2022-07-19 |
Family
ID=75437497
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022006205A BR112022006205A2 (pt) | 2019-10-06 | 2020-10-06 | Oligonucleotídeo; composição farmacêutica; composição de oligonucleotídeo; fosforamidita; método de preparação de um oligonucleotídeo ou composição; método para caracterizar um oligonucleotídeo ou uma composição; método para modificar uma adenosina alvo em um ácido nucleico alvo; método para desaminar uma adenosina alvo em um ácido nucleico alvo; método para prevenção ou tratamento de uma afecção, distúrbio ou doença passível de uma mutação de g para a; e composto, oligonucleotídeo, composição ou método |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230220384A1 (https=) |
| EP (1) | EP4022059A4 (https=) |
| JP (2) | JP7766589B2 (https=) |
| KR (1) | KR20220076508A (https=) |
| CN (1) | CN114585737A (https=) |
| AU (1) | AU2020363391A1 (https=) |
| BR (1) | BR112022006205A2 (https=) |
| CA (1) | CA3154768A1 (https=) |
| IL (1) | IL291933A (https=) |
| MX (1) | MX2022004101A (https=) |
| WO (1) | WO2021071858A1 (https=) |
Families Citing this family (62)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102213609B1 (ko) | 2012-07-13 | 2021-02-08 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| PL2872485T3 (pl) | 2012-07-13 | 2021-05-31 | Wave Life Sciences Ltd. | Asymetryczna grupa pomocnicza |
| BR112016016400A2 (pt) | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos |
| IL320434A (en) | 2015-07-22 | 2025-06-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods |
| CN108779132B (zh) | 2016-03-13 | 2022-04-15 | 波涛生命科学有限公司 | 用于亚磷酰胺和寡核苷酸合成的组合物和方法 |
| JP2019520339A (ja) | 2016-06-03 | 2019-07-18 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | オリゴヌクレオチド、その組成物および方法 |
| WO2017220751A1 (en) | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| CN110352244B (zh) | 2016-09-01 | 2023-03-21 | ProQR治疗上市公司Ⅱ | 化学修饰的编辑rna的单链寡核苷酸 |
| US11873316B2 (en) | 2016-11-23 | 2024-01-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| CA3065523A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| JP2020522510A (ja) | 2017-06-02 | 2020-07-30 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | オリゴヌクレオチド組成物及びその使用方法 |
| US11718638B2 (en) | 2017-06-21 | 2023-08-08 | Wave Life Sciences Ltd. | Compounds, compositions and methods for synthesis |
| EP3664816A4 (en) | 2017-08-08 | 2021-05-19 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED PROCESSES |
| CA3072110A1 (en) | 2017-09-18 | 2019-03-21 | Wave Life Sciences Ltd. | Technologies for oligonucleotide preparation |
| EP3694530A4 (en) | 2017-10-12 | 2021-06-30 | Wave Life Sciences Ltd. | COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES |
| SG11202009877XA (en) | 2018-04-12 | 2020-11-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods of use thereof |
| US12391942B2 (en) | 2018-05-11 | 2025-08-19 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| JP2022525541A (ja) * | 2019-03-20 | 2022-05-17 | ウェイブ ライフ サイエンシズ リミテッド | オリゴヌクレオチド調製に有用な技術 |
| WO2022046667A1 (en) * | 2020-08-24 | 2022-03-03 | Wave Life Sciences Ltd. | Cells and non-human animals engineered to express adar1 and uses thereof |
| KR20230118716A (ko) * | 2020-11-08 | 2023-08-11 | 웨이브 라이프 사이언시스 리미티드 | 올리고뉴클레오티드 조성물 및 이의 방법 |
| EP4363574A1 (en) * | 2021-06-29 | 2024-05-08 | Korro Bio, Inc. | Methods and compositions for adar-mediated editing |
| US20230194709A9 (en) * | 2021-06-29 | 2023-06-22 | Seagate Technology Llc | Range information detection using coherent pulse sets with selected waveform characteristics |
| EP4396354A4 (en) * | 2021-09-26 | 2025-12-03 | Wave Life Sciences Ltd | OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED PROCESSES |
| EP4479535A1 (en) | 2022-02-14 | 2024-12-25 | ProQR Therapeutics II B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| WO2023220428A1 (en) * | 2022-05-12 | 2023-11-16 | Wave Life Sciences Ltd. | Compositions for editing ass1 transcripts and methods thereof |
| US20250302995A1 (en) * | 2022-05-12 | 2025-10-02 | Chikdu Shakti Shivalila | Oligonucleotide compositions and methods thereof |
| US20260027237A1 (en) | 2022-07-15 | 2026-01-29 | 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 |
| JP2025525564A (ja) * | 2022-07-18 | 2025-08-05 | エフ． ホフマン－ラ ロシュ アーゲー | オリゴヌクレオチドの編集 |
| 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 |
| WO2024114908A1 (en) | 2022-11-30 | 2024-06-06 | Eberhard Karls Universität Tübingen | Chemically modified antisense oligonucleotides (asos) and compositions comprising the same for rna editing |
| GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
| AU2023390632A1 (en) | 2022-12-09 | 2025-07-10 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of cardiovascular disease |
| GB202300865D0 (en) | 2023-01-20 | 2023-03-08 | Proqr Therapeutics Ii Bv | Delivery of oligonucleotides |
| WO2024175550A1 (en) | 2023-02-20 | 2024-08-29 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease |
| EP4669750A2 (en) | 2023-02-21 | 2025-12-31 | Vib Vzw | SYNAPTOGYRIN-3 EXPRESSION INHIBITORS |
| WO2024175588A1 (en) | 2023-02-21 | 2024-08-29 | Vib Vzw | Oligonucleotides for modulating synaptogyrin-3 expression |
| EP4432289A1 (en) | 2023-03-15 | 2024-09-18 | Shape Therapeutics Inc. | Generative sequence screening with conditional gans, diffusion models, and denoising diffusion conditional gans |
| EP4689110A1 (en) | 2023-03-24 | 2026-02-11 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for the treatment of neurological disorders |
| GB202304363D0 (en) | 2023-03-24 | 2023-05-10 | Proqr Therapeutics Ii Bv | Chemically modified antisense oligonucleotides for use in RNA editing |
| EP4688143A1 (en) | 2023-03-27 | 2026-02-11 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for the treatment of liver disease |
| TW202516003A (zh) | 2023-06-16 | 2025-04-16 | 荷蘭商Ｐｒｏｑｒ治療上市公司Ｉｉ | 用於治療神經退化性疾病之反義寡核苷酸 |
| WO2025015335A1 (en) | 2023-07-13 | 2025-01-16 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
| CN121794378A (zh) | 2023-08-15 | 2026-04-03 | 艾尔纳公司 | 用于rna编辑的化学修饰的反义寡核苷酸（aso）及组合物 |
| WO2025051946A1 (en) | 2023-09-07 | 2025-03-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| AU2024354150A1 (en) * | 2023-09-28 | 2026-03-12 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| WO2025073902A1 (en) | 2023-10-06 | 2025-04-10 | Eberhard Karls Universität Tübingen | Chemical and sequence optimisation of antisense oligonucleotides for adar-mediated rna editing |
| WO2025083268A1 (en) | 2023-10-20 | 2025-04-24 | Airna Corporation | Chemically modified antisense oligonucleotides (asos) and compositions comprising the same for rna editing |
| WO2025096809A1 (en) | 2023-10-31 | 2025-05-08 | Korro Bio, Inc. | Oligonucleotides comprising phosphoramidate internucleotide linkages |
| WO2025104239A1 (en) | 2023-11-16 | 2025-05-22 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of classic galactosemia |
| WO2025128799A1 (en) | 2023-12-12 | 2025-06-19 | Korro Bio, Inc. | Double-stranded rna-editing oligonucleotides and uses thereof |
| WO2025132708A1 (en) | 2023-12-20 | 2025-06-26 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of huntington's disease |
| WO2025172372A1 (en) * | 2024-02-12 | 2025-08-21 | Airna Corporation | Conjugate for targeted delivery of an antisense oligonucleotide |
| 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 |
| WO2026050243A1 (en) | 2024-08-26 | 2026-03-05 | Korro Bio, Inc. | Galnac conjugated oligonucleotides for rna editing |
| WO2026060374A2 (en) | 2024-09-16 | 2026-03-19 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurological disorders |
| WO2026068781A1 (en) | 2024-09-30 | 2026-04-02 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of liver disease |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014169206A2 (en) * | 2013-04-11 | 2014-10-16 | Carnegie Mellon University | Divalent nucleobase compounds and uses therefor |
| BR112016016400A2 (pt) * | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos |
| RU2708237C2 (ru) * | 2014-08-22 | 2019-12-05 | Общество с ограниченной ответственностью "НооГен" | Модифицированные олигонуклеотиды и способ их получения |
| US10787664B2 (en) * | 2015-05-26 | 2020-09-29 | City Of Hope | Compounds of chemically modified oligonucleotides and methods of use thereof |
| IL295755A (en) * | 2015-10-09 | 2022-10-01 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| JP2019520339A (ja) * | 2016-06-03 | 2019-07-18 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | オリゴヌクレオチド、その組成物および方法 |
| CN110352244B (zh) * | 2016-09-01 | 2023-03-21 | ProQR治疗上市公司Ⅱ | 化学修饰的编辑rna的单链寡核苷酸 |
| US11718638B2 (en) * | 2017-06-21 | 2023-08-08 | Wave Life Sciences Ltd. | Compounds, compositions and methods for synthesis |
-
2020
- 2020-10-06 JP JP2022520901A patent/JP7766589B2/ja active Active
- 2020-10-06 US US17/766,677 patent/US20230220384A1/en active Pending
- 2020-10-06 CA CA3154768A patent/CA3154768A1/en active Pending
- 2020-10-06 EP EP20873624.9A patent/EP4022059A4/en active Pending
- 2020-10-06 CN CN202080069504.2A patent/CN114585737A/zh active Pending
- 2020-10-06 BR BR112022006205A patent/BR112022006205A2/pt unknown
- 2020-10-06 KR KR1020227015085A patent/KR20220076508A/ko active Pending
- 2020-10-06 MX MX2022004101A patent/MX2022004101A/es unknown
- 2020-10-06 AU AU2020363391A patent/AU2020363391A1/en active Pending
- 2020-10-06 WO PCT/US2020/054436 patent/WO2021071858A1/en not_active Ceased
-
2022
- 2022-04-04 IL IL291933A patent/IL291933A/en unknown
-
2025
- 2025-07-17 JP JP2025120874A patent/JP2025165976A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| MX2022004101A (es) | 2022-04-26 |
| IL291933A (en) | 2022-06-01 |
| AU2020363391A1 (en) | 2022-03-24 |
| US20230220384A1 (en) | 2023-07-13 |
| WO2021071858A1 (en) | 2021-04-15 |
| CN114585737A (zh) | 2022-06-03 |
| EP4022059A1 (en) | 2022-07-06 |
| JP2022551124A (ja) | 2022-12-07 |
| CA3154768A1 (en) | 2021-04-15 |
| EP4022059A4 (en) | 2023-11-01 |
| JP2025165976A (ja) | 2025-11-05 |
| KR20220076508A (ko) | 2022-06-08 |
| JP7766589B2 (ja) | 2025-11-10 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| BR112022006205A2 (pt) | Oligonucleotídeo; composição farmacêutica; composição de oligonucleotídeo; fosforamidita; método de preparação de um oligonucleotídeo ou composição; método para caracterizar um oligonucleotídeo ou uma composição; método para modificar uma adenosina alvo em um ácido nucleico alvo; método para desaminar uma adenosina alvo em um ácido nucleico alvo; método para prevenção ou tratamento de uma afecção, distúrbio ou doença passível de uma mutação de g para a; e composto, oligonucleotídeo, composição ou método | |
| BR112021017430A2 (pt) | Inibidores da proteína tirosina fosfatase e métodos de uso dos mesmos | |
| BR112021021224A2 (pt) | Anticorpos ou fragmentos de anticorpos, composição farmacêutica, kit, ácido nucleico ou conjunto de ácidos nucleicos, célula hospedeira recombinante e método para o tratamento ou a prevenção de uma doença em um paciente em necessidade do mesmo | |
| BR112017013597A2 (pt) | molécula de ácido nucleico, composição farmacêutica, vetor ou célula, e, método para tratamento de uma doença. | |
| MX2023008469A (es) | Oligonucleotidos de doble hebra modificados. | |
| PH12020552228A1 (en) | Protein tyrosine phosphatase inhibitors and methods of use thereof | |
| BR112022006207A2 (pt) | Oligonucleotídeo, composição de oligonucleotídeo, composição farmacêutica, método, método para diminuir a atividade, expressão e/ou nível de um gene alvo ou seu produto de gene em uma célula, método para reduzir focos em uma população de células, método para reduzir o nível de uma proteína de repetição de dipeptídeo (dpr), método para knockdown preferencial de uma transcrição de rna, e método para preparar um oligonucleotídeo ou composição | |
| EA202191630A1 (ru) | ХИМИЧЕСКИ МОДИФИЦИРОВАННЫЕ КОНСТРУКЦИИ ДЛЯ RNAi И ПУТИ ИХ ПРИМЕНЕНИЯ | |
| BR112022001324A2 (pt) | Composto, composição farmacêutica e método para tratamento de uma doença mediada por p38 map quinase em um indivíduo necessitando do mesmo | |
| MX2021012126A (es) | Composiciones y métodos para inhibir la expresión génica en el sistema nervioso central. | |
| BR112021025781A2 (pt) | Composto, composição farmacêutica, método de tratamento de uma doença ou transtorno associado com um defeito no metabolismo de glioxilato, método de tratamento de uma doença ou transtorno associado com a enzima glicolato oxidase (go) ou alterações no metabolismo de oxalato e método para inibição da enzima go | |
| BR112018008344A2 (pt) | molécula de ácido nucleico, composição farmacêutica, vetor ou célula, método para prevenir, tratar ou melhorar uma doença, e, uso de uma composição | |
| US20240327835A1 (en) | Methods for reactivating genes on the inactive x chromosome | |
| MA47079B1 (fr) | Composés amino-triazolopyidines et leur utilisation pour traiter le cancer | |
| MX2022016422A (es) | Inhibidores de glicolato oxidasa para el tratamiento de enfermedades. | |
| BRPI0622054B8 (pt) | composto e composição farmacêutica | |
| BR112018015413A2 (pt) | inibidores de indoleamina-2,3-dioxigenase (ido) | |
| BRPI0912475B8 (pt) | compostos como inibidores de quinase, método para produção dos ditos compostos e composição farmacêutica | |
| BR112019022280A2 (pt) | tratamento de cânceres de her2 positivo | |
| CL2021001488A1 (es) | Transposasa de piggybac mutada | |
| MX2022007441A (es) | Inhibidores de proteína tirosina fosfatasa y sus métodos de uso. | |
| CR20240360A (es) | Inhibidores de proteína tirosina fosfatasa y usos de estos | |
| EA201891429A1 (ru) | 5'-фосфоротиоатные аналоги 5'-конца (кэпа) мрнк, содержащая их мрнк, способ их получения и применение | |
| WO2022192594A3 (en) | Nucleic acid molecules and vaccines comprising same for the prevention and treatment of coronavirus infections and disease | |
| BR112018014525A2 (pt) | compostos que antagonizam receptor de adenosina a3, método para preparação dos mesmos e aplicação médica dos mesmos |