NI201800009A - Composiciones de oligonecleótidos y métodos de los mismos. - Google Patents
Composiciones de oligonecleótidos y métodos de los mismos.Info
- Publication number
- NI201800009A NI201800009A NI201800009A NI201800009A NI201800009A NI 201800009 A NI201800009 A NI 201800009A NI 201800009 A NI201800009 A NI 201800009A NI 201800009 A NI201800009 A NI 201800009A NI 201800009 A NI201800009 A NI 201800009A
- Authority
- NI
- Nicaragua
- Prior art keywords
- chirally controlled
- oligonucleotide composition
- controlled oligonucleotide
- modifications
- methods
- 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
- C12N15/113—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
-
- 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
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
-
- 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
- C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Epidemiology (AREA)
- Psychology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562195779P | 2015-07-22 | 2015-07-22 | |
| US201562236847P | 2015-10-02 | 2015-10-02 | |
| US201662331960P | 2016-05-04 | 2016-05-04 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| NI201800009A true NI201800009A (es) | 2018-06-12 |
Family
ID=57835219
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| NI201800009A NI201800009A (es) | 2015-07-22 | 2018-01-15 | Composiciones de oligonecleótidos y métodos de los mismos. |
Country Status (23)
| Country | Link |
|---|---|
| US (6) | US10479995B2 (enExample) |
| EP (3) | EP4344744A3 (enExample) |
| JP (6) | JP7296724B2 (enExample) |
| KR (1) | KR20180028516A (enExample) |
| CN (3) | CN108025089A (enExample) |
| AU (3) | AU2016297155B2 (enExample) |
| BR (1) | BR112017028636A2 (enExample) |
| CA (1) | CA2989682A1 (enExample) |
| CL (1) | CL2018000145A1 (enExample) |
| CO (1) | CO2018001484A2 (enExample) |
| CR (1) | CR20180107A (enExample) |
| EC (1) | ECSP18010463A (enExample) |
| HK (2) | HK1255369A1 (enExample) |
| IL (2) | IL320434A (enExample) |
| MA (1) | MA43072A (enExample) |
| MX (2) | MX2018000796A (enExample) |
| NI (1) | NI201800009A (enExample) |
| PE (1) | PE20181174A1 (enExample) |
| PH (1) | PH12018500145A1 (enExample) |
| SG (1) | SG10201912900XA (enExample) |
| SV (1) | SV2018005619A (enExample) |
| TW (1) | TW201707711A (enExample) |
| WO (2) | WO2017015575A1 (enExample) |
Families Citing this family (157)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8158768B2 (en) * | 2002-12-23 | 2012-04-17 | Dynavax Technologies Corporation | Immunostimulatory sequence oligonucleotides and methods of using the same |
| US9394333B2 (en) | 2008-12-02 | 2016-07-19 | Wave Life Sciences Japan | Method for the synthesis of phosphorus atom modified nucleic acids |
| MX342945B (es) | 2009-07-06 | 2016-10-18 | Ontorii Inc * | Profármacos de ácido nucleico novedosos y métodos de uso de los mismos. |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| US10273474B2 (en) | 2012-03-30 | 2019-04-30 | Washington University | Methods for modulating Tau expression for reducing seizure and modifying a neurodegenerative syndrome |
| KR20240148947A (ko) | 2012-07-13 | 2024-10-11 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| TWI772856B (zh) | 2013-07-19 | 2022-08-01 | 美商百健Ｍａ公司 | 用於調節τ蛋白表現之組合物 |
| WO2015108048A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| ES2917473T3 (es) | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| EP3234131B1 (en) * | 2014-12-16 | 2020-04-22 | Roche Innovation Center Copenhagen A/S | Chiral toxicity screening method |
| CA2968336C (en) | 2014-12-17 | 2021-11-23 | Proqr Therapeutics Ii B.V. | Construct for site directed editing of an adenosine nucleotide in target rna |
| US11129844B2 (en) | 2015-03-03 | 2021-09-28 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating MECP2 expression |
| WO2016161388A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| CN107980062B (zh) | 2015-04-03 | 2022-11-25 | 马萨诸塞大学 | 用于靶向亨廷汀mRNA的寡核苷酸化合物 |
| CN108025089A (zh) | 2015-07-22 | 2018-05-11 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| EP3334499A4 (en) * | 2015-08-14 | 2019-04-17 | University of Massachusetts | BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES |
| SG10201912902TA (en) | 2015-10-09 | 2020-02-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| CN110946865B (zh) | 2015-12-10 | 2024-01-26 | Ptc医疗公司 | 用于治疗亨廷顿病的方法 |
| US10478503B2 (en) | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| AU2017234150B2 (en) | 2016-03-13 | 2021-09-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| CN109477108A (zh) | 2016-05-04 | 2019-03-15 | 波涛生命科学有限公司 | 寡核苷酸组合物和其方法 |
| WO2017210647A1 (en) | 2016-06-03 | 2017-12-07 | Wave Life Sciences Ltd. | Oligonucleotides, compositions and methods thereof |
| CN109477103A (zh) | 2016-06-22 | 2019-03-15 | ProQR治疗上市公司Ⅱ | 单链rna-编辑寡核苷酸 |
| WO2018031933A2 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| EP3507366B1 (en) | 2016-09-01 | 2020-10-07 | ProQR Therapeutics II B.V. | Chemically modified single-stranded rna-editing oligonucleotides |
| JOP20190065A1 (ar) | 2016-09-29 | 2019-03-28 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن tau |
| JOP20190104A1 (ar) | 2016-11-10 | 2019-05-07 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن atxn3 |
| JP7296882B2 (ja) | 2016-11-23 | 2023-06-23 | ウェイブ ライフ サイエンシズ リミテッド | ホスホラミダイト及びオリゴヌクレオチド合成のための組成物及び方法 |
| KR102646318B1 (ko) | 2016-12-19 | 2024-03-12 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| HRP20240705T1 (hr) | 2016-12-19 | 2024-08-30 | Sarepta Therapeutics, Inc. | Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju |
| KR102810425B1 (ko) | 2016-12-19 | 2025-05-21 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| EP3571300A1 (en) | 2017-01-19 | 2019-11-27 | ProQR Therapeutics II B.V. | Oligonucleotide complexes for use in rna editing |
| WO2018145009A1 (en) * | 2017-02-06 | 2018-08-09 | University Of Tennessee Research Foundation | Dna-zyme based methods & compositions for treating huntington's disease |
| EP3589751A4 (en) | 2017-03-03 | 2021-11-17 | The Regents of The University of California | RNA TARGETING OF MUTATIONS VIA SUPPRESSOR RNA AND DEAMINASES |
| WO2018223056A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US11597927B2 (en) | 2017-06-02 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| EP3634953B1 (en) | 2017-06-05 | 2024-01-03 | PTC Therapeutics, Inc. | Compounds for treating huntington's disease |
| WO2018237194A1 (en) | 2017-06-21 | 2018-12-27 | Wave Life Sciences Ltd. | Compounds, compositions and methods for synthesis |
| CN110799647A (zh) | 2017-06-23 | 2020-02-14 | 马萨诸塞大学 | 双尾自递送sirna及相关的方法 |
| MX2019015578A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
| CN111163838B (zh) | 2017-06-28 | 2023-03-28 | Ptc医疗公司 | 用于治疗亨廷顿氏病的方法 |
| CA3072076A1 (en) * | 2017-08-08 | 2019-02-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| SG11202000276YA (en) | 2017-09-18 | 2020-04-29 | Wave Life Sciences Ltd | Technologies for oligonucleotide preparation |
| EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| EP3687577A1 (en) | 2017-09-28 | 2020-08-05 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| EP3687519A1 (en) | 2017-09-28 | 2020-08-05 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| JP2020536060A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
| WO2019075357A1 (en) | 2017-10-12 | 2019-04-18 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
| TWI809004B (zh) | 2017-11-09 | 2023-07-21 | 美商Ionis製藥公司 | 用於降低snca表現之化合物及方法 |
| EA202091693A1 (ru) | 2018-01-12 | 2021-04-14 | Бристол-Маерс Сквибб Компани | Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения |
| MX2020007433A (es) | 2018-01-12 | 2020-09-14 | Bristol Myers Squibb Co | Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos. |
| MX2020007369A (es) | 2018-01-15 | 2020-10-28 | Ionis Pharmaceuticals Inc | Moduladores de la expresion de dnm2. |
| AU2019218987B2 (en) | 2018-02-12 | 2025-04-24 | Ionis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
| US11661601B2 (en) | 2018-03-22 | 2023-05-30 | Ionis Pharmaceuticals, Inc. | Methods for modulating FMR1 expression |
| JP7399870B2 (ja) | 2018-03-27 | 2023-12-18 | ピーティーシー セラピューティクス， インコーポレイテッド | ハンチントン病を処置するための化合物 |
| BR112020020670A2 (pt) * | 2018-04-12 | 2021-03-02 | Wave Life Sciences Ltd. | composição de oligonucleotídeo, composição farmacêutica, método para alterar o splicing de uma transcrição alvo, método para tratar distrofia muscular, método para preparar um oligonucleotídeo ou uma composição de oligonucleotídeo do mesmo e oligonucleotídeo |
| MX2020011570A (es) | 2018-05-07 | 2020-11-24 | Alnylam Pharmaceuticals Inc | Administracion extrahepatica. |
| SG11202010012PA (en) | 2018-05-09 | 2020-11-27 | Ionis Pharmaceuticals Inc | Compounds and methods for reducing fxi expression |
| WO2019217708A1 (en) | 2018-05-09 | 2019-11-14 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing atxn3 expression |
| TWI844541B (zh) * | 2018-05-11 | 2024-06-11 | 新加坡商波濤生命科學有限公司 | 寡核苷酸組成物及其使用方法 |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| JP2021525507A (ja) * | 2018-05-25 | 2021-09-27 | ニューヨーク インスティチュート オブ テクノロジーＮｅｗ Ｙｏｒｋ Ｉｎｓｔｉｔｕｔｅ Ｏｆ Ｔｅｃｈｎｏｌｏｇｙ | 直接核酸配列決定方法 |
| US10765760B2 (en) | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| EP3806868A4 (en) | 2018-06-13 | 2022-06-22 | Sarepta Therapeutics, Inc. | EXON SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY |
| AU2019287635B2 (en) * | 2018-06-14 | 2026-02-12 | Ionis Pharmaceuticals, Inc. | Compounds and methods for increasing STMN2 expression |
| PL3814357T3 (pl) | 2018-06-27 | 2024-09-16 | Ptc Therapeutics, Inc. | Związki heterocykliczne i heteroarylowe do leczenia choroby huntingtona |
| KR20210038845A (ko) | 2018-06-27 | 2021-04-08 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료를 위한 헤테로아릴 화합물 |
| CN109030440B (zh) * | 2018-07-18 | 2020-12-04 | 西北农林科技大学 | 一种基于三氧化钼量子点检测单宁酸含量的方法 |
| CA3106986A1 (en) | 2018-07-25 | 2020-01-30 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing atxn2 expression |
| TW202449155A (zh) | 2018-07-27 | 2024-12-16 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
| AU2019316640B2 (en) | 2018-08-10 | 2026-03-12 | University Of Massachusetts | Modified oligonucleotides targeting SNPs |
| EP3840759A4 (en) | 2018-08-23 | 2022-06-01 | University Of Massachusetts | O-METHYL RICH FULLY STABILIZED OLIGONUCLEOTIDES |
| TWI869213B (zh) | 2018-09-19 | 2025-01-01 | 美商Ionis製藥公司 | Pnpla3表現之調節劑 |
| US20210380976A1 (en) * | 2018-10-05 | 2021-12-09 | Ionis Pharmaceuticals, Inc. | Chirally enriched oligomeric compounds |
| AU2019380940A1 (en) | 2018-11-15 | 2021-06-03 | Ionis Pharmaceuticals, Inc. | Modulators of IRF5 expression |
| EP3884053A4 (en) | 2018-11-21 | 2023-02-01 | Ionis Pharmaceuticals, Inc. | COMPOUNDS AND METHODS FOR REDUCING PRION EXPRESSION |
| EP3894559A4 (en) | 2018-12-03 | 2023-04-05 | Triplet Therapeutics, Inc. | METHODS OF TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS RELATED TO MLH3 ACTIVITY |
| SG11202104960PA (en) | 2018-12-13 | 2021-06-29 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| SG11202106378VA (en) | 2018-12-21 | 2021-07-29 | Ionis Pharmaceuticals Inc | Modulators of hsd17b13 expression |
| AU2020207935A1 (en) | 2019-01-18 | 2021-08-26 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| SG11202107318YA (en) * | 2019-02-01 | 2021-08-30 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| MX2021010152A (es) | 2019-02-27 | 2021-09-14 | Ionis Pharmaceuticals Inc | Moduladores de la expresion de malat1. |
| 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 |
| MD3947684T2 (ro) | 2019-03-29 | 2025-10-31 | Ionis Pharmaceuticals Inc | Compuși și metode pentru modularea UBE3A-ATS |
| JP2022528725A (ja) | 2019-04-18 | 2022-06-15 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを治療するための組成物 |
| BR112021021203A2 (pt) * | 2019-04-25 | 2022-01-04 | Wave Life Sciences Ltd | Oligonucleotídeo; composição de oligonucleotídeo quiralmente controlada; composição farmacêutica; método para prevenir, tratar ou melhorar uma afecção, distúrbio ou doença relacionada a ush2a e/ou prevenir, atrasar o início, desenvolvimento e/ou progresso, e/ou tratar uma afecção, distúrbio ou doença relacionada a ush2a em um indivíduo suscetível à mesma ou que sofre da mesma; método para aumentar o nível de salto de um éxon prejudicial em uma transcrição de gene ush2a ou seu produto de gene em uma célula; método para prevenir, atrasar o início ou progressão de, tratar ou melhorar uma afecção, distúrbio ou doença relacionada a ush2a em um indivíduo suscetível à mesma ou que sofre da mesma; método para saltar um éxon prejudicial 13 em um alelo de ush2a em um indivíduo; método para produzir ou aumentar o nível de um éxon de proteína ush2a com salto 13 em um sistema; e composto, oligonucleotídeo, composição ou método |
| US20230145795A1 (en) * | 2019-04-25 | 2023-05-11 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| CN114245794B (zh) | 2019-05-13 | 2024-09-13 | Ptc医疗公司 | 用于治疗亨廷顿氏病的化合物 |
| US20220243203A1 (en) | 2019-05-28 | 2022-08-04 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing fus expression |
| EP4660307A2 (en) | 2019-07-26 | 2025-12-10 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating gfap |
| KR20220047989A (ko) | 2019-08-09 | 2022-04-19 | 유니버시티 오브 매사추세츠 | Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드 |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| JP7757277B2 (ja) | 2019-10-14 | 2025-10-21 | アストラゼネカ・アクチエボラーグ | Ｐｎｐｌａ３発現のモジュレーター |
| US20240216369A1 (en) | 2019-11-01 | 2024-07-04 | Novartis Ag | The use of a splicing modulator for a treatment slowing progression of huntington's disease |
| CN115038789A (zh) | 2019-12-02 | 2022-09-09 | 塑造治疗公司 | 治疗性编辑 |
| WO2021168183A1 (en) * | 2020-02-21 | 2021-08-26 | Ionis Pharmaceuticals, Inc. | Methods for reducing htt expression |
| CN120505310A (zh) | 2020-02-28 | 2025-08-19 | Ionis制药公司 | 用于调节smn2的化合物和方法 |
| US20230203486A1 (en) * | 2020-03-30 | 2023-06-29 | Alnylam Pharmaceuticals, Inc. | MICROTUBULE ASSOCIATED PROTEIN TAU (MAPT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
| JP2023522957A (ja) | 2020-04-21 | 2023-06-01 | フラッグシップ パイオニアリング， インコーポレイテッド | 二機能性分子およびその使用方法 |
| IL298406A (en) | 2020-05-22 | 2023-01-01 | Wave Life Sciences Ltd | Double stranded oligonucleotide compositions and methods relating thereto |
| US12534724B2 (en) | 2020-05-26 | 2026-01-27 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| AR122534A1 (es) | 2020-06-03 | 2022-09-21 | Triplet Therapeutics Inc | Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3 |
| TW202227102A (zh) | 2020-09-22 | 2022-07-16 | 瑞典商阿斯特捷利康公司 | 治療脂肪肝病之方法 |
| MX2023005736A (es) | 2020-11-18 | 2023-05-25 | Ionis Pharmaceuticals Inc | Compuestos y metodos para modular la expresion de angiotensinogeno. |
| GB2603454A (en) | 2020-12-09 | 2022-08-10 | Ucl Business Ltd | Novel therapeutics for the treatment of neurodegenerative disorders |
| PE20240696A1 (es) | 2021-03-08 | 2024-04-10 | Servier Lab | Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina |
| TW202304446A (zh) | 2021-03-29 | 2023-02-01 | 瑞士商諾華公司 | 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途 |
| CN113122615B (zh) * | 2021-05-24 | 2024-07-09 | 湖南赛哲智造科技有限公司 | 一种应用于绝对定量高通量测序的多重pcr扩增的单分子标签引物及其应用 |
| KR20240037226A (ko) | 2021-06-22 | 2024-03-21 | 아큐라스템 인코포레이티드 | Pikfyve 안티센스 올리고뉴클레오티드 |
| IL309334A (en) | 2021-06-23 | 2024-02-01 | Univ Massachusetts | Anti-FLT1 optimized oligonucleotide compounds for the treatment of preeclampsia and other angiogenic disorders |
| KR20240095157A (ko) | 2021-07-21 | 2024-06-25 | 아큐라스템 인코포레이티드 | Unc13a 안티센스 올리고뉴클레오타이드 |
| AU2022358322A1 (en) | 2021-09-30 | 2024-05-16 | Sarepta Therapeutics, Inc. | Antisense oligonucleotides having one or more abasic units |
| IL311518A (en) | 2021-10-01 | 2024-05-01 | Adarx Pharmaceuticals Inc | Preparations that modulate perkalkerine and methods of using them |
| GB202117758D0 (en) | 2021-12-09 | 2022-01-26 | Ucl Business Ltd | Therapeutics for the treatment of neurodegenerative disorders |
| US20250154504A1 (en) | 2022-02-14 | 2025-05-15 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| KR20250007559A (ko) | 2022-04-28 | 2025-01-14 | 아큐라스템 인코포레이티드 | Syf2 안티센스 올리고뉴클레오타이드 |
| US20260027237A1 (en) | 2022-07-15 | 2026-01-29 | Proqr Therapeutics Ii B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
| EP4555086A1 (en) | 2022-07-15 | 2025-05-21 | ProQR Therapeutics II B.V. | Oligonucleotides for adar-mediated rna editing and use thereof |
| CN120476205A (zh) * | 2022-08-11 | 2025-08-12 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| EP4590311A2 (en) | 2022-09-21 | 2025-07-30 | Sarepta Therapeutics, Inc. | Dmd antisense oligonucleotide-mediated exon skipping efficiency |
| US12152052B2 (en) | 2022-09-23 | 2024-11-26 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing MECP2 expression |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| JP2025536808A (ja) | 2022-11-24 | 2025-11-07 | プロキューアール・セラピューティクス・セカンド・ベスローテン・フェンノートシャップ | 遺伝性ｈｆｅヘモクロマトーシスの治療のためのアンチセンスオリゴヌクレオチド |
| GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
| JP2025540146A (ja) | 2022-12-09 | 2025-12-11 | 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 | 心血管疾患の治療のためのアンチセンスオリゴヌクレオチド |
| WO2024155986A2 (en) | 2023-01-20 | 2024-07-25 | AcuraStem Incorporated | Unc13a antisense oligonucleotides |
| GB202300865D0 (en) | 2023-01-20 | 2023-03-08 | Proqr Therapeutics Ii Bv | Delivery of oligonucleotides |
| IL322500A (en) | 2023-01-31 | 2025-09-01 | AcuraStem Incorporated | syf2 molecular-readable oligonucleotides |
| AU2024217638A1 (en) | 2023-02-06 | 2025-07-31 | AcuraStem Incorporated | Pikfyve antisense oligonucleotides |
| EP4669753A1 (en) | 2023-02-20 | 2025-12-31 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease |
| WO2024206175A1 (en) | 2023-03-24 | 2024-10-03 | 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 |
| CN121002181A (zh) | 2023-03-27 | 2025-11-21 | ProQR治疗上市公司Ⅱ | 用于治疗肝脏疾病的反义寡核苷酸 |
| EP4698652A2 (en) | 2023-04-20 | 2026-02-25 | Adarx Pharmaceuticals, Inc. | Mapt-modulating compositions and methods of use thereof |
| WO2024227040A1 (en) | 2023-04-27 | 2024-10-31 | Sarepta Therapeutics, Inc. | Antisense oligomers for treatment of chronic kidney disease |
| KR20260021635A (ko) | 2023-05-12 | 2026-02-13 | 아다르엑스 파마슈티컬스, 인크. | Nmda 리간드 접합 화합물 및 이의 용도 |
| CN121335980A (zh) | 2023-05-26 | 2026-01-13 | 阿达尔克斯制药有限公司 | Sod1调节组合物及其使用方法 |
| 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 |
| TW202506137A (zh) | 2023-06-20 | 2025-02-16 | 美商雅迪克斯製藥公司 | Ｌｒｒｋ２調節組合物及其使用方法 |
| AU2024287308A1 (en) | 2023-07-13 | 2025-12-18 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
| AU2024323589A1 (en) | 2023-08-16 | 2026-03-05 | Les Laboratoires Servier | Oligonucleotides for modulating kcnt1 expression |
| WO2025051946A1 (en) | 2023-09-07 | 2025-03-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| US20250243487A1 (en) | 2023-09-14 | 2025-07-31 | Ionis Pharmaceuticals, Inc. | Compounds and Methods for Reducing APOCIII Expression |
| WO2025064821A2 (en) | 2023-09-21 | 2025-03-27 | Ionis Pharmaceuticals, Inc. | Compounds and methods for inhibiting lpa |
| GB202314724D0 (en) | 2023-09-26 | 2023-11-08 | Astrazeneca Ab | compounds and methods for reducing psd3 expression |
| WO2025085810A2 (en) | 2023-10-18 | 2025-04-24 | Sarepta Therapeutics, Inc. | Antisense oligomers for treatment of centronuclear myopathies |
| AU2024369609A1 (en) | 2023-10-31 | 2025-12-11 | 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 |
| 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 |
Family Cites Families (211)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US513030A (en) | 1894-01-16 | Machine for waxing or coating paper | ||
| US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
| US5132418A (en) | 1980-02-29 | 1992-07-21 | University Patents, Inc. | Process for preparing polynucleotides |
| US4500707A (en) | 1980-02-29 | 1985-02-19 | University Patents, Inc. | Nucleosides useful in the preparation of polynucleotides |
| US4973679A (en) | 1981-03-27 | 1990-11-27 | University Patents, Inc. | Process for oligonucleo tide synthesis using phosphormidite intermediates |
| US4668777A (en) | 1981-03-27 | 1987-05-26 | University Patents, Inc. | Phosphoramidite nucleoside compounds |
| US4415732A (en) | 1981-03-27 | 1983-11-15 | University Patents, Inc. | Phosphoramidite compounds and processes |
| DE3329892A1 (de) | 1983-08-18 | 1985-03-07 | Köster, Hubert, Prof. Dr., 2000 Hamburg | Verfahren zur herstellung von oligonucleotiden |
| US5118800A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | Oligonucleotides possessing a primary amino group in the terminal nucleotide |
| FR2567892B1 (fr) | 1984-07-19 | 1989-02-17 | Centre Nat Rech Scient | Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons |
| US5367066A (en) | 1984-10-16 | 1994-11-22 | Chiron Corporation | Oligonucleotides with selectably cleavable and/or abasic sites |
| FR2575751B1 (fr) | 1985-01-08 | 1987-04-03 | Pasteur Institut | Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques |
| US4659774A (en) | 1985-11-01 | 1987-04-21 | American Hoechst Corporation | Support for solid-phase oligonucleotide synthesis |
| US5130300A (en) | 1986-03-07 | 1992-07-14 | Monsanto Company | Method for enhancing growth of mammary parenchyma |
| EP0366685B1 (en) | 1987-06-24 | 1994-10-19 | Howard Florey Institute Of Experimental Physiology And Medicine | Nucleoside derivatives |
| US4923901A (en) | 1987-09-04 | 1990-05-08 | Millipore Corporation | Membranes with bound oligonucleotides and peptides |
| US5175273A (en) | 1988-07-01 | 1992-12-29 | Genentech, Inc. | Nucleic acid intercalating agents |
| US5047524A (en) | 1988-12-21 | 1991-09-10 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
| US5262530A (en) | 1988-12-21 | 1993-11-16 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
| US5141813A (en) | 1989-08-28 | 1992-08-25 | Clontech Laboratories, Inc. | Multifunctional controlled pore glass reagent for solid phase oligonucleotide synthesis |
| US5134066A (en) | 1989-08-29 | 1992-07-28 | Monsanto Company | Improved probes using nucleosides containing 3-dezauracil analogs |
| CA2029273A1 (en) | 1989-12-04 | 1991-06-05 | Christine L. Brakel | Modified nucleotide compounds |
| US5681941A (en) | 1990-01-11 | 1997-10-28 | Isis Pharmaceuticals, Inc. | Substituted purines and oligonucleotide cross-linking |
| US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
| US5587361A (en) | 1991-10-15 | 1996-12-24 | Isis Pharmaceuticals, Inc. | Oligonucleotides having phosphorothioate linkages of high chiral purity |
| US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5212295A (en) | 1990-01-11 | 1993-05-18 | Isis Pharmaceuticals | Monomers for preparation of oligonucleotides having chiral phosphorus linkages |
| US5457191A (en) | 1990-01-11 | 1995-10-10 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| EP0544824B1 (en) | 1990-07-27 | 1997-06-11 | Isis Pharmaceuticals, Inc. | Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression |
| US5432272A (en) | 1990-10-09 | 1995-07-11 | Benner; Steven A. | Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases |
| US5512668A (en) * | 1991-03-06 | 1996-04-30 | Polish Academy Of Sciences | Solid phase oligonucleotide synthesis using phospholane intermediates |
| US7015315B1 (en) | 1991-12-24 | 2006-03-21 | Isis Pharmaceuticals, Inc. | Gapped oligonucleotides |
| US20020183502A1 (en) | 1991-05-21 | 2002-12-05 | Mesmaeker Alain De | Backbone-modified oligonucleotide analogs and methods for using same |
| ATE221127T1 (de) | 1991-10-15 | 2002-08-15 | Isis Pharmaceuticals Inc | Über chirale phosphoratome gebundene oligonukleotide |
| ES2103918T3 (es) | 1991-10-17 | 1997-10-01 | Ciba Geigy Ag | Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios. |
| US5594121A (en) | 1991-11-07 | 1997-01-14 | Gilead Sciences, Inc. | Enhanced triple-helix and double-helix formation with oligomers containing modified purines |
| US6235887B1 (en) | 1991-11-26 | 2001-05-22 | Isis Pharmaceuticals, Inc. | Enhanced triple-helix and double-helix formation directed by oligonucleotides containing modified pyrimidines |
| US5484908A (en) | 1991-11-26 | 1996-01-16 | Gilead Sciences, Inc. | Oligonucleotides containing 5-propynyl pyrimidines |
| US5359044A (en) | 1991-12-13 | 1994-10-25 | Isis Pharmaceuticals | Cyclobutyl oligonucleotide surrogates |
| EP1044987B1 (en) | 1991-12-24 | 2006-02-15 | Isis Pharmaceuticals, Inc. | Gapped 2'-modified oligonucleotides |
| US6015886A (en) | 1993-05-24 | 2000-01-18 | Chemgenes Corporation | Oligonucleotide phosphate esters |
| US5502177A (en) | 1993-09-17 | 1996-03-26 | Gilead Sciences, Inc. | Pyrimidine derivatives for labeled binding partners |
| JPH09507836A (ja) | 1993-11-16 | 1997-08-12 | ジンタ・インコーポレイテッド | 非ホスホネート・ヌクレオシジル間結合と混合したキラリティー的に純粋なホスホネート・ヌクレオシジル間結合を有する合成オリゴマー |
| US5457187A (en) | 1993-12-08 | 1995-10-10 | Board Of Regents University Of Nebraska | Oligonucleotides containing 5-fluorouracil |
| US5596091A (en) | 1994-03-18 | 1997-01-21 | The Regents Of The University Of California | Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides |
| US5525711A (en) | 1994-05-18 | 1996-06-11 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Pteridine nucleotide analogs as fluorescent DNA probes |
| ATE327244T1 (de) | 1995-03-06 | 2006-06-15 | Isis Pharmaceuticals Inc | Verfahren zur synthese von 2'-0-substituierten pyrimidinen und oligomere davon |
| US6166197A (en) | 1995-03-06 | 2000-12-26 | Isis Pharmaceuticals, Inc. | Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions |
| US6639062B2 (en) | 1997-02-14 | 2003-10-28 | Isis Pharmaceuticals, Inc. | Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom |
| US6172209B1 (en) | 1997-02-14 | 2001-01-09 | Isis Pharmaceuticals Inc. | Aminooxy-modified oligonucleotides and methods for making same |
| WO1999005160A2 (en) | 1997-07-25 | 1999-02-04 | Hybridon, Inc. | Oligonuclotides having 3' terminal stereospecific phosphorothioates |
| US6528640B1 (en) | 1997-11-05 | 2003-03-04 | Ribozyme Pharmaceuticals, Incorporated | Synthetic ribonucleic acids with RNAse activity |
| US6617438B1 (en) | 1997-11-05 | 2003-09-09 | Sirna Therapeutics, Inc. | Oligoribonucleotides with enzymatic activity |
| CN101298481A (zh) * | 1998-02-20 | 2008-11-05 | 吉宁特有限公司 | 补体活化的抑制剂 |
| US7045610B2 (en) | 1998-04-03 | 2006-05-16 | Epoch Biosciences, Inc. | Modified oligonucleotides for mismatch discrimination |
| US6242589B1 (en) * | 1998-07-14 | 2001-06-05 | Isis Pharmaceuticals, Inc. | Phosphorothioate oligonucleotides having modified internucleoside linkages |
| US6867294B1 (en) * | 1998-07-14 | 2005-03-15 | Isis Pharmaceuticals, Inc. | Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages |
| US6066500A (en) | 1999-06-25 | 2000-05-23 | Isis Pharmaceuticals Inc. | Antisense modulation of Beta catenin expression |
| US6147200A (en) | 1999-08-19 | 2000-11-14 | Isis Pharmaceuticals, Inc. | 2'-O-acetamido modified monomers and oligomers |
| US6949520B1 (en) | 1999-09-27 | 2005-09-27 | Coley Pharmaceutical Group, Inc. | Methods related to immunostimulatory nucleic acid-induced interferon |
| US20020082227A1 (en) * | 1999-09-30 | 2002-06-27 | Scott Henry | Use of oligonucleotides for inhibition of complement activation |
| US20010055761A1 (en) | 1999-10-29 | 2001-12-27 | Agilent Technologies | Small scale dna synthesis using polymeric solid support with functionalized regions |
| DE10019756A1 (de) | 2000-04-20 | 2001-10-25 | Bayer Ag | Verfahren zur Herstellung von superabsorbierenden Polymeren aus Polyacrylnitrilen |
| US6492171B2 (en) | 2000-05-16 | 2002-12-10 | Isis Pharmaceuticals, Inc. | Antisense modulation of TERT expression |
| GB0024752D0 (en) | 2000-10-10 | 2000-11-22 | Univ Belfast | Oxidative halogenation of aromatic compounds |
| US20050277133A1 (en) | 2001-05-18 | 2005-12-15 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
| US6440739B1 (en) | 2001-07-17 | 2002-08-27 | Isis Pharmaceuticals, Inc. | Antisense modulation of glioma-associated oncogene-2 expression |
| JP4348044B2 (ja) | 2002-02-12 | 2009-10-21 | 株式会社キラルジェン | 立体規則性の高いジヌクレオシドホスホロチオエートの製造法 |
| US8232383B2 (en) | 2002-02-20 | 2012-07-31 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| US20050096284A1 (en) | 2002-02-20 | 2005-05-05 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
| WO2003085110A2 (en) | 2002-04-05 | 2003-10-16 | Santaris Pharma A/S | Oligomeric compounds for the modulation hif-1alpha expression |
| US8101348B2 (en) | 2002-07-10 | 2012-01-24 | Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften E.V. | RNA-interference by single-stranded RNA molecules |
| US20080274989A1 (en) | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
| US20050042646A1 (en) | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
| US7045306B2 (en) | 2003-04-28 | 2006-05-16 | The General Hospital Corporation | Method for identifying compounds in vitro that modulate the dysregulation of transcription of transcription mediated by mutant huntingtin protein |
| AU2004239114B2 (en) | 2003-05-14 | 2008-03-13 | Japan Science And Technology Agency | Inhibition of the expression of huntingtin gene |
| JP2005089441A (ja) | 2003-08-08 | 2005-04-07 | Toudai Tlo Ltd | 立体規則性の高いリン原子修飾ヌクレオチド類縁体の製造法 |
| DK1661905T3 (da) | 2003-08-28 | 2012-07-23 | Takeshi Imanishi | Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype |
| JP4616175B2 (ja) | 2003-09-02 | 2011-01-19 | 株式会社キラルジェン | ５’−ホスフィチル化モノマーおよびｈ−ホスホネートオリゴヌクレオチド誘導体の製造方法 |
| JP4580870B2 (ja) | 2003-09-02 | 2010-11-17 | 株式会社キラルジェン | リボヌクレオチド又はリボヌクレオチド誘導体の製造方法 |
| ES2485848T3 (es) | 2003-09-12 | 2014-08-14 | University Of Massachusetts | ARN de interferencia para el tratamiento de trastornos relacionados con la ganancia de función |
| US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
| WO2008005562A2 (en) | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Rna silencing compositions and methods for the treatment of huntington's disease |
| WO2005070859A1 (ja) | 2004-01-27 | 2005-08-04 | Takeshi Wada | フルオラス担体およびそれを用いたオリゴヌクレオチド誘導体の製造方法 |
| WO2005085272A1 (ja) | 2004-03-05 | 2005-09-15 | Takeshi Wada | ボラノホスフェートモノマーおよびそれを用いたオリゴヌクレオチド誘導体の製造方法 |
| JP4865544B2 (ja) | 2004-03-25 | 2012-02-01 | 株式会社キラルジェン | 立体規則性の高いリボヌクレオチド類縁体及びデオキシリボヌクレオチド類縁体の製造法 |
| US20060019916A1 (en) | 2004-04-02 | 2006-01-26 | Coley Pharmaceutical Group, Inc. | Immunostimulatory nucleic acids for inducing IL-10 responses |
| EP2397563A3 (en) | 2004-09-17 | 2012-07-18 | Isis Pharmaceuticals, Inc. | Enhanced antisense oligonucleotides |
| WO2006066260A2 (en) | 2004-12-17 | 2006-06-22 | Thiosense, Inc. | Compositions of and methods for producing phosphorus-chiral monomers and oligomers |
| US7902352B2 (en) | 2005-05-06 | 2011-03-08 | Medtronic, Inc. | Isolated nucleic acid duplex for reducing huntington gene expression |
| US20060257912A1 (en) | 2005-05-06 | 2006-11-16 | Medtronic, Inc. | Methods and sequences to suppress primate huntington gene expression |
| US9133517B2 (en) | 2005-06-28 | 2015-09-15 | Medtronics, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin |
| EP2316967A1 (en) | 2005-06-28 | 2011-05-04 | Medtronic, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin gene. |
| WO2007028065A2 (en) | 2005-08-30 | 2007-03-08 | Isis Pharmaceuticals, Inc. | Chimeric oligomeric compounds for modulation of splicing |
| CN101365801B (zh) | 2005-10-28 | 2013-03-27 | 阿尔尼拉姆医药品有限公司 | 抑制亨廷顿基因表达的组合物和方法 |
| JP5425474B2 (ja) | 2006-01-26 | 2014-02-26 | アイシス ファーマシューティカルズ， インコーポレーテッド | ハンチンチン対する、組成物及びその使用 |
| CA2640171C (en) | 2006-01-27 | 2014-10-28 | Isis Pharmaceuticals, Inc. | 6-modified bicyclic nucleic acid analogs |
| CA3044969A1 (en) | 2006-05-05 | 2007-12-21 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating gene expression |
| JP5441688B2 (ja) | 2006-05-11 | 2014-03-12 | アイシス ファーマシューティカルズ， インコーポレーテッド | ５’修飾二環式核酸類似体 |
| ES2389112T3 (es) | 2006-05-31 | 2012-10-23 | Toray Industries, Inc. | Oligonucleótido inmunoestimulador y aplicación farmacéutica del mismo |
| DK2092065T4 (da) * | 2006-10-18 | 2019-10-21 | Ionis Pharmaceuticals Inc | Antisense-forbindelser |
| WO2008151833A2 (en) | 2007-06-13 | 2008-12-18 | Hochschule Mannheim | Compounds for the modulation of huntingtin aggregation, methods and means for identifying such compounds |
| DE602007005629D1 (de) | 2007-06-18 | 2010-05-12 | Commissariat Energie Atomique | Reversibles siRNA-Silencing eines mutierten und endogenen Huntington-Wildtypgens und dessen Anwendung zur Behandlung von Morbus Huntington |
| JP5737937B2 (ja) | 2007-07-09 | 2015-06-17 | イデラ ファーマシューティカルズ インコーポレイテッドＩｄｅｒａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． | 安定化免疫調節ｒｎａ（ｓｉｍｒａ）化合物 |
| EP2224912B1 (en) * | 2008-01-02 | 2016-05-11 | TEKMIRA Pharmaceuticals Corporation | Improved compositions and methods for the delivery of nucleic acids |
| US20110105587A1 (en) | 2008-02-04 | 2011-05-05 | David Frederik Fishcher | Target sequences and methods to identify the same, useful in treatment of neurodegenerative diseases |
| US8679750B2 (en) * | 2008-05-09 | 2014-03-25 | The University Of British Columbia | Methods and compositions for the treatment of Huntington'S disease |
| WO2009135322A1 (en) | 2008-05-09 | 2009-11-12 | The Universtity Of British Columbia | Methods and compositions for the treatment of huntington's disease |
| WO2009143391A2 (en) | 2008-05-22 | 2009-11-26 | Isis Pharmaceuticals, Inc | Methods for modulation expression of creb |
| WO2009148605A2 (en) | 2008-06-04 | 2009-12-10 | Isis Pharmaceuticals, Inc. | Methods for treating hypercholesterolemia |
| KR20110044983A (ko) * | 2008-07-10 | 2011-05-03 | 아카데미쉬 지에켄후이스 비즈 드 유니버시테이트 반 암스테르담 | 보체 길항제 및 그 용도 |
| US9394333B2 (en) | 2008-12-02 | 2016-07-19 | Wave Life Sciences Japan | Method for the synthesis of phosphorus atom modified nucleic acids |
| WO2010091301A1 (en) | 2009-02-06 | 2010-08-12 | Isis Pharmaceuticals, Inc. | Oligomeric compounds and excipients |
| US8987222B2 (en) * | 2009-04-08 | 2015-03-24 | University Of Massachusetts | Single nucleotide polymorphism (SNP) targeting therapies for the treatment of huntington'S disease |
| MX342945B (es) | 2009-07-06 | 2016-10-18 | Ontorii Inc * | Profármacos de ácido nucleico novedosos y métodos de uso de los mismos. |
| WO2011015573A1 (en) | 2009-08-03 | 2011-02-10 | Galapagos Nv | Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases |
| WO2011015572A1 (en) | 2009-08-03 | 2011-02-10 | Galapagos Nv | Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases |
| US8927553B2 (en) | 2009-08-10 | 2015-01-06 | Daljit Singh Dhanoa | Deuterium-enriched alkyl sulfonamides and uses thereof |
| NZ599032A (en) | 2009-09-11 | 2014-09-26 | Isis Pharmaceuticals Inc | Modulation of huntingtin expression |
| EP2479182B8 (en) | 2009-09-16 | 2016-07-13 | Wave Life Sciences Japan, Inc. | Novel protecting group for synthesizing rna and derivative thereof |
| CN102791862B (zh) | 2009-12-31 | 2017-04-05 | 库尔纳公司 | 通过抑制胰岛素受体底物2(irs2)和转录因子e3(tfe3)的天然反义转录物而治疗irs2相关疾病 |
| WO2011097644A2 (en) | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| EP3561060A1 (en) | 2010-02-08 | 2019-10-30 | Ionis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| JP2011184318A (ja) | 2010-03-05 | 2011-09-22 | Univ Of Tokyo | リボヌクレシドｈ−ボラノホスホネート |
| JP5847700B2 (ja) | 2010-03-05 | 2016-01-27 | 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ | リボヌクレオシドホスホロチオエートの製造方法 |
| KR20130103662A (ko) | 2010-04-19 | 2013-09-24 | 엔라이프 떼라퓨틱스, 에스.엘. | 올리고뉴클레오티드 분자들의 특정 뉴런 유형들로의 선택적인 전달을 위한 조성물들 및 방법 |
| EA201390145A1 (ru) * | 2010-08-20 | 2013-11-29 | Серулин Фарма Инк. | Конъюгаты, частицы, композиции и связанные с ними способы |
| EP2611927B1 (en) | 2010-08-31 | 2018-08-01 | Sirna Therapeutics, Inc. | Novel single chemical entities and methods for delivery of oligonucleotides |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| AU2011325956B2 (en) | 2010-11-12 | 2016-07-14 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| WO2012073857A1 (ja) | 2010-11-30 | 2012-06-07 | 株式会社キラルジェン | ２'－ｏ－修飾ｒｎａ |
| EP3467109A1 (en) | 2011-02-08 | 2019-04-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| ES2739804T3 (es) | 2011-02-12 | 2020-02-04 | Univ Iowa Res Found | Compuestos terapéuticos |
| US20150038549A1 (en) * | 2011-04-20 | 2015-02-05 | Larry J. Smith | Methods and Compositions for Modulating Gene Expression Using Components That Self Assemble in Cells and Produce RNAi Activity |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| EP2742056B2 (en) | 2011-08-11 | 2020-06-10 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| EP2785729B1 (en) | 2011-11-30 | 2020-11-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| ES2907250T3 (es) * | 2012-01-27 | 2022-04-22 | Biomarin Tech Bv | Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker |
| KR20240148947A (ko) * | 2012-07-13 | 2024-10-11 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| SG11201500243WA (en) * | 2012-07-13 | 2015-04-29 | Shin Nippon Biomedical Lab Ltd | Chiral nucleic acid adjuvant |
| SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| CA2887884A1 (en) | 2012-10-12 | 2014-04-17 | Isis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| WO2014064258A1 (en) | 2012-10-26 | 2014-05-01 | Nlife Therapeutics, S.L. | Compositions and methods for selective delivery of oligonucleotide molecules to cell types |
| HK1214179A1 (zh) | 2012-10-26 | 2016-07-22 | 恩莱弗医疗有限公司 | 通过选择性递送寡核苷酸分子至特定神经元类型治疗帕金森病的组合物和方法 |
| DK2951305T3 (en) | 2013-01-30 | 2018-10-29 | Hoffmann La Roche | LNA oligonucleotide KULHYDRATKONJUGATER |
| WO2014121287A2 (en) | 2013-02-04 | 2014-08-07 | Isis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| PE20152002A1 (es) * | 2013-05-01 | 2016-01-21 | Isis Pharmaceuticals Inc | Composiciones y metodos para modular la expresion de ttr y vhb |
| EP3052521A1 (en) * | 2013-10-03 | 2016-08-10 | Moderna Therapeutics, Inc. | Polynucleotides encoding low density lipoprotein receptor |
| NZ719477A (en) | 2013-11-11 | 2022-05-27 | Sangamo Therapeutics Inc | Methods and compositions for treating huntington’s disease |
| EP3095460A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
| WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| WO2015108048A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| ES2917473T3 (es) | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| EP3647318B1 (en) | 2014-04-28 | 2021-06-30 | Ionis Pharmaceuticals, Inc. | Linkage modified oligomeric compounds |
| MX378789B (es) | 2014-05-08 | 2025-03-11 | Sangamo Biosciences Inc | Métodos y composiciones para tratar la enfermedad de huntington. |
| JP6663859B2 (ja) | 2014-05-20 | 2020-03-13 | ユニバーシティー オブ アイオワ リサーチ ファウンデーション | ハンチントン病の治療化合物 |
| US20160017327A1 (en) | 2014-07-11 | 2016-01-21 | The Johns Hopkins University | Phosphorodiamidate morpholino oligomers (pmos) and their use in suppression of mutant huntingtin expression and attenuation of neurotoxicity |
| EP3169693B1 (en) | 2014-07-16 | 2022-03-09 | ModernaTX, Inc. | Chimeric polynucleotides |
| JP2017536119A (ja) | 2014-11-19 | 2017-12-07 | ロシュ イノベーション センター コペンハーゲン エーエス | キラルホスホロチオエート連結を含むｌｎａギャップマーオリゴヌクレオチド |
| EP3234131B1 (en) | 2014-12-16 | 2020-04-22 | Roche Innovation Center Copenhagen A/S | Chiral toxicity screening method |
| SMT201900347T1 (it) | 2014-12-24 | 2019-07-11 | Uniqure Ip Bv | Soppressione del gene huntingtina indotta da rnai |
| US11761951B2 (en) | 2015-02-04 | 2023-09-19 | Bristol-Myers Squibb Company | Methods of selecting therapeutic molecules |
| EP3253871A1 (en) | 2015-02-04 | 2017-12-13 | Bristol-Myers Squibb Company | Lna oligonucleotides with alternating flanks |
| SG11201706444TA (en) | 2015-02-10 | 2017-09-28 | Genzyme Corp | VARIANT RNAi |
| CN107980062B (zh) | 2015-04-03 | 2022-11-25 | 马萨诸塞大学 | 用于靶向亨廷汀mRNA的寡核苷酸化合物 |
| CN108025089A (zh) | 2015-07-22 | 2018-05-11 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| SG10201912902TA (en) | 2015-10-09 | 2020-02-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| WO2017067970A1 (en) | 2015-10-22 | 2017-04-27 | Roche Innovation Center Copenhagen A/S | In vitro toxicity screening assay |
| AU2017234150B2 (en) | 2016-03-13 | 2021-09-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| MA45290A (fr) | 2016-05-04 | 2019-03-13 | Wave Life Sciences Ltd | Procédés et compositions d'agents biologiquement actifs |
| CN109477108A (zh) | 2016-05-04 | 2019-03-15 | 波涛生命科学有限公司 | 寡核苷酸组合物和其方法 |
| WO2017210647A1 (en) | 2016-06-03 | 2017-12-07 | Wave Life Sciences Ltd. | Oligonucleotides, compositions and methods thereof |
| US20190264267A1 (en) | 2016-07-25 | 2019-08-29 | Wave Life Sciences Ltd. | Phasing |
| JP7296882B2 (ja) | 2016-11-23 | 2023-06-23 | ウェイブ ライフ サイエンシズ リミテッド | ホスホラミダイト及びオリゴヌクレオチド合成のための組成物及び方法 |
| WO2018223056A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US11597927B2 (en) | 2017-06-02 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| CN120330183A (zh) | 2017-06-02 | 2025-07-18 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
| WO2018237194A1 (en) | 2017-06-21 | 2018-12-27 | Wave Life Sciences Ltd. | Compounds, compositions and methods for synthesis |
| CA3072076A1 (en) | 2017-08-08 | 2019-02-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| SG11202000276YA (en) | 2017-09-18 | 2020-04-29 | Wave Life Sciences Ltd | Technologies for oligonucleotide preparation |
| WO2019075357A1 (en) | 2017-10-12 | 2019-04-18 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
| BR112020020670A2 (pt) | 2018-04-12 | 2021-03-02 | Wave Life Sciences Ltd. | composição de oligonucleotídeo, composição farmacêutica, método para alterar o splicing de uma transcrição alvo, método para tratar distrofia muscular, método para preparar um oligonucleotídeo ou uma composição de oligonucleotídeo do mesmo e oligonucleotídeo |
| TWI844541B (zh) | 2018-05-11 | 2024-06-11 | 新加坡商波濤生命科學有限公司 | 寡核苷酸組成物及其使用方法 |
| AU2019392928A1 (en) | 2018-12-06 | 2021-06-17 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| SG11202107318YA (en) | 2019-02-01 | 2021-08-30 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| 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 |
| JPWO2020196662A1 (enExample) | 2019-03-25 | 2020-10-01 | ||
| BR112021021203A2 (pt) | 2019-04-25 | 2022-01-04 | Wave Life Sciences Ltd | Oligonucleotídeo; composição de oligonucleotídeo quiralmente controlada; composição farmacêutica; método para prevenir, tratar ou melhorar uma afecção, distúrbio ou doença relacionada a ush2a e/ou prevenir, atrasar o início, desenvolvimento e/ou progresso, e/ou tratar uma afecção, distúrbio ou doença relacionada a ush2a em um indivíduo suscetível à mesma ou que sofre da mesma; método para aumentar o nível de salto de um éxon prejudicial em uma transcrição de gene ush2a ou seu produto de gene em uma célula; método para prevenir, atrasar o início ou progressão de, tratar ou melhorar uma afecção, distúrbio ou doença relacionada a ush2a em um indivíduo suscetível à mesma ou que sofre da mesma; método para saltar um éxon prejudicial 13 em um alelo de ush2a em um indivíduo; método para produzir ou aumentar o nível de um éxon de proteína ush2a com salto 13 em um sistema; e composto, oligonucleotídeo, composição ou método |
| US20230145795A1 (en) | 2019-04-25 | 2023-05-11 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| CN114502177A (zh) | 2019-05-09 | 2022-05-13 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
| CA3156176A1 (en) | 2019-10-06 | 2021-04-15 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| BR112022006205A2 (pt) | 2019-10-06 | 2022-07-19 | Wave Life Sciences Ltd | 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 |
| WO2021178237A2 (en) | 2020-03-01 | 2021-09-10 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| AU2021277407A1 (en) | 2020-05-22 | 2022-11-17 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| IL298406A (en) | 2020-05-22 | 2023-01-01 | Wave Life Sciences Ltd | Double stranded oligonucleotide compositions and methods relating thereto |
| WO2022046667A1 (en) | 2020-08-24 | 2022-03-03 | Wave Life Sciences Ltd. | Cells and non-human animals engineered to express adar1 and uses thereof |
| EP4200616A1 (en) | 2020-08-24 | 2023-06-28 | Wave Life Sciences Ltd. | Immunoassays for detecting wild type huntingtin protein and methods of treatment employing such immunoassays |
| WO2022067023A1 (en) | 2020-09-24 | 2022-03-31 | Mayo Foundation For Medical Education And Research | Screening platforms |
| CA3197311A1 (en) | 2020-11-08 | 2022-05-12 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| CN116507724A (zh) | 2020-11-08 | 2023-07-28 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| WO2023049477A2 (en) | 2021-09-26 | 2023-03-30 | Wave Life Sciences Ltd. | Compositions for editing mecp2 transcripts and methods thereof |
| KR20240063964A (ko) | 2021-09-26 | 2024-05-10 | 웨이브 라이프 사이언시스 리미티드 | 올리고뉴클레오티드 조성물 및 이의 방법 |
| WO2023075766A1 (en) | 2021-10-27 | 2023-05-04 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| WO2023076352A2 (en) | 2021-10-27 | 2023-05-04 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| WO2023154528A1 (en) | 2022-02-11 | 2023-08-17 | Wave Life Sciences Ltd. | Stereoselective technologies for chiral compounds |
| AU2023227808A1 (en) | 2022-03-02 | 2024-09-19 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof for exon skipping |
| EP4507700A2 (en) | 2022-04-15 | 2025-02-19 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods relating thereto |
| WO2023220440A1 (en) | 2022-05-12 | 2023-11-16 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| CN120476205A (zh) | 2022-08-11 | 2025-08-12 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| AU2024315761A1 (en) | 2023-08-03 | 2026-02-12 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
-
2016
- 2016-07-22 CN CN201680043299.6A patent/CN108025089A/zh active Pending
- 2016-07-22 CN CN202311216358.3A patent/CN117965536A/zh active Pending
- 2016-07-22 PE PE2018000099A patent/PE20181174A1/es unknown
- 2016-07-22 JP JP2018502637A patent/JP7296724B2/ja active Active
- 2016-07-22 JP JP2018502647A patent/JP2018525357A/ja active Pending
- 2016-07-22 HK HK18114508.6A patent/HK1255369A1/zh unknown
- 2016-07-22 HK HK18114509.5A patent/HK1255370A1/zh unknown
- 2016-07-22 CN CN201680042413.3A patent/CN108135921B/zh active Active
- 2016-07-22 US US15/746,220 patent/US10479995B2/en active Active
- 2016-07-22 MX MX2018000796A patent/MX2018000796A/es unknown
- 2016-07-22 TW TW105123348A patent/TW201707711A/zh unknown
- 2016-07-22 SG SG10201912900XA patent/SG10201912900XA/en unknown
- 2016-07-22 EP EP23206565.6A patent/EP4344744A3/en active Pending
- 2016-07-22 IL IL320434A patent/IL320434A/en unknown
- 2016-07-22 US US15/746,199 patent/US20180216107A1/en not_active Abandoned
- 2016-07-22 MA MA43072A patent/MA43072A/fr unknown
- 2016-07-22 WO PCT/US2016/043598 patent/WO2017015575A1/en not_active Ceased
- 2016-07-22 WO PCT/US2016/043542 patent/WO2017015555A1/en not_active Ceased
- 2016-07-22 AU AU2016297155A patent/AU2016297155B2/en active Active
- 2016-07-22 KR KR1020187004483A patent/KR20180028516A/ko not_active Withdrawn
- 2016-07-22 EP EP16828604.5A patent/EP3325017A4/en not_active Withdrawn
- 2016-07-22 EP EP16828617.7A patent/EP3324978A4/en active Pending
- 2016-07-22 BR BR112017028636A patent/BR112017028636A2/pt not_active IP Right Cessation
- 2016-07-22 CA CA2989682A patent/CA2989682A1/en active Pending
- 2016-07-22 CR CR20180107A patent/CR20180107A/es unknown
-
2017
- 2017-12-26 IL IL256603A patent/IL256603A/en unknown
-
2018
- 2018-01-15 NI NI201800009A patent/NI201800009A/es unknown
- 2018-01-18 CL CL2018000145A patent/CL2018000145A1/es unknown
- 2018-01-18 MX MX2025000894A patent/MX2025000894A/es unknown
- 2018-01-18 PH PH12018500145A patent/PH12018500145A1/en unknown
- 2018-01-22 SV SV2018005619A patent/SV2018005619A/es unknown
- 2018-02-09 EC ECIEPI201810463A patent/ECSP18010463A/es unknown
- 2018-02-15 CO CONC2018/0001484A patent/CO2018001484A2/es unknown
-
2019
- 2019-08-26 US US16/551,503 patent/US20200080083A1/en not_active Abandoned
-
2020
- 2020-02-04 US US16/782,021 patent/US11634710B2/en active Active
-
2021
- 2021-07-14 US US17/375,658 patent/US20220195429A1/en not_active Abandoned
- 2021-08-20 JP JP2021134582A patent/JP7441816B2/ja active Active
- 2021-10-18 JP JP2021170541A patent/JP7606954B2/ja active Active
-
2023
- 2023-02-14 AU AU2023200807A patent/AU2023200807A1/en not_active Abandoned
- 2023-04-21 US US18/305,195 patent/US12486505B2/en active Active
- 2023-12-08 JP JP2023207761A patent/JP7818563B2/ja active Active
-
2024
- 2024-12-13 JP JP2024219444A patent/JP2025038091A/ja active Pending
-
2025
- 2025-08-25 AU AU2025220872A patent/AU2025220872A1/en active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| NI201800009A (es) | Composiciones de oligonecleótidos y métodos de los mismos. | |
| MX2016009290A (es) | Diseño quiral. | |
| AR132094A2 (es) | Composiciones detergentes, variantes de lipasa y polinucleótidos que las codifican | |
| CR20170564A (es) | Nuevos derivados hidroxiéster, un proceso para su preparación y composiciones farmacéuticas que los contienen | |
| MX389703B (es) | Anticuerpos anti-c1s humanizados y metodos para usarlos. | |
| MX2020003841A (es) | Métodos y composiciones para la producción de nucleósido trifosfatos y ácidos ribonucleicos. | |
| CL2018003474A1 (es) | Nuevos derivados piperidinilo, un proceso para su preparación y composiciones farmacéuticas que los contienen. | |
| CR20190199A (es) | Anticuerpos anti-pd-1 y sus usos | |
| CO2017008600A2 (es) | Análogos de desacetoxitubulisina h, composiciones y método de producción de los mismos | |
| AR110677A1 (es) | Anticuerpos anti-c1s y métodos para usarlos | |
| CU24466B1 (es) | Compuestos derivados de d-fenilalanina-n-sustituido con biciclos aromáticos, método de preparación de las mismas y composiciones farmacéuticas que los contienen | |
| UY37523A (es) | Anticuerpos anti-ox40 y sus usos | |
| BR112018007017A2 (pt) | polipeptídeos | |
| CO2017002253A2 (es) | Compuesto dirigido a il-23a y tnf-alfa | |
| MX2021011676A (es) | Polimorfos de sepiapterina y sales de los mismos. | |
| MX2016016992A (es) | Aptameros especificos de tlr-4 y usos de los mismos. | |
| ECSP20021132A (es) | Enantiómeros de tiazoles sustituidos compuestos antivirales | |
| ECSP16074478A (es) | Compuestos novedosos | |
| BR112018001255A2 (pt) | composto direcionado à il-23a e fator de ativação de células b (baff) e seus usos | |
| MX2021010060A (es) | Produccion de virus en huevos aviares. | |
| BR112017019750A2 (pt) | processo para preparação de uma forma cristalina de menaquinol, forma cristalina de menaquinol, processo para preparação de uma forma sólida do menaquinol, composição farmacêutica ou nutracêutica e uso da forma cristalina de menaquinol | |
| BR112017025872A2 (pt) | proteínas de ligação multiespecíficas | |
| MX2023009432A (es) | Metodo para crear un compuesto a base de mercurio, compuesto a base de mercurio, metodos de uso del compuesto a base de mercurio y usos del compuesto a base de mercurio. | |
| AR105488A1 (es) | Composiciones de oligonucleótidos y métodos de los mismos | |
| AR105066A1 (es) | Enzimas y sistemas crispr |