CO2018001484A2 - Oligonucleótidos quiralmente controlados que comprenden azúcares modificados y ligaciones internucleotídicos modificadas y estereocontroladas, omposiciones y métodos de los mismos - Google Patents
Oligonucleótidos quiralmente controlados que comprenden azúcares modificados y ligaciones internucleotídicos modificadas y estereocontroladas, omposiciones y métodos de los mismosInfo
- Publication number
- CO2018001484A2 CO2018001484A2 CONC2018/0001484A CO2018001484A CO2018001484A2 CO 2018001484 A2 CO2018001484 A2 CO 2018001484A2 CO 2018001484 A CO2018001484 A CO 2018001484A CO 2018001484 A2 CO2018001484 A2 CO 2018001484A2
- Authority
- CO
- Colombia
- Prior art keywords
- modified
- methods
- chirally controlled
- internucleotide linkages
- ompositions
- Prior art date
Links
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- 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
-
- 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
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C—CHEMISTRY; METALLURGY
- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—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)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (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 (4)
| 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 | |
| PCT/US2016/043542 WO2017015555A1 (en) | 2015-07-22 | 2016-07-22 | Oligonucleotide compositions and methods thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CO2018001484A2 true CO2018001484A2 (es) | 2018-07-19 |
Family
ID=57835219
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2018/0001484A CO2018001484A2 (es) | 2015-07-22 | 2018-02-15 | Oligonucleótidos quiralmente controlados que comprenden azúcares modificados y ligaciones internucleotídicos modificadas y estereocontroladas, omposiciones y métodos de los mismos |
Country Status (23)
| Country | Link |
|---|---|
| US (6) | US20180216107A1 (enExample) |
| EP (3) | EP4344744A3 (enExample) |
| JP (7) | JP2018525357A (enExample) |
| KR (1) | KR20180028516A (enExample) |
| CN (3) | CN117965536A (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 (168)
| 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 |
| BRPI0923225A2 (pt) | 2008-12-02 | 2016-10-04 | Chiralgen Ltd | metodo para sintese de acidos nucleicos modificados no atomo de fosforo |
| EP2451461A4 (en) | 2009-07-06 | 2013-05-29 | Ontorii Inc | NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| CN107365339A (zh) | 2011-07-19 | 2017-11-21 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| CA2866392C (en) | 2012-03-30 | 2023-10-24 | Washington University | Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome |
| WO2014010250A1 (en) | 2012-07-13 | 2014-01-16 | Chiralgen, Ltd. | Asymmetric auxiliary group |
| DK2872147T3 (da) | 2012-07-13 | 2023-02-20 | Wave Life Sciences Ltd | Fremgangsmåde til fremstilling af kirale oligonukleotider |
| TWI702046B (zh) | 2013-07-19 | 2020-08-21 | 美商Ionis製藥公司 | 用於調節τ蛋白表現之組合物 |
| EP3095461A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| EP3095459A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having antitumor effect and antitumor agent |
| EP3094728B1 (en) | 2014-01-16 | 2022-03-09 | Wave Life Sciences Ltd. | Chiral design |
| DK3234131T3 (da) * | 2014-12-16 | 2020-06-29 | Roche Innovation Ct Copenhagen As | Fremgangsmåde til screening af kiral toksicitet |
| AU2015367378A1 (en) | 2014-12-17 | 2017-06-15 | Proqr Therapeutics Ii B.V. | Targeted RNA editing |
| US11129844B2 (en) | 2015-03-03 | 2021-09-28 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating MECP2 expression |
| RS60762B1 (sr) | 2015-04-03 | 2020-10-30 | Univ Massachusetts | Oligonukleotidna jedinjenja za ciljane irnk hantingtina |
| EP3929293A3 (en) | 2015-04-03 | 2022-03-16 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| CN117965536A (zh) | 2015-07-22 | 2024-05-03 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| MA46427A (fr) | 2015-10-09 | 2018-08-15 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| CA3119019C (en) | 2015-12-10 | 2024-06-11 | Ptc Therapeutics, Inc. | Compounds and methods for treating huntington's disease |
| WO2017132669A1 (en) | 2016-01-31 | 2017-08-03 | University Of Massachusetts | Branched oligonucleotides |
| MA43822A (fr) | 2016-03-13 | 2018-11-28 | Wave Life Sciences Ltd | Compositions et procédés de synthèse de phosphoramidite et d'oligonucléotides |
| EP3452596A4 (en) * | 2016-05-04 | 2020-03-18 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
| US11013757B2 (en) | 2016-06-03 | 2021-05-25 | Wave Life Sciences Ltd. | Oligonucleotides, compositions and methods thereof |
| EP3475424A1 (en) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Single-stranded rna-editing oligonucleotides |
| WO2018031933A2 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| WO2018041973A1 (en) | 2016-09-01 | 2018-03-08 | Proqr Therapeutics Ii B.V. | Chemically modified single-stranded rna-editing oligonucleotides |
| MD3519572T2 (ro) | 2016-09-29 | 2024-03-31 | Biogen Ma Inc | Compuși și procedee de reducere a expresiei Tau |
| JOP20190104A1 (ar) | 2016-11-10 | 2019-05-07 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن atxn3 |
| WO2018098264A1 (en) | 2016-11-23 | 2018-05-31 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| NZ755416A (en) | 2016-12-19 | 2023-05-26 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| ES3051834T3 (en) | 2016-12-19 | 2025-12-30 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| PT4122497T (pt) | 2016-12-19 | 2024-06-12 | Sarepta Therapeutics Inc | Conjugados oligoméricos de salto de exão para a distrofia muscular |
| US11274300B2 (en) | 2017-01-19 | 2022-03-15 | 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 |
| CN110612353A (zh) | 2017-03-03 | 2019-12-24 | 加利福尼亚大学董事会 | 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向 |
| US11603532B2 (en) | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| CA3065523A1 (en) * | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| EP4151627A1 (en) | 2017-06-05 | 2023-03-22 | 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 |
| WO2018237245A1 (en) | 2017-06-23 | 2018-12-27 | University Of Massachusetts | TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS |
| CN111182898B (zh) | 2017-06-28 | 2024-04-16 | Ptc医疗公司 | 用于治疗亨廷顿氏病的方法 |
| 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 |
| AU2018316159A1 (en) | 2017-08-08 | 2020-02-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| WO2019046316A1 (en) | 2017-08-28 | 2019-03-07 | Acurastem Inc. | PIKFYVE KINASE INHIBITORS |
| CN111108096A (zh) | 2017-09-18 | 2020-05-05 | 波涛生命科学有限公司 | 寡核苷酸制备技术 |
| EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| JP2020536058A (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 |
| 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 |
| EP3687519A1 (en) | 2017-09-28 | 2020-08-05 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| EP3694530A4 (en) | 2017-10-12 | 2021-06-30 | Wave Life Sciences Ltd. | COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES |
| 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 |
| MY203356A (en) | 2018-01-12 | 2024-06-25 | Bristol Myers Squibb Co | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| WO2019140236A1 (en) | 2018-01-12 | 2019-07-18 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| CA3088522A1 (en) | 2018-01-15 | 2019-07-18 | Ionis Pharmaceuticals, Inc. | Modulators of dnm2 expression |
| AU2019218987B2 (en) | 2018-02-12 | 2025-04-24 | Ionis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
| WO2019183440A1 (en) * | 2018-03-22 | 2019-09-26 | Ionis Pharmaceuticals, Inc. | Methods for modulating fmr1 expression |
| BR112020019373A2 (pt) | 2018-03-27 | 2020-12-29 | Ptc Therapeutics, Inc. | Compostos para o tratamento da doença de hutington |
| JP2021521140A (ja) * | 2018-04-12 | 2021-08-26 | 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 |
| CA3098623A1 (en) | 2018-05-07 | 2019-11-14 | Alnylam Pharmaceuticals, Inc. | Extrahepatic delivery |
| JP2021522800A (ja) | 2018-05-09 | 2021-09-02 | アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． | Ａｔｘｎ３発現を低減するための化合物及び方法 |
| IL277970B2 (en) | 2018-05-09 | 2025-03-01 | Ionis Pharmaceuticals Inc | Oligomeric compounds comprising modified oligonucleotides and conjugated groups for use in the treatment of end-stage renal disease, chronic kidney disease and coronary artery disease |
| CA3098624A1 (en) | 2018-05-11 | 2019-11-14 | 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 |
| 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 |
| EP4219717A3 (en) | 2018-06-13 | 2023-12-20 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| CA3103429A1 (en) * | 2018-06-14 | 2019-12-19 | Don W. Cleveland | Compounds and methods for increasing stmn2 expression |
| IL279688B2 (en) | 2018-06-27 | 2025-01-01 | Ptc Therapeutics Inc | Heterocyclic and heteroaryl compounds for the treatment of Huntington's disease |
| 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 |
| AU2019310097A1 (en) | 2018-07-25 | 2021-02-04 | 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 |
| BR112021002440A2 (pt) | 2018-08-10 | 2021-05-04 | University Of Massachusetts | oligonucleotídeos modificados visando snps |
| WO2020041769A1 (en) | 2018-08-23 | 2020-02-27 | 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 |
| MX2021005758A (es) | 2018-11-15 | 2021-09-21 | Ionis Pharmaceuticals Inc | Moduladores de la expresión de irf5. |
| US12281305B2 (en) | 2018-11-21 | 2025-04-22 | 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 |
| BR112021011018A2 (pt) | 2018-12-13 | 2021-08-31 | Sarepta Therapeutics, Inc. | Conjugados de oligômero para exon skipping para distrofia muscular |
| SG11202106378VA (en) | 2018-12-21 | 2021-07-29 | Ionis Pharmaceuticals Inc | Modulators of hsd17b13 expression |
| CN113614232A (zh) | 2019-01-18 | 2021-11-05 | 马萨诸塞大学 | 动态药代动力学修饰锚 |
| EP3917497A4 (en) * | 2019-02-01 | 2023-06-07 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF |
| WO2020176771A1 (en) | 2019-02-27 | 2020-09-03 | Ionis Pharmaceuticals, Inc. | Modulators of malat1 expression |
| AU2020241559A1 (en) * | 2019-03-20 | 2021-11-11 | Wave Life Sciences Ltd. | Technologies useful for oligonucleotide preparation |
| TW202536179A (zh) | 2019-03-29 | 2025-09-16 | 美商Ionis製藥公司 | 用於調節ube3a-ats之化合物及方法 |
| WO2020214763A1 (en) | 2019-04-18 | 2020-10-22 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| EP3958872A4 (en) * | 2019-04-25 | 2024-07-03 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| MX2021012981A (es) * | 2019-04-25 | 2022-01-18 | Wave Life Sciences Ltd | Composiciones de oligonucleotido y metodos de uso de las mismas. |
| CA3139821A1 (en) | 2019-05-13 | 2020-11-19 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
| WO2020243292A1 (en) | 2019-05-28 | 2020-12-03 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing fus expression |
| PL3956450T3 (pl) | 2019-07-26 | 2025-12-22 | Ionis Pharmaceuticals, Inc. | Związki i sposoby modulowania 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 |
| CN114728017B (zh) | 2019-10-14 | 2025-10-28 | 阿斯利康(瑞典)有限公司 | Pnpla3表达的调节剂 |
| TW202131920A (zh) | 2019-11-01 | 2021-09-01 | 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 |
| US11827880B2 (en) | 2019-12-02 | 2023-11-28 | Shape Therapeutics Inc. | Therapeutic editing |
| US20230108340A1 (en) * | 2020-02-21 | 2023-04-06 | Ionis Pharmaceuticals, Inc. | Methods for reducing htt expression |
| AU2021225957A1 (en) | 2020-02-28 | 2022-09-08 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating SMN2 |
| JP2023521604A (ja) * | 2020-03-30 | 2023-05-25 | アルナイラム ファーマシューティカルズ， インコーポレイテッド | 微小管関連タンパク質タウ（ＭＡＰＴ）ｉＲＮＡ剤組成物およびその使用方法 |
| 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 |
| MX2022014606A (es) | 2020-05-22 | 2023-03-08 | Wave Life Sciences Ltd | Composiciones de oligonucleótidos bicatenarios y métodos relacionados con las mismas. |
| CA3174079A1 (en) | 2020-05-26 | 2021-12-02 | Anastasia Khvorova | Synthetic oligonucleotides having regions of block and cluster modifications |
| US11408000B2 (en) | 2020-06-03 | 2022-08-09 | Triplet Therapeutics, Inc. | Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity |
| 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 |
| KR20230108728A (ko) | 2020-11-18 | 2023-07-18 | 아이오니스 파마수티컬즈, 인코포레이티드 | 앤지오텐시노겐 발현을 조절하기 위한 화합물 및 방법 |
| 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 |
| IL309445A (en) | 2021-06-22 | 2024-02-01 | Acurastem Inc | PIKFYVE antisense oligonucleotides |
| IL309334A (en) | 2021-06-23 | 2024-02-01 | Univ Massachusetts | Anti-FLT1 optimized oligonucleotide compounds for the treatment of preeclampsia and other angiogenic disorders |
| WO2023004049A1 (en) | 2021-07-21 | 2023-01-26 | AcuraStem, Inc. | Unc13a antisense oligonucleotides |
| US20240425864A1 (en) | 2021-09-30 | 2024-12-26 | Sarepta Therapeutics, Inc. | Antisense oligonucleotides having one or more abasic units |
| US20240401045A1 (en) | 2021-10-01 | 2024-12-05 | Adarx Pharmaceuticals, Inc. | Angiotensinogen-modulating compositions and methods of use thereof |
| AU2022357561A1 (en) | 2021-10-01 | 2024-04-18 | Adarx Pharmaceuticals, Inc. | Prekallikrein-modulating compositions and methods of use thereof |
| GB202117758D0 (en) | 2021-12-09 | 2022-01-26 | Ucl Business Ltd | Therapeutics for the treatment of neurodegenerative disorders |
| EP4479535A1 (en) | 2022-02-14 | 2024-12-25 | ProQR Therapeutics II B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| WO2023212625A1 (en) | 2022-04-28 | 2023-11-02 | AcuraStem Incorporated | Syf2 antisense oligonucleotides |
| EP4555086A1 (en) | 2022-07-15 | 2025-05-21 | ProQR Therapeutics II B.V. | Oligonucleotides for adar-mediated rna editing and use thereof |
| EP4555085A1 (en) | 2022-07-15 | 2025-05-21 | ProQR Therapeutics II B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
| EP4569115A4 (en) * | 2022-08-11 | 2026-07-15 | Wave Life Sciences Ltd | OLIGONUCLEOTID COMPOSITIONS AND ASSOCIATED PROCESSES |
| EP4590311A2 (en) | 2022-09-21 | 2025-07-30 | Sarepta Therapeutics, Inc. | Dmd antisense oligonucleotide-mediated exon skipping efficiency |
| JP2025532127A (ja) | 2022-09-23 | 2025-09-29 | アイオーニス ファーマシューティカルズ， インコーポレーテッド | Ｍｅｃｐ２発現を低減する化合物及び方法 |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| KR20250113455A (ko) | 2022-11-24 | 2025-07-25 | 프로큐알 테라퓨틱스 Ⅱ 비.브이. | 유전성 hfe-혈색소침착증의 치료를 위한 안티센스 올리고뉴클레오티드 |
| GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
| WO2024121373A1 (en) | 2022-12-09 | 2024-06-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of cardiovascular disease |
| EP4652276A2 (en) | 2023-01-20 | 2025-11-26 | AcuraStem Incorporated | Unc13a antisense oligonucleotides |
| GB202300865D0 (en) | 2023-01-20 | 2023-03-08 | Proqr Therapeutics Ii Bv | Delivery of oligonucleotides |
| WO2024163651A2 (en) | 2023-01-31 | 2024-08-08 | AcuraStem Incorporated | Syf2 antisense oligonucleotides |
| WO2024167945A1 (en) | 2023-02-06 | 2024-08-15 | AcuraStem Incorporated | Pikfyve antisense oligonucleotides |
| WO2024175550A1 (en) | 2023-02-20 | 2024-08-29 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease |
| GB202304363D0 (en) | 2023-03-24 | 2023-05-10 | Proqr Therapeutics Ii Bv | Chemically modified antisense oligonucleotides for use in RNA editing |
| JP2026510408A (ja) | 2023-03-24 | 2026-04-02 | 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 | 神経障害の処置のためのアンチセンスオリゴヌクレオチド |
| EP4688143A1 (en) | 2023-03-27 | 2026-02-11 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for the treatment of liver disease |
| KR20260021596A (ko) | 2023-04-05 | 2026-02-13 | 아이오니스 파마수티컬즈, 인코포레이티드 | Pln 발현을 감소시키는 화합물 및 방법 |
| EP4698652A2 (en) | 2023-04-20 | 2026-02-25 | Adarx Pharmaceuticals, Inc. | Mapt-modulating compositions and methods of use thereof |
| EP4702144A1 (en) | 2023-04-27 | 2026-03-04 | Sarepta Therapeutics, Inc. | Antisense oligomers for treatment of chronic kidney disease |
| WO2024238396A1 (en) | 2023-05-12 | 2024-11-21 | Adarx Pharmaceuticals, Inc. | Nmda ligand conjugated compounds and uses thereof |
| WO2024249328A2 (en) | 2023-05-26 | 2024-12-05 | Adarx Pharmaceuticals, Inc. | Sod1-modulating compositions and methods of use thereof |
| WO2024256620A1 (en) | 2023-06-16 | 2024-12-19 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurodegenerative disease |
| CN121712891A (zh) | 2023-06-20 | 2026-03-20 | 阿达尔克斯制药有限公司 | Lrrk2调节组合物及其使用方法 |
| 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 |
| EP4774393A1 (en) | 2023-09-07 | 2026-07-15 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| KR20260089666A (ko) | 2023-09-14 | 2026-06-15 | 아이오니스 파마수티컬즈, 인코포레이티드 | Apociii 발현을 감소시키기 위한 화합물 및 방법 |
| UY40897A (es) | 2023-09-21 | 2025-03-31 | Ionis Pharmaceuticals Inc | Compuestos y métodos para inhibir 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 |
| WO2025096809A1 (en) | 2023-10-31 | 2025-05-08 | Korro Bio, Inc. | Oligonucleotides comprising phosphoramidate internucleotide linkages |
| AU2024379264A1 (en) | 2023-11-16 | 2026-05-14 | 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 |
| WO2026060374A2 (en) | 2024-09-16 | 2026-03-19 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurological disorders |
| WO2026062247A1 (en) | 2024-09-20 | 2026-03-26 | Astrazeneca Ab | Liquid-phase oligonucleotide synthesis using 2-(2-nitrophenyl)propyloxycarbonyl (nppoc) as a protecting group |
| WO2026068781A1 (en) | 2024-09-30 | 2026-04-02 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of liver disease |
| WO2026093339A1 (en) | 2024-10-29 | 2026-05-07 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of peroxisome biogenesis disorders in the zellweger spectrum (pbd-zsd) |
| WO2026099316A1 (en) | 2024-11-06 | 2026-05-15 | Astrazeneca Ab | Compounds and methods for reducing arhgef6 expression |
| WO2026112585A1 (en) | 2024-11-22 | 2026-05-28 | AcuraStem Incorporated | Syf2 antisense oligonucleotides |
| WO2026122478A1 (en) | 2024-12-02 | 2026-06-11 | Korro Bio, Inc. | Extrahepatic delivery formulations and methods |
| WO2026136663A1 (en) | 2024-12-18 | 2026-06-25 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurodegenerative disease |
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 |
| US4500707A (en) | 1980-02-29 | 1985-02-19 | University Patents, Inc. | Nucleosides useful in the preparation of polynucleotides |
| US5132418A (en) | 1980-02-29 | 1992-07-21 | University Patents, Inc. | Process for preparing polynucleotides |
| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing 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 |
| US5457191A (en) | 1990-01-11 | 1995-10-10 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5681941A (en) | 1990-01-11 | 1997-10-28 | Isis Pharmaceuticals, Inc. | Substituted purines and oligonucleotide cross-linking |
| US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5587361A (en) | 1991-10-15 | 1996-12-24 | Isis Pharmaceuticals, Inc. | Oligonucleotides having phosphorothioate linkages of high chiral purity |
| US5212295A (en) | 1990-01-11 | 1993-05-18 | Isis Pharmaceuticals | Monomers for preparation of oligonucleotides having chiral phosphorus linkages |
| US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
| JPH0874B2 (ja) | 1990-07-27 | 1996-01-10 | アイシス・ファーマシューティカルス・インコーポレーテッド | 遺伝子発現を検出および変調するヌクレアーゼ耐性、ピリミジン修飾オリゴヌクレオチド |
| 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 |
| US20020183502A1 (en) | 1991-05-21 | 2002-12-05 | Mesmaeker Alain De | Backbone-modified oligonucleotide analogs and methods for using same |
| US7015315B1 (en) | 1991-12-24 | 2006-03-21 | Isis Pharmaceuticals, Inc. | Gapped oligonucleotides |
| AU2874892A (en) | 1991-10-15 | 1993-05-21 | Isis Pharmaceuticals, Inc. | Oligonucleotides having chiral phosphorus linkages |
| DE59208572D1 (de) | 1991-10-17 | 1997-07-10 | Ciba Geigy Ag | Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte |
| 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 |
| ATE515510T1 (de) | 1991-12-24 | 2011-07-15 | Isis Pharmaceuticals Inc | Durch dna-abschnitte unterbrochene modifizierte oligonukleotide |
| 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 |
| DE69636160D1 (de) | 1995-03-06 | 2006-06-29 | 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 |
| AU8512598A (en) | 1997-07-25 | 1999-02-16 | 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 |
| EP3056516A1 (en) * | 1998-02-20 | 2016-08-17 | Genentech, Inc. | Inhibitors of complement activation |
| 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 |
| 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) |
| 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) |
| ES2649817T3 (es) | 2002-04-05 | 2018-01-15 | Roche Innovation Center Copenhagen A/S | Compuestos oligom¿¿ricos para la modulaci¿®n de la expresi¿®n de HIF-1¿Á |
| CA2489174C (en) | 2002-07-10 | 2013-02-05 | Thomas Tuschl | Rna-interference by single-stranded rna molecules |
| US20050042646A1 (en) | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
| US20080274989A1 (en) | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression 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 |
| 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 |
| 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 |
| WO2005027980A1 (en) | 2003-09-12 | 2005-03-31 | University Of Massachusetts | Rna interference for the treatment of gain-of-function disorders |
| US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
| JP4945129B2 (ja) | 2004-01-27 | 2012-06-06 | 株式会社キラルジェン | 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 |
| WO2005092909A1 (ja) | 2004-03-25 | 2005-10-06 | Toudai Tlo, Ltd. | 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 |
| JP2007531746A (ja) * | 2004-04-02 | 2007-11-08 | コーリー ファーマシューティカル グループ，インコーポレイテッド | Ｉｌ−１０応答を誘導するための免疫活性化核酸 |
| EP1799859B1 (en) | 2004-09-17 | 2014-07-02 | 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 |
| US20060257912A1 (en) | 2005-05-06 | 2006-11-16 | Medtronic, Inc. | Methods and sequences to suppress primate huntington gene expression |
| US7902352B2 (en) | 2005-05-06 | 2011-03-08 | Medtronic, Inc. | Isolated nucleic acid duplex for reducing huntington gene expression |
| US9133517B2 (en) | 2005-06-28 | 2015-09-15 | Medtronics, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin |
| ATE522626T1 (de) | 2005-06-28 | 2011-09-15 | Medtronic Inc | Verfahren und nukleotid-sequenzen, die bevorzugt die expression eines mutierten huntingtin-genes unterdrücken |
| WO2007028065A2 (en) | 2005-08-30 | 2007-03-08 | Isis Pharmaceuticals, Inc. | Chimeric oligomeric compounds for modulation of splicing |
| EP2325315B1 (en) * | 2005-10-28 | 2014-05-07 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of huntingtin gene |
| EP2422819B1 (en) | 2006-01-26 | 2017-03-01 | Ionis Pharmaceuticals, Inc. | Compositions and their uses directed to Huntingtin |
| DE602007009487D1 (de) | 2006-01-27 | 2010-11-11 | Isis Pharmaceutical Inc | 6-modifizierte bicyclische nukleinsäureanaloga |
| EP2505646A1 (en) | 2006-05-05 | 2012-10-03 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating expression of CRP |
| CA2651453C (en) | 2006-05-11 | 2014-10-14 | Isis Pharmaceuticals, Inc. | 5'-modified bicyclic nucleic acid analogs |
| DK2034015T3 (da) | 2006-05-31 | 2012-10-15 | Toray Industries | Immunstimulerende oligonucleotid og farmaceutisk anvendelse heraf |
| EP2046993A4 (en) | 2006-07-07 | 2010-11-17 | Univ Massachusetts | RNA SILENCING COMPOSITIONS, AND METHODS OF TREATING HUNTINGTON CHOREA |
| EP2410053B2 (en) | 2006-10-18 | 2020-07-15 | Ionis Pharmaceuticals, Inc. | Antisense compounds |
| 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 |
| KR101343489B1 (ko) | 2007-07-09 | 2013-12-20 | 이데라 파마슈티칼즈, 인코포레이티드 | 안정화된 면역 조절성 ｒｎａ（ｓｉｍｒａ）화합물 |
| EP3100718B1 (en) * | 2008-01-02 | 2019-11-27 | Arbutus Biopharma Corporation | Improved compositions and methods for the delivery of nucleic acids |
| JP2011515653A (ja) | 2008-02-04 | 2011-05-19 | ガラパゴス・ナムローゼ・フェンノートシャップ | 神経変性疾患の治療に有用な標的配列及びそれらの同定方法 |
| AU2009244013B2 (en) | 2008-05-09 | 2015-06-25 | The University Of British Columbia | Methods and compositions for the treatment of Huntington's disease |
| US8679750B2 (en) | 2008-05-09 | 2014-03-25 | The University 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 |
| CN103740713A (zh) | 2008-07-10 | 2014-04-23 | 阿姆斯特丹大学附属医院 | 补体拮抗剂及其应用 |
| BRPI0923225A2 (pt) | 2008-12-02 | 2016-10-04 | Chiralgen Ltd | metodo para sintese de acidos nucleicos modificados no atomo de fosforo |
| US20120264806A1 (en) | 2009-02-06 | 2012-10-18 | Bennett C Frank | 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 |
| EP2451461A4 (en) | 2009-07-06 | 2013-05-29 | Ontorii Inc | NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF |
| 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 |
| EP3626823A1 (en) | 2009-09-11 | 2020-03-25 | Ionis 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 |
| CA2785727C (en) | 2009-12-31 | 2020-01-07 | Curna, Inc. | Treatment of insulin receptor substrate 2 (irs2) related diseases by inhibition of natural antisense transcript to irs2 and transcription factor e3 (tfe3) |
| ES2733708T3 (es) | 2010-02-08 | 2019-12-02 | Ionis Pharmaceuticals Inc | Reducción selectiva de variantes alélicas |
| JP6018506B2 (ja) | 2010-02-08 | 2016-11-02 | アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． | 対立遺伝子多様体の選択的低減 |
| 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 |
| WO2011108682A1 (ja) | 2010-03-05 | 2011-09-09 | 国立大学法人 東京大学 | 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 |
| US20120225129A1 (en) * | 2010-08-20 | 2012-09-06 | Cerulean Pharma Inc. | Conjugates, particles, compositions, and related methods |
| US20130253168A1 (en) | 2010-08-31 | 2013-09-26 | Steven L. Colletti | Novel single chemical entities and methods for delivery of oligonucleotides |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| EP3702460A1 (en) | 2010-11-12 | 2020-09-02 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| EP2647644B1 (en) | 2010-11-30 | 2020-09-09 | Wave Life Sciences Japan, Inc. | 2'-o-modified rna |
| US10017764B2 (en) | 2011-02-08 | 2018-07-10 | 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 |
| CN104271740A (zh) * | 2011-04-20 | 2015-01-07 | L·J·史密斯 | 利用在细胞中自组装和产生RNAi活性的成分来调节基因表达的方法和组合物 |
| CN107365339A (zh) | 2011-07-19 | 2017-11-21 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| US20140303235A1 (en) | 2011-08-11 | 2014-10-09 | Isis Pharmaceuticals, Inc. | Linkage modified gapped oligomeric compounds and uses thereof |
| WO2013082548A1 (en) | 2011-11-30 | 2013-06-06 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| CA2862628C (en) | 2012-01-27 | 2021-08-24 | Prosensa Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| SG11201500243WA (en) * | 2012-07-13 | 2015-04-29 | Shin Nippon Biomedical Lab Ltd | Chiral nucleic acid adjuvant |
| DK2872147T3 (da) | 2012-07-13 | 2023-02-20 | Wave Life Sciences Ltd | Fremgangsmåde til fremstilling af kirale oligonukleotider |
| WO2014010250A1 (en) | 2012-07-13 | 2014-01-16 | Chiralgen, Ltd. | Asymmetric auxiliary group |
| EP4086347A3 (en) | 2012-10-12 | 2023-01-11 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| WO2014064257A1 (en) | 2012-10-26 | 2014-05-01 | Nlife Therapeutics, S.L. | Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types |
| MX2015005328A (es) | 2012-10-26 | 2015-09-25 | Nlife Therapeutics S L | Composiciones y metodos para la administracion selectiva de moleculas de oligonucleotidos a tipos de celulas. |
| CA2893801A1 (en) | 2013-01-30 | 2014-08-07 | F. Hoffmann-La Roche Ag | Lna oligonucleotide carbohydrate conjugates |
| EP2951304B1 (en) | 2013-02-04 | 2020-07-08 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| PL2992098T3 (pl) * | 2013-05-01 | 2019-09-30 | Ionis Pharmaceuticals, Inc. | Kompozycje i sposoby modulowania ekspresji hbv i ttr |
| KR20160067219A (ko) | 2013-10-03 | 2016-06-13 | 모더나 세라퓨틱스, 인코포레이티드 | 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드 |
| MX391904B (es) | 2013-11-11 | 2025-03-12 | Sangamo Biosciences Inc | Un represor genetico para usarse en el tratamiento de la enfermedad de huntington. |
| WO2015108046A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤 |
| EP3095461A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| EP3095459A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having antitumor effect and antitumor agent |
| EP3094728B1 (en) | 2014-01-16 | 2022-03-09 | Wave Life Sciences Ltd. | Chiral design |
| US9926556B2 (en) | 2014-04-28 | 2018-03-27 | Ionis Pharmaceuticals, Inc. | Linkage modified oligomeric compounds |
| BR112016025849A2 (pt) | 2014-05-08 | 2017-10-17 | Chdi Foundation Inc | métodos e composições para o tratamento da doença de huntington |
| AU2015264263B2 (en) | 2014-05-20 | 2021-08-05 | University Of Iowa Research Foundation | Huntington's disease therapeutic compounds |
| 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 |
| WO2016011226A1 (en) * | 2014-07-16 | 2016-01-21 | Moderna Therapeutics, Inc. | Chimeric polynucleotides |
| EP3221329A1 (en) | 2014-11-19 | 2017-09-27 | Roche Innovation Center Copenhagen A/S | Lna gapmer oligonucleotides comprising chiral phosphorothioate linkages |
| DK3234131T3 (da) | 2014-12-16 | 2020-06-29 | Roche Innovation Ct Copenhagen As | Fremgangsmåde til screening af kiral toksicitet |
| AU2015370903B2 (en) | 2014-12-24 | 2021-06-17 | Uniqure Ip B.V. | RNAi induced huntingtin gene suppression |
| ES2893114T3 (es) | 2015-02-04 | 2022-02-08 | Bristol Myers Squibb Co | Métodos para seleccionar moléculas terapéuticas |
| US20180023081A1 (en) | 2015-02-04 | 2018-01-25 | Bristol-Myers Squibb Company | Lna oligonucleotides with alternating flanks |
| CA3255657A1 (en) | 2015-02-10 | 2025-12-01 | Genzyme Corporation | VARIANT RNAi |
| RS60762B1 (sr) | 2015-04-03 | 2020-10-30 | Univ Massachusetts | Oligonukleotidna jedinjenja za ciljane irnk hantingtina |
| CN117965536A (zh) | 2015-07-22 | 2024-05-03 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| MA46427A (fr) | 2015-10-09 | 2018-08-15 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| WO2017067970A1 (en) | 2015-10-22 | 2017-04-27 | Roche Innovation Center Copenhagen A/S | In vitro toxicity screening assay |
| MA43822A (fr) | 2016-03-13 | 2018-11-28 | Wave Life Sciences Ltd | Compositions et procédés de synthèse de phosphoramidite et d'oligonucléotides |
| JP2019516679A (ja) | 2016-05-04 | 2019-06-20 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | 生物活性剤に関する方法および組成物 |
| EP3452596A4 (en) | 2016-05-04 | 2020-03-18 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
| US11013757B2 (en) | 2016-06-03 | 2021-05-25 | Wave Life Sciences Ltd. | Oligonucleotides, compositions and methods thereof |
| US20190264267A1 (en) | 2016-07-25 | 2019-08-29 | Wave Life Sciences Ltd. | Phasing |
| WO2018098264A1 (en) | 2016-11-23 | 2018-05-31 | 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 |
| CN111164091B (zh) | 2017-06-02 | 2025-01-07 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
| US11603532B2 (en) | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| WO2018237194A1 (en) | 2017-06-21 | 2018-12-27 | Wave Life Sciences Ltd. | Compounds, compositions and methods for synthesis |
| AU2018316159A1 (en) | 2017-08-08 | 2020-02-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| CN111108096A (zh) | 2017-09-18 | 2020-05-05 | 波涛生命科学有限公司 | 寡核苷酸制备技术 |
| EP3694530A4 (en) | 2017-10-12 | 2021-06-30 | Wave Life Sciences Ltd. | COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES |
| JP2021521140A (ja) | 2018-04-12 | 2021-08-26 | 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 |
| CA3098624A1 (en) | 2018-05-11 | 2019-11-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| AU2019392928A1 (en) | 2018-12-06 | 2021-06-17 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| EP3917497A4 (en) | 2019-02-01 | 2023-06-07 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF |
| AU2020241559A1 (en) | 2019-03-20 | 2021-11-11 | Wave Life Sciences Ltd. | Technologies useful for oligonucleotide preparation |
| JPWO2020196662A1 (enExample) | 2019-03-25 | 2020-10-01 | ||
| MX2021012981A (es) | 2019-04-25 | 2022-01-18 | Wave Life Sciences Ltd | Composiciones de oligonucleotido y metodos de uso de las mismas. |
| EP3958872A4 (en) | 2019-04-25 | 2024-07-03 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| EP3965780A4 (en) | 2019-05-09 | 2023-10-25 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE |
| CA3156176A1 (en) | 2019-10-06 | 2021-04-15 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| CA3154768A1 (en) | 2019-10-06 | 2021-04-15 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| AU2021230473A1 (en) | 2020-03-01 | 2022-09-22 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| MX2022014606A (es) | 2020-05-22 | 2023-03-08 | Wave Life Sciences Ltd | Composiciones de oligonucleótidos bicatenarios y métodos relacionados con las mismas. |
| AU2021277407A1 (en) | 2020-05-22 | 2022-11-17 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods 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 |
| CN115989041A (zh) | 2020-08-24 | 2023-04-18 | 波涛生命科学有限公司 | 经工程化为表达adar1的细胞和非人动物及其用途 |
| US20230416847A1 (en) | 2020-09-24 | 2023-12-28 | Mayo Foundation For Medical Education And Research | Screening platforms |
| JP2023548584A (ja) | 2020-11-08 | 2023-11-17 | 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組成物及びその方法 |
| 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 |
| CA3232070A1 (en) | 2021-09-26 | 2023-03-30 | Prashant MONIAN | Oligonucleotide compositions and methods thereof |
| EP4404942A4 (en) | 2021-09-26 | 2026-06-17 | Wave Life Sciences Ltd | Compositions for the Editing of Transcripts MECP2 and Related Processes |
| EP4422645A4 (en) | 2021-10-27 | 2025-12-03 | Wave Life Sciences Ltd | OLIGONUCLEOTID COMPOSITIONS AND THEIR METHODS OF USE |
| AU2021471586A1 (en) | 2021-10-27 | 2024-05-09 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| EP4476202A4 (en) | 2022-02-11 | 2026-06-10 | Wave Life Sciences Ltd | STEREOSELECTIVE TECHNOLOGIES FOR CHIRICAL COMPOUNDS |
| US20250197857A1 (en) | 2022-03-02 | 2025-06-19 | 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 |
| CN119452084A (zh) | 2022-05-12 | 2025-02-14 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| EP4569115A4 (en) | 2022-08-11 | 2026-07-15 | Wave Life Sciences Ltd | OLIGONUCLEOTID COMPOSITIONS AND ASSOCIATED PROCESSES |
| EP4753719A1 (en) | 2023-08-03 | 2026-06-10 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
-
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