CA2878945A1 - Chiral control - Google Patents
Chiral control Download PDFInfo
- Publication number
- CA2878945A1 CA2878945A1 CA2878945A CA2878945A CA2878945A1 CA 2878945 A1 CA2878945 A1 CA 2878945A1 CA 2878945 A CA2878945 A CA 2878945A CA 2878945 A CA2878945 A CA 2878945A CA 2878945 A1 CA2878945 A1 CA 2878945A1
- Authority
- CA
- Canada
- Prior art keywords
- oligonucleotide
- chirally controlled
- composition
- optionally substituted
- linkage
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 0 CC(C(N1)=O)=CN([C@@](C2)O[C@](C*)C2OP2O[C@@](C(c3ccccc3)c3ccccc3)[C@@]3N2CCC3)C1=O Chemical compound CC(C(N1)=O)=CN([C@@](C2)O[C@](C*)C2OP2O[C@@](C(c3ccccc3)c3ccccc3)[C@@]3N2CCC3)C1=O 0.000 description 2
- HPPYXPVTGSTCCW-NWDGAFQWSA-N [O-][N+](c1ccc(C[C@H]2O[P-]N3[C@H]2CCC3)cc1)=O Chemical compound [O-][N+](c1ccc(C[C@H]2O[P-]N3[C@H]2CCC3)cc1)=O HPPYXPVTGSTCCW-NWDGAFQWSA-N 0.000 description 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- 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
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C317/00—Sulfones; Sulfoxides
- C07C317/26—Sulfones; Sulfoxides having sulfone or sulfoxide groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton
- C07C317/28—Sulfones; Sulfoxides having sulfone or sulfoxide groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton with sulfone or sulfoxide groups bound to acyclic carbon atoms of the carbon skeleton
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
- C07D295/04—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
- C07D295/08—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly bound oxygen or sulfur atoms
- C07D295/084—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly bound oxygen or sulfur atoms with the ring nitrogen atoms and the oxygen or sulfur atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
- C07D295/088—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly bound oxygen or sulfur atoms with the ring nitrogen atoms and the oxygen or sulfur atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H1/00—Processes for the preparation of sugar derivatives
- C07H1/02—Phosphorylation
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
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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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—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
- C12N2330/00—Production
- C12N2330/30—Production chemically synthesised
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- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Analytical Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Gastroenterology & Hepatology (AREA)
- Massaging Devices (AREA)
- Paper (AREA)
- Extrusion Moulding Of Plastics Or The Like (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261671656P | 2012-07-13 | 2012-07-13 | |
| US201261671655P | 2012-07-13 | 2012-07-13 | |
| US61/671,655 | 2012-07-13 | ||
| US61/671,656 | 2012-07-13 | ||
| US201261671724P | 2012-07-14 | 2012-07-14 | |
| US201261671722P | 2012-07-14 | 2012-07-14 | |
| US61/671,722 | 2012-07-14 | ||
| US61/671,724 | 2012-07-14 | ||
| PCT/US2013/050407 WO2014012081A2 (en) | 2012-07-13 | 2013-07-12 | Chiral control |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2878945A1 true CA2878945A1 (en) | 2014-01-16 |
Family
ID=49916709
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2878945A Pending CA2878945A1 (en) | 2012-07-13 | 2013-07-12 | Chiral control |
Country Status (16)
| Country | Link |
|---|---|
| US (4) | US9982257B2 (enExample) |
| EP (2) | EP2872147B1 (enExample) |
| JP (4) | JP6453212B2 (enExample) |
| KR (4) | KR20240148947A (enExample) |
| CN (2) | CN112007045B (enExample) |
| AU (5) | AU2013289880B2 (enExample) |
| CA (1) | CA2878945A1 (enExample) |
| CL (1) | CL2015000092A1 (enExample) |
| DK (1) | DK2872147T3 (enExample) |
| ES (1) | ES2940887T3 (enExample) |
| IL (1) | IL236621B (enExample) |
| MX (1) | MX2015000577A (enExample) |
| PL (1) | PL2872147T3 (enExample) |
| RU (1) | RU2015104762A (enExample) |
| SG (3) | SG10201700283WA (enExample) |
| WO (1) | WO2014012081A2 (enExample) |
Families Citing this family (158)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| WO2012068405A2 (en) | 2010-11-17 | 2012-05-24 | Isis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| 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 |
| 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 |
| RU2745324C2 (ru) | 2013-03-14 | 2021-03-23 | Ионис Фармасьютикалз, Инк. | Композиции и способы модулирования экспрессии tau |
| 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 |
| 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 |
| 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 |
| WO2015125845A1 (ja) * | 2014-02-20 | 2015-08-27 | 塩野義製薬株式会社 | 含窒素非芳香族複素環を含む核酸のリン酸部位修飾 |
| US10214743B2 (en) | 2014-03-04 | 2019-02-26 | Hirofumi Yamamoto | Colorectal cancer drug, and method for predicting prognosis of colorectal cancer patient |
| US10619156B2 (en) | 2014-05-28 | 2020-04-14 | The Regents Of The University Of California | Hybrid tRNA/pre-miRNA molecules and methods of use |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| RU2708237C2 (ru) | 2014-08-22 | 2019-12-05 | Общество с ограниченной ответственностью "НооГен" | Модифицированные олигонуклеотиды и способ их получения |
| AU2015327836B2 (en) | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| 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 |
| 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 |
| 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 |
| US10533175B2 (en) | 2015-09-25 | 2020-01-14 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating Ataxin 3 expression |
| CN108603230A (zh) | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| SG10201912902TA (en) * | 2015-10-09 | 2020-02-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| EP3390636B1 (en) | 2015-12-14 | 2021-05-19 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of dravet syndrome |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| WO2017131208A1 (ja) * | 2016-01-28 | 2017-08-03 | 東レ株式会社 | 癌の治療及び/又は予防用医薬組成物 |
| AU2017234150B2 (en) * | 2016-03-13 | 2021-09-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| US11267843B2 (en) * | 2016-03-18 | 2022-03-08 | Roche Innovation Center Copenhagen A/S | Stereodefining L-monomers |
| MA45470A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Acides nucléiques kras et leurs utilisations |
| WO2017173301A1 (en) | 2016-04-01 | 2017-10-05 | Avidity Biosciences Llc | Egfr nucleic acids and uses thereof |
| KR102775461B1 (ko) | 2016-04-01 | 2025-02-28 | 어비디티 바이오사이언시스 인크. | 핵산-폴리펩타이드 조성물 및 이의 용도 |
| EP3436582B1 (en) | 2016-04-01 | 2021-10-13 | Avidity Biosciences, Inc. | Beta-catenin nucleic acids and uses thereof |
| EP3436587B1 (en) | 2016-04-01 | 2021-05-19 | Avidity Biosciences, Inc. | Phosphatidylinositol-3-kinase nucleic acids and uses thereof |
| WO2017184529A1 (en) | 2016-04-18 | 2017-10-26 | Sarepta Therapeutics, Inc. | Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene |
| NZ747685A (en) | 2016-04-29 | 2023-05-26 | Sarepta Therapeutics Inc | Oligonucleotide analogues targeting human lmna |
| 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 |
| CN109311925B (zh) * | 2016-05-12 | 2022-06-03 | 罗氏创新中心哥本哈根有限公司 | 立体限定的氧杂氮杂磷杂环戊烷亚磷酰胺单体与核苷或寡核苷酸的增强的偶联 |
| EP3458464A1 (en) * | 2016-05-18 | 2019-03-27 | ETH Zurich | Stereoselective synthesis of phosphorothioate oligoribonucleotides |
| WO2017210647A1 (en) * | 2016-06-03 | 2017-12-07 | Wave Life Sciences Ltd. | Oligonucleotides, compositions and methods thereof |
| EP3472346B1 (en) | 2016-06-17 | 2023-01-18 | F. Hoffmann-La Roche AG | Papd5 and papd7 inhibitors for treating a hepatitis b infection |
| 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 |
| US20190262375A1 (en) | 2016-06-30 | 2019-08-29 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| EP3500581A4 (en) | 2016-08-17 | 2021-10-06 | Solstice Biologics, Ltd. | POLYNUCLEOTIDE CONSTRUCTS |
| 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 |
| WO2018102397A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| HRP20240705T1 (hr) | 2016-12-19 | 2024-08-30 | Sarepta Therapeutics, Inc. | Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju |
| KR102646318B1 (ko) | 2016-12-19 | 2024-03-12 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| KR102810425B1 (ko) | 2016-12-19 | 2025-05-21 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
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