JP6382344B2 - キラルデザイン - Google Patents
キラルデザイン Download PDFInfo
- Publication number
- JP6382344B2 JP6382344B2 JP2016564442A JP2016564442A JP6382344B2 JP 6382344 B2 JP6382344 B2 JP 6382344B2 JP 2016564442 A JP2016564442 A JP 2016564442A JP 2016564442 A JP2016564442 A JP 2016564442A JP 6382344 B2 JP6382344 B2 JP 6382344B2
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- JP
- Japan
- Prior art keywords
- composition
- oligonucleotide
- chiral
- oligonucleotides
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- Prior art date
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- 0 CC*C*1CCCC1 Chemical compound CC*C*1CCCC1 0.000 description 20
- MUUOWCYFVHIANC-UHFFFAOYSA-N N#CC[N+]1(CCCC1)[O-] Chemical compound N#CC[N+]1(CCCC1)[O-] MUUOWCYFVHIANC-UHFFFAOYSA-N 0.000 description 2
- XDTMQSROBMDMFD-UHFFFAOYSA-N C1CCCCC1 Chemical compound C1CCCCC1 XDTMQSROBMDMFD-UHFFFAOYSA-N 0.000 description 1
- BYSKVCHIRBYMLX-UHFFFAOYSA-N C=[S](c1ccccc11)(SC1=O)(=O)=O Chemical compound C=[S](c1ccccc11)(SC1=O)(=O)=O BYSKVCHIRBYMLX-UHFFFAOYSA-N 0.000 description 1
- LUQAVTVEQGRYDY-UHFFFAOYSA-N CC(C)(C)SCN1CCOCC1 Chemical compound CC(C)(C)SCN1CCOCC1 LUQAVTVEQGRYDY-UHFFFAOYSA-N 0.000 description 1
- OMTNTFDKNPQPFZ-UHFFFAOYSA-N CC(C)[N+](CC#N)(C(C)C)[O-] Chemical compound CC(C)[N+](CC#N)(C(C)C)[O-] OMTNTFDKNPQPFZ-UHFFFAOYSA-N 0.000 description 1
- OQTLVPVYEVOARR-UHFFFAOYSA-N CCCCC[NH+](CC#N)[O-] Chemical compound CCCCC[NH+](CC#N)[O-] OQTLVPVYEVOARR-UHFFFAOYSA-N 0.000 description 1
- ABZHPJSWYVYVLX-UHFFFAOYSA-N CC[O](C)C(SSC(N)O)=C Chemical compound CC[O](C)C(SSC(N)O)=C ABZHPJSWYVYVLX-UHFFFAOYSA-N 0.000 description 1
- LOXSULYIHSQCAQ-ONEGZZNKSA-N CN(C)/C=C/NC(SS1)=NC1=S Chemical compound CN(C)/C=C/NC(SS1)=NC1=S LOXSULYIHSQCAQ-ONEGZZNKSA-N 0.000 description 1
- KDXLBTLIGMNWCQ-UHFFFAOYSA-N CNC(CSSC(NC)=O)=O Chemical compound CNC(CSSC(NC)=O)=O KDXLBTLIGMNWCQ-UHFFFAOYSA-N 0.000 description 1
- XXLKSGUTIFHVAY-UHFFFAOYSA-N COC(C1N(CCS(C)=C)CCC1)=O Chemical compound COC(C1N(CCS(C)=C)CCC1)=O XXLKSGUTIFHVAY-UHFFFAOYSA-N 0.000 description 1
- HVOXUDKLFYOUJM-UHFFFAOYSA-N CSS(c1ccccc1Cl)(=O)=O Chemical compound CSS(c1ccccc1Cl)(=O)=O HVOXUDKLFYOUJM-UHFFFAOYSA-N 0.000 description 1
- LQBCCZNDSBKPIU-UHFFFAOYSA-N C[N+](C)(CC#N)[O-] Chemical compound C[N+](C)(CC#N)[O-] LQBCCZNDSBKPIU-UHFFFAOYSA-N 0.000 description 1
- XTQWAJAVRPQUQE-UHFFFAOYSA-N C[N](C)(C)CSN Chemical compound C[N](C)(C)CSN XTQWAJAVRPQUQE-UHFFFAOYSA-N 0.000 description 1
- YSWBFLWKAIRHEI-UHFFFAOYSA-N Cc1c(C)nc[nH]1 Chemical compound Cc1c(C)nc[nH]1 YSWBFLWKAIRHEI-UHFFFAOYSA-N 0.000 description 1
- SAUOXXLEGIELMN-UHFFFAOYSA-N N#CC[N+]1(CCCCC1)[O-] Chemical compound N#CC[N+]1(CCCCC1)[O-] SAUOXXLEGIELMN-UHFFFAOYSA-N 0.000 description 1
- XGDRLCRGKUCBQL-UHFFFAOYSA-N N#Cc1c(C#N)nc[nH]1 Chemical compound N#Cc1c(C#N)nc[nH]1 XGDRLCRGKUCBQL-UHFFFAOYSA-N 0.000 description 1
- YWZHEXZIISFIDA-UHFFFAOYSA-N NC(SS1)=NC1=S Chemical compound NC(SS1)=NC1=S YWZHEXZIISFIDA-UHFFFAOYSA-N 0.000 description 1
- KUEFXPHXHHANKS-UHFFFAOYSA-N [O-][N+](c1n[nH]cn1)=O Chemical compound [O-][N+](c1n[nH]cn1)=O KUEFXPHXHHANKS-UHFFFAOYSA-N 0.000 description 1
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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
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
-
- 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
-
- 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
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- Genetics & Genomics (AREA)
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Window Of Vehicle (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Liquid Crystal Substances (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461928405P | 2014-01-16 | 2014-01-16 | |
| US61/928,405 | 2014-01-16 | ||
| US201462063359P | 2014-10-13 | 2014-10-13 | |
| US62/063,359 | 2014-10-13 | ||
| PCT/IB2015/000395 WO2015107425A2 (en) | 2014-01-16 | 2015-01-16 | Chiral design |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018145256A Division JP6757373B2 (ja) | 2014-01-16 | 2018-08-01 | キラルデザイン |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2017506911A JP2017506911A (ja) | 2017-03-16 |
| JP6382344B2 true JP6382344B2 (ja) | 2018-08-29 |
Family
ID=53039535
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016564442A Active JP6382344B2 (ja) | 2014-01-16 | 2015-01-16 | キラルデザイン |
| JP2018145256A Active JP6757373B2 (ja) | 2014-01-16 | 2018-08-01 | キラルデザイン |
| JP2020144129A Active JP7224319B2 (ja) | 2014-01-16 | 2020-08-28 | キラルデザイン |
| JP2023016863A Active JP7662687B2 (ja) | 2014-01-16 | 2023-02-07 | キラルデザイン |
| JP2025061939A Pending JP2025106395A (ja) | 2014-01-16 | 2025-04-03 | キラルデザイン |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018145256A Active JP6757373B2 (ja) | 2014-01-16 | 2018-08-01 | キラルデザイン |
| JP2020144129A Active JP7224319B2 (ja) | 2014-01-16 | 2020-08-28 | キラルデザイン |
| JP2023016863A Active JP7662687B2 (ja) | 2014-01-16 | 2023-02-07 | キラルデザイン |
| JP2025061939A Pending JP2025106395A (ja) | 2014-01-16 | 2025-04-03 | キラルデザイン |
Country Status (19)
| Country | Link |
|---|---|
| US (4) | US10160969B2 (enExample) |
| EP (2) | EP3094728B1 (enExample) |
| JP (5) | JP6382344B2 (enExample) |
| KR (5) | KR102423317B1 (enExample) |
| CN (2) | CN113278617A (enExample) |
| AU (3) | AU2015207773B2 (enExample) |
| BR (1) | BR112016016400A2 (enExample) |
| CA (1) | CA2936712A1 (enExample) |
| CL (1) | CL2016001814A1 (enExample) |
| DK (1) | DK3094728T3 (enExample) |
| ES (1) | ES2917473T3 (enExample) |
| IL (1) | IL246703A0 (enExample) |
| MX (1) | MX2016009290A (enExample) |
| MY (1) | MY193116A (enExample) |
| PL (1) | PL3094728T3 (enExample) |
| PT (1) | PT3094728T (enExample) |
| RU (1) | RU2016133035A (enExample) |
| SG (2) | SG10201912897UA (enExample) |
| WO (1) | WO2015107425A2 (enExample) |
Families Citing this family (128)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG171914A1 (en) | 2008-12-02 | 2011-07-28 | Chiralgen Ltd | Method for the synthesis of phosphorus atom modified nucleic acids |
| US9744183B2 (en) | 2009-07-06 | 2017-08-29 | Wave Life Sciences Ltd. | Nucleic acid prodrugs and methods of use thereof |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| RU2014105311A (ru) | 2011-07-19 | 2015-08-27 | Уэйв Лайф Сайенсес Пте. Лтд. | Способы синтеза функционализованных нуклеиновых кислот |
| PT2872485T (pt) | 2012-07-13 | 2021-03-05 | Wave Life Sciences Ltd | Grupo auxiliar assimétrico |
| BR112015000723A2 (pt) | 2012-07-13 | 2017-06-27 | Shin Nippon Biomedical Laboratories Ltd | adjuvante de ácido nucléico quiral |
| KR102712879B1 (ko) | 2012-07-13 | 2024-10-04 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| JPWO2015108046A1 (ja) | 2014-01-15 | 2017-03-23 | 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 |
| JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 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 |
| JPWO2015108047A1 (ja) | 2014-01-15 | 2017-03-23 | 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 |
| PT3094728T (pt) | 2014-01-16 | 2022-05-19 | Wave Life Sciences Ltd | Desenho quiral |
| RU2708237C2 (ru) | 2014-08-22 | 2019-12-05 | Общество с ограниченной ответственностью "НооГен" | Модифицированные олигонуклеотиды и способ их получения |
| EP3234131B1 (en) | 2014-12-16 | 2020-04-22 | Roche Innovation Center Copenhagen A/S | Chiral toxicity screening method |
| EP3712269A1 (en) | 2014-12-17 | 2020-09-23 | ProQR Therapeutics II B.V. | Targeted rna editing |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| US20190127733A1 (en) * | 2015-10-09 | 2019-05-02 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| EP3848035B1 (en) | 2015-12-10 | 2025-02-19 | PTC Therapeutics, Inc. | 1,4-disubstituted pyridazine compounds for treating huntington's disease |
| AU2017234150B2 (en) | 2016-03-13 | 2021-09-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
| MA45469A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Acides nucléiques de bêta-caténine et leurs utilisations |
| MA45349A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Acides nucléiques egfr et leurs utilisations |
| MA45471A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Acides nucléiques de phosphatidylinositol-3-kinase et leurs utilisations |
| MA45470A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Acides nucléiques kras et leurs utilisations |
| CA3021267A1 (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 |
| CA3022303A1 (en) | 2016-04-29 | 2017-11-02 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues targeting human lmna |
| MA45270A (fr) * | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| MA45290A (fr) * | 2016-05-04 | 2019-03-13 | Wave Life Sciences Ltd | Procédés et compositions d'agents biologiquement actifs |
| CN109562122A (zh) * | 2016-06-03 | 2019-04-02 | 波涛生命科学有限公司 | 寡核苷酸、组合物及其方法 |
| 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 |
| CA3025575A1 (en) | 2016-06-30 | 2018-01-04 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| KR102501980B1 (ko) | 2016-09-01 | 2023-02-20 | 프로큐알 테라퓨틱스 Ⅱ 비.브이. | 화학적으로 변형된 단일 가닥 rna-편집 올리고뉴클레오타이드 |
| US11873316B2 (en) | 2016-11-23 | 2024-01-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| CA3043768A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| KR102646318B1 (ko) | 2016-12-19 | 2024-03-12 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| AU2017382741B2 (en) | 2016-12-19 | 2025-01-30 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| AU2017382723B2 (en) | 2016-12-19 | 2025-01-30 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| SG11201906200WA (en) | 2017-01-06 | 2019-08-27 | Avidity Biosciences Llc | Nucleic acid-polypeptide compositions and methods of inducing exon skipping |
| MX2019012295A (es) | 2017-04-14 | 2020-02-07 | Tollnine Inc | Polinucleotidos inmunomoduladores, conjugados de anticuerpos de los mismos y metodos para su uso. |
| CA3065523A1 (en) * | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US20210198305A1 (en) * | 2017-06-02 | 2021-07-01 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| WO2018223056A1 (en) * | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| WO2018226622A1 (en) | 2017-06-05 | 2018-12-13 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
| CN111051281A (zh) | 2017-06-21 | 2020-04-21 | 波涛生命科学有限公司 | 用于合成的化合物、组合物和方法 |
| WO2019005993A1 (en) | 2017-06-28 | 2019-01-03 | Ptc Therapeutics, Inc. | METHODS OF TREATING HUNTINGTON'S DISEASE |
| BR112019027717A2 (pt) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics, Inc. | métodos para tratar a doença de huntington |
| GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| US20200362337A1 (en) * | 2017-08-08 | 2020-11-19 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| EP3684755A4 (en) | 2017-09-18 | 2022-03-02 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE PREPARATION TECHNOLOGIES |
| 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 |
| US20200248178A1 (en) | 2017-09-28 | 2020-08-06 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| US20200254002A1 (en) | 2017-09-28 | 2020-08-13 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| WO2019071028A1 (en) | 2017-10-04 | 2019-04-11 | Avidity Biosciences Llc | NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF |
| JP2020537518A (ja) * | 2017-10-12 | 2020-12-24 | ウェーブ ライフ サイエンシーズ リミテッド | オリゴヌクレオチド組成物及びその方法 |
| EP3694995A1 (en) | 2017-10-13 | 2020-08-19 | Roche Innovation Center Copenhagen A/S | Methods for identifying improved stereodefined phosphorothioate oligonucleotide variants of antisense oligonucleotides utilising sub-libraries of partially stereodefined oligonucleotides |
| EP3697910A4 (en) | 2017-10-18 | 2021-07-14 | Sarepta Therapeutics, Inc. | ANTISENSE OLIGOMER COMPOUNDS |
| CN118291458A (zh) | 2017-12-01 | 2024-07-05 | 德克萨斯A&M大学体系 | 天使综合征反义治疗 |
| SG11202005022YA (en) | 2017-12-06 | 2020-06-29 | Avidity Biosciences Inc | Compositions and methods of treating muscle atrophy and myotonic dystrophy |
| CA3086485A1 (en) * | 2017-12-21 | 2019-06-27 | Alnylam Pharmaceuticals, Inc. | Chirally-enriched double-stranded rna agents |
| CN111902537A (zh) * | 2018-01-15 | 2020-11-06 | Ionis制药公司 | Dnm2表达的调节剂 |
| BR112020019373A2 (pt) | 2018-03-27 | 2020-12-29 | Ptc Therapeutics, Inc. | Compostos para o tratamento da doença de hutington |
| KR20210005057A (ko) * | 2018-04-12 | 2021-01-13 | 웨이브 라이프 사이언시스 리미티드 | 올리고뉴클레오티드 조성물 및 이의 이용 방법 |
| MX2020011570A (es) | 2018-05-07 | 2020-11-24 | Alnylam Pharmaceuticals Inc | Administracion extrahepatica. |
| JP7427608B2 (ja) | 2018-05-11 | 2024-02-05 | 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使用方法 |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| US10758629B2 (en) | 2018-05-29 | 2020-09-01 | 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 |
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