JP2016526529A5 - - Google Patents
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- Publication number
- JP2016526529A5 JP2016526529A5 JP2015560881A JP2015560881A JP2016526529A5 JP 2016526529 A5 JP2016526529 A5 JP 2016526529A5 JP 2015560881 A JP2015560881 A JP 2015560881A JP 2015560881 A JP2015560881 A JP 2015560881A JP 2016526529 A5 JP2016526529 A5 JP 2016526529A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleotides
- strand
- independently selected
- nucleotide analogs
- natural
- 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.)
- Granted
Links
- 125000003729 nucleotide group Chemical group 0.000 description 9
- 239000002773 nucleotide Substances 0.000 description 7
- 108091034117 Oligonucleotide Proteins 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361835634P | 2013-06-16 | 2013-06-16 | |
| US61/835,634 | 2013-06-16 | ||
| US201361836672P | 2013-06-19 | 2013-06-19 | |
| US61/836,672 | 2013-06-19 | ||
| PCT/JP2014/003208 WO2014203518A1 (en) | 2013-06-16 | 2014-06-16 | Double-stranded antisense nucleic acid with exon-skipping effect |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016526529A JP2016526529A (ja) | 2016-09-05 |
| JP2016526529A5 true JP2016526529A5 (enExample) | 2017-08-17 |
| JP6604544B2 JP6604544B2 (ja) | 2019-11-13 |
Family
ID=52104269
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015560881A Active JP6604544B2 (ja) | 2013-06-16 | 2014-06-16 | エクソンスキッピング効果を有する二本鎖アンチセンス核酸 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US10190117B2 (enExample) |
| EP (1) | EP3010514B1 (enExample) |
| JP (1) | JP6604544B2 (enExample) |
| CN (1) | CN105324119A (enExample) |
| AU (1) | AU2014282666A1 (enExample) |
| CA (1) | CA2915443A1 (enExample) |
| WO (1) | WO2014203518A1 (enExample) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| AU2013262649A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating smn gene family expression |
| EA201492116A1 (ru) | 2012-05-16 | 2015-05-29 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии mecp2 |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| PT2872485T (pt) | 2012-07-13 | 2021-03-05 | Wave Life Sciences Ltd | Grupo auxiliar assimétrico |
| EP3052632A4 (en) | 2013-10-04 | 2017-03-29 | Rana Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| EP3712269A1 (en) | 2014-12-17 | 2020-09-23 | ProQR Therapeutics II B.V. | Targeted rna editing |
| JP6892433B2 (ja) | 2015-04-03 | 2021-06-23 | ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts | 十分に安定化された非対称sirna |
| CA2995110A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| US11116843B2 (en) | 2015-09-25 | 2021-09-14 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| US10478503B2 (en) | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| IL262728B2 (en) | 2016-05-06 | 2023-10-01 | Astrazeneca Ab | Oligonucleotides conjugated to the GLP-1 receptor ligand complex and their uses |
| 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 |
| BR112019000061A2 (pt) * | 2016-07-05 | 2019-11-26 | Biomarin Technologies B.V. | comutação e modulação de splicing de pré-mrna compreendendo porções de arcabouço bicíclico, com características melhoradas para o tratamento de distúrbios genéticos |
| CA3033368A1 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| KR102501980B1 (ko) | 2016-09-01 | 2023-02-20 | 프로큐알 테라퓨틱스 Ⅱ 비.브이. | 화학적으로 변형된 단일 가닥 rna-편집 올리고뉴클레오타이드 |
| WO2018056442A1 (ja) | 2016-09-23 | 2018-03-29 | 国立大学法人東京医科歯科大学 | 血液脳関門通過型ヘテロ2本鎖核酸 |
| US11260134B2 (en) | 2016-09-29 | 2022-03-01 | National University Corporation Tokyo Medical And Dental University | Double-stranded nucleic acid complex having overhang |
| EP3571300A1 (en) | 2017-01-19 | 2019-11-27 | ProQR Therapeutics II B.V. | Oligonucleotide complexes for use in rna editing |
| EP3642341A4 (en) | 2017-06-23 | 2021-06-16 | University Of Massachusetts | TWO-DAY SELF-RELEASING SIRNA AND RELATED PROCEDURES |
| EP3647423A4 (en) | 2017-06-30 | 2021-03-24 | National University Corporation Tokyo Medical and Dental University | STRAIGHT DOUBLE-STRANDED ANTI-MIRROR |
| CN119876131A (zh) * | 2017-11-08 | 2025-04-25 | Ionis制药公司 | Glp-1受体配体部分缀合的寡核苷酸及其用途 |
| JP7360170B2 (ja) | 2018-02-28 | 2023-10-12 | 国立大学法人 東京医科歯科大学 | 虚血病変部位特異的な遺伝子治療法 |
| WO2019177061A1 (ja) * | 2018-03-14 | 2019-09-19 | 国立大学法人東京医科歯科大学 | 核酸複合体 |
| EP3769769A4 (en) | 2018-03-19 | 2022-05-04 | National University Corporation Tokyo Medical and Dental University | NUCLEIC ACID WITH REDUCED TOXICITY |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| KR20210093227A (ko) | 2018-08-10 | 2021-07-27 | 유니버시티 오브 매사추세츠 | Snp를 표적화하는 변형된 올리고뉴클레오티드 |
| JP7627042B2 (ja) | 2018-08-23 | 2025-02-05 | ユニバーシティー オブ マサチューセッツ | O-メチルリッチ完全安定化オリゴヌクレオチド |
| JP2022523467A (ja) * | 2019-01-18 | 2022-04-25 | ユニバーシティ・オブ・マサチューセッツ | 動的な薬物動態を修飾するアンカー |
| EP3954395A4 (en) * | 2019-04-08 | 2023-06-28 | National University Corporation Tokyo Medical and Dental University | Pharmaceutical composition for muscle disease treatment |
| CA3149835A1 (en) | 2019-08-09 | 2021-02-18 | University Of Massachusetts | Chemically modified oligonucleotides targeting snps |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| US20240117346A1 (en) * | 2019-10-11 | 2024-04-11 | National University Corporation Tokyo Medical And Dental University | Modified hetero nucleic acids |
| US20230061751A1 (en) * | 2020-01-17 | 2023-03-02 | University Of Massachusetts | Universal dynamic pharmacokinetic-modifying anchors |
| US20230174981A1 (en) * | 2020-03-16 | 2023-06-08 | National University Corporation Tokyo Medical And Dental University | Heteronucleic acid containing morpholino nucleic acid |
| US20240002853A1 (en) | 2020-11-23 | 2024-01-04 | Alpha Anomeric Sas | Nucleic acid duplexes |
| WO2022271786A1 (en) | 2021-06-23 | 2022-12-29 | University Of Massachusetts | Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders |
| EP4389891A4 (en) * | 2021-08-19 | 2025-10-29 | Univ Nat Corp Tokyo Medical & Dental | MODIFIED HETERONUCLEIC ACID CONTAINING MORPHOLINO NUCLEIC ACID |
| CN118660965A (zh) * | 2021-12-07 | 2024-09-17 | Liid制药股份有限公司 | 反义寡核苷酸复合体 |
| CN115806984B (zh) * | 2022-10-18 | 2023-10-10 | 昆明理工大学 | 环状rna及载体和载体的应用 |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
Family Cites Families (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPH09110894A (ja) * | 1995-10-17 | 1997-04-28 | Soyaku Gijutsu Kenkyusho:Kk | ハイブリッドdna/rnaオリゴヌクレオチド及び抗ウイルス剤 |
| JP3781879B2 (ja) | 1996-11-18 | 2006-05-31 | 武 今西 | 新規ヌクレオチド類縁体 |
| WO1998022489A1 (en) | 1996-11-18 | 1998-05-28 | Takeshi Imanishi | Novel nucleotide analogues |
| US6770748B2 (en) | 1997-03-07 | 2004-08-03 | Takeshi Imanishi | Bicyclonucleoside and oligonucleotide analogue |
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| CA2303299C (en) | 1997-09-12 | 2016-02-23 | Exiqon A/S | Oligonucleotide analogues |
| US20010024808A1 (en) * | 1998-09-10 | 2001-09-27 | Millennium Pharmaceuticals, Inc., A Delaware Corporation | Leptin induced genes |
| US6933105B2 (en) * | 2000-01-31 | 2005-08-23 | Millennium Pharmaceuticals, Inc. | Resistance sequences and uses thereof |
| US20010039331A1 (en) * | 2000-03-31 | 2001-11-08 | Hunter John J. | 16836, a novel human phospholipase C family member and uses thereof |
| WO2004041889A2 (en) * | 2002-11-05 | 2004-05-21 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| EP2336318B1 (en) * | 2002-11-13 | 2013-04-24 | Genzyme Corporation | Antisense modulation of apolipoprotein b expression |
| DK1661905T3 (da) | 2003-08-28 | 2012-07-23 | Takeshi Imanishi | Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype |
| WO2005113571A2 (en) * | 2004-05-13 | 2005-12-01 | The University Of North Carolina At Chapel Hill | Methods for the delivery of oligomeric compounds |
| WO2006059507A1 (ja) | 2004-11-30 | 2006-06-08 | Sankyo Company, Limited | 11β-HSD1アンチセンス化合物 |
| DK2002004T3 (en) * | 2006-03-23 | 2015-11-30 | Roche Innovation Ct Copenhagen As | LITTLE INTERNAL SEGMENTED INTERFERENCE RNA |
| DK2363481T3 (en) | 2006-05-05 | 2017-06-26 | Ionis Pharmaceuticals Inc | Compounds and methods for modulating gene expression |
| WO2008029619A1 (fr) | 2006-09-07 | 2008-03-13 | Daiichi Sankyo Company, Limited | Oligonucléotide antisens ena ayant une action spécifique de la séquence |
| US20100216864A1 (en) | 2006-10-09 | 2010-08-26 | Ellen Marie Straarup | RNA Antagonist Compounds for the Modulation of PCSK9 |
| US8470791B2 (en) * | 2007-03-22 | 2013-06-25 | Santaris Pharma A/S | RNA antagonist compounds for the inhibition of Apo-B100 expression |
| US8399248B2 (en) * | 2007-05-03 | 2013-03-19 | Merck Sharp & Dohme Corp. | Methods of using MIR34 as a biomarker for TP53 functional status |
| DE102007021870A1 (de) | 2007-05-10 | 2008-11-20 | Clariant International Limited | Wässrige Pigmentpräparationen |
| AU2008292091A1 (en) | 2007-08-30 | 2009-03-05 | Santaris Pharma A/S | RNA antagonist compounds for the modulation of FABP4/aP2 |
| US20100249214A1 (en) * | 2009-02-11 | 2010-09-30 | Dicerna Pharmaceuticals | Multiplex dicer substrate rna interference molecules having joining sequences |
| US20130190383A1 (en) | 2010-04-26 | 2013-07-25 | Marina Biotech, Inc. | Nucleic acid compounds with conformationally restricted monomers and uses thereof |
| US20140128449A1 (en) * | 2011-04-07 | 2014-05-08 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
| CN103747805B (zh) * | 2011-04-22 | 2017-08-29 | 比奥马林技术公司 | 用于治疗、延缓和/或预防人类遗传性疾病如强直性肌营养不良症1型(dm1)的化合物 |
| CA2859581C (en) * | 2011-12-16 | 2022-03-22 | National University Corporation Tokyo Medical And Dental University | Chimeric double-stranded nucleic acid |
-
2014
- 2014-06-16 CN CN201480034378.1A patent/CN105324119A/zh active Pending
- 2014-06-16 CA CA2915443A patent/CA2915443A1/en not_active Abandoned
- 2014-06-16 WO PCT/JP2014/003208 patent/WO2014203518A1/en not_active Ceased
- 2014-06-16 US US14/898,630 patent/US10190117B2/en active Active
- 2014-06-16 AU AU2014282666A patent/AU2014282666A1/en not_active Abandoned
- 2014-06-16 JP JP2015560881A patent/JP6604544B2/ja active Active
- 2014-06-16 EP EP14814254.0A patent/EP3010514B1/en active Active
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