JP2018110601A5 - - Google Patents
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- Publication number
- JP2018110601A5 JP2018110601A5 JP2018081272A JP2018081272A JP2018110601A5 JP 2018110601 A5 JP2018110601 A5 JP 2018110601A5 JP 2018081272 A JP2018081272 A JP 2018081272A JP 2018081272 A JP2018081272 A JP 2018081272A JP 2018110601 A5 JP2018110601 A5 JP 2018110601A5
- Authority
- JP
- Japan
- Prior art keywords
- antisense oligonucleotide
- oligonucleotide according
- base
- pharmaceutically acceptable
- acceptable salt
- 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
- 108091034117 Oligonucleotide Proteins 0.000 claims 15
- 239000000074 antisense oligonucleotide Substances 0.000 claims 15
- 238000012230 antisense oligonucleotides Methods 0.000 claims 15
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 4
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 4
- 150000003839 salts Chemical class 0.000 claims 3
- 239000000203 mixture Substances 0.000 claims 2
- 229940113082 thymine Drugs 0.000 claims 2
- 229940035893 uracil Drugs 0.000 claims 2
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 1
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 claims 1
- 102220492049 Phospholipid scramblase 1_H53A_mutation Human genes 0.000 claims 1
- 239000002202 Polyethylene glycol Substances 0.000 claims 1
- 238000000137 annealing Methods 0.000 claims 1
- 230000004700 cellular uptake Effects 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 125000003827 glycol group Chemical group 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 229920001223 polyethylene glycol Polymers 0.000 claims 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261739968P | 2012-12-20 | 2012-12-20 | |
| US61/739,968 | 2012-12-20 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015549818A Division JP2016502858A (ja) | 2012-12-20 | 2013-12-20 | 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019003308A Division JP2019050834A (ja) | 2012-12-20 | 2019-01-11 | 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018110601A JP2018110601A (ja) | 2018-07-19 |
| JP2018110601A5 true JP2018110601A5 (https=) | 2019-02-21 |
Family
ID=49950080
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015549818A Withdrawn JP2016502858A (ja) | 2012-12-20 | 2013-12-20 | 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 |
| JP2018081272A Pending JP2018110601A (ja) | 2012-12-20 | 2018-04-20 | 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 |
| JP2019003308A Pending JP2019050834A (ja) | 2012-12-20 | 2019-01-11 | 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015549818A Withdrawn JP2016502858A (ja) | 2012-12-20 | 2013-12-20 | 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019003308A Pending JP2019050834A (ja) | 2012-12-20 | 2019-01-11 | 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 |
Country Status (13)
| Country | Link |
|---|---|
| US (5) | US20150361428A1 (https=) |
| EP (2) | EP3885439A1 (https=) |
| JP (3) | JP2016502858A (https=) |
| KR (2) | KR20150099804A (https=) |
| CN (2) | CN111440796A (https=) |
| AU (3) | AU2013364158A1 (https=) |
| BR (1) | BR112015014987A2 (https=) |
| CA (1) | CA2894899A1 (https=) |
| EA (1) | EA201591178A1 (https=) |
| HK (1) | HK1216902A1 (https=) |
| IL (1) | IL239491A0 (https=) |
| MX (2) | MX2015008035A (https=) |
| WO (1) | WO2014100714A1 (https=) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP1855694B1 (en) | 2005-02-09 | 2020-12-02 | Sarepta Therapeutics, Inc. | Antisense composition for treating muscle atrophy |
| BR122020021379B1 (pt) | 2008-10-24 | 2021-05-11 | Sarepta Therapeutics, Inc. | oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular |
| TR201816523T4 (tr) | 2009-11-12 | 2018-11-21 | Univ Western Australia | Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler. |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| US20140315977A1 (en) * | 2013-03-14 | 2014-10-23 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| CN110218727A (zh) | 2013-03-14 | 2019-09-10 | 萨勒普塔医疗公司 | 用于治疗肌营养不良的外显子跳跃组合物 |
| US20140329762A1 (en) | 2013-03-15 | 2014-11-06 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| PT3283500T (pt) * | 2015-04-08 | 2021-01-28 | Univ Chicago | Composições e métodos para corrigir distrofia muscular das cinturas tipo 2c com utilização de salto do exão |
| EP3858993A1 (en) | 2015-10-09 | 2021-08-04 | Sarepta Therapeutics, Inc. | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
| FR3044926B1 (fr) | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
| PH12018501207B1 (en) | 2015-12-21 | 2024-02-23 | Novartis Ag | Compositions and methods for decreasing tau expression |
| WO2017136435A1 (en) | 2016-02-01 | 2017-08-10 | The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health | Compounds for modulating fc-epsilon-ri-beta expression and uses thereof |
| JP2019513371A (ja) | 2016-04-01 | 2019-05-30 | アビディティー バイオサイエンシーズ エルエルシー | 核酸ポリペプチド組成物とその使用 |
| JP2019525742A (ja) | 2016-06-30 | 2019-09-12 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマー |
| EP3481432A4 (en) | 2016-07-05 | 2020-05-06 | The U.S.A. as represented by the Secretary, Department of Health and Human Services | Diagnosing col6-related disorders and methods for treating same |
| US10898566B2 (en) | 2016-09-19 | 2021-01-26 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Zika virus vaccines |
| KR20240006023A (ko) | 2016-12-19 | 2024-01-12 | 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 |
| PT3554552T (pt) | 2016-12-19 | 2022-10-03 | Sarepta Therapeutics Inc | Conjugados de oligómero de skipping de exão para distrofia muscular |
| MX2019008199A (es) | 2017-01-06 | 2019-11-25 | Avidity Biosciences Llc | Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon. |
| WO2018152526A1 (en) | 2017-02-20 | 2018-08-23 | The Usa, As Represented By The Secretary, Dept. Of Health And Human Services | Zika virus vaccines |
| GB201711809D0 (en) * | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| EA201991450A1 (ru) * | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| JP2021521794A (ja) * | 2018-04-26 | 2021-08-30 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート |
| US10758629B2 (en) | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| IL318474A (en) * | 2018-12-13 | 2025-03-01 | Sarepta Therapeutics Inc | Axon-skipping oligomer complexes for muscular dystrophy |
| CN118516348B (zh) * | 2024-07-23 | 2025-01-17 | 深川生物科技(山东)股份公司 | 单链核酸或双链核酸及在制备治疗杜氏肌营养不良的药物中的应用 |
Family Cites Families (42)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CH445129A (fr) | 1964-04-29 | 1967-10-15 | Nestle Sa | Procédé pour la préparation de composés d'inclusion à poids moléculaire élevé |
| US3459731A (en) | 1966-12-16 | 1969-08-05 | Corn Products Co | Cyclodextrin polyethers and their production |
| US3426011A (en) | 1967-02-13 | 1969-02-04 | Corn Products Co | Cyclodextrins with anionic properties |
| US3453257A (en) | 1967-02-13 | 1969-07-01 | Corn Products Co | Cyclodextrin with cationic properties |
| US3453259A (en) | 1967-03-22 | 1969-07-01 | Corn Products Co | Cyclodextrin polyol ethers and their oxidation products |
| US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| WO1986005519A1 (en) | 1985-03-15 | 1986-09-25 | James Summerton | Polynucleotide assay reagent and method |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| US4737323A (en) | 1986-02-13 | 1988-04-12 | Liposome Technology, Inc. | Liposome extrusion method |
| KR0166088B1 (ko) | 1990-01-23 | 1999-01-15 | . | 수용해도가 증가된 시클로덱스트린 유도체 및 이의 용도 |
| US5149797A (en) | 1990-02-15 | 1992-09-22 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of rna and production of encoded polypeptides |
| EP0786522A2 (en) | 1992-07-17 | 1997-07-30 | Ribozyme Pharmaceuticals, Inc. | Enzymatic RNA molecules for treatment of stenotic conditions |
| DE69435005T2 (de) | 1993-05-11 | 2008-04-17 | The University Of North Carolina At Chapel Hill | Antisense Oligonukleotide die anomales Splicing verhindern und deren Verwendung |
| US5753613A (en) | 1994-09-30 | 1998-05-19 | Inex Pharmaceuticals Corporation | Compositions for the introduction of polyanionic materials into cells |
| US5885613A (en) | 1994-09-30 | 1999-03-23 | The University Of British Columbia | Bilayer stabilizing components and their use in forming programmable fusogenic liposomes |
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| IL115849A0 (en) | 1994-11-03 | 1996-01-31 | Merz & Co Gmbh & Co | Tangential filtration preparation of liposomal drugs and liposome product thereof |
| WO1999042091A2 (en) | 1998-02-19 | 1999-08-26 | Massachusetts Institute Of Technology | Use of polycations as endosomolytic agents |
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| EP1390490B1 (en) | 2001-05-24 | 2009-04-15 | Genzyme Corporation | Muscle-specific expression vectors |
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| WO2007058894A2 (en) | 2005-11-10 | 2007-05-24 | The University Of North Carolina At Chapel Hill | Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease |
| PL2735568T3 (pl) | 2006-05-10 | 2018-02-28 | Sarepta Therapeutics, Inc. | Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| US8076476B2 (en) | 2007-11-15 | 2011-12-13 | Avi Biopharma, Inc. | Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits |
| US8299206B2 (en) | 2007-11-15 | 2012-10-30 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| WO2009127230A1 (en) | 2008-04-16 | 2009-10-22 | Curevac Gmbh | MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION |
| US8084601B2 (en) * | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| BR122020021379B1 (pt) * | 2008-10-24 | 2021-05-11 | Sarepta Therapeutics, Inc. | oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular |
| TR201816523T4 (tr) * | 2009-11-12 | 2018-11-21 | Univ Western Australia | Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler. |
| KR20200133284A (ko) | 2010-05-28 | 2020-11-26 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| CA3092114A1 (en) | 2011-05-05 | 2012-11-08 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
-
2013
- 2013-12-20 HK HK16104638.2A patent/HK1216902A1/zh unknown
- 2013-12-20 CA CA2894899A patent/CA2894899A1/en not_active Abandoned
- 2013-12-20 KR KR1020157019523A patent/KR20150099804A/ko not_active Ceased
- 2013-12-20 CN CN202010276936.2A patent/CN111440796A/zh active Pending
- 2013-12-20 EA EA201591178A patent/EA201591178A1/ru unknown
- 2013-12-20 MX MX2015008035A patent/MX2015008035A/es unknown
- 2013-12-20 EP EP21152285.9A patent/EP3885439A1/en not_active Withdrawn
- 2013-12-20 WO PCT/US2013/077216 patent/WO2014100714A1/en not_active Ceased
- 2013-12-20 BR BR112015014987A patent/BR112015014987A2/pt not_active Application Discontinuation
- 2013-12-20 AU AU2013364158A patent/AU2013364158A1/en not_active Abandoned
- 2013-12-20 JP JP2015549818A patent/JP2016502858A/ja not_active Withdrawn
- 2013-12-20 CN CN201380073536.XA patent/CN106414739A/zh active Pending
- 2013-12-20 KR KR1020207033842A patent/KR20200143739A/ko not_active Ceased
- 2013-12-20 EP EP13819166.3A patent/EP2935584A1/en not_active Withdrawn
-
2015
- 2015-06-18 IL IL239491A patent/IL239491A0/en unknown
- 2015-06-18 US US14/743,856 patent/US20150361428A1/en not_active Abandoned
- 2015-06-19 MX MX2019008164A patent/MX2019008164A/es unknown
-
2017
- 2017-01-31 US US15/420,823 patent/US20170369875A1/en not_active Abandoned
-
2018
- 2018-04-20 JP JP2018081272A patent/JP2018110601A/ja active Pending
-
2019
- 2019-01-11 JP JP2019003308A patent/JP2019050834A/ja active Pending
- 2019-10-21 AU AU2019253778A patent/AU2019253778A1/en not_active Abandoned
- 2019-12-04 US US16/703,110 patent/US20200339985A1/en not_active Abandoned
-
2021
- 2021-10-11 US US17/498,115 patent/US20220259592A1/en not_active Abandoned
-
2022
- 2022-03-09 AU AU2022201628A patent/AU2022201628A1/en not_active Abandoned
- 2022-05-11 US US17/741,758 patent/US20230104708A1/en not_active Abandoned
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