JP6496549B2 - 脊髄性筋萎縮症における誘発されたエクソン包含 - Google Patents
脊髄性筋萎縮症における誘発されたエクソン包含 Download PDFInfo
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- JP6496549B2 JP6496549B2 JP2014544962A JP2014544962A JP6496549B2 JP 6496549 B2 JP6496549 B2 JP 6496549B2 JP 2014544962 A JP2014544962 A JP 2014544962A JP 2014544962 A JP2014544962 A JP 2014544962A JP 6496549 B2 JP6496549 B2 JP 6496549B2
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- antisense oligonucleotide
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- 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
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- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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- 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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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/31—Chemical structure of the backbone
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/30—Chemical structure
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- C12N2310/314—Phosphoramidates
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- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
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| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| ES2727481T3 (es) | 2011-11-30 | 2019-10-16 | Sarepta Therapeutics Inc | Inclusión inducida de exón en atrofia muscular espinal |
| WO2013082548A1 (en) | 2011-11-30 | 2013-06-06 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| WO2014113540A1 (en) | 2013-01-16 | 2014-07-24 | Iowa State University Research Foundation, Inc. | A deep intronic target for splicing correction on spinal muscular atrophy gene |
| AU2014317961B2 (en) * | 2013-09-05 | 2020-07-30 | Murdoch University | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| WO2016040748A1 (en) | 2014-09-12 | 2016-03-17 | Ionis Pharmaceuticals, Inc. | Compositions and methods for detection of smn protein in a subject and treatment of a subject |
| WO2017040271A1 (en) * | 2015-08-28 | 2017-03-09 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
| US10357543B2 (en) | 2015-11-16 | 2019-07-23 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using Survival Motor Neuron (SMN) protein |
| US11060089B2 (en) | 2016-04-18 | 2021-07-13 | Sarepta Therapeutics, Inc. | Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene |
| US10947533B2 (en) | 2016-05-24 | 2021-03-16 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| TWI737736B (zh) * | 2016-05-24 | 2021-09-01 | 美商薩羅塔治療公司 | 製備磷醯二胺嗎啉代寡聚物之方法 |
| MA45158A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Composition pharmaceutique comprenant de l'éteplirsen |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| MX384727B (es) | 2016-05-24 | 2025-03-14 | Sarepta Therapeutics Inc | Procesos para preparar oligómeros |
| MX390906B (es) * | 2016-05-24 | 2025-03-21 | Sarepta Therapeutics Inc | Procesos para preparar oligómeros de morfolino fosforodiamidato. |
| US11198867B2 (en) | 2016-06-16 | 2021-12-14 | Ionis Pharmaceuticals, Inc. | Combinations for the modulation of SMN expression |
| CN109152775B (zh) * | 2016-06-30 | 2022-04-26 | 萨勒普塔医疗公司 | 制备磷酸二酰胺吗啉代寡聚物的方法 |
| CA3024153A1 (en) | 2016-06-30 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| AU2017330276B2 (en) * | 2016-09-20 | 2022-01-20 | The Regents Of The University Of Colorado, A Body Corporate | Synthesis of backbone modified morpholino oligonucleotides and chimeras using phosphoramidite chemistry |
| PT4122497T (pt) | 2016-12-19 | 2024-06-12 | Sarepta Therapeutics Inc | Conjugados oligoméricos de salto de exão para a distrofia muscular |
| ES3051834T3 (en) | 2016-12-19 | 2025-12-30 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| NZ755416A (en) | 2016-12-19 | 2023-05-26 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| TWI812647B (zh) | 2017-09-25 | 2023-08-21 | 美商薩羅塔治療公司 | 經由速流合成以製備磷醯二胺嗎啉代寡聚物之製程 |
| US11555189B2 (en) * | 2017-10-18 | 2023-01-17 | Sarepta Therapeutics, Inc. | Antisense oligomer compounds |
| EP3765030A4 (en) * | 2018-03-16 | 2022-01-26 | Sarepta Therapeutics, Inc. | CHIMERIC PEPTIDES FOR ANTISENSE ADMINISTRATION |
| WO2019183570A1 (en) * | 2018-03-22 | 2019-09-26 | Board Of Regents, The University Of Texas System | Soluble interleukin-7 receptor (sil7r) molulating therapy to treat autoimmune diseases and cancer |
| EP4219717A3 (en) | 2018-06-13 | 2023-12-20 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| 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 |
| BR112021011018A2 (pt) | 2018-12-13 | 2021-08-31 | Sarepta Therapeutics, Inc. | Conjugados de oligômero para exon skipping para distrofia muscular |
| WO2021025899A1 (en) | 2019-08-02 | 2021-02-11 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer pharmaceutical compositions |
| AU2021225957A1 (en) * | 2020-02-28 | 2022-09-08 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating SMN2 |
| US12378267B2 (en) | 2021-06-17 | 2025-08-05 | Entrada Therapeutics, Inc. | Synthesis of FMOC-protected morpholino monomers and oligomers |
| US20250289851A1 (en) | 2022-04-22 | 2025-09-18 | Entrada Therapeutics, Inc. | Cyclic peptides for delivering therapeutics |
| US20260207778A1 (en) * | 2022-12-23 | 2026-07-23 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Treatment of GEMIN5-Mediated Disorders |
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| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| JP2528107B2 (ja) | 1985-03-15 | 1996-08-28 | サマ−トン，ジエ−ムス | ポリヌクレオチド測定試薬と方法 |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| AU659482B2 (en) | 1991-06-28 | 1995-05-18 | Massachusetts Institute Of Technology | Localized oligonucleotide therapy |
| WO1997022371A1 (en) | 1995-12-18 | 1997-06-26 | Collagen Corporation | Crosslinked polymer compositions and methods for their use |
| US6245747B1 (en) | 1996-03-12 | 2001-06-12 | The Board Of Regents Of The University Of Nebraska | Targeted site specific antisense oligodeoxynucleotide delivery method |
| UA67760C2 (uk) | 1997-12-11 | 2004-07-15 | Міннесота Майнінг Енд Мануфакчурінг Компані | Імідазонафтиридин та тетрагідроімідазонафтиридин, фармацевтична композиція, спосіб індукування біосинтезу цитокінів та спосіб лікування вірусної інфекції, проміжні сполуки |
| FR2802206B1 (fr) * | 1999-12-14 | 2005-04-22 | Sod Conseils Rech Applic | Derives de 4-aminopiperidine et leur utilisation en tant que medicament |
| AU5752601A (en) | 2000-05-04 | 2001-11-12 | Avi Biopharma Inc | Splice-region antisense composition and method |
| US6965025B2 (en) | 2001-12-10 | 2005-11-15 | Isis Pharmaceuticals, Inc. | Antisense modulation of connective tissue growth factor expression |
| US7838657B2 (en) | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
| PT2548560E (pt) | 2005-06-23 | 2015-09-22 | Isis Pharmaceuticals Inc | Composições e métodos de modulação da excisão-união de smn2 |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| LT2735568T (lt) * | 2006-05-10 | 2017-11-27 | Sarepta Therapeutics, Inc. | Oligonukleotido analogai, turintys katijonines jungtis tarp subvienetų |
| JP6047270B2 (ja) | 2006-08-11 | 2016-12-21 | バイオマリン テクノロジーズ ベー．フェー． | Ｄｎａリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段 |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| MX2010004955A (es) | 2007-11-15 | 2010-06-30 | Avi Biopharma Inc | Metodo de sintesis de oligomeros de morfolina. |
| US20120149757A1 (en) | 2009-04-13 | 2012-06-14 | Krainer Adrian R | Compositions and methods for modulation of smn2 splicing |
| KR20220084437A (ko) | 2009-06-17 | 2022-06-21 | 바이오젠 엠에이 인코포레이티드 | 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법 |
| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| JP5585822B2 (ja) | 2010-05-11 | 2014-09-10 | 東レ・ファインケミカル株式会社 | 光学活性ニペコチン酸誘導体の製造方法 |
| KR101981705B1 (ko) | 2010-05-28 | 2019-05-24 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| AU2011367230B2 (en) | 2011-05-05 | 2017-08-10 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| WO2013082548A1 (en) | 2011-11-30 | 2013-06-06 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| ES2727481T3 (es) | 2011-11-30 | 2019-10-16 | Sarepta Therapeutics Inc | Inclusión inducida de exón en atrofia muscular espinal |
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| ES2727481T3 (es) | 2019-10-16 |
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| CA2857664A1 (en) | 2013-06-06 |
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