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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Classifications
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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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- 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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- 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
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
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- Molecular Biology (AREA)
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- Biophysics (AREA)
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- Microbiology (AREA)
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- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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- Micro-Organisms Or Cultivation Processes Thereof (AREA)
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| US201161565499P | 2011-11-30 | 2011-11-30 | |
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| PCT/US2012/067475 WO2013082551A1 (en) | 2011-11-30 | 2012-11-30 | Induced exon inclusion in spinal muscle atrophy |
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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 |
| US10066228B2 (en) | 2011-11-30 | 2018-09-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| US9944926B2 (en) | 2011-11-30 | 2018-04-17 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
| US9856474B2 (en) | 2013-01-16 | 2018-01-02 | Iowa State University Research Foundation, Inc. | 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 |
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| JP6987041B2 (ja) * | 2015-08-28 | 2021-12-22 | サレプタ セラピューティクス，インコーポレイテッド | 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー |
| EP3377073A4 (en) * | 2015-11-16 | 2019-06-26 | Ohio State Innovation Foundation | METHOD AND COMPOSITIONS FOR TREATING DISORDERS AND DISEASES WITH THE SURVIVAL MOTOR NEURON (SMN) PROTEIN |
| JP7033547B2 (ja) | 2016-04-18 | 2022-03-10 | サレプタ セラピューティクス， インコーポレイテッド | 酸性アルファ－グルコシダーゼ遺伝子に関連する疾患を処置するためのアンチセンスオリゴマーおよびそれを用いる方法 |
| SG11201809494VA (en) | 2016-05-24 | 2018-12-28 | Sarepta Therapeutics Inc | Pharmaceutical composition comprising eteplirsen |
| MA45362A (fr) * | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| HRP20241428T1 (hr) * | 2016-05-24 | 2025-01-03 | Sarepta Therapeutics, Inc. | Postupci za pripremu oligomera |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| SG10202101836TA (en) * | 2016-05-24 | 2021-03-30 | Sarepta Therapeutics Inc | Processes for preparing phosphorodiamidate morpholino oligomers |
| AU2017270598B2 (en) * | 2016-05-24 | 2022-12-01 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| BR112018074270B1 (pt) | 2016-05-24 | 2021-02-02 | Sarepta Therapeutics, Inc | processo para preparar compostos oligoméricos bem como os ditos compostos |
| EP3471779A4 (en) | 2016-06-16 | 2020-07-08 | Ionis Pharmaceuticals, Inc. | COMBINATIONS FOR MODULATING SMN EXPRESSION |
| KR102523527B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| CN116804031A (zh) * | 2016-09-20 | 2023-09-26 | 科罗拉多州立大学董事会法人团体 | 使用亚磷酰胺化学法合成主链修饰的吗啉代寡核苷酸和嵌合体 |
| JP7160807B2 (ja) | 2016-12-19 | 2022-10-25 | 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コンジュゲート |
| FI4122497T3 (fi) | 2016-12-19 | 2024-06-04 | Sarepta Therapeutics Inc | Eksonin ohittavia oligomeerikonjugaatteja lihasdystrofiaan |
| LT3554553T (lt) | 2016-12-19 | 2022-08-25 | Sarepta Therapeutics, Inc. | Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos |
| US12370261B2 (en) | 2018-03-16 | 2025-07-29 | Sarepta Therapeutics, Inc. | Chimeric peptides for antisense delivery |
| JP7576835B2 (ja) * | 2018-03-22 | 2024-11-01 | ボード・オブ・リージェンツ・オブ・ザ・ユニバーシティ・オブ・テキサス・システム | 自己免疫疾患および癌を治療するための可溶性インターロイキン７受容体（ｓＩＬ７Ｒ）調節療法 |
| 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 |
| WO2020123574A1 (en) | 2018-12-13 | 2020-06-18 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| WO2021025899A1 (en) | 2019-08-02 | 2021-02-11 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer pharmaceutical compositions |
| BR112022016238A2 (pt) * | 2020-02-28 | 2022-10-11 | Ionis Pharmaceuticals Inc | Compostos e métodos para modular smn2 |
| US12378267B2 (en) | 2021-06-17 | 2025-08-05 | Entrada Therapeutics, Inc. | Synthesis of FMOC-protected morpholino monomers and oligomers |
| CA3249036A1 (en) | 2022-04-22 | 2023-10-26 | Entrada Therapeutics, Inc. | CYCLIC PEPTIDES FOR ADMINISTERING THERAPEUTIC AGENTS |
| WO2024138018A1 (en) * | 2022-12-23 | 2024-06-27 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Treatment of gemin5-mediated disorders |
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| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| ATE124999T1 (de) | 1985-03-15 | 1995-07-15 | Antivirals Inc | Immunotestmittel für polynukleotid und verfahren. |
| 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 |
| JPH07501204A (ja) | 1991-06-28 | 1995-02-09 | マサチューセッツ インスティテュート オブ テクノロジー | 局所的オリゴヌクレオチド療法 |
| PT876165E (pt) | 1995-12-18 | 2006-10-31 | Angiotech Biomaterials Corp | Composicoes de polimeros reticulados e processos para a sua utilizacao |
| 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 |
| WO2001083740A2 (en) * | 2000-05-04 | 2001-11-08 | 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 |
| EP3470072A1 (en) | 2005-06-23 | 2019-04-17 | Biogen MA Inc. | Compositions and methods for modulation of smn2 splicing |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| CA2651881A1 (en) * | 2006-05-10 | 2008-03-27 | Avi Biopharma, Inc. | Oligonucleotide analogs having cationic intersubunit linkages |
| DK2049664T3 (da) | 2006-08-11 | 2012-01-02 | Prosensa Technologies Bv | Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| KR101617472B1 (ko) | 2007-11-15 | 2016-05-02 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 모르폴리노 올리고머의 합성 방법 |
| WO2010120820A1 (en) | 2009-04-13 | 2010-10-21 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing |
| JP5707396B2 (ja) | 2009-06-17 | 2015-04-30 | アイシス ファーマシューティカルズ， インコーポレーテッド | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| 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 |
| KR102182663B1 (ko) | 2010-05-28 | 2020-11-25 | 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 |
| CA2834128A1 (en) | 2011-05-05 | 2012-11-08 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| US9944926B2 (en) | 2011-11-30 | 2018-04-17 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
| US10066228B2 (en) | 2011-11-30 | 2018-09-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
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| ES2727481T3 (es) | 2019-10-16 |
| WO2013082551A1 (en) | 2013-06-06 |
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