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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- 125000001981 tert-butyldimethylsilyl group Chemical group [H]C([H])([H])[Si]([H])(C([H])([H])[H])[*]C(C([H])([H])[H])(C([H])([H])[H])C([H])([H])[H] 0.000 description 1
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Classifications
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- C—CHEMISTRY; METALLURGY
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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
-
- 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
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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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- 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
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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
- 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
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- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
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- Wood Science & Technology (AREA)
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- General Engineering & Computer Science (AREA)
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- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
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- Veterinary Medicine (AREA)
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- Orthopedic Medicine & Surgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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- Medicinal Preparation (AREA)
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Applications Claiming Priority (3)
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|---|---|---|---|
| US201161565499P | 2011-11-30 | 2011-11-30 | |
| US61/565,499 | 2011-11-30 | ||
| PCT/US2012/067475 WO2013082551A1 (en) | 2011-11-30 | 2012-11-30 | Induced exon inclusion in spinal muscle atrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
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| JP2018232429A Division JP2019059769A (ja) | 2011-11-30 | 2018-12-12 | 脊髄性筋萎縮症における誘発されたエクソン包含 |
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| JP2015504650A JP2015504650A (ja) | 2015-02-16 |
| JP2015504650A5 JP2015504650A5 (https=) | 2016-01-21 |
| JP6496549B2 true JP6496549B2 (ja) | 2019-04-03 |
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| JP2018232429A Pending JP2019059769A (ja) | 2011-11-30 | 2018-12-12 | 脊髄性筋萎縮症における誘発されたエクソン包含 |
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| US (2) | US9944926B2 (https=) |
| EP (1) | EP2785840B8 (https=) |
| JP (2) | JP6496549B2 (https=) |
| AU (1) | AU2012345638C1 (https=) |
| CA (1) | CA2857664A1 (https=) |
| ES (1) | ES2727481T3 (https=) |
| WO (1) | WO2013082551A1 (https=) |
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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 |
| JP6496549B2 (ja) | 2011-11-30 | 2019-04-03 | 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誘発されたエクソン包含 |
| US10066228B2 (en) | 2011-11-30 | 2018-09-04 | 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 |
| US11236338B2 (en) * | 2013-09-05 | 2022-02-01 | Sarepta Therapeutics, Inc. | 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 |
| US10905709B2 (en) | 2015-08-28 | 2021-02-02 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
| 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 |
| WO2017184529A1 (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 |
| BR112018074346B1 (pt) * | 2016-05-24 | 2023-01-03 | Sarepta Therapeutics, Inc. | Composto oligomérico e processo para preparar um composto oligomérico |
| CA3024456A1 (en) * | 2016-05-24 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| US10947533B2 (en) | 2016-05-24 | 2021-03-16 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| AU2017278699A1 (en) | 2016-05-24 | 2018-11-15 | Sarepta Therapeutics, Inc. | Pharmaceutical composition comprising Eteplirsen |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| MD3464306T2 (ro) * | 2016-05-24 | 2024-08-31 | Sarepta Therapeutics Inc | Procedee de preparare a oligomerilor morfolino fosforodiamidați |
| US11198867B2 (en) | 2016-06-16 | 2021-12-14 | Ionis Pharmaceuticals, Inc. | Combinations for the modulation of SMN expression |
| KR102523527B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| KR102523522B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| 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 |
| KR102810425B1 (ko) | 2016-12-19 | 2025-05-21 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| KR20240006023A (ko) | 2016-12-19 | 2024-01-12 | 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 |
| WO2019060862A1 (en) | 2017-09-25 | 2019-03-28 | Sarepta Therapeutics, Inc. | METHODS FOR PREPARING MORPHOLINO PHOSPHORODIAMIDATE OLIGOMERS VIA FAST FLOW SYNTHESIS |
| EP3765030A4 (en) * | 2018-03-16 | 2022-01-26 | Sarepta Therapeutics, Inc. | CHIMERIC PEPTIDES FOR ANTISENSE ADMINISTRATION |
| 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 |
| EP3806868A4 (en) | 2018-06-13 | 2022-06-22 | 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 |
| IL318474A (en) | 2018-12-13 | 2025-03-01 | Sarepta Therapeutics Inc | Axon-skipping oligomer complexes for muscular dystrophy |
| WO2021025899A1 (en) | 2019-08-02 | 2021-02-11 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer pharmaceutical compositions |
| CN120505310A (zh) * | 2020-02-28 | 2025-08-19 | Ionis制药公司 | 用于调节smn2的化合物和方法 |
| US12378267B2 (en) | 2021-06-17 | 2025-08-05 | Entrada Therapeutics, Inc. | Synthesis of FMOC-protected morpholino monomers and oligomers |
| CN119156395A (zh) | 2022-04-22 | 2024-12-17 | 恩特拉达治疗学股份有限公司 | 用于递送治疗剂的环肽 |
| 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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| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| WO1986005519A1 (en) | 1985-03-15 | 1986-09-25 | James Summerton | Polynucleotide assay reagent and method |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| 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 |
| US6784291B2 (en) * | 2000-05-04 | 2004-08-31 | 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 |
| PL2735568T3 (pl) | 2006-05-10 | 2018-02-28 | Sarepta Therapeutics, Inc. | Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami |
| SI2049664T1 (sl) | 2006-08-11 | 2012-04-30 | Prosensa Technologies Bv | Enojna veriga oligonukleotidov, komplementarna repetitivnim elementom, za zdravljenje genetskih bolezni, povezanih z nestabilnostjo dnk ponovitev |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| RU2606627C2 (ru) | 2007-11-15 | 2017-01-10 | Серепта Терапьютикс,Инк. | Способ синтеза морфолиновых олигомеров |
| WO2010120820A1 (en) | 2009-04-13 | 2010-10-21 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing |
| ES2699827T3 (es) | 2009-06-17 | 2019-02-13 | Biogen Ma Inc | Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto |
| 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 |
| KR20200133284A (ko) | 2010-05-28 | 2020-11-26 | 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 |
| CA3092114A1 (en) | 2011-05-05 | 2012-11-08 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| JP6496549B2 (ja) | 2011-11-30 | 2019-04-03 | 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誘発されたエクソン包含 |
| US10066228B2 (en) | 2011-11-30 | 2018-09-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
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- 2012-11-30 CA CA2857664A patent/CA2857664A1/en not_active Abandoned
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| US20180273954A1 (en) | 2018-09-27 |
| AU2012345638A1 (en) | 2014-07-24 |
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| US9944926B2 (en) | 2018-04-17 |
| US10577605B2 (en) | 2020-03-03 |
| WO2013082551A1 (en) | 2013-06-06 |
| US20140329772A1 (en) | 2014-11-06 |
| JP2015504650A (ja) | 2015-02-16 |
| EP2785840A1 (en) | 2014-10-08 |
| CA2857664A1 (en) | 2013-06-06 |
| ES2727481T3 (es) | 2019-10-16 |
| EP2785840B8 (en) | 2019-05-22 |
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