JP2015512254A5 - - Google Patents
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- Publication number
- JP2015512254A5 JP2015512254A5 JP2015501712A JP2015501712A JP2015512254A5 JP 2015512254 A5 JP2015512254 A5 JP 2015512254A5 JP 2015501712 A JP2015501712 A JP 2015501712A JP 2015501712 A JP2015501712 A JP 2015501712A JP 2015512254 A5 JP2015512254 A5 JP 2015512254A5
- Authority
- JP
- Japan
- Prior art keywords
- oligonucleotide analogue
- analogue according
- motomeko
- oligonucleotide
- independently
- 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
- 108091034117 Oligonucleotide Proteins 0.000 claims description 46
- 125000000217 alkyl group Chemical group 0.000 claims description 14
- 125000000623 heterocyclic group Chemical group 0.000 claims description 10
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 10
- 125000003282 alkyl amino group Chemical group 0.000 claims description 8
- 125000001072 heteroaryl group Chemical group 0.000 claims description 8
- 239000000203 mixture Substances 0.000 claims description 8
- 125000003545 alkoxy group Chemical group 0.000 claims description 6
- 125000004103 aminoalkyl group Chemical group 0.000 claims description 6
- 125000003118 aryl group Chemical group 0.000 claims description 6
- -1 cyano, amidyl Chemical group 0.000 claims description 6
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims description 6
- RTZKZFJDLAIYFH-UHFFFAOYSA-N Diethyl ether Chemical compound CCOCC RTZKZFJDLAIYFH-UHFFFAOYSA-N 0.000 claims description 4
- GLUUGHFHXGJENI-UHFFFAOYSA-N Piperazine Chemical group C1CNCCN1 GLUUGHFHXGJENI-UHFFFAOYSA-N 0.000 claims description 4
- 125000004453 alkoxycarbonyl group Chemical group 0.000 claims description 4
- 125000004104 aryloxy group Chemical group 0.000 claims description 4
- ZADPBFCGQRWHPN-UHFFFAOYSA-N boronic acid Chemical compound OBO ZADPBFCGQRWHPN-UHFFFAOYSA-N 0.000 claims description 4
- 125000002915 carbonyl group Chemical group [*:2]C([*:1])=O 0.000 claims description 4
- 201000010099 disease Diseases 0.000 claims description 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 4
- 150000002148 esters Chemical group 0.000 claims description 4
- 125000001475 halogen functional group Chemical group 0.000 claims description 4
- 229910052739 hydrogen Inorganic materials 0.000 claims description 4
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 4
- 125000002768 hydroxyalkyl group Chemical group 0.000 claims description 4
- 208000018360 neuromuscular disease Diseases 0.000 claims description 4
- 125000000449 nitro group Chemical group [O-][N+](*)=O 0.000 claims description 4
- 150000003839 salts Chemical class 0.000 claims description 4
- KXDHJXZQYSOELW-UHFFFAOYSA-M Carbamate Chemical group NC([O-])=O KXDHJXZQYSOELW-UHFFFAOYSA-M 0.000 claims description 2
- BWGNESOTFCXPMA-UHFFFAOYSA-N Dihydrogen disulfide Chemical group SS BWGNESOTFCXPMA-UHFFFAOYSA-N 0.000 claims description 2
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 2
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims description 2
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 claims description 2
- UCKMPCXJQFINFW-UHFFFAOYSA-N Sulphide Chemical group [S-2] UCKMPCXJQFINFW-UHFFFAOYSA-N 0.000 claims description 2
- 125000004183 alkoxy alkyl group Chemical group 0.000 claims description 2
- 125000004457 alkyl amino carbonyl group Chemical group 0.000 claims description 2
- 125000004448 alkyl carbonyl group Chemical group 0.000 claims description 2
- 150000001408 amides Chemical group 0.000 claims description 2
- 125000005125 aryl alkyl amino carbonyl group Chemical group 0.000 claims description 2
- 125000001691 aryl alkyl amino group Chemical group 0.000 claims description 2
- 125000005099 aryl alkyl carbonyl group Chemical group 0.000 claims description 2
- 125000003710 aryl alkyl group Chemical group 0.000 claims description 2
- 125000005100 aryl amino carbonyl group Chemical group 0.000 claims description 2
- 125000001769 aryl amino group Chemical group 0.000 claims description 2
- 125000005129 aryl carbonyl group Chemical group 0.000 claims description 2
- 125000005161 aryl oxy carbonyl group Chemical group 0.000 claims description 2
- 125000004429 atom Chemical group 0.000 claims description 2
- 125000003917 carbamoyl group Chemical group [H]N([H])C(*)=O 0.000 claims description 2
- ANCLJVISBRWUTR-UHFFFAOYSA-N diaminophosphinic acid Chemical group NP(N)(O)=O ANCLJVISBRWUTR-UHFFFAOYSA-N 0.000 claims description 2
- 239000001257 hydrogen Substances 0.000 claims description 2
- 125000004356 hydroxy functional group Chemical group O* 0.000 claims description 2
- 150000004713 phosphodiesters Chemical group 0.000 claims description 2
- PTMHPRAIXMAOOB-UHFFFAOYSA-L phosphoramidate Chemical group NP([O-])([O-])=O PTMHPRAIXMAOOB-UHFFFAOYSA-L 0.000 claims description 2
- 229910052698 phosphorus Inorganic materials 0.000 claims description 2
- 239000011574 phosphorus Substances 0.000 claims description 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical group [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 2
- 238000000034 method Methods 0.000 description 5
- 150000001875 compounds Chemical class 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261613385P | 2012-03-20 | 2012-03-20 | |
| US61/613,385 | 2012-03-20 | ||
| PCT/US2013/029684 WO2013142087A1 (en) | 2012-03-20 | 2013-03-07 | Boronic acid conjugates of oligonucleotide analogues |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015512254A JP2015512254A (ja) | 2015-04-27 |
| JP2015512254A5 true JP2015512254A5 (enExample) | 2016-04-21 |
| JP6542662B2 JP6542662B2 (ja) | 2019-07-10 |
Family
ID=49223176
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015501712A Active JP6542662B2 (ja) | 2012-03-20 | 2013-03-07 | オリゴヌクレオチドアナログのボロン酸結合体 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US9920085B2 (enExample) |
| EP (1) | EP2828395B1 (enExample) |
| JP (1) | JP6542662B2 (enExample) |
| KR (1) | KR102079284B1 (enExample) |
| CN (1) | CN104411831B (enExample) |
| AU (3) | AU2013235691B2 (enExample) |
| BR (1) | BR112014023347A2 (enExample) |
| CA (1) | CA2868174A1 (enExample) |
| EA (1) | EA031110B1 (enExample) |
| ES (1) | ES2706198T3 (enExample) |
| IN (1) | IN2014DN08451A (enExample) |
| MX (1) | MX358497B (enExample) |
| NZ (1) | NZ700399A (enExample) |
| WO (1) | WO2013142087A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2627917T3 (es) | 2004-06-28 | 2017-08-01 | The University Of Western Australia | Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismos |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| HUE040445T2 (hu) | 2009-11-12 | 2019-03-28 | Univ Western Australia | Antiszensz molekulák és eljárások betegségek kezelésére |
| KR101981705B1 (ko) | 2010-05-28 | 2019-05-24 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| WO2012031243A2 (en) | 2010-09-03 | 2012-03-08 | Avi Biopharma, Inc. | dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS |
| KR20140097398A (ko) | 2011-11-18 | 2014-08-06 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 기능적으로-변형된 올리고뉴클레오티드 및 이의 서브유니트 |
| US9217148B2 (en) * | 2013-03-14 | 2015-12-22 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| EP3760720A1 (en) * | 2013-03-14 | 2021-01-06 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| HK1220154A1 (zh) | 2013-03-15 | 2017-04-28 | Sarepta Therapeutics, Inc. | 改进的用於治疗肌营养不良的组合物 |
| KR102644053B1 (ko) * | 2015-05-19 | 2024-03-06 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 펩티드 올리고뉴클레오티드 콘주게이트 |
| US11020417B2 (en) | 2015-06-04 | 2021-06-01 | Sarepta Therapeutics, Inc | Methods and compounds for treatment of lymphocyte-related diseases and conditions |
| WO2017040271A1 (en) * | 2015-08-28 | 2017-03-09 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
| AU2016335032B2 (en) * | 2015-10-05 | 2022-04-14 | Proqr Therapeutics Ii B.V. | Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion |
Family Cites Families (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | 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 |
| ATE312834T1 (de) | 1989-12-20 | 2005-12-15 | Avi Biopharma Inc | Ungeladene, auf morpholin basierende polymere mit chiralen, phosphor enthaltenden brücken zwischen den untereinheiten |
| AU659482B2 (en) | 1991-06-28 | 1995-05-18 | Massachusetts Institute Of Technology | Localized oligonucleotide therapy |
| US6030954A (en) | 1991-09-05 | 2000-02-29 | University Of Connecticut | Targeted delivery of poly- or oligonucleotides to cells |
| US6245747B1 (en) | 1996-03-12 | 2001-06-12 | The Board Of Regents Of The University Of Nebraska | Targeted site specific antisense oligodeoxynucleotide delivery method |
| AU729643B2 (en) | 1996-05-01 | 2001-02-08 | Antivirals Inc. | Polypeptide conjugates for transporting substances across cell membranes |
| DE19716073A1 (de) | 1997-04-17 | 1998-10-22 | Boehringer Mannheim Gmbh | Dosiervorrichtung zur Abgabe kleiner Flüssigkeitsmengen |
| US6548651B1 (en) | 1998-11-11 | 2003-04-15 | Pantheco A/S | Modified peptide nucleic acid (PNA) molecules |
| CN1354752A (zh) | 1999-03-19 | 2002-06-19 | 坡林克斯股份有限公司 | 含硼酸的试剂和寡核苷酸 |
| WO2001049775A2 (en) | 2000-01-04 | 2001-07-12 | Avi Biopharma, Inc. | Antisense antibacterial cell division composition and method |
| ATE356231T1 (de) | 2000-01-11 | 2007-03-15 | Nanogen Recognomics Gmbh | Biomoleküle mit multiplen anbindungsabschnitten zur bindung an eine substratoberfläche |
| US7833992B2 (en) | 2001-05-18 | 2010-11-16 | Merck Sharpe & Dohme | Conjugates and compositions for cellular delivery |
| AU2001248281A1 (en) | 2000-04-06 | 2001-10-23 | Pantheco A/S | Pharmaceutical composition of modified pna molecules |
| US7939087B2 (en) | 2000-10-27 | 2011-05-10 | Novartis Vaccines And Diagnostics, Inc. | Nucleic acids and proteins from Streptococcus groups A & B |
| WO2002079467A2 (en) | 2001-03-29 | 2002-10-10 | Københavns Universitet | Antibiotic-free bacterial strain selection with antisense molecules |
| US20030125241A1 (en) | 2001-05-18 | 2003-07-03 | Margit Wissenbach | Therapeutic uses of LNA-modified oligonucleotides in infectious diseases |
| WO2003078583A2 (en) | 2002-03-09 | 2003-09-25 | Maxygen, Inc. | Optimization of crossover points for directed evolution |
| WO2004041854A2 (en) | 2002-11-05 | 2004-05-21 | Affinium Pharmaceuticals, Inc. | Essential novel bacterial polypeptides |
| AU2004235396B2 (en) | 2003-04-29 | 2010-11-25 | Sarepta Therapeutics, Inc. | Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells |
| CA2526579A1 (en) | 2003-05-23 | 2004-12-02 | Epfl-Ecole Polytechnique Federale De Lausanne | Methods for protein labeling based on acyl carrier protein |
| US7402574B2 (en) | 2004-03-12 | 2008-07-22 | Avi Biopharma, Inc. | Antisense composition and method for treating cancer |
| ES2627917T3 (es) | 2004-06-28 | 2017-08-01 | The University Of Western Australia | Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismos |
| AU2005327188C9 (en) | 2004-07-02 | 2011-09-22 | Avi Biopharma, Inc. | Antisense antibacterial method and compound |
| US8129352B2 (en) | 2004-09-16 | 2012-03-06 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating ssRNA viral infection |
| GB0501944D0 (en) * | 2005-01-31 | 2005-03-09 | Univ Cambridge Tech | Compounds for use in chemical detection and/or quantitation |
| WO2006086667A2 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
| US20060293268A1 (en) | 2005-05-05 | 2006-12-28 | Rieder Aida E | Antisense antiviral compounds and methods for treating foot and mouth disease |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| US7790694B2 (en) | 2005-07-13 | 2010-09-07 | Avi Biopharma Inc. | Antisense antibacterial method and compound |
| 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 |
| EP2066812A4 (en) | 2006-09-21 | 2010-04-28 | Univ Rochester | COMPOSITIONS AND METHODS RELATED TO PROTEIN SHIFT THERAPY FOR MYOTONE DYSTROPHY |
| 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. |
| 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 |
| WO2010019847A2 (en) | 2008-08-15 | 2010-02-18 | Georgia State University Research Foundation, Inc. | Aptamer inhibition of thrombus formation |
| EP3133160B1 (en) | 2008-10-24 | 2018-12-12 | Sarepta Therapeutics, Inc. | Exon skipping compositions for dmd |
| US20110269665A1 (en) | 2009-06-26 | 2011-11-03 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| KR101981705B1 (ko) | 2010-05-28 | 2019-05-24 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| CN103154009B (zh) | 2010-09-30 | 2015-06-10 | 日本新药株式会社 | 吗啉基核酸衍生物 |
| US9061960B2 (en) | 2012-05-30 | 2015-06-23 | Basf Se | Method for working up mixtures |
-
2013
- 2013-03-07 US US14/386,720 patent/US9920085B2/en active Active
- 2013-03-07 CN CN201380023605.6A patent/CN104411831B/zh not_active Expired - Fee Related
- 2013-03-07 ES ES13765016T patent/ES2706198T3/es active Active
- 2013-03-07 WO PCT/US2013/029684 patent/WO2013142087A1/en not_active Ceased
- 2013-03-07 NZ NZ700399A patent/NZ700399A/en not_active IP Right Cessation
- 2013-03-07 EP EP13765016.4A patent/EP2828395B1/en active Active
- 2013-03-07 BR BR112014023347A patent/BR112014023347A2/pt active Search and Examination
- 2013-03-07 EA EA201491726A patent/EA031110B1/ru unknown
- 2013-03-07 MX MX2014011276A patent/MX358497B/es active IP Right Grant
- 2013-03-07 JP JP2015501712A patent/JP6542662B2/ja active Active
- 2013-03-07 CA CA2868174A patent/CA2868174A1/en not_active Abandoned
- 2013-03-07 KR KR1020147029235A patent/KR102079284B1/ko not_active Expired - Fee Related
- 2013-03-07 AU AU2013235691A patent/AU2013235691B2/en not_active Ceased
-
2014
- 2014-10-09 IN IN8451DEN2014 patent/IN2014DN08451A/en unknown
-
2017
- 2017-05-01 AU AU2017202883A patent/AU2017202883A1/en not_active Abandoned
-
2019
- 2019-03-22 AU AU2019201989A patent/AU2019201989A1/en not_active Abandoned
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