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 (https=) | 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 (https=) |
| EP (1) | EP2828395B1 (https=) |
| JP (1) | JP6542662B2 (https=) |
| KR (1) | KR102079284B1 (https=) |
| CN (1) | CN104411831B (https=) |
| AU (3) | AU2013235691B2 (https=) |
| BR (1) | BR112014023347A2 (https=) |
| CA (1) | CA2868174A1 (https=) |
| EA (1) | EA031110B1 (https=) |
| ES (1) | ES2706198T3 (https=) |
| IN (1) | IN2014DN08451A (https=) |
| MX (1) | MX358497B (https=) |
| NZ (1) | NZ700399A (https=) |
| WO (1) | WO2013142087A1 (https=) |
Families Citing this family (13)
| 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 |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| 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 |
| US10017763B2 (en) | 2010-09-03 | 2018-07-10 | Sarepta Therapeutics, Inc. | dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups |
| HK1201514A1 (en) | 2011-11-18 | 2015-09-04 | Sarepta Therapeutics, Inc. | Functionally-modified oligonucleotides and subunits thereof |
| 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 |
| AU2016264471B2 (en) * | 2015-05-19 | 2022-01-27 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| US11020417B2 (en) | 2015-06-04 | 2021-06-01 | Sarepta Therapeutics, Inc | Methods and compounds for treatment of lymphocyte-related diseases and conditions |
| US10905709B2 (en) | 2015-08-28 | 2021-02-02 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
| EP3359667A1 (en) * | 2015-10-05 | 2018-08-15 | 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 |
|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| AU655164B2 (en) | 1989-12-20 | 1994-12-08 | Antivirals Inc. | Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages |
| 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 |
| ATE301664T1 (de) | 1999-03-19 | 2005-08-15 | Agilent Technologies Inc | Boronsäurehaltige reagenzien und oligonukleotide |
| AU781676B2 (en) | 2000-01-04 | 2005-06-02 | Avi Biopharma, Inc. | Antisense antibacterial cell division composition and method |
| CA2397091A1 (en) | 2000-01-11 | 2001-07-19 | Markus Schweitzer | Biomolecules having multiple attachment moieties for binding to a substrate surface |
| US7833992B2 (en) | 2001-05-18 | 2010-11-16 | Merck Sharpe & Dohme | Conjugates and compositions for cellular delivery |
| WO2001076636A2 (en) | 2000-04-06 | 2001-10-18 | Pantheco A/S | Pharmaceutical composition of modified pna molecules |
| CA2881568C (en) | 2000-10-27 | 2019-09-24 | 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 |
| AU2002317437A1 (en) | 2001-05-18 | 2002-12-03 | Cureon A/S | Therapeutic uses of lna-modified oligonucleotides in infectious diseases |
| EP1488335A4 (en) | 2002-03-09 | 2006-11-15 | Maxygen Inc | OPTIMIZING CROSSOVER POINTS FOR THE ADJUSTED EVOLUTION |
| WO2004041854A2 (en) | 2002-11-05 | 2004-05-21 | Affinium Pharmaceuticals, Inc. | Essential novel bacterial polypeptides |
| US7468418B2 (en) | 2003-04-29 | 2008-12-23 | Avi Biopharma., Inc. | Compositions for enhancing transport of molecules into cells |
| JP2007509608A (ja) | 2003-05-23 | 2007-04-19 | ウペエフエル・エコル・ポリテクニック・フェデラル・ドゥ・ローザンヌ | アシル担体タンパク質に基づくタンパク質をラベルするための方法 |
| US7402574B2 (en) | 2004-03-12 | 2008-07-22 | Avi Biopharma, Inc. | Antisense composition and method for treating cancer |
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP2351839A3 (en) | 2004-07-02 | 2011-10-05 | 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 |
| EP1855694B1 (en) | 2005-02-09 | 2020-12-02 | Sarepta Therapeutics, Inc. | Antisense composition 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 |
| US7790694B2 (en) | 2005-07-13 | 2010-09-07 | Avi Biopharma Inc. | Antisense antibacterial method and compound |
| 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 |
| EP2560001B1 (en) | 2006-09-21 | 2016-04-13 | University of Rochester | Compositions and methods related to protein displacement therapy for myotonic distrophy |
| 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 |
| 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 |
| 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 |
| US20110269665A1 (en) | 2009-06-26 | 2011-11-03 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| KR20200133284A (ko) | 2010-05-28 | 2020-11-26 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| ES2607603T3 (es) | 2010-09-30 | 2017-04-03 | Nippon Shinyaku Co., Ltd. | Derivado de ácido morfolino nucleico |
| US9061960B2 (en) | 2012-05-30 | 2015-06-23 | Basf Se | Method for working up mixtures |
-
2013
- 2013-03-07 CN CN201380023605.6A patent/CN104411831B/zh not_active Expired - Fee Related
- 2013-03-07 CA CA2868174A patent/CA2868174A1/en not_active Abandoned
- 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 KR KR1020147029235A patent/KR102079284B1/ko not_active Expired - Fee Related
- 2013-03-07 AU AU2013235691A patent/AU2013235691B2/en not_active Ceased
- 2013-03-07 US US14/386,720 patent/US9920085B2/en active Active
- 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 JP JP2015501712A patent/JP6542662B2/ja active Active
- 2013-03-07 NZ NZ700399A patent/NZ700399A/en not_active IP Right Cessation
- 2013-03-07 EA EA201491726A patent/EA031110B1/ru unknown
- 2013-03-07 MX MX2014011276A patent/MX358497B/es active IP Right Grant
-
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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