BR112014023347A2 - conjugados de ácido borônico de análogos oligonucleotídeos - Google Patents
conjugados de ácido borônico de análogos oligonucleotídeosInfo
- Publication number
- BR112014023347A2 BR112014023347A2 BR112014023347A BR112014023347A BR112014023347A2 BR 112014023347 A2 BR112014023347 A2 BR 112014023347A2 BR 112014023347 A BR112014023347 A BR 112014023347A BR 112014023347 A BR112014023347 A BR 112014023347A BR 112014023347 A2 BR112014023347 A2 BR 112014023347A2
- Authority
- BR
- Brazil
- Prior art keywords
- boronic acid
- acid conjugates
- oligonucleotide analogs
- oligonucleotide analogues
- boronic
- Prior art date
Links
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 3
- ZADPBFCGQRWHPN-UHFFFAOYSA-N boronic acid Chemical compound OBO ZADPBFCGQRWHPN-UHFFFAOYSA-N 0.000 title abstract 3
- 230000001594 aberrant effect Effects 0.000 abstract 1
- 230000009286 beneficial effect Effects 0.000 abstract 1
- 150000001875 compounds Chemical class 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 150000002148 esters Chemical group 0.000 abstract 1
- 230000005764 inhibitory process Effects 0.000 abstract 1
- 108020004999 messenger RNA Proteins 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
- C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
- C07F9/02—Phosphorus compounds
- C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
- C07F9/6596—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having atoms other than oxygen, sulfur, selenium, tellurium, nitrogen or phosphorus as ring hetero atoms
-
- 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
-
- 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
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
- C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
- C07F9/02—Phosphorus compounds
- C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
- C07F9/6558—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system
- C07F9/65583—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system each of the hetero rings containing nitrogen as ring hetero atom
-
- 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
- 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
-
- 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
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/331—Universal or degenerate base
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Veterinary Medicine (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurosurgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261613385P | 2012-03-20 | 2012-03-20 | |
| PCT/US2013/029684 WO2013142087A1 (en) | 2012-03-20 | 2013-03-07 | Boronic acid conjugates of oligonucleotide analogues |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112014023347A2 true BR112014023347A2 (pt) | 2017-07-18 |
Family
ID=49223176
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112014023347A BR112014023347A2 (pt) | 2012-03-20 | 2013-03-07 | conjugados de ácido borônico de análogos oligonucleotídeos |
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 |
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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 |
| 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 |
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| WO2004041854A2 (en) | 2002-11-05 | 2004-05-21 | Affinium Pharmaceuticals, Inc. | Essential novel bacterial polypeptides |
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| 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 |
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| 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
Also Published As
| Publication number | Publication date |
|---|---|
| MX2014011276A (es) | 2014-10-06 |
| EP2828395A1 (en) | 2015-01-28 |
| KR20140138995A (ko) | 2014-12-04 |
| US9920085B2 (en) | 2018-03-20 |
| ES2706198T3 (es) | 2019-03-27 |
| JP2015512254A (ja) | 2015-04-27 |
| US20150080340A1 (en) | 2015-03-19 |
| CN104411831A (zh) | 2015-03-11 |
| KR102079284B1 (ko) | 2020-02-19 |
| JP6542662B2 (ja) | 2019-07-10 |
| AU2013235691B2 (en) | 2017-02-02 |
| MX358497B (es) | 2018-08-23 |
| WO2013142087A1 (en) | 2013-09-26 |
| EA201491726A1 (ru) | 2015-03-31 |
| AU2017202883A1 (en) | 2017-05-18 |
| IN2014DN08451A (https=) | 2015-05-08 |
| HK1206064A1 (en) | 2015-12-31 |
| CA2868174A1 (en) | 2013-09-26 |
| EP2828395B1 (en) | 2018-10-24 |
| EA031110B1 (ru) | 2018-11-30 |
| AU2019201989A1 (en) | 2019-04-11 |
| CN104411831B (zh) | 2020-08-11 |
| EP2828395A4 (en) | 2015-11-18 |
| NZ700399A (en) | 2016-07-29 |
| AU2013235691A1 (en) | 2014-10-16 |
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