JP2017522044A5 - - Google Patents
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- Publication number
- JP2017522044A5 JP2017522044A5 JP2017512874A JP2017512874A JP2017522044A5 JP 2017522044 A5 JP2017522044 A5 JP 2017522044A5 JP 2017512874 A JP2017512874 A JP 2017512874A JP 2017512874 A JP2017512874 A JP 2017512874A JP 2017522044 A5 JP2017522044 A5 JP 2017522044A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- antisense morpholino
- morpholino oligomer
- formula
- cpp
- 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
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 59
- 230000000692 anti-sense effect Effects 0.000 claims description 58
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims description 30
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims description 30
- 230000008685 targeting Effects 0.000 claims description 24
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims description 22
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims description 22
- 125000000217 alkyl group Chemical group 0.000 claims description 19
- 229940113082 thymine Drugs 0.000 claims description 15
- 229940035893 uracil Drugs 0.000 claims description 15
- 125000003236 benzoyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C(*)=O 0.000 claims description 14
- 125000003696 stearoyl group Chemical group O=C([*])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 claims description 14
- -1 4-methoxytrityl Chemical group 0.000 claims description 12
- 230000001580 bacterial effect Effects 0.000 claims description 12
- 229910052799 carbon Inorganic materials 0.000 claims description 12
- 108020004999 messenger RNA Proteins 0.000 claims description 12
- 125000002221 trityl group Chemical group [H]C1=C([H])C([H])=C([H])C([H])=C1C([*])(C1=C(C(=C(C(=C1[H])[H])[H])[H])[H])C1=C([H])C([H])=C([H])C([H])=C1[H] 0.000 claims description 12
- 230000007923 virulence factor Effects 0.000 claims description 11
- 239000000304 virulence factor Substances 0.000 claims description 11
- 125000002252 acyl group Chemical group 0.000 claims description 10
- 239000002773 nucleotide Substances 0.000 claims description 10
- 125000003729 nucleotide group Chemical group 0.000 claims description 10
- 239000008194 pharmaceutical composition Substances 0.000 claims description 10
- 241000894006 Bacteria Species 0.000 claims description 9
- 125000002777 acetyl group Chemical group [H]C([H])([H])C(*)=O 0.000 claims description 9
- 125000005647 linker group Chemical group 0.000 claims description 8
- 150000003839 salts Chemical class 0.000 claims description 6
- 239000003795 chemical substances by application Substances 0.000 claims description 5
- MHCMWGPPLOSCJY-UHFFFAOYSA-N 4-$l^{1}-azanylmorpholine Chemical compound [N]N1CCOCC1 MHCMWGPPLOSCJY-UHFFFAOYSA-N 0.000 claims description 4
- OKTJSMMVPCPJKN-UHFFFAOYSA-N Carbon Chemical compound [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 claims description 4
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 claims description 4
- 210000004899 c-terminal region Anatomy 0.000 claims description 4
- 230000000295 complement effect Effects 0.000 claims description 4
- 230000001717 pathogenic effect Effects 0.000 claims description 4
- 229910052698 phosphorus Inorganic materials 0.000 claims description 4
- 239000011574 phosphorus Substances 0.000 claims description 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 4
- 239000003937 drug carrier Substances 0.000 claims description 3
- 230000000694 effects Effects 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 1
- 238000000034 method Methods 0.000 description 10
- 108090000623 proteins and genes Proteins 0.000 description 7
- 102000004169 proteins and genes Human genes 0.000 description 7
- 239000012634 fragment Substances 0.000 description 6
- 108091081024 Start codon Proteins 0.000 description 4
- 230000014621 translational initiation Effects 0.000 description 4
- 101710146995 Acyl carrier protein Proteins 0.000 description 3
- 241000589513 Burkholderia cepacia Species 0.000 description 3
- 108010034752 beta-lactamase NDM-1 Proteins 0.000 description 3
- 241000894007 species Species 0.000 description 3
- 241000588626 Acinetobacter baumannii Species 0.000 description 2
- 241000588724 Escherichia coli Species 0.000 description 2
- 241000588747 Klebsiella pneumoniae Species 0.000 description 2
- 230000003115 biocidal effect Effects 0.000 description 2
- 230000015572 biosynthetic process Effects 0.000 description 2
- 235000014113 dietary fatty acids Nutrition 0.000 description 2
- 229930195729 fatty acid Natural products 0.000 description 2
- 239000000194 fatty acid Substances 0.000 description 2
- 150000004665 fatty acids Chemical class 0.000 description 2
- 238000011144 upstream manufacturing Methods 0.000 description 2
- 101150004974 ACP3 gene Proteins 0.000 description 1
- 241000589291 Acinetobacter Species 0.000 description 1
- 101100000756 Bacillus subtilis (strain 168) acpA gene Proteins 0.000 description 1
- 241001453380 Burkholderia Species 0.000 description 1
- 241000588722 Escherichia Species 0.000 description 1
- 241000588748 Klebsiella Species 0.000 description 1
- 108700043533 SuhB Proteins 0.000 description 1
- 101150023061 acpP gene Proteins 0.000 description 1
- 101150051130 acpP1 gene Proteins 0.000 description 1
- 239000003242 anti bacterial agent Substances 0.000 description 1
- 239000004599 antimicrobial Substances 0.000 description 1
- 239000000126 substance Substances 0.000 description 1
- 101150032811 suhB gene Proteins 0.000 description 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461994750P | 2014-05-16 | 2014-05-16 | |
| US61/994,750 | 2014-05-16 | ||
| US201462099046P | 2014-12-31 | 2014-12-31 | |
| US62/099,046 | 2014-12-31 | ||
| US201562129746P | 2015-03-06 | 2015-03-06 | |
| US62/129,746 | 2015-03-06 | ||
| PCT/US2015/031150 WO2015175977A2 (en) | 2014-05-16 | 2015-05-15 | Antisense antibacterial compounds and methods |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017522044A JP2017522044A (ja) | 2017-08-10 |
| JP2017522044A5 true JP2017522044A5 (enExample) | 2018-06-28 |
| JP6901966B2 JP6901966B2 (ja) | 2021-07-14 |
Family
ID=54480938
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017512874A Active JP6901966B2 (ja) | 2014-05-16 | 2015-05-15 | アンチセンス抗菌化合物および方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US9790495B2 (enExample) |
| EP (2) | EP3620178A3 (enExample) |
| JP (1) | JP6901966B2 (enExample) |
| AU (1) | AU2015258895A1 (enExample) |
| CA (1) | CA2948373A1 (enExample) |
| WO (1) | WO2015175977A2 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2015258895A1 (en) | 2014-05-16 | 2016-11-24 | Board Of Regents, The University Of Texas System | Antisense antibacterial compounds and methods |
| AU2015264449B2 (en) | 2014-05-19 | 2022-05-05 | Board Of Regents, The University Of Texas System | Antisense antibacterial compounds and methods |
| WO2016108930A2 (en) | 2014-12-31 | 2016-07-07 | Geller Bruce L | Antisense antibacterial compounds and methods |
| MX2017011004A (es) * | 2015-02-27 | 2018-02-09 | Sarepta Therapeutics Inc | Inclusion del exon2 inducida por antisentido en alfa-glucosidasa.acida. |
| EP3394261A4 (en) * | 2015-12-23 | 2019-11-27 | Oregon State University | ANTIBACTERIAL ANTISENSE COMPOUNDS AND METHOD |
| EP3394262B1 (en) | 2015-12-23 | 2024-08-28 | Oregon State University | Antisense antibacterial compounds and methods |
| KR102522059B1 (ko) | 2016-04-18 | 2023-04-14 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 안티센스 올리고머, 및 산성 알파-글루코시다제 유전자와 연관된 질환을 치료하기 위한 이의 사용 방법 |
| EA201892467A1 (ru) * | 2016-04-29 | 2019-05-31 | Сарепта Терапьютикс, Инк. | Олигонуклеотидные аналоги, нацеленные на lmna человека |
| WO2018161027A1 (en) * | 2017-03-03 | 2018-09-07 | Oregon State University | Antisense antibacterial compounds and methods |
| US10758629B2 (en) * | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates 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 |
| GB2579253A (en) | 2018-11-28 | 2020-06-17 | Pedanius Therapeutics Ltd | Antibacterial antisense agents |
Family Cites Families (42)
| 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 |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| 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 |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| EP0215942B1 (en) | 1985-03-15 | 1995-07-12 | Antivirals Inc. | Polynucleotide assay reagent and method |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| AU659482B2 (en) | 1991-06-28 | 1995-05-18 | Massachusetts Institute Of Technology | Localized oligonucleotide therapy |
| 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 |
| US20060241075A1 (en) * | 2001-05-18 | 2006-10-26 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of desmoglein gene expression using short interfering nucleic acid (siNA) |
| US20040029129A1 (en) * | 2001-10-25 | 2004-02-12 | Liangsu Wang | Identification of essential genes in microorganisms |
| US6965025B2 (en) | 2001-12-10 | 2005-11-15 | Isis Pharmaceuticals, Inc. | Antisense modulation of connective tissue growth factor expression |
| WO2004093788A2 (en) * | 2003-04-17 | 2004-11-04 | The Trustees Of Columbia University In The City Ofnew York | Desmoglein 4 is a novel gene involved in hair growth |
| US7468418B2 (en) | 2003-04-29 | 2008-12-23 | Avi Biopharma., Inc. | Compositions for enhancing transport of molecules into cells |
| US20050288246A1 (en) * | 2004-05-24 | 2005-12-29 | Iversen Patrick L | Peptide conjugated, inosine-substituted antisense oligomer compound and method |
| AU2005327188C8 (en) | 2004-07-02 | 2011-09-22 | Avi Biopharma, Inc. | Antisense antibacterial method and compound |
| US20080125583A1 (en) * | 2005-02-11 | 2008-05-29 | International Business Machines Corporation | Ribonucleic acid interference molecules |
| US7790694B2 (en) | 2005-07-13 | 2010-09-07 | Avi Biopharma Inc. | Antisense antibacterial method and compound |
| AU2006267051B2 (en) | 2005-07-13 | 2013-03-07 | Sarepta Therapeutics, Inc. | Antisense antibacterial method and compound |
| US8067571B2 (en) * | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| US8143389B2 (en) * | 2005-08-25 | 2012-03-27 | Texas Tech University | Inhibiton of metallo-β-lactamase by double-stranded DNA |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| ES2694726T3 (es) | 2007-06-29 | 2018-12-26 | Sarepta Therapeutics, Inc. | Conjugados peptídicos específicos de tejido y métodos |
| US8299206B2 (en) | 2007-11-15 | 2012-10-30 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| HRP20140646T1 (hr) | 2007-11-15 | 2014-09-26 | Sarepta Therapeutics, Inc. | Postupak sinteze morfolino oligomera |
| US8076476B2 (en) | 2007-11-15 | 2011-12-13 | Avi Biopharma, Inc. | Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits |
| US20110269665A1 (en) | 2009-06-26 | 2011-11-03 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| US9914745B2 (en) | 2009-08-14 | 2018-03-13 | Indian Association For The Cultivation Of Science | Morpholino-based antisense agent |
| WO2012031243A2 (en) | 2010-09-03 | 2012-03-08 | Avi Biopharma, Inc. | dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS |
| PT2623507T (pt) | 2010-09-30 | 2016-12-23 | Nippon Shinyaku Co Ltd | Derivado de ácido morfolino-nucleico |
| EP3067420A1 (en) | 2010-11-12 | 2016-09-14 | Sarepta Therapeutics, Inc. | Antisense antibacterial compounds and methods |
| US20120213663A1 (en) | 2011-02-23 | 2012-08-23 | King Fahd University Of Petroleum And Minerals | Method of removing e. coli bacteria from an aqueous solution |
| US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| KR102183273B1 (ko) | 2011-05-05 | 2020-11-27 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 펩타이드 올리고뉴클레오타이드 접합체 |
| KR101317263B1 (ko) | 2011-07-05 | 2013-10-10 | (주)지노첵 | 리얼타임 pcr을 이용한 카바페넴 내성 장내균 검사 방법 및 키트 |
| WO2013011072A1 (en) * | 2011-07-20 | 2013-01-24 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Carbapenemase and antibacterial treatment |
| AU2015258895A1 (en) | 2014-05-16 | 2016-11-24 | Board Of Regents, The University Of Texas System | Antisense antibacterial compounds and methods |
| AU2015264449B2 (en) | 2014-05-19 | 2022-05-05 | Board Of Regents, The University Of Texas System | Antisense antibacterial compounds and methods |
| WO2016108930A2 (en) | 2014-12-31 | 2016-07-07 | Geller Bruce L | Antisense antibacterial compounds and methods |
| EP3394261A4 (en) | 2015-12-23 | 2019-11-27 | Oregon State University | ANTIBACTERIAL ANTISENSE COMPOUNDS AND METHOD |
| EP3394262B1 (en) | 2015-12-23 | 2024-08-28 | Oregon State University | Antisense antibacterial compounds and methods |
-
2015
- 2015-05-15 AU AU2015258895A patent/AU2015258895A1/en not_active Abandoned
- 2015-05-15 JP JP2017512874A patent/JP6901966B2/ja active Active
- 2015-05-15 CA CA2948373A patent/CA2948373A1/en active Pending
- 2015-05-15 WO PCT/US2015/031150 patent/WO2015175977A2/en not_active Ceased
- 2015-05-15 EP EP19198465.7A patent/EP3620178A3/en not_active Withdrawn
- 2015-05-15 EP EP15792493.7A patent/EP3143141B1/en active Active
- 2015-05-15 US US14/713,857 patent/US9790495B2/en active Active
-
2017
- 2017-09-01 US US15/693,796 patent/US11021706B2/en active Active
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