JP2013530154A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2013530154A5 JP2013530154A5 JP2013512062A JP2013512062A JP2013530154A5 JP 2013530154 A5 JP2013530154 A5 JP 2013530154A5 JP 2013512062 A JP2013512062 A JP 2013512062A JP 2013512062 A JP2013512062 A JP 2013512062A JP 2013530154 A5 JP2013530154 A5 JP 2013530154A5
- Authority
- JP
- Japan
- Prior art keywords
- oligomer
- independently
- alkyl
- hydrogen
- location
- 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.)
- Withdrawn
Links
- 125000000217 alkyl group Chemical group 0.000 claims description 93
- 229910052739 hydrogen Inorganic materials 0.000 claims description 86
- 239000001257 hydrogen Substances 0.000 claims description 84
- -1 trifluoromethylguanidinyl Chemical group 0.000 claims description 57
- 150000002431 hydrogen Chemical class 0.000 claims description 54
- 125000000623 heterocyclic group Chemical group 0.000 claims description 47
- 125000003118 aryl group Chemical group 0.000 claims description 46
- 125000004448 alkyl carbonyl group Chemical group 0.000 claims description 29
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims description 28
- 125000003545 alkoxy group Chemical group 0.000 claims description 28
- 125000003710 aryl alkyl group Chemical group 0.000 claims description 28
- 125000001072 heteroaryl group Chemical group 0.000 claims description 28
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 24
- 125000004453 alkoxycarbonyl group Chemical group 0.000 claims description 20
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 20
- 125000005843 halogen group Chemical group 0.000 claims description 16
- XEEYBQQBJWHFJM-UHFFFAOYSA-N Iron Chemical compound [Fe] XEEYBQQBJWHFJM-UHFFFAOYSA-N 0.000 claims description 14
- 229910052760 oxygen Inorganic materials 0.000 claims description 14
- 125000004103 aminoalkyl group Chemical group 0.000 claims description 12
- 125000005099 aryl alkyl carbonyl group Chemical group 0.000 claims description 12
- 125000005129 aryl carbonyl group Chemical group 0.000 claims description 12
- 125000006254 cycloalkyl carbonyl group Chemical group 0.000 claims description 12
- 150000003839 salts Chemical class 0.000 claims description 12
- 125000005098 aryl alkoxy carbonyl group Chemical group 0.000 claims description 10
- 125000005161 aryl oxy carbonyl group Chemical group 0.000 claims description 10
- 125000005170 cycloalkyloxycarbonyl group Chemical group 0.000 claims description 10
- 125000004415 heterocyclylalkyl group Chemical group 0.000 claims description 10
- 229910052717 sulfur Inorganic materials 0.000 claims description 10
- GLUUGHFHXGJENI-UHFFFAOYSA-N Piperazine Chemical compound C1CNCCN1 GLUUGHFHXGJENI-UHFFFAOYSA-N 0.000 claims description 8
- 125000003282 alkyl amino group Chemical group 0.000 claims description 8
- 150000001413 amino acids Chemical class 0.000 claims description 8
- 125000004429 atom Chemical group 0.000 claims description 8
- 125000002618 bicyclic heterocycle group Chemical group 0.000 claims description 8
- 125000002915 carbonyl group Chemical group [*:2]C([*:1])=O 0.000 claims description 8
- 125000004093 cyano group Chemical group *C#N 0.000 claims description 8
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 8
- 125000002950 monocyclic group Chemical group 0.000 claims description 8
- 125000003341 7 membered heterocyclic group Chemical group 0.000 claims description 7
- 229940099352 cholate Drugs 0.000 claims description 7
- BHQCQFFYRZLCQQ-OELDTZBJSA-N cholic acid Chemical compound C([C@H]1C[C@H]2O)[C@H](O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H]([C@@H](CCC(O)=O)C)[C@@]2(C)[C@@H](O)C1 BHQCQFFYRZLCQQ-OELDTZBJSA-N 0.000 claims description 7
- 125000005647 linker group Chemical group 0.000 claims description 7
- 125000000747 amidyl group Chemical group [H][N-]* 0.000 claims description 6
- 125000004104 aryloxy group Chemical group 0.000 claims description 6
- 229910052799 carbon Inorganic materials 0.000 claims description 6
- 229940009976 deoxycholate Drugs 0.000 claims description 6
- KXGVEGMKQFWNSR-LLQZFEROSA-N deoxycholic acid Chemical compound C([C@H]1CC2)[C@H](O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H]([C@@H](CCC(O)=O)C)[C@@]2(C)[C@@H](O)C1 KXGVEGMKQFWNSR-LLQZFEROSA-N 0.000 claims description 6
- ANCLJVISBRWUTR-UHFFFAOYSA-N diaminophosphinic acid Chemical compound NP(N)(O)=O ANCLJVISBRWUTR-UHFFFAOYSA-N 0.000 claims description 6
- 201000010099 disease Diseases 0.000 claims description 6
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 6
- 125000004043 oxo group Chemical group O=* 0.000 claims description 6
- 125000003386 piperidinyl group Chemical group 0.000 claims description 6
- 125000002023 trifluoromethyl group Chemical group FC(F)(F)* 0.000 claims description 6
- 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 6
- KXDHJXZQYSOELW-UHFFFAOYSA-M Carbamate Chemical compound NC([O-])=O KXDHJXZQYSOELW-UHFFFAOYSA-M 0.000 claims description 4
- OKTJSMMVPCPJKN-UHFFFAOYSA-N Carbon Chemical compound [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 claims description 4
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims description 4
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims description 4
- 208000036142 Viral infection Diseases 0.000 claims description 4
- 150000001408 amides Chemical class 0.000 claims description 4
- QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical compound [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 claims description 4
- 150000002148 esters Chemical class 0.000 claims description 4
- KWIUHFFTVRNATP-UHFFFAOYSA-N glycine betaine Chemical compound C[N+](C)(C)CC([O-])=O KWIUHFFTVRNATP-UHFFFAOYSA-N 0.000 claims description 4
- 208000018360 neuromuscular disease Diseases 0.000 claims description 4
- 108020004707 nucleic acids Proteins 0.000 claims description 4
- 150000007523 nucleic acids Chemical class 0.000 claims description 4
- 102000039446 nucleic acids Human genes 0.000 claims description 4
- 239000001301 oxygen Substances 0.000 claims description 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 4
- 230000009385 viral infection Effects 0.000 claims description 4
- 150000004713 phosphodiesters Chemical class 0.000 claims description 3
- PTMHPRAIXMAOOB-UHFFFAOYSA-L phosphoramidate Chemical compound NP([O-])([O-])=O PTMHPRAIXMAOOB-UHFFFAOYSA-L 0.000 claims description 3
- 125000004482 piperidin-4-yl group Chemical group N1CCC(CC1)* 0.000 claims description 3
- XIBPCLQLEGQADN-UHFFFAOYSA-N 4-acetyloxy-4-oxobutanoic acid Chemical compound CC(=O)OC(=O)CCC(O)=O XIBPCLQLEGQADN-UHFFFAOYSA-N 0.000 claims description 2
- NJYVEMPWNAYQQN-UHFFFAOYSA-N 5-carboxyfluorescein Chemical compound C12=CC=C(O)C=C2OC2=CC(O)=CC=C2C21OC(=O)C1=CC(C(=O)O)=CC=C21 NJYVEMPWNAYQQN-UHFFFAOYSA-N 0.000 claims description 2
- 208000035143 Bacterial infection Diseases 0.000 claims description 2
- BWGNESOTFCXPMA-UHFFFAOYSA-N Dihydrogen disulfide Chemical compound SS BWGNESOTFCXPMA-UHFFFAOYSA-N 0.000 claims description 2
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 2
- 206010061218 Inflammation Diseases 0.000 claims description 2
- 208000022362 bacterial infectious disease Diseases 0.000 claims description 2
- 125000003236 benzoyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C(*)=O 0.000 claims description 2
- 125000001309 chloro group Chemical group Cl* 0.000 claims description 2
- 150000001875 compounds Chemical class 0.000 claims description 2
- 150000003983 crown ethers Chemical class 0.000 claims description 2
- ZZVUWRFHKOJYTH-UHFFFAOYSA-N diphenhydramine Chemical group C=1C=CC=CC=1C(OCCN(C)C)C1=CC=CC=C1 ZZVUWRFHKOJYTH-UHFFFAOYSA-N 0.000 claims description 2
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims description 2
- 125000004030 farnesyl group Chemical group [H]C([*])([H])C([H])=C(C([H])([H])[H])C([H])([H])C([H])([H])C([H])=C(C([H])([H])[H])C([H])([H])C([H])([H])C([H])=C(C([H])([H])[H])C([H])([H])[H] 0.000 claims description 2
- 125000002350 geranyl group Chemical group [H]C([*])([H])/C([H])=C(C([H])([H])[H])/C([H])([H])C([H])([H])C([H])=C(C([H])([H])[H])C([H])([H])[H] 0.000 claims description 2
- 230000004054 inflammatory process Effects 0.000 claims description 2
- 206010022000 influenza Diseases 0.000 claims description 2
- 125000001449 isopropyl group Chemical group [H]C([H])([H])C([H])(*)C([H])([H])[H] 0.000 claims description 2
- 125000000400 lauroyl 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])[H] 0.000 claims description 2
- 125000002757 morpholinyl group Chemical group 0.000 claims description 2
- 150000002825 nitriles Chemical class 0.000 claims description 2
- 125000000538 pentafluorophenyl group Chemical group FC1=C(F)C(F)=C(*)C(F)=C1F 0.000 claims description 2
- 125000005008 perfluoropentyl group Chemical group FC(C(C(C(C(F)(F)F)(F)F)(F)F)(F)F)(F)* 0.000 claims description 2
- 125000004194 piperazin-1-yl group Chemical group [H]N1C([H])([H])C([H])([H])N(*)C([H])([H])C1([H])[H] 0.000 claims description 2
- 208000030761 polycystic kidney disease Diseases 0.000 claims description 2
- 125000001844 prenyl group Chemical group [H]C([*])([H])C([H])=C(C([H])([H])[H])C([H])([H])[H] 0.000 claims description 2
- 125000000714 pyrimidinyl group Chemical group 0.000 claims description 2
- 125000000719 pyrrolidinyl group Chemical group 0.000 claims description 2
- 239000007787 solid Substances 0.000 claims description 2
- FIQMHBFVRAXMOP-UHFFFAOYSA-N triphenylphosphane oxide Chemical group C=1C=CC=CC=1P(C=1C=CC=CC=1)(=O)C1=CC=CC=C1 FIQMHBFVRAXMOP-UHFFFAOYSA-N 0.000 claims description 2
- 244000007835 Cyamopsis tetragonoloba Species 0.000 claims 8
- 125000006552 (C3-C8) cycloalkyl group Chemical group 0.000 claims 5
- 125000004642 (C1-C12) alkoxy group Chemical group 0.000 claims 4
- 125000004435 hydrogen atom Chemical group [H]* 0.000 claims 2
- 208000026372 Congenital cystic kidney disease Diseases 0.000 claims 1
- 239000004305 biphenyl Substances 0.000 claims 1
- 0 CCC(C)C(*C)c1ccccc1 Chemical compound CCC(C)C(*C)c1ccccc1 0.000 description 11
- 125000000753 cycloalkyl group Chemical group 0.000 description 5
- 125000004183 alkoxy alkyl group Chemical group 0.000 description 2
- ZAMBXEPXMJVNCP-UHFFFAOYSA-N CC(C)(C)P(N1CCOCC1)(OCS)=O Chemical compound CC(C)(C)P(N1CCOCC1)(OCS)=O ZAMBXEPXMJVNCP-UHFFFAOYSA-N 0.000 description 1
- BOQSEVFINDZVDK-UHFFFAOYSA-N CC(C)(CN(CCCO1)P1=O)S Chemical compound CC(C)(CN(CCCO1)P1=O)S BOQSEVFINDZVDK-UHFFFAOYSA-N 0.000 description 1
- VRKKZOILOGTZSA-UHFFFAOYSA-N CC(C)C(c(cc1)ccc1-[n]1ncc2c1CC(C)(C)CC2=O)=O Chemical compound CC(C)C(c(cc1)ccc1-[n]1ncc2c1CC(C)(C)CC2=O)=O VRKKZOILOGTZSA-UHFFFAOYSA-N 0.000 description 1
- WCOTWXLJGZKQQE-UHFFFAOYSA-N CC(C)C(c(cc1OC)cc(OC)c1OC)=O Chemical compound CC(C)C(c(cc1OC)cc(OC)c1OC)=O WCOTWXLJGZKQQE-UHFFFAOYSA-N 0.000 description 1
- RWGFKTVRMDUZSP-UHFFFAOYSA-N CC(C)c1ccccc1 Chemical compound CC(C)c1ccccc1 RWGFKTVRMDUZSP-UHFFFAOYSA-N 0.000 description 1
- CZEAOJQCXNACGZ-UHFFFAOYSA-N CC(COP(C)(N(CC1)CCC1NC(c1cc(-c2cc(Cl)cc(Cl)c2)ccc1)=O)=O)S Chemical compound CC(COP(C)(N(CC1)CCC1NC(c1cc(-c2cc(Cl)cc(Cl)c2)ccc1)=O)=O)S CZEAOJQCXNACGZ-UHFFFAOYSA-N 0.000 description 1
- MROKJEYKOKZQEK-UHFFFAOYSA-N CC(c(cc1)ccc1-[n]1c2ccccc2c2ccccc12)=O Chemical compound CC(c(cc1)ccc1-[n]1c2ccccc2c2ccccc12)=O MROKJEYKOKZQEK-UHFFFAOYSA-N 0.000 description 1
- VLWKOVOYTAGSDU-UHFFFAOYSA-N CCOc1ccc(C)cc1OCCOCCOc1ccccc1OCCOC Chemical compound CCOc1ccc(C)cc1OCCOCCOc1ccccc1OCCOC VLWKOVOYTAGSDU-UHFFFAOYSA-N 0.000 description 1
- PTMHPRAIXMAOOB-UHFFFAOYSA-N phosphoramidic acid Chemical compound NP(O)(O)=O PTMHPRAIXMAOOB-UHFFFAOYSA-N 0.000 description 1
- RQCNHUCCQJMSRG-UHFFFAOYSA-N tert-butyl piperidine-1-carboxylate Chemical compound CC(C)(C)OC(=O)N1CCCCC1 RQCNHUCCQJMSRG-UHFFFAOYSA-N 0.000 description 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US34978310P | 2010-05-28 | 2010-05-28 | |
| US61/349,783 | 2010-05-28 | ||
| US36187810P | 2010-07-06 | 2010-07-06 | |
| US61/361,878 | 2010-07-06 | ||
| US38642810P | 2010-09-24 | 2010-09-24 | |
| US61/386,428 | 2010-09-24 | ||
| PCT/US2011/038459 WO2011150408A2 (en) | 2010-05-28 | 2011-05-27 | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015196646A Division JP2016000059A (ja) | 2010-05-28 | 2015-10-02 | 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2013530154A JP2013530154A (ja) | 2013-07-25 |
| JP2013530154A5 true JP2013530154A5 (https=) | 2014-06-26 |
Family
ID=44627075
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013512062A Withdrawn JP2013530154A (ja) | 2010-05-28 | 2011-05-27 | 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ |
| JP2015196646A Withdrawn JP2016000059A (ja) | 2010-05-28 | 2015-10-02 | 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ |
| JP2017192810A Active JP6634424B2 (ja) | 2010-05-28 | 2017-10-02 | 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ |
| JP2019150398A Active JP7008056B2 (ja) | 2010-05-28 | 2019-08-20 | 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ |
| JP2021002181A Pending JP2021048886A (ja) | 2010-05-28 | 2021-01-08 | 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015196646A Withdrawn JP2016000059A (ja) | 2010-05-28 | 2015-10-02 | 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ |
| JP2017192810A Active JP6634424B2 (ja) | 2010-05-28 | 2017-10-02 | 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ |
| JP2019150398A Active JP7008056B2 (ja) | 2010-05-28 | 2019-08-20 | 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ |
| JP2021002181A Pending JP2021048886A (ja) | 2010-05-28 | 2021-01-08 | 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ |
Country Status (12)
| Country | Link |
|---|---|
| US (5) | US8779128B2 (https=) |
| EP (1) | EP2576574A2 (https=) |
| JP (5) | JP2013530154A (https=) |
| KR (5) | KR20200133284A (https=) |
| CN (2) | CN103003288B (https=) |
| AU (2) | AU2011257980B2 (https=) |
| BR (1) | BR112012031363A2 (https=) |
| CA (1) | CA2799501C (https=) |
| IL (4) | IL223298B (https=) |
| NZ (1) | NZ603606A (https=) |
| TW (2) | TWI616454B (https=) |
| WO (1) | WO2011150408A2 (https=) |
Families Citing this family (94)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE498685T1 (de) | 2004-06-28 | 2011-03-15 | Univ Western Australia | Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon |
| AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| CN112574988A (zh) | 2008-10-24 | 2021-03-30 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
| LT2499249T (lt) | 2009-11-12 | 2018-12-27 | The University Of Western Australia | Priešprasmės molekulės ir patologijų gydymo būdai |
| EP2545173A2 (en) * | 2010-03-12 | 2013-01-16 | Sarepta Therapeutics, Inc. | Antisense modulation of nuclear hormone receptors |
| KR20200133284A (ko) | 2010-05-28 | 2020-11-26 | 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 |
| US9161948B2 (en) * | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| EP2732035A2 (en) | 2011-07-15 | 2014-05-21 | Sarepta Therapeutics, Inc. | Methods and compositions for manipulating translation of protein isoforms from alternative initiation start sites |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| EA202090338A1 (ru) * | 2011-11-18 | 2021-04-30 | Сарепта Терапьютикс, Инк. | Функционально-модифицированные олигонуклеотиды и их субъединицы |
| US10066228B2 (en) | 2011-11-30 | 2018-09-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| ES2727481T3 (es) | 2011-11-30 | 2019-10-16 | Sarepta Therapeutics Inc | Inclusión inducida de exón en atrofia muscular espinal |
| EP2788487B1 (en) | 2011-12-08 | 2018-04-04 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues targeting human lmna |
| CN117721110A (zh) * | 2011-12-28 | 2024-03-19 | 日本新药株式会社 | 反义核酸 |
| CN112251436A (zh) | 2012-01-27 | 2021-01-22 | 比奥马林技术公司 | 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
| JP6542662B2 (ja) * | 2012-03-20 | 2019-07-10 | サレプタ セラピューティクス， インコーポレイテッド | オリゴヌクレオチドアナログのボロン酸結合体 |
| SMT201700339T1 (it) | 2012-04-23 | 2017-09-07 | Biomarin Tech Bv | Oligonucleotidi di modulazione dell'rna con caratteristiche migliorate per il trattamento dei disturbi neuromuscolari |
| EP2870246B1 (en) | 2012-07-03 | 2019-09-11 | BioMarin Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
| EP2852668B1 (en) | 2012-07-12 | 2016-04-27 | ProQR Therapeutics II B.V. | Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell |
| US9175291B2 (en) | 2012-10-11 | 2015-11-03 | Isis Pharmaceuticals Inc. | Modulation of androgen receptor expression |
| CA2894899A1 (en) | 2012-12-20 | 2014-06-26 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
| 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 |
| RS58529B1 (sr) | 2013-03-14 | 2019-04-30 | Sarepta Therapeutics Inc | Sastavi koji preskaču egzon za tretiranje mišićne distrofije |
| NZ731587A (en) | 2013-03-14 | 2021-07-30 | Sarepta Therapeutics Inc | Exon skipping compositions for treating muscular dystrophy |
| CA2906812A1 (en) | 2013-03-15 | 2014-09-18 | Sarepta Therapeutics, Inc. | Improved compositions for treating muscular dystrophy |
| JP6618910B2 (ja) | 2013-09-05 | 2019-12-11 | サレプタ セラピューティクス，インコーポレイテッド | 酸性α−グルコシダーゼにおけるアンチセンス誘導エクソン２包含 |
| SMT201900169T1 (it) | 2014-03-12 | 2019-05-10 | Nippon Shinyaku Co Ltd | Acido nucleico antisenso |
| BR112017004056A2 (pt) | 2014-09-12 | 2017-12-05 | Biogen Ma Inc | composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo |
| WO2016138534A2 (en) * | 2015-02-27 | 2016-09-01 | Sarepta Therapeutics, Inc. | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| MA41795A (fr) * | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| US10849917B2 (en) | 2015-06-01 | 2020-12-01 | Sarepta Therapeutics, Inc. | Antisense-induced exon exclusion in type VII collagen |
| EP3302489A4 (en) | 2015-06-04 | 2019-02-06 | Sarepta Therapeutics, Inc. | METHOD AND COMPOUNDS FOR THE TREATMENT OF DISEASES AND SUFFERING IN CONNECTION WITH LYMPHOCYTES |
| SI3331891T1 (sl) * | 2015-08-05 | 2022-04-29 | Eisai R&D Management Co., Ltd. | Postopek priprave v bistvu diastereomerno čistega fosforodiamidatnega oligomera, fosforodiamidatni oligomer izdelan po takem postopku in farmacevtski sestavek, ki vsebuje tak fosforodiamidatni oligomer |
| JP6987041B2 (ja) | 2015-08-28 | 2021-12-22 | 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エクソン包含のための修飾アンチセンスオリゴマー |
| EA201890811A1 (ru) | 2015-09-30 | 2018-09-28 | Сарепта Терапьютикс, Инк. | Способы лечения мышечной дистрофии |
| EP3858993A1 (en) | 2015-10-09 | 2021-08-04 | Sarepta Therapeutics, Inc. | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
| JP2019513371A (ja) | 2016-04-01 | 2019-05-30 | アビディティー バイオサイエンシーズ エルエルシー | 核酸ポリペプチド組成物とその使用 |
| CA3021267A1 (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 |
| CN109477109B (zh) | 2016-04-29 | 2022-09-23 | 萨勒普塔医疗公司 | 靶向人lmna的寡核苷酸类似物 |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| MA45183A (fr) * | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| TWI737736B (zh) * | 2016-05-24 | 2021-09-01 | 美商薩羅塔治療公司 | 製備磷醯二胺嗎啉代寡聚物之方法 |
| MA45362A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| MA49775A (fr) * | 2016-05-24 | 2020-06-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| TW201811807A (zh) * | 2016-06-30 | 2018-04-01 | 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 |
| KR102523522B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| CN109152775B (zh) * | 2016-06-30 | 2022-04-26 | 萨勒普塔医疗公司 | 制备磷酸二酰胺吗啉代寡聚物的方法 |
| JP2019534862A (ja) * | 2016-09-20 | 2019-12-05 | ザ リージェンツ オブ ザ ユニヴァーシティ オブ コロラド，ア ボディ コーポレイト | ホスホルアミダイト化学を使用する骨格修飾モルホリノオリゴヌクレオチド及びキメラの合成 |
| EP3330276A1 (en) | 2016-11-30 | 2018-06-06 | Universität Bern | Novel bicyclic nucleosides and oligomers prepared therefrom |
| SI3554554T1 (sl) | 2016-12-19 | 2023-02-28 | Sarepta Therapeutics, Inc. | Oligomerni konjugati, ki preskakujejo ekson za mišično distrofijo |
| SMT202200366T1 (it) | 2016-12-19 | 2022-11-18 | Sarepta Therapeutics Inc | Coniugati di oligomeri per salto di esone per distrofia muscolare |
| MX2019008199A (es) | 2017-01-06 | 2019-11-25 | Avidity Biosciences Llc | Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon. |
| WO2018193428A1 (en) | 2017-04-20 | 2018-10-25 | Synthena Ag | Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof |
| KR102778110B1 (ko) | 2017-04-20 | 2025-03-10 | 신테나 아게 | 트리시클로-dna 뉴클레오시드를 포함하는 변형 올리고머 화합물 및 그의 용도 |
| GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| WO2019071028A1 (en) | 2017-10-04 | 2019-04-11 | Avidity Biosciences Llc | NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF |
| WO2019079637A2 (en) | 2017-10-18 | 2019-04-25 | Sarepta Therapeutics, Inc. | ANTISENSE OLIGOMERIC COMPOUNDS |
| JP7506601B2 (ja) | 2017-12-06 | 2024-06-26 | アビディティー バイオサイエンシーズ，インク． | 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法 |
| JP7652567B2 (ja) | 2018-01-19 | 2025-03-27 | シンセナ アーゲー | トリシクロ－ｄｎａヌクレオシド前駆体およびそれを調製するためのプロセス |
| CA3098266A1 (en) | 2018-05-11 | 2019-11-14 | Alpha Anomeric Sas | Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| EP3829595A4 (en) | 2018-08-02 | 2022-08-24 | Dyne Therapeutics, Inc. | MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| IL312067B2 (en) | 2018-12-21 | 2025-08-01 | Avidity Biosciences Inc | Anti-transferrin receptor antibodies and uses thereof |
| US12497615B2 (en) | 2019-04-25 | 2025-12-16 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
| WO2020231206A1 (ko) | 2019-05-15 | 2020-11-19 | 주식회사 엘지화학 | 배터리 팩 및 이를 포함하는 전력 저장 장치 |
| JP7592034B2 (ja) | 2019-06-06 | 2024-11-29 | アビディティー バイオサイエンシーズ，インク． | 核酸ポリペプチド組成物およびその使用 |
| KR20220019259A (ko) | 2019-06-06 | 2022-02-16 | 어비디티 바이오사이언시스 인크. | Una 아미다이트 및 이의 용도 |
| KR20220147575A (ko) * | 2019-11-27 | 2022-11-03 | 디티엑스 파마, 인크. | 뒤시엔느 근위축증의 치료를 위한 화합물 및 방법 |
| CR20220485A (es) | 2020-02-28 | 2022-11-10 | Ionis Pharmaceuticals Inc | Compuestos y métodos para modular smn2 |
| US11555190B2 (en) | 2020-03-19 | 2023-01-17 | Avidity Biosciences, Inc. | Compositions and methods of treating Facioscapulohumeral muscular dystrophy |
| AU2021241682B2 (en) | 2020-03-27 | 2026-02-12 | Avidity Biosciences, Inc. | Compositions and methods of treating muscle dystrophy |
| EP3978608A1 (en) | 2020-10-05 | 2022-04-06 | SQY Therapeutics | Oligomeric compound for dystrophin rescue in dmd patients throughout skipping of exon-51 |
| WO2022106695A1 (en) | 2020-11-23 | 2022-05-27 | Alpha Anomeric Sas | Nucleic acid duplexes |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| IL309936A (en) | 2021-07-09 | 2024-03-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of dystrophinopathy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| MX2024003266A (es) | 2021-09-16 | 2024-04-03 | Avidity Biosciences Inc | Composiciones y metodos de tratamiento de la distrofia muscular facioescapulohumeral. |
| MX2024003690A (es) | 2021-09-30 | 2024-06-19 | Sarepta Therapeutics Inc | Oligonucleótidos antisentido que tienen una o más unidades abásicas. |
| JP2024539223A (ja) | 2021-10-22 | 2024-10-28 | サレプタ セラピューティクス， インコーポレイテッド | 末梢ミエリンタンパク質２２関連疾患の処置のためのモルフォリノオリゴマー |
| JPWO2023145755A1 (https=) | 2022-01-25 | 2023-08-03 | ||
| US12071621B2 (en) | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
| US20230357763A1 (en) * | 2022-05-06 | 2023-11-09 | James E. Summerton | Morpholinos with Increased Delivery Efficiency |
| WO2023246935A1 (zh) * | 2022-06-23 | 2023-12-28 | 安沛治疗有限公司 | 包含喹啉修饰的靶特异性核酸分子 |
| WO2024064237A2 (en) | 2022-09-21 | 2024-03-28 | Sarepta Therapeutics, Inc. | Dmd antisense oligonucleotide-mediated exon skipping efficiency |
| CN121194983A (zh) | 2023-04-06 | 2025-12-23 | 上海舶望制药有限公司 | 5'-膦酸酯修饰的核苷类似物及其制备的寡核苷酸 |
| WO2024262593A1 (ja) | 2023-06-22 | 2024-12-26 | 公益財団法人川崎市産業振興財団 | 核酸の血中安定性を向上させ、標的化効率を向上させる方法 |
| WO2025085810A2 (en) | 2023-10-18 | 2025-04-24 | Sarepta Therapeutics, Inc. | Antisense oligomers for treatment of centronuclear myopathies |
| CN121358717A (zh) * | 2023-12-15 | 2026-01-16 | 厦门赛诺邦格生物科技股份有限公司 | 一种含二硫键的可降解阳离子脂质及其应用 |
| CN119745845A (zh) * | 2025-01-16 | 2025-04-04 | 兰州大学 | 小分子n-甲基十二烷基胺在制备防治慢性肾病和高尿酸血症药物中的应用 |
Family Cites Families (126)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| JP3022967B2 (ja) | 1985-03-15 | 2000-03-21 | アンチバイラルズ インコーポレイテッド | 立体規則性ポリヌクレオチド結合ポリマー |
| 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 |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| US5749847A (en) | 1988-01-21 | 1998-05-12 | Massachusetts Institute Of Technology | Delivery of nucleotides into organisms by electroporation |
| US5223168A (en) | 1989-12-12 | 1993-06-29 | Gary Holt | Surface cleaner and treatment |
| JP3398378B2 (ja) | 1989-12-20 | 2003-04-21 | アンチビラルス・インコーポレイテツド | リン含有キラルインターサブユニットリンケージを有する非電荷モルホリノ―基体ポリマー |
| US5212295A (en) | 1990-01-11 | 1993-05-18 | Isis Pharmaceuticals | Monomers for preparation of oligonucleotides having chiral phosphorus linkages |
| US5578718A (en) | 1990-01-11 | 1996-11-26 | Isis Pharmaceuticals, Inc. | Thiol-derivatized nucleosides |
| US5587361A (en) | 1991-10-15 | 1996-12-24 | Isis Pharmaceuticals, Inc. | Oligonucleotides having phosphorothioate linkages of high chiral purity |
| US5457191A (en) | 1990-01-11 | 1995-10-10 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5506351A (en) | 1992-07-23 | 1996-04-09 | Isis Pharmaceuticals | Process for the preparation of 2'-O-alkyl guanosine and related compounds |
| US5852188A (en) | 1990-01-11 | 1998-12-22 | Isis Pharmaceuticals, Inc. | Oligonucleotides having chiral phosphorus linkages |
| US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
| US5138045A (en) | 1990-07-27 | 1992-08-11 | Isis Pharmaceuticals | Polyamine conjugated oligonucleotides |
| US5378825A (en) | 1990-07-27 | 1995-01-03 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs |
| US5386023A (en) | 1990-07-27 | 1995-01-31 | Isis Pharmaceuticals | Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling |
| US5218105A (en) | 1990-07-27 | 1993-06-08 | Isis Pharmaceuticals | Polyamine conjugated oligonucleotides |
| US5602240A (en) | 1990-07-27 | 1997-02-11 | Ciba Geigy Ag. | Backbone modified oligonucleotide analogs |
| US5610289A (en) | 1990-07-27 | 1997-03-11 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogues |
| US5608046A (en) | 1990-07-27 | 1997-03-04 | Isis Pharmaceuticals, Inc. | Conjugated 4'-desmethyl nucleoside analog compounds |
| US5541307A (en) | 1990-07-27 | 1996-07-30 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs and solid phase synthesis thereof |
| US6262241B1 (en) | 1990-08-13 | 2001-07-17 | Isis Pharmaceuticals, Inc. | Compound for detecting and modulating RNA activity and gene expression |
| KR970005274B1 (ko) | 1990-08-14 | 1997-04-15 | 아이시스 파마슈티칼스, 인코포레이티드 | 안티센스 올리고뉴클레오티드에 의한 a형 인플루엔자 바이러스, 안 아아보스트레인(ann arbor strain) h2n2의 억제 |
| US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
| WO1993001286A2 (en) | 1991-06-28 | 1993-01-21 | 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 |
| US5599797A (en) | 1991-10-15 | 1997-02-04 | Isis Pharmaceuticals, Inc. | Oligonucleotides having phosphorothioate linkages of high chiral purity |
| US5576302A (en) | 1991-10-15 | 1996-11-19 | Isis Pharmaceuticals, Inc. | Oligonucleotides for modulating hepatitis C virus having phosphorothioate linkages of high chiral purity |
| WO1993013226A1 (en) | 1991-12-23 | 1993-07-08 | Chiron Corporation | Hav probes for use in solution phase sandwich hybridization assays |
| US6391542B1 (en) | 1992-09-10 | 2002-05-21 | Isis Pharmaceuticals, Inc. | Compositions and methods for treatment of Hepatitis C virus-associated diseases |
| US6174868B1 (en) | 1992-09-10 | 2001-01-16 | Isis Pharmaceuticals, Inc. | Compositions and methods for treatment of hepatitis C virus-associated diseases |
| US5571902A (en) | 1993-07-29 | 1996-11-05 | Isis Pharmaceuticals, Inc. | Synthesis of oligonucleotides |
| US5801154A (en) | 1993-10-18 | 1998-09-01 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of multidrug resistance-associated protein |
| US6060456A (en) | 1993-11-16 | 2000-05-09 | Genta Incorporated | Chimeric oligonucleoside compounds |
| US5554746A (en) | 1994-05-16 | 1996-09-10 | Isis Pharmaceuticals, Inc. | Lactam nucleic acids |
| US5734039A (en) | 1994-09-15 | 1998-03-31 | Thomas Jefferson University | Antisense oligonucleotides targeting cooperating oncogenes |
| GB9510718D0 (en) | 1995-05-26 | 1995-07-19 | Sod Conseils Rech Applic | Antisense oligonucleotides |
| JPH0915828A (ja) | 1995-07-03 | 1997-01-17 | Fuji Photo Film Co Ltd | 写真処理システム用ペーパーカッター |
| US5955318A (en) | 1995-08-14 | 1999-09-21 | Abbott Laboratories | Reagents and methods useful for controlling the translation of hepatitis GBV proteins |
| 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 |
| US20040161844A1 (en) | 1996-06-06 | 2004-08-19 | Baker Brenda F. | Sugar and backbone-surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| DE19716073A1 (de) | 1997-04-17 | 1998-10-22 | Boehringer Mannheim Gmbh | Dosiervorrichtung zur Abgabe kleiner Flüssigkeitsmengen |
| IL132941A0 (en) | 1997-05-21 | 2001-03-19 | Univ Leland Stanford Junior | Composition and method for enhancing transport across biological membranes |
| US6133246A (en) | 1997-08-13 | 2000-10-17 | Isis Pharmaceuticals Inc. | Antisense oligonucleotide compositions and methods for the modulation of JNK proteins |
| US6228579B1 (en) | 1997-11-14 | 2001-05-08 | San Diego State University Foundation | Method for identifying microbial proliferation genes |
| US6548651B1 (en) | 1998-11-11 | 2003-04-15 | Pantheco A/S | Modified peptide nucleic acid (PNA) molecules |
| US7094765B1 (en) | 1999-01-29 | 2006-08-22 | Avi Biopharma, Inc. | Antisense restenosis composition and method |
| JP2002535015A (ja) | 1999-01-29 | 2002-10-22 | エイブイアイ バイオファーマ， インコーポレイテッド | 標的ｒｎａを検出するための非侵襲性方法 |
| US6521438B1 (en) | 1999-11-05 | 2003-02-18 | North Carolina State University | Chemoreceptors in plant parasitic nematodes |
| US20030095953A1 (en) | 1999-11-12 | 2003-05-22 | Myles C. Cabot | Methods of reversing drug resistance in cancer cells |
| KR20020064915A (ko) | 1999-11-29 | 2002-08-10 | 에이브이아이 바이오파마 인코포레이티드 | 안티센스 항박테리아 방법 및 조성물 |
| WO2001049775A2 (en) | 2000-01-04 | 2001-07-12 | Avi Biopharma, Inc. | Antisense antibacterial cell division composition and method |
| 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 |
| US6784291B2 (en) | 2000-05-04 | 2004-08-31 | Avi Biopharma, Inc. | Splice-region antisense composition and method |
| AU2001271873A1 (en) | 2000-07-06 | 2002-01-21 | Avi Biopharma, Inc. | Transforming growth factor beta (TGF-beta) blocking agent-treated stem cell composition and method |
| DK1328543T3 (da) | 2000-10-27 | 2009-11-23 | Novartis Vaccines & Diagnostic | Nukleinsyrer og proteiner fra streptococcus-gruppe A & B |
| WO2002079467A2 (en) | 2001-03-29 | 2002-10-10 | Københavns Universitet | Antibiotic-free bacterial strain selection with antisense molecules |
| JP2004537517A (ja) | 2001-05-17 | 2004-12-16 | エイブイアイ バイオファーマ， インコーポレイテッド | ｃ−ｍｙｃアンチセンスオリゴマーを使用した、癌を処置するための併用アプローチ |
| AU2002317437A1 (en) | 2001-05-18 | 2002-12-03 | Cureon A/S | Therapeutic uses of lna-modified oligonucleotides in infectious diseases |
| US20030224353A1 (en) | 2001-10-16 | 2003-12-04 | Stein David A. | Antisense antiviral agent and method for treating ssRNA viral infection |
| AU2002342057B2 (en) | 2001-10-16 | 2009-01-22 | Avi Biopharma, Inc. | Antisense antiviral agent and method for treating ssRNA viral infection |
| EP2330194A3 (en) | 2002-09-13 | 2011-10-12 | Replicor, Inc. | Non-sequence complementary antiviral oligonucleotides |
| CA2868618C (en) | 2002-10-16 | 2017-05-23 | Gen-Probe Incorporated | Compositions and methods for detecting west nile virus |
| WO2004041854A2 (en) | 2002-11-05 | 2004-05-21 | Affinium Pharmaceuticals, Inc. | Essential novel bacterial polypeptides |
| EP1585755B1 (en) | 2002-12-31 | 2015-08-05 | Sigma-Aldrich Co. LLC | Methods and compositions for the tandem synthesis of two or more oligonuleotides on the same solid support |
| CA2523672C (en) | 2003-04-29 | 2012-07-17 | Avi Biopharma, Inc. | Compositions for enhancing transport of molecules into cells |
| AU2004242249A1 (en) | 2003-05-23 | 2004-12-02 | Epfl-Ecole Polytechnique Federale De Lausanne | Methods for protein labeling based on acyl carrier protein |
| DE602004030583D1 (de) | 2003-08-05 | 2011-01-27 | Avi Biopharma Inc | Oligonukleotidanalog und verfahren zur behandlung von flavivirusinfektionen |
| JP2007509939A (ja) | 2003-10-27 | 2007-04-19 | ジェネラブズ テクノロジーズ インコーポレーティッド | ウイルス感染を治療するためのヌクレオシド化合物 |
| US20050171044A1 (en) | 2003-12-24 | 2005-08-04 | Stein David A. | Oligonucleotide compound and method for treating nidovirus infections |
| EP1713332A4 (en) | 2004-01-23 | 2010-08-18 | Avi Biopharma Inc | ANTISENSE OLIGOMERS AND METHOD FOR INDUCTION OF IMMUNOTHERAPY AND IMMUNOSUPPRESSION |
| US7402574B2 (en) | 2004-03-12 | 2008-07-22 | Avi Biopharma, Inc. | Antisense composition and method for treating cancer |
| US20050288246A1 (en) | 2004-05-24 | 2005-12-29 | Iversen Patrick L | Peptide conjugated, inosine-substituted antisense oligomer compound and method |
| ATE498685T1 (de) | 2004-06-28 | 2011-03-15 | Univ Western Australia | Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon |
| ATE510914T1 (de) | 2004-07-02 | 2011-06-15 | Avi Biopharma Inc | Antisinn antibakterielle verfahren und verbindungen |
| US8129352B2 (en) | 2004-09-16 | 2012-03-06 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating ssRNA viral infection |
| US8357664B2 (en) | 2004-10-26 | 2013-01-22 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating influenza viral infection |
| US7524829B2 (en) | 2004-11-01 | 2009-04-28 | Avi Biopharma, Inc. | Antisense antiviral compounds and methods for treating a filovirus infection |
| US7838657B2 (en) | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
| GB0501944D0 (en) | 2005-01-31 | 2005-03-09 | Univ Cambridge Tech | Compounds for use in chemical detection and/or quantitation |
| AU2006213686A1 (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 |
| SI2548560T1 (sl) | 2005-06-23 | 2015-12-31 | Isis Pharmaceuticals, Inc. | Sestavki in postopki za moduliranje izrezovanja smn2 |
| CA2614191C (en) * | 2005-07-13 | 2015-06-30 | Avi Biopharma, Inc. | Antisense antibacterial method and compound |
| 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 |
| US8329668B2 (en) | 2005-09-08 | 2012-12-11 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating picornavirus infection |
| US8524676B2 (en) | 2005-09-08 | 2013-09-03 | Sarepta Therapeutics, Inc. | Method for treating enterovirus or rhinovirus infection using antisense antiviral compounds |
| CA2644262C (en) | 2006-03-07 | 2016-01-05 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating arenavirus infection |
| US8785407B2 (en) | 2006-05-10 | 2014-07-22 | Sarepta Therapeutics, Inc. | Antisense antiviral agent and method for treating ssRNA viral infection |
| DK2735568T3 (da) | 2006-05-10 | 2017-11-13 | Sarepta Therapeutics Inc | Oligonukleotidanaloger med kationiske bindinger mellem underenheder |
| AU2007282224B2 (en) | 2006-08-11 | 2013-08-29 | Vico Therapeutics B.V. | Methods and means for treating DNA repeat instability associated genetic disorders |
| EP3034083B1 (en) | 2006-09-21 | 2020-12-09 | University of Rochester | Antisense oligonucleotides for use in treating myotonic dystrophy |
| JP5227803B2 (ja) | 2006-11-24 | 2013-07-03 | ハイクス ラボラトリーズ合同会社 | スピロキノン化合物及び医薬組成物 |
| EP3443976A1 (en) | 2007-06-29 | 2019-02-20 | Sarepta Therapeutics, Inc. | Tissue specific peptide conjugates and methods |
| 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 |
| US9000142B2 (en) | 2008-07-07 | 2015-04-07 | Syntrix Biosystems, Inc. | Photocleavable sense-antisense complex |
| CN112574988A (zh) | 2008-10-24 | 2021-03-30 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
| WO2010120820A1 (en) | 2009-04-13 | 2010-10-21 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing |
| PT3305302T (pt) | 2009-06-17 | 2018-12-14 | Biogen Ma Inc | Composições e métodos de modulação de excisões de smn2 em um sujeito |
| US20110269665A1 (en) | 2009-06-26 | 2011-11-03 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| AU2010319314C1 (en) | 2009-11-13 | 2016-09-01 | Sarepta Therapeutics, Inc. | Antisense antiviral compound and method for treating influenza viral infection |
| EP2545173A2 (en) | 2010-03-12 | 2013-01-16 | Sarepta Therapeutics, Inc. | Antisense modulation of nuclear hormone receptors |
| ES2816898T3 (es) * | 2010-05-13 | 2021-04-06 | Sarepta Therapeutics Inc | Compuestos que modulan la actividad de señalización de las interleucinas 17 y 23 |
| KR20200133284A (ko) | 2010-05-28 | 2020-11-26 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| US8198429B2 (en) | 2010-08-09 | 2012-06-12 | Avi Biopharma, Inc. | Antisense antiviral compounds and methods for treating a filovirus infection |
| WO2012031243A2 (en) | 2010-09-03 | 2012-03-08 | Avi Biopharma, Inc. | dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS |
| RS55610B1 (sr) * | 2010-09-30 | 2017-06-30 | Nippon Shinyaku Co Ltd | Derivat morfolino nukleinske kiseline |
| EP3067420A1 (en) | 2010-11-12 | 2016-09-14 | Sarepta Therapeutics, Inc. | Antisense antibacterial compounds and methods |
| US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| EP2732035A2 (en) | 2011-07-15 | 2014-05-21 | Sarepta Therapeutics, Inc. | Methods and compositions for manipulating translation of protein isoforms from alternative initiation start sites |
| EA202090338A1 (ru) | 2011-11-18 | 2021-04-30 | Сарепта Терапьютикс, Инк. | Функционально-модифицированные олигонуклеотиды и их субъединицы |
| EP2788487B1 (en) | 2011-12-08 | 2018-04-04 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues targeting human lmna |
| JP6542662B2 (ja) | 2012-03-20 | 2019-07-10 | サレプタ セラピューティクス， インコーポレイテッド | オリゴヌクレオチドアナログのボロン酸結合体 |
| US20150036865A1 (en) | 2013-07-30 | 2015-02-05 | Lai-Shi Huang | Electrical signal to audio signal transducer with a deformable diaphragm unit |
| JP2016011512A (ja) | 2014-06-27 | 2016-01-21 | サントップエンジニア株式会社 | 排水シート材 |
-
2011
- 2011-05-27 KR KR1020207033269A patent/KR20200133284A/ko not_active Ceased
- 2011-05-27 US US13/118,298 patent/US8779128B2/en active Active
- 2011-05-27 AU AU2011257980A patent/AU2011257980B2/en not_active Ceased
- 2011-05-27 TW TW100118810A patent/TWI616454B/zh not_active IP Right Cessation
- 2011-05-27 KR KR1020207008683A patent/KR102182663B1/ko not_active Expired - Fee Related
- 2011-05-27 KR KR1020187003799A patent/KR101981705B1/ko not_active Expired - Fee Related
- 2011-05-27 CN CN201180034599.5A patent/CN103003288B/zh not_active Expired - Fee Related
- 2011-05-27 EP EP11725839.2A patent/EP2576574A2/en not_active Withdrawn
- 2011-05-27 CN CN201710590274.4A patent/CN107353317A/zh active Pending
- 2011-05-27 KR KR1020197014303A patent/KR102095478B1/ko not_active Expired - Fee Related
- 2011-05-27 KR KR1020127033727A patent/KR101829644B1/ko not_active Expired - Fee Related
- 2011-05-27 WO PCT/US2011/038459 patent/WO2011150408A2/en not_active Ceased
- 2011-05-27 CA CA2799501A patent/CA2799501C/en not_active Expired - Fee Related
- 2011-05-27 TW TW105121412A patent/TWI620756B/zh not_active IP Right Cessation
- 2011-05-27 JP JP2013512062A patent/JP2013530154A/ja not_active Withdrawn
- 2011-05-27 NZ NZ603606A patent/NZ603606A/en not_active IP Right Cessation
- 2011-05-27 BR BR112012031363-0A patent/BR112012031363A2/pt not_active Application Discontinuation
-
2012
- 2012-11-27 IL IL223298A patent/IL223298B/en active IP Right Grant
-
2014
- 2014-06-06 US US14/298,655 patent/US9469664B2/en active Active
-
2015
- 2015-10-02 JP JP2015196646A patent/JP2016000059A/ja not_active Withdrawn
-
2016
- 2016-01-06 IL IL243467A patent/IL243467A/en active IP Right Grant
- 2016-08-25 US US15/247,584 patent/US10202602B2/en active Active
- 2016-09-29 AU AU2016234953A patent/AU2016234953B2/en not_active Ceased
-
2017
- 2017-10-02 JP JP2017192810A patent/JP6634424B2/ja active Active
-
2018
- 2018-12-19 US US16/225,909 patent/US10760078B2/en active Active
-
2019
- 2019-08-20 JP JP2019150398A patent/JP7008056B2/ja active Active
-
2020
- 2020-02-13 IL IL272656A patent/IL272656B/en unknown
- 2020-07-23 US US16/936,906 patent/US20210139897A1/en not_active Abandoned
-
2021
- 2021-01-08 JP JP2021002181A patent/JP2021048886A/ja active Pending
- 2021-06-17 IL IL284154A patent/IL284154A/en unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2013530154A5 (https=) | ||
| JP2014515762A5 (https=) | ||
| AU2016234953B2 (en) | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups | |
| AU2017282459B2 (en) | Cationic lipid | |
| JP2018511590A5 (https=) | ||
| US10947193B2 (en) | Cationic lipid | |
| JP2019512478A5 (https=) | ||
| IL303195A (en) | Lipid nanoparticles for delivery of nucleic acids and related methods of use | |
| JP2020525429A5 (https=) | ||
| CA2834128A1 (en) | Peptide oligonucleotide conjugates | |
| JP2018035142A5 (https=) | ||
| EP4245369A3 (en) | Neuroactive steroids, compositions, and uses thereof | |
| CY1119827T1 (el) | Ακυλαμινο-υποκατεστημενα συντηγμενα παραγωγα κυκλοπεντανο καρβοξυλικου οξεος και η χρηση αυτων ως φαρμακευτικων ουσιων | |
| JP2017538682A5 (https=) | ||
| JP2015501817A5 (https=) | ||
| JP2017536344A5 (https=) | ||
| RU2015100198A (ru) | Хиральный адъювант нуклеиновой кислоты | |
| WO1994018987A1 (fr) | Composition medicinale renfermant un copolymere d'acide nucleique | |
| DK2806896T3 (en) | Chitosan covalently bound to small molecule integrin antagonist for targeted delivery | |
| JP2012510502A5 (https=) | ||
| JP2010527913A5 (https=) | ||
| JP2013505327A5 (https=) | ||
| JP2020514299A5 (https=) | ||
| JP2011500656A (ja) | カチオン性脂質 | |
| JP2011510009A5 (https=) |