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- Withdrawn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) | CN107353317A (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 |
|---|---|---|---|---|
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP1855694B1 (en) | 2005-02-09 | 2020-12-02 | Sarepta Therapeutics, Inc. | Antisense composition for treating muscle atrophy |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| 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 |
| TR201816523T4 (tr) | 2009-11-12 | 2018-11-21 | Univ Western Australia | Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler. |
| US9068185B2 (en) * | 2010-03-12 | 2015-06-30 | 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 |
| US10017763B2 (en) | 2010-09-03 | 2018-07-10 | Sarepta Therapeutics, 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 |
| US10100305B2 (en) | 2011-07-15 | 2018-10-16 | Sarepta Therapeutics, Inc. | Methods and compositions for manipulating translation of protein isoforms from alternative initiation of start sites |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| HK1201514A1 (en) * | 2011-11-18 | 2015-09-04 | Sarepta Therapeutics, Inc. | Functionally-modified oligonucleotides and subunits thereof |
| US10066228B2 (en) | 2011-11-30 | 2018-09-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| JP6496549B2 (ja) | 2011-11-30 | 2019-04-03 | 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誘発されたエクソン包含 |
| ES2935606T3 (es) | 2011-12-08 | 2023-03-08 | Sarepta Therapeutics Inc | Análogos de oligonucleótidos dirigidos a LMNA humana |
| CA2861247C (en) * | 2011-12-28 | 2021-11-16 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acids |
| NZ627896A (en) | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| CN104411831B (zh) * | 2012-03-20 | 2020-08-11 | 萨勒普塔医疗公司 | 寡核苷酸类似物的硼酸缀合物 |
| LT2841578T (lt) | 2012-04-23 | 2017-09-25 | Biomarin Technologies B.V. | Rnr moduliuojantys oligonukleotidai su pagerintomis savybėmis, skirti neuromuskulinių susirgimų gydymui |
| EP2870246B1 (en) | 2012-07-03 | 2019-09-11 | BioMarin Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
| RS54944B1 (sr) | 2012-07-12 | 2016-11-30 | Proqr Therapeutics Ii Bv | Oligonukleotidi za pravljenje promena u sekvencama ciljanih rnk molekula prisutnih u živoj ćeliji |
| AR092982A1 (es) | 2012-10-11 | 2015-05-13 | Isis Pharmaceuticals Inc | Modulacion de la expresion de receptores androgenicos |
| HK1216902A1 (zh) | 2012-12-20 | 2016-12-09 | Sarepta Therapeutics, Inc. | 用作治疗肌肉萎缩的改进外显子跳跃组合物 |
| 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 |
| 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 |
| US11236338B2 (en) | 2013-09-05 | 2022-02-01 | Sarepta Therapeutics, Inc. | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| DK3118311T3 (en) * | 2014-03-12 | 2019-03-11 | Nippon Shinyaku Co Ltd | Antisense nucleic acid |
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| EP3262056A4 (en) * | 2015-02-27 | 2018-09-19 | 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 |
| MA50829A (fr) | 2015-06-01 | 2018-04-11 | Sarepta Therapeutics Inc | Exclusion d'exon induite pat technologie antisens dans le collagène de type vii |
| US11020417B2 (en) | 2015-06-04 | 2021-06-01 | Sarepta Therapeutics, Inc | Methods and compounds for treatment of lymphocyte-related diseases and conditions |
| AU2016302009B2 (en) * | 2015-08-05 | 2021-09-30 | Eisai R&D Management Co., Ltd. | Chiral reagents for preparation of homogeneous oligomers |
| US10905709B2 (en) | 2015-08-28 | 2021-02-02 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
| WO2017059131A1 (en) | 2015-09-30 | 2017-04-06 | Sarepta Therapeutics, Inc. | Methods for treating muscular dystrophy |
| 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 |
| WO2017184529A1 (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 |
| MX2018013157A (es) | 2016-04-29 | 2019-05-23 | Sarepta Therapeutics Inc | Analogos de oligonucleotidos dirigidos a lmna humana. |
| CA3024456A1 (en) * | 2016-05-24 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| BR112018074346B1 (pt) | 2016-05-24 | 2023-01-03 | Sarepta Therapeutics, Inc. | Composto oligomérico e processo para preparar um composto oligomérico |
| MD3464306T2 (ro) * | 2016-05-24 | 2024-08-31 | Sarepta Therapeutics Inc | Procedee de preparare a oligomerilor morfolino fosforodiamidați |
| US10947533B2 (en) | 2016-05-24 | 2021-03-16 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| KR102523527B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| KR102523522B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| JP2019525742A (ja) * | 2016-06-30 | 2019-09-12 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマー |
| AU2017330276B2 (en) * | 2016-09-20 | 2022-01-20 | The Regents Of The University Of Colorado, A Body Corporate | Synthesis of backbone modified morpholino oligonucleotides and chimeras using phosphoramidite chemistry |
| EP3330276A1 (en) | 2016-11-30 | 2018-06-06 | Universität Bern | Novel bicyclic nucleosides and oligomers prepared therefrom |
| KR20240006023A (ko) | 2016-12-19 | 2024-01-12 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| KR102810425B1 (ko) | 2016-12-19 | 2025-05-21 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| 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. |
| EP4036101A1 (en) | 2017-04-20 | 2022-08-03 | Synthena AG | Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof |
| WO2018193428A1 (en) | 2017-04-20 | 2018-10-25 | Synthena Ag | Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof |
| GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| EP3691657A4 (en) | 2017-10-04 | 2021-07-21 | Avidity Biosciences, Inc. | NUCLEIC ACID POLYPEPTIDE COMPOSITIONS AND USES THEREOF |
| US11555189B2 (en) | 2017-10-18 | 2023-01-17 | Sarepta Therapeutics, Inc. | Antisense oligomer compounds |
| AU2018378812B2 (en) | 2017-12-06 | 2025-05-22 | Avidity Biosciences, Inc. | Compositions and methods of treating muscle atrophy and myotonic dystrophy |
| WO2019142135A1 (en) | 2018-01-19 | 2019-07-25 | Synthena Ag | Tricyclo-dna nucleoside precursors and processes for preparing the same |
| JP7665509B2 (ja) | 2018-05-11 | 2025-04-21 | アルファ アノマリック エスアエス | ７’－５’－アルファ－アノマー二環式糖ヌクレオシドを含むオリゴヌクレオチドコンジュゲート |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| 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 |
| CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | 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 |
| WO2020219820A1 (en) | 2019-04-25 | 2020-10-29 | 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 |
| CN114375296A (zh) | 2019-06-06 | 2022-04-19 | 艾维迪提生物科学公司 | 核酸多肽组合物及其用途 |
| US12006499B2 (en) | 2019-06-06 | 2024-06-11 | Avidity Biosciences, Inc. | Una amidites and uses thereof |
| CA3166370A1 (en) * | 2019-11-27 | 2021-06-03 | Dtx Pharma, Inc. | Compounds and methods for the treatment of duchenne muscular dystrophy |
| CN120505310A (zh) | 2020-02-28 | 2025-08-19 | Ionis制药公司 | 用于调节smn2的化合物和方法 |
| WO2021188390A1 (en) | 2020-03-19 | 2021-09-23 | Avidity Biosciences, Inc. | Compositions and methods of treating facioscapulohumeral muscular dystrophy |
| SI4126066T1 (sl) | 2020-03-27 | 2026-04-30 | Avidity Biosciences, Inc. | Sestavki in postopki zdravljenja mišične distrofije |
| EP3978608A1 (en) | 2020-10-05 | 2022-04-06 | SQY Therapeutics | Oligomeric compound for dystrophin rescue in dmd patients throughout skipping of exon-51 |
| EP4247949A1 (en) | 2020-11-23 | 2023-09-27 | Alpha Anomeric SAS | Nucleic acid duplexes |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| JP2024525608A (ja) | 2021-07-09 | 2024-07-12 | ダイン セラピューティクス，インコーポレーテッド | ジストロフィン異常症を処置するための筋標的化複合体および製剤 |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| CN118265544A (zh) | 2021-09-16 | 2024-06-28 | 艾维迪提生物科学公司 | 治疗面肩肱型肌营养不良的组合物和方法 |
| WO2023055774A1 (en) | 2021-09-30 | 2023-04-06 | Sarepta Therapeutics, Inc. | Antisense oligonucleotides having one or more abasic units |
| EP4419680A1 (en) | 2021-10-22 | 2024-08-28 | Sarepta Therapeutics, Inc. | Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases |
| CN118660708A (zh) | 2022-01-25 | 2024-09-17 | 公益财团法人川崎市产业振兴财团 | 用于经皮给药的含有rna的组合物和该组合物的给药方法 |
| 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 |
| CN119032177A (zh) * | 2022-06-23 | 2024-11-26 | 安沛治疗有限公司 | 包含喹啉修饰的靶特异性核酸分子 |
| JP2025533454A (ja) | 2022-09-21 | 2025-10-07 | サレプタ セラピューティクス， インコーポレイテッド | Ｄｍｄアンチセンスオリゴヌクレオチド介在性エクソンスキッピング効率 |
| KR20250174651A (ko) | 2023-04-06 | 2025-12-12 | 상하이 아르고 바이오파마슈티칼 씨오., 엘티디. | 5'-포스포네이트 변형 뉴클레오시드 유사체 및 이로부터 제조된 올리고뉴클레오티드 |
| EP4732849A1 (en) | 2023-06-22 | 2026-04-29 | Kawasaki Institute of Industrial Promotion | Method for improving stability of nucleic acid in blood and improving targeting efficiency of nucleic acid |
| 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 |
|---|---|---|---|---|
| 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 |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| WO1986005519A1 (en) | 1985-03-15 | 1986-09-25 | James Summerton | Polynucleotide assay reagent and method |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| 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 |
| AU655164B2 (en) * | 1989-12-20 | 1994-12-08 | Antivirals Inc. | Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages |
| US5506351A (en) | 1992-07-23 | 1996-04-09 | Isis Pharmaceuticals | Process for the preparation of 2'-O-alkyl guanosine and related compounds |
| US5578718A (en) | 1990-01-11 | 1996-11-26 | Isis Pharmaceuticals, Inc. | Thiol-derivatized nucleosides |
| US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
| 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 |
| US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5212295A (en) | 1990-01-11 | 1993-05-18 | Isis Pharmaceuticals | Monomers for preparation of oligonucleotides having chiral phosphorus linkages |
| US5852188A (en) | 1990-01-11 | 1998-12-22 | Isis Pharmaceuticals, Inc. | Oligonucleotides having chiral phosphorus linkages |
| US5541307A (en) | 1990-07-27 | 1996-07-30 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs and solid phase synthesis thereof |
| US5602240A (en) | 1990-07-27 | 1997-02-11 | Ciba Geigy Ag. | Backbone modified oligonucleotide analogs |
| US5378825A (en) | 1990-07-27 | 1995-01-03 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs |
| US5610289A (en) | 1990-07-27 | 1997-03-11 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogues |
| US5138045A (en) | 1990-07-27 | 1992-08-11 | Isis Pharmaceuticals | Polyamine conjugated oligonucleotides |
| US5218105A (en) | 1990-07-27 | 1993-06-08 | Isis Pharmaceuticals | Polyamine conjugated oligonucleotides |
| US5386023A (en) | 1990-07-27 | 1995-01-31 | Isis Pharmaceuticals | Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling |
| US5608046A (en) | 1990-07-27 | 1997-03-04 | Isis Pharmaceuticals, Inc. | Conjugated 4'-desmethyl nucleoside analog compounds |
| US6262241B1 (en) | 1990-08-13 | 2001-07-17 | Isis Pharmaceuticals, Inc. | Compound for detecting and modulating RNA activity and gene expression |
| HUT69956A (en) | 1990-08-14 | 1995-09-28 | Isis Pharmaceuticals Inc | Methode for inhibition of influenza virus by antiseuse oligonucleotides |
| US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
| 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 |
| US5576302A (en) | 1991-10-15 | 1996-11-19 | Isis Pharmaceuticals, Inc. | Oligonucleotides for modulating hepatitis C virus having phosphorothioate linkages of high chiral purity |
| US5599797A (en) | 1991-10-15 | 1997-02-04 | Isis Pharmaceuticals, Inc. | Oligonucleotides having phosphorothioate linkages of high chiral purity |
| JPH07502658A (ja) | 1991-12-23 | 1995-03-23 | バイエル コーポレイション | 溶液相サンドイッチハイブリダイゼーションアッセイに用いるためのｈａｖプローブ |
| US6174868B1 (en) | 1992-09-10 | 2001-01-16 | Isis Pharmaceuticals, Inc. | Compositions and methods for treatment of hepatitis C virus-associated diseases |
| US6391542B1 (en) | 1992-09-10 | 2002-05-21 | 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 |
| CN1263473A (zh) | 1997-05-21 | 2000-08-16 | 利兰·斯坦福青年大学托管委员会 | 增加跨生物膜转运的组合物和方法 |
| 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 |
| KR20010102992A (ko) | 1999-01-29 | 2001-11-17 | 추후보정 | 표적 ｒｎａ를 검출하는 비-침입적 방법 |
| US7094765B1 (en) | 1999-01-29 | 2006-08-22 | Avi Biopharma, Inc. | Antisense restenosis composition and method |
| 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 |
| JP2003516151A (ja) | 1999-11-29 | 2003-05-13 | エイブイアイ バイオファーマ， インコーポレイテッド | 細菌１６Ｓおよび２３ＳｒＲＮＡに標的化された、荷電していないアンチセンスオリゴヌクレオチド、ならびにその使用 |
| AU781676B2 (en) | 2000-01-04 | 2005-06-02 | 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 |
| EP1301525B1 (en) | 2000-07-06 | 2015-09-02 | Sarepta Therapeutics, Inc. | Transforming growth factor beta (tgf-beta) blocking agent-treated stem cell composition and method |
| 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 |
| NZ530101A (en) | 2001-05-17 | 2007-01-26 | Avi Biopharma Inc | Methods for treating cancer by administering an oligomer antisense to c-myc together with a cancer chemotherapeutic agent |
| AU2002317437A1 (en) | 2001-05-18 | 2002-12-03 | Cureon A/S | Therapeutic uses of lna-modified oligonucleotides in infectious diseases |
| JP2005507660A (ja) | 2001-10-16 | 2005-03-24 | アビ バイオファーマ， インコーポレイテッド | ｓｓＲＮＡウイルス感染の処置のためのアンチセンス抗ウイルス剤および方法 |
| US20030224353A1 (en) | 2001-10-16 | 2003-12-04 | Stein David A. | Antisense antiviral agent and method for treating ssRNA viral infection |
| BR0314236A (pt) | 2002-09-13 | 2005-08-09 | Replicor Inc | Formulação de oligonucleotìdeo, composição farmacêutica, kit, composto antiviral, preparação de oligonucleotìdeo e métodos para seleção de um oligonucleotìdeo antiviral para uso como um agente antiviral, para profilaxia ou tratamento de uma infecção viral em um paciente, para tratamento profilático de câncer causado por oncovìrus, para identificação de um composto que altera a ligação de um oligonucleotìdeo a pelo menos um componente viral, para purificação da ligação de oligonucleotìdeos a pelo menos um componente viral e para enriquecimento de oligonucleotìdeos a partir de um agrupamento de oligonucleotìdeos |
| US7115374B2 (en) | 2002-10-16 | 2006-10-03 | 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 |
| JP4791043B2 (ja) | 2002-12-31 | 2011-10-12 | プロリゴ・エルエルシー | 同一固体支持体上で、２以上のオリゴヌクレオチドをタンデムに合成するための方法および組成物 |
| 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 |
| CA2533218C (en) | 2003-08-05 | 2014-11-18 | Avi Biopharma, Inc. | Oligonucleotide analog and method for treating flavivirus infections |
| CA2542776A1 (en) | 2003-10-27 | 2005-05-12 | Genelabs Technologies, Inc. | Nucleoside compounds for treating viral infections |
| US20050171044A1 (en) | 2003-12-24 | 2005-08-04 | Stein David A. | Oligonucleotide compound and method for treating nidovirus infections |
| CA2553104A1 (en) | 2004-01-23 | 2005-08-11 | Avi Biopharma, Inc. | Antisense oligomers and methods for inducing immune tolerance 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 |
| 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 |
| 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 |
| 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 |
| EP3470072A1 (en) | 2005-06-23 | 2019-04-17 | Biogen MA Inc. | Compositions and methods for modulation of smn2 splicing |
| US7790694B2 (en) | 2005-07-13 | 2010-09-07 | Avi Biopharma Inc. | Antisense antibacterial method and compound |
| WO2007009094A2 (en) * | 2005-07-13 | 2007-01-18 | Avi Biopharma, Inc. | Antisense antibacterial method and compound |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| US8524676B2 (en) | 2005-09-08 | 2013-09-03 | Sarepta Therapeutics, Inc. | Method for treating enterovirus or rhinovirus infection using antisense antiviral compounds |
| WO2007030576A2 (en) | 2005-09-08 | 2007-03-15 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating picornavirus infection |
| US7582615B2 (en) | 2006-03-07 | 2009-09-01 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating arenavirus infection |
| PL2735568T3 (pl) * | 2006-05-10 | 2018-02-28 | Sarepta Therapeutics, Inc. | Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami |
| US8785407B2 (en) | 2006-05-10 | 2014-07-22 | Sarepta Therapeutics, Inc. | Antisense antiviral agent and method for treating ssRNA viral infection |
| 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 |
| US8119686B2 (en) | 2006-11-24 | 2012-02-21 | Hykes Laboratories Llc | Spiroquinone compound and pharmaceutical composition |
| WO2009005793A2 (en) | 2007-06-29 | 2009-01-08 | Avi Biopharma, 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 |
| US8076476B2 (en) | 2007-11-15 | 2011-12-13 | Avi Biopharma, Inc. | Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits |
| RU2606627C2 (ru) | 2007-11-15 | 2017-01-10 | Серепта Терапьютикс,Инк. | Способ синтеза морфолиновых олигомеров |
| 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 |
| 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 |
| WO2010120820A1 (en) | 2009-04-13 | 2010-10-21 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing |
| ES2699827T3 (es) | 2009-06-17 | 2019-02-13 | Biogen Ma Inc | Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto |
| US20110269665A1 (en) | 2009-06-26 | 2011-11-03 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| EP2499248B1 (en) | 2009-11-13 | 2017-01-04 | Sarepta Therapeutics, Inc. | Antisense antiviral compound and method for treating influenza viral infection |
| US9068185B2 (en) | 2010-03-12 | 2015-06-30 | Sarepta Therapeutics, Inc. | Antisense modulation of nuclear hormone receptors |
| CA3090304A1 (en) * | 2010-05-13 | 2011-11-17 | Sarepta Therapeutics, Inc. | Antisense modulation of interleukins 17 and 23 signaling |
| 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 |
| US10017763B2 (en) | 2010-09-03 | 2018-07-10 | Sarepta Therapeutics, Inc. | dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups |
| ES2607603T3 (es) * | 2010-09-30 | 2017-04-03 | Nippon Shinyaku Co., Ltd. | Derivado de ácido morfolino nucleico |
| AU2011326364B2 (en) | 2010-11-12 | 2016-08-11 | Sarepta Therapeutics, Inc. | Antisense antibacterial compounds and methods |
| US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| US10100305B2 (en) | 2011-07-15 | 2018-10-16 | Sarepta Therapeutics, Inc. | Methods and compositions for manipulating translation of protein isoforms from alternative initiation of start sites |
| HK1201514A1 (en) | 2011-11-18 | 2015-09-04 | Sarepta Therapeutics, Inc. | Functionally-modified oligonucleotides and subunits thereof |
| ES2935606T3 (es) | 2011-12-08 | 2023-03-08 | Sarepta Therapeutics Inc | Análogos de oligonucleótidos dirigidos a LMNA humana |
| CN104411831B (zh) | 2012-03-20 | 2020-08-11 | 萨勒普塔医疗公司 | 寡核苷酸类似物的硼酸缀合物 |
| 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 CN CN201710590274.4A patent/CN107353317A/zh active Pending
- 2011-05-27 AU AU2011257980A patent/AU2011257980B2/en not_active Ceased
- 2011-05-27 KR KR1020187003799A patent/KR101981705B1/ko not_active Expired - Fee Related
- 2011-05-27 JP JP2013512062A patent/JP2013530154A/ja not_active Withdrawn
- 2011-05-27 BR BR112012031363-0A patent/BR112012031363A2/pt not_active Application Discontinuation
- 2011-05-27 EP EP11725839.2A patent/EP2576574A2/en not_active Withdrawn
- 2011-05-27 TW TW100118810A patent/TWI616454B/zh not_active IP Right Cessation
- 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 TW TW105121412A patent/TWI620756B/zh not_active IP Right Cessation
- 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 KR KR1020207008683A patent/KR102182663B1/ko not_active Expired - Fee Related
- 2011-05-27 US US13/118,298 patent/US8779128B2/en active Active
- 2011-05-27 CN CN201180034599.5A patent/CN103003288B/zh not_active Expired - Fee Related
- 2011-05-27 NZ NZ603606A patent/NZ603606A/en not_active IP Right Cessation
-
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=) | ||
| AU2021202224A1 (en) | Peptide oligonucleotide conjugates | |
| AU2016234953B2 (en) | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups | |
| AU2017282459B2 (en) | Cationic lipid | |
| JP2018511590A5 (https=) | ||
| US10947193B2 (en) | Cationic lipid | |
| JP2022505234A5 (https=) | ||
| JP2020525429A5 (https=) | ||
| 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 | |
| JP2015501817A5 (https=) | ||
| JP2019513734A5 (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 | |
| EP2581376A4 (en) | MATRIC ACID / MATRIN DERIVATIVES AND MANUFACTURING METHOD AND USES THEREOF | |
| JP2012510502A5 (https=) | ||
| JP2010527913A5 (https=) | ||
| JP2013505327A5 (https=) | ||
| JP2020514299A5 (https=) | ||
| JP2011500656A (ja) | カチオン性脂質 | |
| JP2011510009A5 (https=) |