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- Withdrawn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 (enExample) | 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 (enExample) |
| EP (1) | EP2576574A2 (enExample) |
| JP (5) | JP2013530154A (enExample) |
| KR (5) | KR102182663B1 (enExample) |
| CN (2) | CN107353317A (enExample) |
| AU (2) | AU2011257980B2 (enExample) |
| BR (1) | BR112012031363A2 (enExample) |
| CA (1) | CA2799501C (enExample) |
| IL (4) | IL223298B (enExample) |
| NZ (1) | NZ603606A (enExample) |
| TW (2) | TWI616454B (enExample) |
| WO (1) | WO2011150408A2 (enExample) |
Families Citing this family (92)
| 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 |
| TR201902952T4 (tr) | 2008-10-24 | 2019-03-21 | Sarepta Therapeutics Inc | Dmd için ekson atlama bileşimleri. |
| CA2780563C (en) | 2009-11-12 | 2025-05-06 | Univ Western Australia | Antisense Molecules and Methods for Treating Pathologies |
| EP2545173A2 (en) * | 2010-03-12 | 2013-01-16 | Sarepta Therapeutics, Inc. | Antisense modulation of nuclear hormone receptors |
| EP2576574A2 (en) | 2010-05-28 | 2013-04-10 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups |
| 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 |
| 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 |
| CN108864192A (zh) * | 2011-11-18 | 2018-11-23 | 萨勒普塔医疗公司 | 功能改性的寡核苷酸及其亚单元 |
| ES2727481T3 (es) | 2011-11-30 | 2019-10-16 | Sarepta Therapeutics Inc | Inclusión inducida de exón en atrofia muscular espinal |
| US10066228B2 (en) | 2011-11-30 | 2018-09-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| WO2013086441A2 (en) | 2011-12-08 | 2013-06-13 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues targeting human lmna |
| CN107881175B (zh) * | 2011-12-28 | 2022-05-13 | 日本新药株式会社 | 反义核酸 |
| 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 |
| US9920085B2 (en) * | 2012-03-20 | 2018-03-20 | Sarepta Therapeutics, Inc. | Boronic acid conjugates of oligonucleotide analogues |
| BR112014026285B1 (pt) | 2012-04-23 | 2021-08-31 | Biomarin Technologies B.V | Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento de distúrbios neuromusculares |
| EP3608407A1 (en) | 2012-07-03 | 2020-02-12 | BioMarin Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
| WO2014011053A1 (en) | 2012-07-12 | 2014-01-16 | Proqr Therapeutics 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 |
| WO2014100714A1 (en) | 2012-12-20 | 2014-06-26 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
| US9856474B2 (en) | 2013-01-16 | 2018-01-02 | Iowa State University Research Foundation, Inc. | Deep intronic target for splicing correction on spinal muscular atrophy gene |
| HUE042218T2 (hu) | 2013-03-14 | 2019-06-28 | Sarepta Therapeutics Inc | Exonátugrást elõsegítõ készítmények izomdisztrófia kezelésére |
| NZ775701A (en) * | 2013-03-14 | 2022-08-26 | Sarepta Therapeutics Inc | Exon skipping compositions for treating muscular dystrophy |
| NZ732507A (en) | 2013-03-15 | 2018-08-31 | Sarepta Therapeutics Inc | Improved compositions for treating muscular dystrophy |
| CN105793422B (zh) | 2013-09-05 | 2020-03-03 | 萨罗塔治疗公司(美国) | 酸性α-葡糖苷酶中反义诱导的外显子2纳入 |
| TR201901939T4 (tr) * | 2014-03-12 | 2019-03-21 | Nat Center Neurology & Psychiatry | Antisens nükleik asit. |
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| US20180216111A1 (en) * | 2015-02-27 | 2018-08-02 | 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 |
| WO2016196670A1 (en) | 2015-06-01 | 2016-12-08 | Sarepta Therapeutics, Inc. | Antisense-induced exon exclusion in type vii collagen |
| US11020417B2 (en) | 2015-06-04 | 2021-06-01 | Sarepta Therapeutics, Inc | Methods and compounds for treatment of lymphocyte-related diseases and conditions |
| PE20231843A1 (es) * | 2015-08-05 | 2023-11-21 | Eisai Randd Man Co Ltd | Reactivos quirales para la preparacion de oligomeros homogeneos |
| AU2016317667A1 (en) | 2015-08-28 | 2018-03-22 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
| BR112018006445A2 (pt) | 2015-09-30 | 2018-12-11 | Sarepta Therapeutics Inc | métodos para tratar distrofia muscular |
| MA45819A (fr) | 2015-10-09 | 2018-08-15 | Sarepta Therapeutics Inc | Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés |
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
| EP3445405A4 (en) | 2016-04-18 | 2019-12-18 | Sarepta Therapeutics, Inc. | ANTISENSE OLIGOMERS AND METHOD FOR USE THEREOF TO TREAT DISEASES RELATING TO 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 |
| EP3464305B1 (en) * | 2016-05-24 | 2024-08-21 | Sarepta Therapeutics, Inc. | Processes for preparing oligomers |
| HRP20240583T1 (hr) | 2016-05-24 | 2024-07-19 | Sarepta Therapeutics, Inc. | Postupci za pripremu fosforodiamidat morfolino oligomera |
| AU2017270598B2 (en) * | 2016-05-24 | 2022-12-01 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| IL263044B2 (en) * | 2016-05-24 | 2024-06-01 | Sarepta Therapeutics Inc | Processes for preparing phosphorodiamidate morpholino oligomers |
| MA45362A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| KR20190024977A (ko) * | 2016-06-30 | 2019-03-08 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 |
| KR102523527B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| EP3515445A4 (en) * | 2016-09-20 | 2020-05-27 | The Regents of The University of Colorado, A Body Corporate | SYNTHESIS OF BACKBONE-MODIFIED MORPHOLINO-OLIGONUCLEOTIDES AND CHIMERES WITH THE AID OF PHOSPHORAMIDITE CHEMISTRY |
| EP3330276A1 (en) | 2016-11-30 | 2018-06-06 | Universität Bern | Novel bicyclic nucleosides and oligomers prepared therefrom |
| MD3554553T2 (ro) | 2016-12-19 | 2022-10-31 | Sarepta Therapeutics Inc | Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară |
| WO2018118627A1 (en) * | 2016-12-19 | 2018-06-28 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| WO2018129384A1 (en) | 2017-01-06 | 2018-07-12 | Avidity Biosciences Llc | Nucleic acid-polypeptide compositions and methods of inducing exon skipping |
| 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 |
| SG11202003126VA (en) | 2017-10-04 | 2020-05-28 | Avidity Biosciences Inc | Nucleic acid-polypeptide compositions and uses thereof |
| US11555189B2 (en) | 2017-10-18 | 2023-01-17 | Sarepta Therapeutics, Inc. | Antisense oligomer compounds |
| CN118667812A (zh) | 2017-12-06 | 2024-09-20 | 艾维迪提生物科学公司 | 治疗肌萎缩和强直性肌营养不良的组合物和方法 |
| US12319667B2 (en) | 2018-01-19 | 2025-06-03 | Synthena Ag | Tricyclo-DNA nucleoside precursors and processes for preparing the same |
| SG11202010841QA (en) | 2018-05-11 | 2020-11-27 | Alpha Anomeric Sas | Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides |
| US20210308273A1 (en) | 2018-08-02 | 2021-10-07 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| 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 |
| JP2021533200A (ja) | 2018-08-02 | 2021-12-02 | ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. | 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| IL319265A (en) | 2018-12-21 | 2025-04-01 | Avidity Biosciences Inc | Anti-transferrin receptor antibodies and their uses |
| WO2020231206A1 (ko) | 2019-05-15 | 2020-11-19 | 주식회사 엘지화학 | 배터리 팩 및 이를 포함하는 전력 저장 장치 |
| KR20220019259A (ko) | 2019-06-06 | 2022-02-16 | 어비디티 바이오사이언시스 인크. | Una 아미다이트 및 이의 용도 |
| 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 |
| EP4065225A1 (en) * | 2019-11-27 | 2022-10-05 | DTX Pharma, Inc. | Compounds and methods for the treatment of duchenne muscular dystrophy |
| AR121446A1 (es) | 2020-02-28 | 2022-06-08 | Ionis Pharmaceuticals Inc | Compuestos y métodos para modular smn2 |
| EP4121063A4 (en) | 2020-03-19 | 2024-07-03 | Avidity Biosciences, Inc. | COMPOSITIONS AND METHODS FOR TREATING FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY |
| JP7748962B2 (ja) | 2020-03-27 | 2025-10-03 | アビディティー バイオサイエンシーズ，インク． | 筋ジストロフィーを処置するための組成物および方法 |
| 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 |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| EP4366784A1 (en) | 2021-07-09 | 2024-05-15 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| KR20240055874A (ko) | 2021-09-16 | 2024-04-29 | 어비디티 바이오사이언시스 인크. | 안면견갑상완 근이영양증을 치료하는 조성물 및 방법 |
| KR20240070615A (ko) | 2021-09-30 | 2024-05-21 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 하나 이상의 비염기성 유닛을 갖는 안티센스 올리고뉴클레오티드 |
| 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 |
| EP4470543A1 (en) | 2022-01-25 | 2024-12-04 | Kawasaki Institute of Industrial Promotion | Rna-containing composition for transdermal administration, and method for administering said composition |
| 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 |
| AU2024244071A1 (en) | 2023-04-06 | 2025-10-30 | Shanghai Argo Biopharmaceutical Co., Ltd. | Nucleoside analog for 5'-phosphonate modification and oligonucleotide prepared therefrom |
| WO2025085810A2 (en) | 2023-10-18 | 2025-04-24 | Sarepta Therapeutics, Inc. | Antisense oligomers for treatment of centronuclear myopathies |
| WO2025124586A1 (zh) * | 2023-12-15 | 2025-06-19 | 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 |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| 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 |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| DE3687030T2 (de) | 1985-03-15 | 1993-03-11 | Eugene Stirchak | Stereoregulare polynukleotiden bindende polymere. |
| 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 |
| ATE312834T1 (de) * | 1989-12-20 | 2005-12-15 | Avi Biopharma Inc | Ungeladene, auf morpholin basierende polymere mit chiralen, phosphor enthaltenden brücken zwischen den untereinheiten |
| US5457191A (en) | 1990-01-11 | 1995-10-10 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5578718A (en) | 1990-01-11 | 1996-11-26 | Isis Pharmaceuticals, Inc. | Thiol-derivatized nucleosides |
| 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 |
| US5852188A (en) | 1990-01-11 | 1998-12-22 | Isis Pharmaceuticals, Inc. | Oligonucleotides having chiral phosphorus linkages |
| US5587361A (en) | 1991-10-15 | 1996-12-24 | Isis Pharmaceuticals, Inc. | Oligonucleotides having phosphorothioate linkages of high chiral purity |
| US5506351A (en) | 1992-07-23 | 1996-04-09 | Isis Pharmaceuticals | Process for the preparation of 2'-O-alkyl guanosine and related compounds |
| US5212295A (en) | 1990-01-11 | 1993-05-18 | Isis Pharmaceuticals | Monomers for preparation of oligonucleotides having chiral phosphorus linkages |
| US5610289A (en) | 1990-07-27 | 1997-03-11 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogues |
| 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 |
| US5138045A (en) | 1990-07-27 | 1992-08-11 | Isis Pharmaceuticals | Polyamine conjugated oligonucleotides |
| 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 |
| US5378825A (en) | 1990-07-27 | 1995-01-03 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs |
| US5602240A (en) | 1990-07-27 | 1997-02-11 | Ciba Geigy Ag. | Backbone modified oligonucleotide analogs |
| US6262241B1 (en) | 1990-08-13 | 2001-07-17 | Isis Pharmaceuticals, Inc. | Compound for detecting and modulating RNA activity and gene expression |
| BR9106747A (pt) | 1990-08-14 | 1993-07-20 | Isis Pharmaceuticals Inc | Oligonucleotideo ou analogo de oligonucleotideo e processo para tratamento de um animal suspeito de estar infectado por virus da influenza |
| US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
| JPH07501204A (ja) | 1991-06-28 | 1995-02-09 | マサチューセッツ インスティテュート オブ テクノロジー | 局所的オリゴヌクレオチド療法 |
| 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 |
| 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 |
| JP2000509394A (ja) | 1996-05-01 | 2000-07-25 | アンティバイラルズ インコーポレイテッド | 細胞膜を横切って物質を輸送するためのポリペプチド結合体 |
| 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 |
| AU734827B2 (en) | 1997-05-21 | 2001-06-21 | Board Of Trustees Of The Leland Stanford Junior University | 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 |
| EP1173561A2 (en) | 1999-01-29 | 2002-01-23 | Avi Biopharma, Inc. | Non-invasive method for detecting target rna |
| 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 |
| US6677153B2 (en) | 1999-11-29 | 2004-01-13 | Avi Biopharma, Inc. | Antisense antibacterial method and composition |
| EP1248813A2 (en) | 2000-01-04 | 2002-10-16 | 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 |
| US20040072743A1 (en) | 2000-04-06 | 2004-04-15 | Christensen Jeppe Viggo | Pharmaceutical composition of modified pna molecules |
| JP2003533986A (ja) | 2000-05-04 | 2003-11-18 | エイブイアイ バイオファーマ， インコーポレイテッド | スプライス領域アンチセンス組成物および方法 |
| 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 |
| WO2002034771A2 (en) | 2000-10-27 | 2002-05-02 | Chiron Srl | 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 |
| CA2447052A1 (en) | 2001-05-17 | 2002-11-21 | Avi Biopharma, Inc. | Combined approach to treatment of cancer using a c-myc antisense oligomer |
| AU2002317437A1 (en) | 2001-05-18 | 2002-12-03 | Cureon A/S | Therapeutic uses of lna-modified oligonucleotides in infectious diseases |
| US6828105B2 (en) | 2001-10-16 | 2004-12-07 | Avi Biopharma, Inc. | Antisense antiviral agent and method for treating ssRNA viral infection |
| US20030224353A1 (en) | 2001-10-16 | 2003-12-04 | Stein David A. | Antisense antiviral agent and method for treating ssRNA viral infection |
| AU2003267785C1 (en) | 2002-09-13 | 2009-12-24 | Replicor, Inc. | Non-sequence complementary antiviral oligonucleotides |
| EP2977470B1 (en) | 2002-10-16 | 2018-01-03 | 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 |
| US7615629B2 (en) | 2002-12-31 | 2009-11-10 | Sigma-Aldrich Co. | Methods and compositions for the tandem synthesis of two or more oligonucleotides on the same solid support |
| EP2351844B1 (en) | 2003-04-29 | 2014-06-11 | Sarepta Therapeutics, Inc. | Compositions for enhancing transport and antisense efficacy of nucleic acid analog 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 |
| ATE491791T1 (de) * | 2003-08-05 | 2011-01-15 | Avi Biopharma Inc | Oligonukleotidanalog und verfahren zur behandlung von flavivirusinfektionen |
| US7157434B2 (en) | 2003-10-27 | 2007-01-02 | 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 |
| WO2005072527A2 (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 |
| ATE498685T1 (de) | 2004-06-28 | 2011-03-15 | Univ Western Australia | Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon |
| CA2875436A1 (en) | 2004-07-02 | 2006-08-17 | Sarepta Therapeutics, 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 |
| 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 |
| DK2548560T3 (en) | 2005-06-23 | 2015-07-13 | Cold Spring Harbor Lab | Compositions and Methods for Modulating SMN2 Splicing |
| 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 |
| US7790694B2 (en) | 2005-07-13 | 2010-09-07 | Avi Biopharma Inc. | Antisense antibacterial method and compound |
| WO2007030576A2 (en) | 2005-09-08 | 2007-03-15 | 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 |
| DE602007006457D1 (de) | 2006-03-07 | 2010-06-24 | Avi Biopharma Inc | Antivirale antisense-verbindung und verfahren zur behandlung von arenavirus-infektion |
| US8785407B2 (en) | 2006-05-10 | 2014-07-22 | Sarepta Therapeutics, Inc. | Antisense antiviral agent and method for treating ssRNA viral infection |
| ES2645410T3 (es) * | 2006-05-10 | 2017-12-05 | Sarepta Therapeutics, Inc. | Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos |
| WO2008018795A1 (en) | 2006-08-11 | 2008-02-14 | Prosensa Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
| EP2066812A4 (en) | 2006-09-21 | 2010-04-28 | Univ Rochester | COMPOSITIONS AND METHODS RELATED TO PROTEIN SHIFT THERAPY FOR MYOTONE 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 |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| JP5864100B2 (ja) | 2007-06-29 | 2016-02-17 | サレプタ セラピューティクス インコーポレイテッド | 組織特異的ペプチドコンジュゲートおよび方法 |
| WO2009064471A1 (en) | 2007-11-15 | 2009-05-22 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| 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 |
| TR201902952T4 (tr) | 2008-10-24 | 2019-03-21 | Sarepta Therapeutics Inc | Dmd için ekson atlama bileşimleri. |
| US20120149757A1 (en) | 2009-04-13 | 2012-06-14 | Krainer Adrian R | Compositions and methods for modulation of smn2 splicing |
| SI3449926T1 (sl) | 2009-06-17 | 2020-04-30 | Biogen Ma Inc. | Sestave in metode za modulacijo združevanja SMN2 pri subjektu |
| US20110269665A1 (en) | 2009-06-26 | 2011-11-03 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| WO2011060320A1 (en) | 2009-11-13 | 2011-05-19 | Avi Biopharma, 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 |
| CA3090304A1 (en) * | 2010-05-13 | 2011-11-17 | Sarepta Therapeutics, Inc. | Antisense modulation of interleukins 17 and 23 signaling |
| EP2576574A2 (en) | 2010-05-28 | 2013-04-10 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups |
| 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 |
| HUE030292T2 (en) | 2010-09-30 | 2017-04-28 | Nippon Shinyaku Co Ltd | Morpholino-nucleic acid derivatives |
| US20120122769A1 (en) | 2010-11-12 | 2012-05-17 | Avi Biopharma, 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 |
| CN108864192A (zh) | 2011-11-18 | 2018-11-23 | 萨勒普塔医疗公司 | 功能改性的寡核苷酸及其亚单元 |
| WO2013086441A2 (en) | 2011-12-08 | 2013-06-13 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues targeting human lmna |
| US9920085B2 (en) | 2012-03-20 | 2018-03-20 | Sarepta Therapeutics, Inc. | Boronic acid conjugates of oligonucleotide analogues |
| 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 EP EP11725839.2A patent/EP2576574A2/en not_active Withdrawn
- 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 KR KR1020197014303A patent/KR102095478B1/ko not_active Expired - Fee Related
- 2011-05-27 WO PCT/US2011/038459 patent/WO2011150408A2/en not_active Ceased
- 2011-05-27 AU AU2011257980A patent/AU2011257980B2/en not_active Ceased
- 2011-05-27 CN CN201710590274.4A patent/CN107353317A/zh active Pending
- 2011-05-27 TW TW100118810A patent/TWI616454B/zh not_active IP Right Cessation
- 2011-05-27 US US13/118,298 patent/US8779128B2/en active Active
- 2011-05-27 NZ NZ603606A patent/NZ603606A/en not_active IP Right Cessation
- 2011-05-27 JP JP2013512062A patent/JP2013530154A/ja not_active Withdrawn
- 2011-05-27 KR KR1020127033727A patent/KR101829644B1/ko not_active Expired - Fee Related
- 2011-05-27 BR BR112012031363-0A patent/BR112012031363A2/pt not_active Application Discontinuation
- 2011-05-27 CN CN201180034599.5A patent/CN103003288B/zh not_active Expired - Fee Related
- 2011-05-27 KR KR1020207033269A patent/KR20200133284A/ko 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
-
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 (enExample) | ||
| JP2014515762A5 (enExample) | ||
| AU2021202224A1 (en) | Peptide oligonucleotide conjugates | |
| AU2016234953B2 (en) | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups | |
| AU2017282459B2 (en) | Cationic lipid | |
| JP2019512478A5 (enExample) | ||
| JP2020525429A5 (enExample) | ||
| IL303195A (en) | Lipid nanoparticles for delivery of nucleic acids and related methods of use | |
| JP2018035142A5 (enExample) | ||
| JP7164547B2 (ja) | カチオン性脂質 | |
| CY1119827T1 (el) | Ακυλαμινο-υποκατεστημενα συντηγμενα παραγωγα κυκλοπεντανο καρβοξυλικου οξεος και η χρηση αυτων ως φαρμακευτικων ουσιων | |
| JP2017538682A5 (enExample) | ||
| JP2019513734A5 (enExample) | ||
| JP2017536344A5 (enExample) | ||
| JP2017515901A5 (enExample) | ||
| RU2015100198A (ru) | Хиральный адъювант нуклеиновой кислоты | |
| DK2806896T3 (en) | Chitosan covalently bound to small molecule integrin antagonist for targeted delivery | |
| JP2012510502A5 (enExample) | ||
| JP2010527913A5 (enExample) | ||
| JP2020514299A5 (enExample) | ||
| JP2011500656A (ja) | カチオン性脂質 | |
| JP2011510009A5 (enExample) | ||
| JP2019518029A5 (enExample) | ||
| RU2016143395A (ru) | Новые гидротропы на основе полимера для доставки гидрофобного лекарственного средства | |
| EP4622673A1 (en) | Novel carriers for nucleic acid and/or protein delivery |