JP2013530154A5 - - Google Patents
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- 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
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- Prior art keywords
- oligomer
- independently
- alkyl
- hydrogen
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- Prior art date
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- 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 (OSRAM) | 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 (OSRAM) |
| EP (1) | EP2576574A2 (OSRAM) |
| JP (5) | JP2013530154A (OSRAM) |
| KR (5) | KR20200133284A (OSRAM) |
| CN (2) | CN107353317A (OSRAM) |
| AU (2) | AU2011257980B2 (OSRAM) |
| BR (1) | BR112012031363A2 (OSRAM) |
| CA (1) | CA2799501C (OSRAM) |
| IL (4) | IL223298B (OSRAM) |
| NZ (1) | NZ603606A (OSRAM) |
| TW (2) | TWI620756B (OSRAM) |
| WO (1) | WO2011150408A2 (OSRAM) |
Families Citing this family (93)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| USRE47769E1 (en) | 2004-06-28 | 2019-12-17 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| CA2596506C (en) | 2005-02-09 | 2021-04-06 | Avi Biopharma, 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. |
| WO2011057350A1 (en) | 2009-11-12 | 2011-05-19 | The University Of Western Australia | Antisense molecules and methods for treating pathologies |
| US9068185B2 (en) * | 2010-03-12 | 2015-06-30 | Sarepta Therapeutics, Inc. | Antisense modulation of nuclear hormone receptors |
| CA2799501C (en) | 2010-05-28 | 2022-02-15 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups |
| 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 |
| AU2012284259A1 (en) | 2011-07-15 | 2014-03-06 | Sarepta Therapeutics, Inc. | Methods and compositions for manipulating translation of protein isoforms from alternative initiation start sites |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| EA035030B1 (ru) * | 2011-11-18 | 2020-04-20 | Сарепта Терапьютикс, Инк. | Модифицированные морфолиновые аналоги олигонуклеотидов |
| JP6317675B2 (ja) * | 2011-11-30 | 2018-04-25 | サレプタ セラピューティクス， インコーポレイテッド | 延長リピート病を処置するためのオリゴヌクレオチド |
| ES2727481T3 (es) | 2011-11-30 | 2019-10-16 | Sarepta Therapeutics Inc | Inclusión inducida de exón en atrofia muscular espinal |
| WO2013086444A2 (en) | 2011-12-08 | 2013-06-13 | Sarepta Therapeutics, Inc. | Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human lmna |
| IN2014DN06220A (OSRAM) | 2011-12-28 | 2015-10-23 | Nippon Shinyaku Co Ltd | |
| CN111893117B (zh) | 2012-01-27 | 2024-06-04 | 比奥马林技术公司 | 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
| EP2828395B1 (en) * | 2012-03-20 | 2018-10-24 | Sarepta Therapeutics, Inc. | Boronic acid conjugates of oligonucleotide analogues |
| HRP20171254T1 (hr) | 2012-04-23 | 2018-01-12 | Biomarin Technologies B.V. | Oligonukleotidi koji moduliraju rnk s poboljšanim karakteristikama za liječenje neuromišićnih poremećaja |
| RU2674600C2 (ru) | 2012-07-03 | 2018-12-11 | Просенса Текнолоджиз Б.В. | Олигонуклеотид для лечения пациентов с мышечной дистрофией |
| NZ703824A (en) | 2012-07-12 | 2018-06-29 | Proqr Therapeutics Ii Bv | 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 |
| BR112015014987A2 (pt) | 2012-12-20 | 2017-08-15 | Sarepta Therapeutics Inc | Aprimoradas composições que pulam éxon para o tratamento da distrofia muscular |
| 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 |
| SMT201900139T1 (it) | 2013-03-14 | 2019-05-10 | Sarepta Therapeutics Inc | Composizioni di salto dell'esone per il trattamento della distrofia muscolare |
| US9217148B2 (en) * | 2013-03-14 | 2015-12-22 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| MX373959B (es) | 2013-03-15 | 2020-07-13 | Sarepta Therapeutics Inc | Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd). |
| MX384377B (es) | 2013-09-05 | 2025-03-14 | Sarepta Therapeutics Inc | Inclusión del exón 2 inducida por antisentido en la alfa-glucosidasa ácida. |
| EP3514234A1 (en) | 2014-03-12 | 2019-07-24 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid |
| WO2016040748A1 (en) | 2014-09-12 | 2016-03-17 | Ionis Pharmaceuticals, Inc. | Compositions and methods for detection of smn protein in a subject and treatment of a subject |
| MA41759A (fr) * | 2015-02-27 | 2018-01-03 | Univ Murdoch | Inclusion de l'exon 2, induite par antisens, dans une alpha-glucosidase acide |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| HK1253335A1 (zh) | 2015-06-01 | 2019-06-14 | Sarepta Therapeutics, Inc. | 在vii型胶原蛋白中反义诱导外显子的排除 |
| US11020417B2 (en) | 2015-06-04 | 2021-06-01 | Sarepta Therapeutics, Inc | Methods and compounds for treatment of lymphocyte-related diseases and conditions |
| MA44209B1 (fr) * | 2015-08-05 | 2022-03-31 | Eisai R&D Man Co Ltd | Procede de preparation d'un oligomere phosphorodiamidate substantiellement diastereomeriquement pure, un oligomere phosphorodiamidate faite par un tel procede et une composition pharmaceutique comprenant un tel oligomere phosphorodiamidate |
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- 2011-05-27 CN CN201710590274.4A patent/CN107353317A/zh active Pending
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