JP2016516066A5 - - Google Patents
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- JP2016516066A5 JP2016516066A5 JP2016503163A JP2016503163A JP2016516066A5 JP 2016516066 A5 JP2016516066 A5 JP 2016516066A5 JP 2016503163 A JP2016503163 A JP 2016503163A JP 2016503163 A JP2016503163 A JP 2016503163A JP 2016516066 A5 JP2016516066 A5 JP 2016516066A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- patient
- dmd
- muscular dystrophy
- duchenne muscular
- 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.)
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- 239000000203 mixture Substances 0.000 claims description 68
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 52
- 238000011282 treatment Methods 0.000 claims description 24
- 108010069091 Dystrophin Proteins 0.000 claims description 13
- 229940124624 oral corticosteroid Drugs 0.000 claims description 10
- 230000005021 gait Effects 0.000 claims description 7
- 238000012360 testing method Methods 0.000 claims description 7
- 210000003019 respiratory muscle Anatomy 0.000 claims description 5
- 201000010099 disease Diseases 0.000 claims description 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 3
- 230000003434 inspiratory effect Effects 0.000 claims description 3
- 238000004519 manufacturing process Methods 0.000 claims description 3
- 230000035772 mutation Effects 0.000 claims 11
- 108090000623 proteins and genes Proteins 0.000 claims 10
- 230000004199 lung function Effects 0.000 claims 6
- 230000004220 muscle function Effects 0.000 claims 4
- 102000001039 Dystrophin Human genes 0.000 claims 3
- 239000003246 corticosteroid Substances 0.000 claims 3
- 230000009325 pulmonary function Effects 0.000 claims 3
- JYGXADMDTFJGBT-VWUMJDOOSA-N hydrocortisone Chemical compound O=C1CC[C@]2(C)[C@H]3[C@@H](O)C[C@](C)([C@@](CC4)(O)C(=O)CO)[C@@H]4[C@@H]3CCC2=C1 JYGXADMDTFJGBT-VWUMJDOOSA-N 0.000 claims 2
- 230000003137 locomotive effect Effects 0.000 claims 2
- 238000003757 reverse transcription PCR Methods 0.000 claims 2
- 230000014616 translation Effects 0.000 claims 2
- FUFLCEKSBBHCMO-UHFFFAOYSA-N 11-dehydrocorticosterone Natural products O=C1CCC2(C)C3C(=O)CC(C)(C(CC4)C(=O)CO)C4C3CCC2=C1 FUFLCEKSBBHCMO-UHFFFAOYSA-N 0.000 claims 1
- VHRSUDSXCMQTMA-PJHHCJLFSA-N 6alpha-methylprednisolone Chemical compound C([C@@]12C)=CC(=O)C=C1[C@@H](C)C[C@@H]1[C@@H]2[C@@H](O)C[C@]2(C)[C@@](O)(C(=O)CO)CC[C@H]21 VHRSUDSXCMQTMA-PJHHCJLFSA-N 0.000 claims 1
- VOVIALXJUBGFJZ-KWVAZRHASA-N Budesonide Chemical compound C1CC2=CC(=O)C=C[C@]2(C)[C@@H]2[C@@H]1[C@@H]1C[C@H]3OC(CCC)O[C@@]3(C(=O)CO)[C@@]1(C)C[C@@H]2O VOVIALXJUBGFJZ-KWVAZRHASA-N 0.000 claims 1
- MFYSYFVPBJMHGN-ZPOLXVRWSA-N Cortisone Chemical compound O=C1CC[C@]2(C)[C@H]3C(=O)C[C@](C)([C@@](CC4)(O)C(=O)CO)[C@@H]4[C@@H]3CCC2=C1 MFYSYFVPBJMHGN-ZPOLXVRWSA-N 0.000 claims 1
- MFYSYFVPBJMHGN-UHFFFAOYSA-N Cortisone Natural products O=C1CCC2(C)C3C(=O)CC(C)(C(CC4)(O)C(=O)CO)C4C3CCC2=C1 MFYSYFVPBJMHGN-UHFFFAOYSA-N 0.000 claims 1
- 229960002537 betamethasone Drugs 0.000 claims 1
- UREBDLICKHMUKA-DVTGEIKXSA-N betamethasone Chemical compound C1CC2=CC(=O)C=C[C@]2(C)[C@]2(F)[C@@H]1[C@@H]1C[C@H](C)[C@@](C(=O)CO)(O)[C@@]1(C)C[C@@H]2O UREBDLICKHMUKA-DVTGEIKXSA-N 0.000 claims 1
- 229960004436 budesonide Drugs 0.000 claims 1
- 229960004544 cortisone Drugs 0.000 claims 1
- 238000001514 detection method Methods 0.000 claims 1
- 229960003957 dexamethasone Drugs 0.000 claims 1
- UREBDLICKHMUKA-CXSFZGCWSA-N dexamethasone Chemical compound C1CC2=CC(=O)C=C[C@]2(C)[C@]2(F)[C@@H]1[C@@H]1C[C@@H](C)[C@@](C(=O)CO)(O)[C@@]1(C)C[C@@H]2O UREBDLICKHMUKA-CXSFZGCWSA-N 0.000 claims 1
- 101150015424 dmd gene Proteins 0.000 claims 1
- 229960000890 hydrocortisone Drugs 0.000 claims 1
- 238000003364 immunohistochemistry Methods 0.000 claims 1
- 230000006698 induction Effects 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 229960004584 methylprednisolone Drugs 0.000 claims 1
- 229960005205 prednisolone Drugs 0.000 claims 1
- OIGNJSKKLXVSLS-VWUMJDOOSA-N prednisolone Chemical compound O=C1C=C[C@]2(C)[C@H]3[C@@H](O)C[C@](C)([C@@](CC4)(O)C(=O)CO)[C@@H]4[C@@H]3CCC2=C1 OIGNJSKKLXVSLS-VWUMJDOOSA-N 0.000 claims 1
- 229960004618 prednisone Drugs 0.000 claims 1
- XOFYZVNMUHMLCC-ZPOLXVRWSA-N prednisone Chemical compound O=C1C=C[C@]2(C)[C@H]3C(=O)C[C@](C)([C@@](CC4)(O)C(=O)CO)[C@@H]4[C@@H]3CCC2=C1 XOFYZVNMUHMLCC-ZPOLXVRWSA-N 0.000 claims 1
- 238000001262 western blot Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 30
- 125000002147 dimethylamino group Chemical group [H]C([H])([H])N(*)C([H])([H])[H] 0.000 description 28
- 108091034117 Oligonucleotide Proteins 0.000 description 14
- 239000000074 antisense oligonucleotide Substances 0.000 description 14
- 238000012230 antisense oligonucleotides Methods 0.000 description 14
- 239000002773 nucleotide Substances 0.000 description 8
- 125000003729 nucleotide group Chemical group 0.000 description 8
- 102100024108 Dystrophin Human genes 0.000 description 6
- 238000012217 deletion Methods 0.000 description 6
- 230000037430 deletion Effects 0.000 description 6
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 description 4
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 description 4
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 description 3
- LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical compound P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 description 3
- 239000003937 drug carrier Substances 0.000 description 3
- 239000002953 phosphate buffered saline Substances 0.000 description 3
- -1 2-hydroxyethoxy Chemical group 0.000 description 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 description 2
- 108091028043 Nucleic acid sequence Proteins 0.000 description 2
- 230000000692 anti-sense effect Effects 0.000 description 2
- 230000007812 deficiency Effects 0.000 description 2
- 230000001934 delay Effects 0.000 description 2
- 239000003814 drug Substances 0.000 description 2
- 125000001301 ethoxy group Chemical group [H]C([H])([H])C([H])([H])O* 0.000 description 2
- 239000000835 fiber Substances 0.000 description 2
- 238000009472 formulation Methods 0.000 description 2
- 238000011947 six minute walk test Methods 0.000 description 2
- 230000001225 therapeutic effect Effects 0.000 description 2
- 229940113082 thymine Drugs 0.000 description 2
- 229940035893 uracil Drugs 0.000 description 2
- 239000004475 Arginine Substances 0.000 description 1
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 1
- 239000000654 additive Substances 0.000 description 1
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 description 1
- 125000002915 carbonyl group Chemical group [*:2]C([*:1])=O 0.000 description 1
- 230000004700 cellular uptake Effects 0.000 description 1
- 239000003795 chemical substances by application Substances 0.000 description 1
- 230000000295 complement effect Effects 0.000 description 1
- 150000001875 compounds Chemical class 0.000 description 1
- 239000003085 diluting agent Substances 0.000 description 1
- 229940079593 drug Drugs 0.000 description 1
- 230000004064 dysfunction Effects 0.000 description 1
- 230000000694 effects Effects 0.000 description 1
- 238000011156 evaluation Methods 0.000 description 1
- 238000005259 measurement Methods 0.000 description 1
- 239000008194 pharmaceutical composition Substances 0.000 description 1
- 239000000902 placebo Substances 0.000 description 1
- 229940068196 placebo Drugs 0.000 description 1
- 108090000765 processed proteins & peptides Proteins 0.000 description 1
- 230000000750 progressive effect Effects 0.000 description 1
- 230000004202 respiratory function Effects 0.000 description 1
- 239000011780 sodium chloride Substances 0.000 description 1
- 239000000126 substance Substances 0.000 description 1
- 230000003519 ventilatory effect Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361793463P | 2013-03-15 | 2013-03-15 | |
| US61/793,463 | 2013-03-15 | ||
| PCT/US2014/029610 WO2014144978A2 (en) | 2013-03-15 | 2014-03-14 | Improved compositions for treating muscular dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019030772A Division JP2019108349A (ja) | 2013-03-15 | 2019-02-22 | 筋ジストロフィを処置するための改善された組成物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016516066A JP2016516066A (ja) | 2016-06-02 |
| JP2016516066A5 true JP2016516066A5 (enExample) | 2017-04-20 |
Family
ID=50678303
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016503163A Withdrawn JP2016516066A (ja) | 2013-03-15 | 2014-03-14 | 筋ジストロフィを処置するための改善された組成物 |
| JP2019030772A Pending JP2019108349A (ja) | 2013-03-15 | 2019-02-22 | 筋ジストロフィを処置するための改善された組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019030772A Pending JP2019108349A (ja) | 2013-03-15 | 2019-02-22 | 筋ジストロフィを処置するための改善された組成物 |
Country Status (14)
| Country | Link |
|---|---|
| US (8) | US20140329762A1 (enExample) |
| EP (2) | EP2968991A2 (enExample) |
| JP (2) | JP2016516066A (enExample) |
| KR (2) | KR20150133768A (enExample) |
| CN (2) | CN113633787A (enExample) |
| AU (3) | AU2014233456B2 (enExample) |
| BR (1) | BR112015022998A2 (enExample) |
| CA (1) | CA2906812A1 (enExample) |
| EA (1) | EA201591792A1 (enExample) |
| HK (1) | HK1220154A1 (enExample) |
| IL (3) | IL241558B (enExample) |
| MX (2) | MX373959B (enExample) |
| NZ (2) | NZ732507A (enExample) |
| WO (1) | WO2014144978A2 (enExample) |
Families Citing this family (87)
| 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 |
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| WO2011057350A1 (en) | 2009-11-12 | 2011-05-19 | The University Of Western Australia | Antisense molecules and methods for treating pathologies |
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| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| EP2841572B1 (en) | 2012-04-27 | 2019-06-19 | Duke University | Genetic correction of mutated genes |
| JP6449231B2 (ja) | 2013-03-14 | 2019-01-09 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
| JP2016516066A (ja) | 2013-03-15 | 2016-06-02 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィを処置するための改善された組成物 |
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| JP6449231B2 (ja) | 2013-03-14 | 2019-01-09 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
| KR20240094032A (ko) | 2013-03-14 | 2024-06-24 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 |
| JP2016516066A (ja) | 2013-03-15 | 2016-06-02 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィを処置するための改善された組成物 |
| KR102268473B1 (ko) | 2013-04-20 | 2021-06-25 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 엑손 2-표적 U7snRNA 폴리뉴클레오티드 작제물의 재조합형 아데노 부속 바이러스 전달 |
| CN105658805B (zh) * | 2013-06-05 | 2021-12-31 | 杜克大学 | Rna指导的基因编辑和基因调节 |
| CN108738310A (zh) | 2015-09-30 | 2018-11-02 | 萨勒普塔医疗公司 | 用于治疗肌营养不良的方法 |
| EP3684933A4 (en) * | 2017-09-22 | 2021-06-23 | The Regents of the University of Colorado, A Body Corporate | THIOMORPHOLINO-OLIGONUCLEOTIDES, FOR TREATMENT OF MUSCULAR DYSTROPHY |
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- 2014-03-14 CN CN202110759294.6A patent/CN113633787A/zh active Pending
- 2014-03-14 KR KR1020157029546A patent/KR20150133768A/ko not_active Ceased
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