JPWO2020004675A5 - - Google Patents
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- JPWO2020004675A5 JPWO2020004675A5 JP2020527712A JP2020527712A JPWO2020004675A5 JP WO2020004675 A5 JPWO2020004675 A5 JP WO2020004675A5 JP 2020527712 A JP2020527712 A JP 2020527712A JP 2020527712 A JP2020527712 A JP 2020527712A JP WO2020004675 A5 JPWO2020004675 A5 JP WO2020004675A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- treatment
- week
- composition according
- weeks
- 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.)
- Granted
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- 239000008194 pharmaceutical composition Substances 0.000 claims 19
- 230000000692 anti-sense effect Effects 0.000 claims 10
- 150000003839 salts Chemical class 0.000 claims 8
- 108010069091 Dystrophin Proteins 0.000 claims 4
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims 4
- 230000032683 aging Effects 0.000 claims 4
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 3
- 102000001039 Dystrophin Human genes 0.000 claims 3
- HEMHJVSKTPXQMS-UHFFFAOYSA-M Sodium hydroxide Chemical compound [OH-].[Na+] HEMHJVSKTPXQMS-UHFFFAOYSA-M 0.000 claims 3
- KRKNYBCHXYNGOX-UHFFFAOYSA-N citric acid Chemical compound OC(=O)CC(O)(C(O)=O)CC(O)=O KRKNYBCHXYNGOX-UHFFFAOYSA-N 0.000 claims 3
- 230000000694 effects Effects 0.000 claims 3
- PEDCQBHIVMGVHV-UHFFFAOYSA-N Glycerine Chemical compound OCC(O)CO PEDCQBHIVMGVHV-UHFFFAOYSA-N 0.000 claims 2
- VEXZGXHMUGYJMC-UHFFFAOYSA-N Hydrochloric acid Chemical compound Cl VEXZGXHMUGYJMC-UHFFFAOYSA-N 0.000 claims 2
- WCUXLLCKKVVCTQ-UHFFFAOYSA-M Potassium chloride Chemical compound [Cl-].[K+] WCUXLLCKKVVCTQ-UHFFFAOYSA-M 0.000 claims 2
- JVTAAEKCZFNVCJ-UHFFFAOYSA-N lactic acid Chemical compound CC(O)C(O)=O JVTAAEKCZFNVCJ-UHFFFAOYSA-N 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 239000003002 pH adjusting agent Substances 0.000 claims 2
- 239000011780 sodium chloride Substances 0.000 claims 2
- 239000002904 solvent Substances 0.000 claims 2
- HDTRYLNUVZCQOY-UHFFFAOYSA-N α-D-glucopyranosyl-α-D-glucopyranoside Natural products OC1C(O)C(O)C(CO)OC1OC1C(O)C(O)C(O)C(CO)O1 HDTRYLNUVZCQOY-UHFFFAOYSA-N 0.000 claims 1
- OWEGMIWEEQEYGQ-UHFFFAOYSA-N 100676-05-9 Natural products OC1C(O)C(O)C(CO)OC1OCC1C(O)C(O)C(O)C(OC2C(OC(O)C(O)C2O)CO)O1 OWEGMIWEEQEYGQ-UHFFFAOYSA-N 0.000 claims 1
- HZAXFHJVJLSVMW-UHFFFAOYSA-N 2-Aminoethan-1-ol Chemical compound NCCO HZAXFHJVJLSVMW-UHFFFAOYSA-N 0.000 claims 1
- GUBGYTABKSRVRQ-XLOQQCSPSA-N Alpha-Lactose Chemical compound O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@H]1O[C@@H]1[C@@H](CO)O[C@H](O)[C@H](O)[C@H]1O GUBGYTABKSRVRQ-XLOQQCSPSA-N 0.000 claims 1
- FBPFZTCFMRRESA-FSIIMWSLSA-N D-Glucitol Natural products OC[C@H](O)[C@H](O)[C@@H](O)[C@H](O)CO FBPFZTCFMRRESA-FSIIMWSLSA-N 0.000 claims 1
- FBPFZTCFMRRESA-KVTDHHQDSA-N D-Mannitol Chemical compound OC[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-KVTDHHQDSA-N 0.000 claims 1
- FBPFZTCFMRRESA-JGWLITMVSA-N D-glucitol Chemical compound OC[C@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-JGWLITMVSA-N 0.000 claims 1
- 108700024394 Exon Proteins 0.000 claims 1
- 229930091371 Fructose Natural products 0.000 claims 1
- 239000005715 Fructose Substances 0.000 claims 1
- RFSUNEUAIZKAJO-ARQDHWQXSA-N Fructose Chemical compound OC[C@H]1O[C@](O)(CO)[C@@H](O)[C@@H]1O RFSUNEUAIZKAJO-ARQDHWQXSA-N 0.000 claims 1
- WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 claims 1
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 claims 1
- GUBGYTABKSRVRQ-QKKXKWKRSA-N Lactose Natural products OC[C@H]1O[C@@H](O[C@H]2[C@H](O)[C@@H](O)C(O)O[C@@H]2CO)[C@H](O)[C@@H](O)[C@H]1O GUBGYTABKSRVRQ-QKKXKWKRSA-N 0.000 claims 1
- GUBGYTABKSRVRQ-PICCSMPSSA-N Maltose Natural products O[C@@H]1[C@@H](O)[C@H](O)[C@@H](CO)O[C@@H]1O[C@@H]1[C@@H](CO)OC(O)[C@H](O)[C@H]1O GUBGYTABKSRVRQ-PICCSMPSSA-N 0.000 claims 1
- 229930195725 Mannitol Natural products 0.000 claims 1
- 229910019142 PO4 Inorganic materials 0.000 claims 1
- 229930006000 Sucrose Natural products 0.000 claims 1
- CZMRCDWAGMRECN-UGDNZRGBSA-N Sucrose Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 CZMRCDWAGMRECN-UGDNZRGBSA-N 0.000 claims 1
- HDTRYLNUVZCQOY-WSWWMNSNSA-N Trehalose Natural products O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@@H]1O[C@@H]1[C@H](O)[C@@H](O)[C@@H](O)[C@@H](CO)O1 HDTRYLNUVZCQOY-WSWWMNSNSA-N 0.000 claims 1
- TVXBFESIOXBWNM-UHFFFAOYSA-N Xylitol Natural products OCCC(O)C(O)C(O)CCO TVXBFESIOXBWNM-UHFFFAOYSA-N 0.000 claims 1
- HDTRYLNUVZCQOY-LIZSDCNHSA-N alpha,alpha-trehalose Chemical compound O[C@@H]1[C@@H](O)[C@H](O)[C@@H](CO)O[C@@H]1O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 HDTRYLNUVZCQOY-LIZSDCNHSA-N 0.000 claims 1
- 239000007864 aqueous solution Substances 0.000 claims 1
- 230000001174 ascending effect Effects 0.000 claims 1
- WQZGKKKJIJFFOK-VFUOTHLCSA-N beta-D-glucose Chemical compound OC[C@H]1O[C@@H](O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-VFUOTHLCSA-N 0.000 claims 1
- GUBGYTABKSRVRQ-QUYVBRFLSA-N beta-maltose Chemical compound OC[C@H]1O[C@H](O[C@H]2[C@H](O)[C@@H](O)[C@H](O)O[C@@H]2CO)[C@H](O)[C@@H](O)[C@@H]1O GUBGYTABKSRVRQ-QUYVBRFLSA-N 0.000 claims 1
- 230000009194 climbing Effects 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 230000003247 decreasing effect Effects 0.000 claims 1
- 230000002950 deficient Effects 0.000 claims 1
- BNIILDVGGAEEIG-UHFFFAOYSA-L disodium hydrogen phosphate Chemical compound [Na+].[Na+].OP([O-])([O-])=O BNIILDVGGAEEIG-UHFFFAOYSA-L 0.000 claims 1
- 229910000397 disodium phosphate Inorganic materials 0.000 claims 1
- 235000019800 disodium phosphate Nutrition 0.000 claims 1
- 239000008103 glucose Substances 0.000 claims 1
- 235000011187 glycerol Nutrition 0.000 claims 1
- 239000007951 isotonicity adjuster Substances 0.000 claims 1
- 239000004310 lactic acid Substances 0.000 claims 1
- 235000014655 lactic acid Nutrition 0.000 claims 1
- 239000008101 lactose Substances 0.000 claims 1
- 239000000594 mannitol Substances 0.000 claims 1
- 235000010355 mannitol Nutrition 0.000 claims 1
- 238000004949 mass spectrometry Methods 0.000 claims 1
- HEBKCHPVOIAQTA-UHFFFAOYSA-N meso ribitol Natural products OCC(O)C(O)C(O)CO HEBKCHPVOIAQTA-UHFFFAOYSA-N 0.000 claims 1
- 229910000402 monopotassium phosphate Inorganic materials 0.000 claims 1
- 235000019796 monopotassium phosphate Nutrition 0.000 claims 1
- 229910000403 monosodium phosphate Inorganic materials 0.000 claims 1
- 235000019799 monosodium phosphate Nutrition 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 239000010452 phosphate Substances 0.000 claims 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 1
- PJNZPQUBCPKICU-UHFFFAOYSA-N phosphoric acid;potassium Chemical compound [K].OP(O)(O)=O PJNZPQUBCPKICU-UHFFFAOYSA-N 0.000 claims 1
- 239000001103 potassium chloride Substances 0.000 claims 1
- 235000011164 potassium chloride Nutrition 0.000 claims 1
- 230000000630 rising effect Effects 0.000 claims 1
- 238000011947 six minute walk test Methods 0.000 claims 1
- 210000002027 skeletal muscle Anatomy 0.000 claims 1
- AJPJDKMHJJGVTQ-UHFFFAOYSA-M sodium dihydrogen phosphate Chemical compound [Na+].OP(O)([O-])=O AJPJDKMHJJGVTQ-UHFFFAOYSA-M 0.000 claims 1
- 239000000600 sorbitol Substances 0.000 claims 1
- 235000010356 sorbitol Nutrition 0.000 claims 1
- 239000005720 sucrose Substances 0.000 claims 1
- 239000012929 tonicity agent Substances 0.000 claims 1
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 claims 1
- 238000001262 western blot Methods 0.000 claims 1
- 239000000811 xylitol Substances 0.000 claims 1
- 235000010447 xylitol Nutrition 0.000 claims 1
- HEBKCHPVOIAQTA-SCDXWVJYSA-N xylitol Chemical compound OC[C@H](O)[C@@H](O)[C@H](O)CO HEBKCHPVOIAQTA-SCDXWVJYSA-N 0.000 claims 1
- 229960002675 xylitol Drugs 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023143451A JP7595719B2 (ja) | 2018-06-26 | 2023-09-05 | アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用 |
| JP2024205093A JP2025028961A (ja) | 2018-06-26 | 2024-11-26 | アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862690270P | 2018-06-26 | 2018-06-26 | |
| US62/690,270 | 2018-06-26 | ||
| US201862739386P | 2018-10-01 | 2018-10-01 | |
| US62/739,386 | 2018-10-01 | ||
| PCT/JP2019/026393 WO2020004675A1 (ja) | 2018-06-26 | 2019-06-26 | アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023143451A Division JP7595719B2 (ja) | 2018-06-26 | 2023-09-05 | アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JPWO2020004675A1 JPWO2020004675A1 (ja) | 2021-07-15 |
| JPWO2020004675A5 true JPWO2020004675A5 (enExample) | 2022-03-03 |
| JP7345466B2 JP7345466B2 (ja) | 2023-09-15 |
Family
ID=68987235
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020527712A Active JP7345466B2 (ja) | 2018-06-26 | 2019-06-26 | アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用 |
| JP2023143451A Active JP7595719B2 (ja) | 2018-06-26 | 2023-09-05 | アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用 |
| JP2024205093A Pending JP2025028961A (ja) | 2018-06-26 | 2024-11-26 | アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023143451A Active JP7595719B2 (ja) | 2018-06-26 | 2023-09-05 | アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用 |
| JP2024205093A Pending JP2025028961A (ja) | 2018-06-26 | 2024-11-26 | アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用 |
Country Status (20)
| Country | Link |
|---|---|
| US (2) | US20210261963A1 (enExample) |
| EP (1) | EP3815696A4 (enExample) |
| JP (3) | JP7345466B2 (enExample) |
| KR (1) | KR20210023988A (enExample) |
| CN (1) | CN112399849A (enExample) |
| AU (1) | AU2019293687B2 (enExample) |
| BR (1) | BR112020026542A2 (enExample) |
| CA (1) | CA3101321A1 (enExample) |
| CL (1) | CL2020003367A1 (enExample) |
| CO (1) | CO2020015685A2 (enExample) |
| EC (1) | ECSP20083454A (enExample) |
| IL (1) | IL279692A (enExample) |
| MX (2) | MX2020013880A (enExample) |
| MY (1) | MY209148A (enExample) |
| PE (1) | PE20210630A1 (enExample) |
| PH (1) | PH12020552078A1 (enExample) |
| SG (1) | SG11202011554PA (enExample) |
| TW (1) | TW202035692A (enExample) |
| WO (1) | WO2020004675A1 (enExample) |
| ZA (1) | ZA202007682B (enExample) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4150092A4 (en) | 2020-05-11 | 2024-11-06 | Stoke Therapeutics, Inc. | OPA1 ANTISENSE OLIGOMERS FOR THE TREATMENT OF DISEASES AND CONDITIONS |
| EP4330395A1 (en) | 2021-04-30 | 2024-03-06 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
| CA3252177A1 (en) | 2022-03-17 | 2023-09-21 | Sarepta Therapeutics, Inc. | MORPHOLINO PHOSPHORODIAMIDATE OLIGOMER CONJUGATE |
| EP4665407A2 (en) * | 2023-02-14 | 2025-12-24 | Stoke Therapeutics, Inc. | Antisense oligomer formulations |
| US20240368596A1 (en) * | 2023-05-05 | 2024-11-07 | Biomarin Pharmaceutical Inc. | Dystrophin exon skipping oligonucleotides |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2069869C (en) | 1989-12-20 | 1995-12-19 | James E. Summerton | Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages |
| CA2796924C (en) | 2002-11-25 | 2016-12-13 | Nonprofit Organization Translational Research Organization Of Duchenne Muscular Dystrophy | Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors |
| EP1766010B1 (en) | 2004-06-28 | 2011-02-16 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| WO2006038608A1 (ja) | 2004-10-05 | 2006-04-13 | Nippon Shinyaku Co., Ltd. | オリゴ二本鎖ｒｎａ及び医薬組成物 |
| WO2006129594A1 (ja) | 2005-05-30 | 2006-12-07 | Nippon Shinyaku Co., Ltd. | 核酸含有複合体製剤の製造方法 |
| MX2010004955A (es) | 2007-11-15 | 2010-06-30 | Avi Biopharma Inc | Metodo de sintesis de oligomeros de morfolina. |
| US8084601B2 (en) * | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| KR20180118828A (ko) | 2008-10-24 | 2018-10-31 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | Dmd를 위한 다중 엑손 스키핑 조성물 |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| JP2015509922A (ja) * | 2012-01-27 | 2015-04-02 | プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． | デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド |
| CN110066793A (zh) * | 2013-03-14 | 2019-07-30 | 萨勒普塔医疗公司 | 用于治疗肌肉萎缩的外显子跳跃组合物 |
| BR112015022998A2 (pt) | 2013-03-15 | 2017-11-14 | Sarepta Therapeutics Inc | composições melhoradas para o tratamento de distrofia muscular |
| EP3015467B1 (en) * | 2013-05-24 | 2024-12-04 | Ajinomoto Co., Inc. | Morpholino oligonucleotide manufacturing method |
| EP3653216A1 (en) * | 2015-09-30 | 2020-05-20 | Sarepta Therapeutics, Inc. | Methods for treating muscular dystrophy |
| FR3044926B1 (fr) * | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
-
2019
- 2019-06-26 AU AU2019293687A patent/AU2019293687B2/en active Active
- 2019-06-26 CN CN201980043482.XA patent/CN112399849A/zh active Pending
- 2019-06-26 MX MX2020013880A patent/MX2020013880A/es unknown
- 2019-06-26 JP JP2020527712A patent/JP7345466B2/ja active Active
- 2019-06-26 EP EP19826094.5A patent/EP3815696A4/en active Pending
- 2019-06-26 KR KR1020217001037A patent/KR20210023988A/ko not_active Ceased
- 2019-06-26 BR BR112020026542-0A patent/BR112020026542A2/pt unknown
- 2019-06-26 PE PE2020002157A patent/PE20210630A1/es unknown
- 2019-06-26 US US17/253,760 patent/US20210261963A1/en not_active Abandoned
- 2019-06-26 SG SG11202011554PA patent/SG11202011554PA/en unknown
- 2019-06-26 WO PCT/JP2019/026393 patent/WO2020004675A1/ja not_active Ceased
- 2019-06-26 MY MYPI2020006568A patent/MY209148A/en unknown
- 2019-06-26 TW TW108122381A patent/TW202035692A/zh unknown
- 2019-06-26 CA CA3101321A patent/CA3101321A1/en active Pending
-
2020
- 2020-12-02 PH PH12020552078A patent/PH12020552078A1/en unknown
- 2020-12-09 ZA ZA2020/07682A patent/ZA202007682B/en unknown
- 2020-12-14 CO CONC2020/0015685A patent/CO2020015685A2/es unknown
- 2020-12-16 MX MX2025001486A patent/MX2025001486A/es unknown
- 2020-12-22 IL IL279692A patent/IL279692A/en unknown
- 2020-12-23 EC ECSENADI202083454A patent/ECSP20083454A/es unknown
- 2020-12-23 CL CL2020003367A patent/CL2020003367A1/es unknown
-
2023
- 2023-09-05 JP JP2023143451A patent/JP7595719B2/ja active Active
- 2023-11-08 US US18/504,781 patent/US20240158792A1/en active Pending
-
2024
- 2024-11-26 JP JP2024205093A patent/JP2025028961A/ja active Pending
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