JP2020503010A5 - - Google Patents
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- JP2020503010A5 JP2020503010A5 JP2019531379A JP2019531379A JP2020503010A5 JP 2020503010 A5 JP2020503010 A5 JP 2020503010A5 JP 2019531379 A JP2019531379 A JP 2019531379A JP 2019531379 A JP2019531379 A JP 2019531379A JP 2020503010 A5 JP2020503010 A5 JP 2020503010A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- antisense oligomer
- oligomer conjugate
- exon
- administered
- 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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- 230000000692 anti-sense effect Effects 0.000 claims description 44
- 239000000203 mixture Substances 0.000 claims description 24
- 108010069091 Dystrophin Proteins 0.000 claims description 22
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims description 12
- 230000035772 mutation Effects 0.000 claims description 12
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims description 12
- 102000001039 Dystrophin Human genes 0.000 claims description 10
- 239000008194 pharmaceutical composition Substances 0.000 claims description 10
- 230000008685 targeting Effects 0.000 claims description 10
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 8
- 108020004999 messenger RNA Proteins 0.000 claims description 8
- 150000003839 salts Chemical class 0.000 claims description 8
- 229940113082 thymine Drugs 0.000 claims description 6
- 229940035893 uracil Drugs 0.000 claims description 6
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 4
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims description 4
- FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 claims description 4
- 238000004519 manufacturing process Methods 0.000 claims description 4
- 230000003442 weekly effect Effects 0.000 claims description 4
- 230000001939 inductive effect Effects 0.000 claims description 3
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 2
- 229930024421 Adenine Natural products 0.000 claims description 2
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims description 2
- 102220479948 Alkaline phosphatase, germ cell type_H45A_mutation Human genes 0.000 claims description 2
- UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 claims description 2
- 229960000643 adenine Drugs 0.000 claims description 2
- 238000000137 annealing Methods 0.000 claims description 2
- 230000000295 complement effect Effects 0.000 claims description 2
- 229940104302 cytosine Drugs 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 108700024394 Exon Proteins 0.000 claims 1
- 230000008878 coupling Effects 0.000 claims 1
- 238000010168 coupling process Methods 0.000 claims 1
- 238000005859 coupling reaction Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 20
- 125000000217 alkyl group Chemical group 0.000 description 1
Priority Applications (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022075930A JP2022101695A (ja) | 2016-12-19 | 2022-05-02 | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
| JP2024095058A JP7728923B2 (ja) | 2016-12-19 | 2024-06-12 | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
| JP2025134907A JP2025166161A (ja) | 2016-12-19 | 2025-08-13 | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662436199P | 2016-12-19 | 2016-12-19 | |
| US62/436,199 | 2016-12-19 | ||
| US201762443481P | 2017-01-06 | 2017-01-06 | |
| US62/443,481 | 2017-01-06 | ||
| US201762479177P | 2017-03-30 | 2017-03-30 | |
| US62/479,177 | 2017-03-30 | ||
| US201762562119P | 2017-09-22 | 2017-09-22 | |
| US62/562,119 | 2017-09-22 | ||
| PCT/US2017/066351 WO2018118627A1 (en) | 2016-12-19 | 2017-12-14 | Exon skipping oligomer conjugates for muscular dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022075930A Division JP2022101695A (ja) | 2016-12-19 | 2022-05-02 | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020503010A JP2020503010A (ja) | 2020-01-30 |
| JP2020503010A5 true JP2020503010A5 (OSRAM) | 2021-01-28 |
| JP7125940B2 JP7125940B2 (ja) | 2022-08-25 |
Family
ID=61006319
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019531379A Active JP7125940B2 (ja) | 2016-12-19 | 2017-12-14 | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
| JP2022075930A Withdrawn JP2022101695A (ja) | 2016-12-19 | 2022-05-02 | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
| JP2024095058A Active JP7728923B2 (ja) | 2016-12-19 | 2024-06-12 | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
| JP2025134907A Pending JP2025166161A (ja) | 2016-12-19 | 2025-08-13 | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022075930A Withdrawn JP2022101695A (ja) | 2016-12-19 | 2022-05-02 | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
| JP2024095058A Active JP7728923B2 (ja) | 2016-12-19 | 2024-06-12 | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
| JP2025134907A Pending JP2025166161A (ja) | 2016-12-19 | 2025-08-13 | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
Country Status (31)
| Country | Link |
|---|---|
| US (2) | US11382981B2 (OSRAM) |
| EP (3) | EP4406601A3 (OSRAM) |
| JP (4) | JP7125940B2 (OSRAM) |
| KR (2) | KR102639633B1 (OSRAM) |
| CN (2) | CN110337308B (OSRAM) |
| AU (1) | AU2017382741B2 (OSRAM) |
| BR (1) | BR112019012651A2 (OSRAM) |
| CA (1) | CA3046801A1 (OSRAM) |
| CO (1) | CO2019007398A2 (OSRAM) |
| DK (2) | DK3554554T3 (OSRAM) |
| ES (2) | ES2929498T3 (OSRAM) |
| FI (1) | FI4122497T3 (OSRAM) |
| HR (2) | HRP20240705T1 (OSRAM) |
| HU (2) | HUE060065T2 (OSRAM) |
| IL (2) | IL304284B2 (OSRAM) |
| LT (2) | LT4122497T (OSRAM) |
| MA (1) | MA65895B1 (OSRAM) |
| MD (2) | MD4122497T2 (OSRAM) |
| MX (1) | MX2019006989A (OSRAM) |
| MY (1) | MY205600A (OSRAM) |
| NZ (1) | NZ755422A (OSRAM) |
| PL (2) | PL3554554T3 (OSRAM) |
| PT (2) | PT4122497T (OSRAM) |
| RS (2) | RS65583B1 (OSRAM) |
| SA (1) | SA519402143B1 (OSRAM) |
| SG (1) | SG10202100491QA (OSRAM) |
| SI (2) | SI3554554T1 (OSRAM) |
| SM (2) | SMT202200440T1 (OSRAM) |
| TW (1) | TWI780095B (OSRAM) |
| WO (1) | WO2018118627A1 (OSRAM) |
| ZA (1) | ZA201903940B (OSRAM) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| BR112018074346B1 (pt) | 2016-05-24 | 2023-01-03 | Sarepta Therapeutics, Inc. | Composto oligomérico e processo para preparar um composto oligomérico |
| MA45362A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| CN110337308B (zh) | 2016-12-19 | 2023-09-01 | 萨勒普塔医疗公司 | 用于肌肉萎缩症的外显子跳跃寡聚体缀合物 |
| KR20210081324A (ko) | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
| SG11202100934PA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | 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 |
| GB201821269D0 (en) | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| MA55515A (fr) * | 2019-03-28 | 2022-02-09 | Sarepta Therapeutics Inc | Procédés de traitement de la dystrophie musculaire avec casimersen |
| EP3955966A1 (en) * | 2019-04-18 | 2022-02-23 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| EP3987029A1 (en) * | 2019-06-19 | 2022-04-27 | Sarepta Therapeutics, Inc. | Methods for treating muscular dystrophy |
| MY209476A (en) | 2019-12-26 | 2025-07-11 | Nippon Shinyaku Co Ltd | Antisense nucleic acid that induces skipping of exon 50 |
| EP4112083A4 (en) | 2020-02-28 | 2024-06-26 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid inducing skipping of exon 51 |
| US20230256047A1 (en) * | 2020-06-17 | 2023-08-17 | Stealth Biotherapeutics Inc. | Methods and compositions for the treatment of muscular dystrophy |
| EP4330395A1 (en) * | 2021-04-30 | 2024-03-06 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
| MX2023014417A (es) | 2021-06-23 | 2023-12-15 | Nippon Shinyaku Co Ltd | Combinacion de oligomeros antisentido. |
| AU2022306542A1 (en) | 2021-07-08 | 2024-01-18 | Nippon Shinyaku Co., Ltd. | Precipitation suppressing agent |
| JPWO2023282345A1 (OSRAM) | 2021-07-08 | 2023-01-12 | ||
| TW202308664A (zh) | 2021-07-08 | 2023-03-01 | 日商日本新藥股份有限公司 | 腎毒性減輕劑 |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof 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 |
| US20250188469A1 (en) | 2022-03-10 | 2025-06-12 | Nippon Shinyaku Co., Ltd. | Antiviral antisense oligomer |
| JP7523012B1 (ja) * | 2023-05-17 | 2024-07-26 | 東亞合成株式会社 | ペプチドフラグメント及びその利用 |
| WO2025030155A1 (en) * | 2023-08-03 | 2025-02-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
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