JPWO2020123574A5 - - Google Patents
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- Publication number
- JPWO2020123574A5 JPWO2020123574A5 JP2021531988A JP2021531988A JPWO2020123574A5 JP WO2020123574 A5 JPWO2020123574 A5 JP WO2020123574A5 JP 2021531988 A JP2021531988 A JP 2021531988A JP 2021531988 A JP2021531988 A JP 2021531988A JP WO2020123574 A5 JPWO2020123574 A5 JP WO2020123574A5
- Authority
- JP
- Japan
- Prior art keywords
- antisense oligomer
- seq
- pharmaceutically acceptable
- cell membrane
- acceptable salt
- 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
Links
- 230000000692 anti-sense effect Effects 0.000 claims description 79
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 30
- 210000000170 cell membrane Anatomy 0.000 claims description 22
- 230000000149 penetrating effect Effects 0.000 claims description 20
- 150000003839 salts Chemical class 0.000 claims description 20
- 239000004475 Arginine Substances 0.000 claims description 12
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 claims description 12
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims description 12
- 108010069091 Dystrophin Proteins 0.000 claims description 9
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 6
- 239000000203 mixture Substances 0.000 claims description 5
- 230000035772 mutation Effects 0.000 claims description 5
- 239000008194 pharmaceutical composition Substances 0.000 claims description 5
- SLXKOJJOQWFEFD-UHFFFAOYSA-N 6-aminohexanoic acid Chemical group NCCCCCC(O)=O SLXKOJJOQWFEFD-UHFFFAOYSA-N 0.000 claims description 4
- 102000001039 Dystrophin Human genes 0.000 claims description 4
- 239000004471 Glycine Substances 0.000 claims description 4
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 claims description 4
- COLNVLDHVKWLRT-QMMMGPOBSA-N L-phenylalanine Chemical compound OC(=O)[C@@H](N)CC1=CC=CC=C1 COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 claims description 4
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims description 4
- 125000002252 acyl group Chemical group 0.000 claims description 4
- 229960002684 aminocaproic acid Drugs 0.000 claims description 4
- 238000000137 annealing Methods 0.000 claims description 4
- 125000000637 arginyl group Chemical group N[C@@H](CCCNC(N)=N)C(=O)* 0.000 claims description 4
- 239000002585 base Substances 0.000 claims description 4
- 125000003236 benzoyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C(*)=O 0.000 claims description 4
- 239000012458 free base Substances 0.000 claims description 4
- 108020004999 messenger RNA Proteins 0.000 claims description 4
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Natural products OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 claims description 4
- 125000003696 stearoyl 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])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 claims description 4
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims description 4
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims description 2
- 230000000295 complement effect Effects 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 229910052739 hydrogen Inorganic materials 0.000 claims description 2
- 239000001257 hydrogen Substances 0.000 claims description 2
- 230000001939 inductive effect Effects 0.000 claims description 2
- 238000004519 manufacturing process Methods 0.000 claims description 2
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 2
- 230000008685 targeting Effects 0.000 claims description 2
- 229940113082 thymine Drugs 0.000 claims description 2
- 229940035893 uracil Drugs 0.000 claims description 2
- UCMIRNVEIXFBKS-UHFFFAOYSA-N beta-alanine Chemical group NCCC(O)=O UCMIRNVEIXFBKS-UHFFFAOYSA-N 0.000 claims 4
- 229940000635 beta-alanine Drugs 0.000 claims 2
- 238000000034 method Methods 0.000 description 8
- 239000003814 drug Substances 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024028361A JP2024051108A (ja) | 2018-12-13 | 2024-02-28 | 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862779028P | 2018-12-13 | 2018-12-13 | |
| US62/779,028 | 2018-12-13 | ||
| PCT/US2019/065581 WO2020123574A1 (en) | 2018-12-13 | 2019-12-11 | Exon skipping oligomer conjugates for muscular dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024028361A Division JP2024051108A (ja) | 2018-12-13 | 2024-02-28 | 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 (4)
| Publication Number | Publication Date |
|---|---|
| JP2022511055A JP2022511055A (ja) | 2022-01-28 |
| JP2022511055A5 JP2022511055A5 (https=) | 2022-12-12 |
| JPWO2020123574A5 true JPWO2020123574A5 (https=) | 2022-12-12 |
| JP7658900B2 JP7658900B2 (ja) | 2025-04-08 |
Family
ID=69570813
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021531988A Active JP7658900B2 (ja) | 2018-12-13 | 2019-12-11 | 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 |
| JP2024028361A Pending JP2024051108A (ja) | 2018-12-13 | 2024-02-28 | 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 (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024028361A Pending JP2024051108A (ja) | 2018-12-13 | 2024-02-28 | 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 (17)
| Country | Link |
|---|---|
| US (3) | US20200190516A1 (https=) |
| EP (1) | EP3894558A1 (https=) |
| JP (2) | JP7658900B2 (https=) |
| KR (1) | KR20210104759A (https=) |
| CN (1) | CN113412330A (https=) |
| AR (1) | AR117320A1 (https=) |
| AU (2) | AU2019397461A1 (https=) |
| BR (1) | BR112021011018A2 (https=) |
| CA (1) | CA3122281A1 (https=) |
| CL (1) | CL2021001523A1 (https=) |
| CO (1) | CO2021008891A2 (https=) |
| EA (1) | EA202191601A1 (https=) |
| IL (3) | IL283646B2 (https=) |
| MX (1) | MX2021006737A (https=) |
| SG (1) | SG11202104960PA (https=) |
| TW (1) | TW202035695A (https=) |
| WO (1) | WO2020123574A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN118109468A (zh) | 2019-12-26 | 2024-05-31 | 日本新药株式会社 | 诱导外显子50的跳读的反义核酸 |
| JP2025509438A (ja) * | 2022-03-17 | 2025-04-11 | サレプタ セラピューティクス， インコーポレイテッド | ホスホロジアミデートモルホリノオリゴマーコンジュゲート |
| WO2025212708A2 (en) * | 2024-04-03 | 2025-10-09 | Avidity Biosciences, Inc. | Antibody-oligonucleotide conjugate compositions and methods of inducing dmd exon 50 skipping |
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-
2019
- 2019-12-11 MX MX2021006737A patent/MX2021006737A/es unknown
- 2019-12-11 SG SG11202104960PA patent/SG11202104960PA/en unknown
- 2019-12-11 EA EA202191601A patent/EA202191601A1/ru unknown
- 2019-12-11 BR BR112021011018-6A patent/BR112021011018A2/pt unknown
- 2019-12-11 WO PCT/US2019/065581 patent/WO2020123574A1/en not_active Ceased
- 2019-12-11 AU AU2019397461A patent/AU2019397461A1/en not_active Abandoned
- 2019-12-11 US US16/710,144 patent/US20200190516A1/en not_active Abandoned
- 2019-12-11 IL IL283646A patent/IL283646B2/en unknown
- 2019-12-11 CA CA3122281A patent/CA3122281A1/en active Pending
- 2019-12-11 IL IL318474A patent/IL318474A/en unknown
- 2019-12-11 IL IL321667A patent/IL321667A/en unknown
- 2019-12-11 EP EP19850744.4A patent/EP3894558A1/en active Pending
- 2019-12-11 JP JP2021531988A patent/JP7658900B2/ja active Active
- 2019-12-11 CN CN201980081396.8A patent/CN113412330A/zh active Pending
- 2019-12-11 KR KR1020217021183A patent/KR20210104759A/ko active Pending
- 2019-12-12 TW TW108145500A patent/TW202035695A/zh unknown
- 2019-12-12 AR ARP190103646A patent/AR117320A1/es unknown
-
2021
- 2021-06-10 CL CL2021001523A patent/CL2021001523A1/es unknown
- 2021-06-11 US US17/345,624 patent/US20220017901A1/en not_active Abandoned
- 2021-07-06 CO CONC2021/0008891A patent/CO2021008891A2/es unknown
-
2024
- 2024-02-28 JP JP2024028361A patent/JP2024051108A/ja active Pending
- 2024-06-14 US US18/744,061 patent/US20250179487A1/en active Pending
-
2026
- 2026-03-24 AU AU2026202254A patent/AU2026202254A1/en active Pending
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