JP2024056778A5 - - Google Patents
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- JP2024056778A5 JP2024056778A5 JP2024015750A JP2024015750A JP2024056778A5 JP 2024056778 A5 JP2024056778 A5 JP 2024056778A5 JP 2024015750 A JP2024015750 A JP 2024015750A JP 2024015750 A JP2024015750 A JP 2024015750A JP 2024056778 A5 JP2024056778 A5 JP 2024056778A5
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- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- target protein
- antisense oligomer
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- allele
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Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762575924P | 2017-10-23 | 2017-10-23 | |
| US62/575,924 | 2017-10-23 | ||
| US201862667200P | 2018-05-04 | 2018-05-04 | |
| US62/667,200 | 2018-05-04 | ||
| PCT/US2018/057165 WO2019084050A1 (en) | 2017-10-23 | 2018-10-23 | ANTISENSE OLIGOMERS FOR THE TREATMENT OF CONDITIONS AND DISEASES BASED ON THE DECLINE OF NON-SENSE MEDIATED MRNA |
| JP2020522905A JP7475272B2 (ja) | 2017-10-23 | 2018-10-23 | ナンセンス変異依存rna分解機構に基づく状態および疾患の処置のためのアンチセンスオリゴマー |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020522905A Division JP7475272B2 (ja) | 2017-10-23 | 2018-10-23 | ナンセンス変異依存rna分解機構に基づく状態および疾患の処置のためのアンチセンスオリゴマー |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2024056778A JP2024056778A (ja) | 2024-04-23 |
| JP2024056778A5 true JP2024056778A5 (https=) | 2024-11-07 |
| JP7783920B2 JP7783920B2 (ja) | 2025-12-10 |
Family
ID=66247689
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020522905A Active JP7475272B2 (ja) | 2017-10-23 | 2018-10-23 | ナンセンス変異依存rna分解機構に基づく状態および疾患の処置のためのアンチセンスオリゴマー |
| JP2024015750A Active JP7783920B2 (ja) | 2017-10-23 | 2024-02-05 | ナンセンス変異依存rna分解機構に基づく状態および疾患の処置のためのアンチセンスオリゴマー |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020522905A Active JP7475272B2 (ja) | 2017-10-23 | 2018-10-23 | ナンセンス変異依存rna分解機構に基づく状態および疾患の処置のためのアンチセンスオリゴマー |
Country Status (23)
| Country | Link |
|---|---|
| US (2) | US20210268667A1 (https=) |
| EP (2) | EP3700570B1 (https=) |
| JP (2) | JP7475272B2 (https=) |
| KR (2) | KR102774070B1 (https=) |
| CN (2) | CN111936163B (https=) |
| AU (2) | AU2018355237B2 (https=) |
| BR (1) | BR112020007881A2 (https=) |
| CA (1) | CA3079729A1 (https=) |
| DK (1) | DK3700570T3 (https=) |
| ES (1) | ES3015767T3 (https=) |
| FI (1) | FI3700570T3 (https=) |
| GB (2) | GB2584204B (https=) |
| HR (1) | HRP20250322T1 (https=) |
| HU (1) | HUE070436T2 (https=) |
| IL (1) | IL274023A (https=) |
| LT (1) | LT3700570T (https=) |
| PL (1) | PL3700570T3 (https=) |
| PT (1) | PT3700570T (https=) |
| RS (1) | RS66633B1 (https=) |
| SG (1) | SG11202003692YA (https=) |
| SI (1) | SI3700570T1 (https=) |
| SM (1) | SMT202500127T1 (https=) |
| WO (1) | WO2019084050A1 (https=) |
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| AU2015327836B2 (en) | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
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| CN108603230A (zh) | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| EP4089175A1 (en) | 2015-10-13 | 2022-11-16 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| KR102787119B1 (ko) | 2015-11-30 | 2025-03-27 | 듀크 유니버시티 | 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법 |
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| US11618900B2 (en) * | 2019-11-01 | 2023-04-04 | The Johns Hopkins University | Modulating SYNGAP |
| BR112022010882A2 (pt) * | 2019-12-06 | 2022-10-04 | Stoke Therapeutics Inc | Oligômeros antissenso para tratamento de condições e doenças |
| CN120505310A (zh) * | 2020-02-28 | 2025-08-19 | Ionis制药公司 | 用于调节smn2的化合物和方法 |
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| WO2021222328A1 (en) * | 2020-04-27 | 2021-11-04 | Duke University | Targeted genomic integration to restore neurofibromin coding sequence in neurofibromatosis type 1 (nf1) |
| CN115916977A (zh) * | 2020-05-11 | 2023-04-04 | 弗洛里神经科学与心理健康研究所 | 用于治疗与syngap1中功能失去突变相关的障碍的组合物和方法 |
| CN115867657A (zh) * | 2020-05-11 | 2023-03-28 | 斯托克制药公司 | 用于治疗疾患和疾病的opa1反义寡聚物 |
| KR20230095056A (ko) * | 2020-07-22 | 2023-06-28 | 더 플로레이 인스티튜트 오브 뉴로사이언스 앤드 멘탈 헬스 | Scn2a에서 기능-상실 돌연변이와 연관된 장애를 치료하기 위한 조성물 및 방법 |
| IL300258A (en) * | 2020-08-07 | 2023-03-01 | Ionis Pharmaceuticals Inc | Compounds and methods for modulating SCN2A |
| AU2021354152A1 (en) * | 2020-10-02 | 2023-06-01 | PYC Therapeutics Limited | Treatment of optic atrophy |
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| CN117413061A (zh) * | 2021-02-03 | 2024-01-16 | 斯托克制药公司 | 用于治疗与多囊蛋白表达相关的疾患和疾病的组合物 |
| JP2024518788A (ja) * | 2021-05-10 | 2024-05-02 | キュー-ステート バイオサイエンシーズ, インコーポレイテッド | ナトリウムチャネル1.6を標的とする組成物 |
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| CN117980479A (zh) * | 2021-06-21 | 2024-05-03 | 斯托克制药公司 | 用于治疗基于无义介导的rna衰变的病状和疾病的反义寡聚体 |
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| CN114032323B (zh) * | 2021-11-17 | 2023-08-08 | 云南省烟草农业科学研究院 | 一种与雪茄烟抗黑胫病基因紧密连锁的共显性ssr标记及其应用 |
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| AU2023212479A1 (en) * | 2022-01-31 | 2024-08-01 | PYC Therapeutics Limited | Method of treatment for optic atrophy |
| WO2023163801A1 (en) * | 2022-02-24 | 2023-08-31 | Q-State Biosciences, Inc. | Therapeutics for syngap haploinsufficiency |
| CN116083427B (zh) * | 2022-04-22 | 2025-10-03 | 齐齐哈尔大学 | 大豆高温诱导型启动子及其应用 |
| CN115029347B (zh) * | 2022-05-11 | 2024-02-20 | 珠海中科先进技术研究院有限公司 | 识别和调控肝肾细胞纤维化的分子监测序列、重组质粒、抑制病毒 |
| EP4532723A2 (en) * | 2022-06-01 | 2025-04-09 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases |
| KR20250021378A (ko) * | 2022-06-07 | 2025-02-12 | 피와이씨 테라퓨틱스 리미티드 | 단일유전자성 신경발달 장애 치료를 위한 조성물 및 방법 |
| US20250368991A1 (en) * | 2022-06-28 | 2025-12-04 | Agency For Science, Technology And Research | Oligonucleotides |
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| WO2025091028A1 (en) * | 2023-10-27 | 2025-05-01 | The Regents Of The University Of Michigan | Compounds and methods for modulating cln3 expression |
| CN117511947B (zh) * | 2024-01-08 | 2024-03-29 | 艾斯拓康医药科技(北京)有限公司 | 一种优化的5`utr序列及其应用 |
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| WO2025255491A1 (en) * | 2024-06-06 | 2025-12-11 | The Regents Of The University Of California | Gene therapy for treatment of syngap1-related intellectual disability |
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| CN119060981B (zh) * | 2024-09-04 | 2025-07-22 | 中国人民解放军军事科学院军事医学研究院 | Perk蛋白突变体及其在预防uvb诱发皮肤光损伤反应中的应用 |
| CN119552958A (zh) * | 2024-11-29 | 2025-03-04 | 福州福瑞医学检验实验室有限公司 | 检测歌舞伎综合征kmt2d基因变异位点的多重荧光定量pcr引物、探针及试剂盒 |
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2018
- 2018-10-23 HU HUE18871437A patent/HUE070436T2/hu unknown
- 2018-10-23 AU AU2018355237A patent/AU2018355237B2/en active Active
- 2018-10-23 JP JP2020522905A patent/JP7475272B2/ja active Active
- 2018-10-23 KR KR1020207014744A patent/KR102774070B1/ko active Active
- 2018-10-23 SI SI201831220T patent/SI3700570T1/sl unknown
- 2018-10-23 PT PT188714372T patent/PT3700570T/pt unknown
- 2018-10-23 EP EP18871437.2A patent/EP3700570B1/en active Active
- 2018-10-23 SM SM20250127T patent/SMT202500127T1/it unknown
- 2018-10-23 EP EP24215260.1A patent/EP4494706A3/en active Pending
- 2018-10-23 CN CN201880084329.7A patent/CN111936163B/zh active Active
- 2018-10-23 CA CA3079729A patent/CA3079729A1/en active Pending
- 2018-10-23 LT LTEPPCT/US2018/057165T patent/LT3700570T/lt unknown
- 2018-10-23 SG SG11202003692YA patent/SG11202003692YA/en unknown
- 2018-10-23 ES ES18871437T patent/ES3015767T3/es active Active
- 2018-10-23 RS RS20250313A patent/RS66633B1/sr unknown
- 2018-10-23 CN CN202510314435.1A patent/CN120290560A/zh active Pending
- 2018-10-23 FI FIEP18871437.2T patent/FI3700570T3/fi active
- 2018-10-23 BR BR112020007881-6A patent/BR112020007881A2/pt unknown
- 2018-10-23 WO PCT/US2018/057165 patent/WO2019084050A1/en not_active Ceased
- 2018-10-23 US US16/758,776 patent/US20210268667A1/en not_active Abandoned
- 2018-10-23 HR HRP20250322TT patent/HRP20250322T1/hr unknown
- 2018-10-23 GB GB2006322.8A patent/GB2584204B/en active Active
- 2018-10-23 KR KR1020257004856A patent/KR20250025519A/ko active Pending
- 2018-10-23 DK DK18871437.2T patent/DK3700570T3/da active
- 2018-10-23 GB GB2216904.9A patent/GB2610100B/en active Active
- 2018-10-23 PL PL18871437.2T patent/PL3700570T3/pl unknown
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2020
- 2020-04-19 IL IL274023A patent/IL274023A/en unknown
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2023
- 2023-04-13 US US18/299,956 patent/US20240033378A1/en active Pending
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2024
- 2024-02-05 JP JP2024015750A patent/JP7783920B2/ja active Active
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2025
- 2025-12-24 AU AU2025287361A patent/AU2025287361A1/en active Pending
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