JPWO2021081225A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2021081225A5 JPWO2021081225A5 JP2022523854A JP2022523854A JPWO2021081225A5 JP WO2021081225 A5 JPWO2021081225 A5 JP WO2021081225A5 JP 2022523854 A JP2022523854 A JP 2022523854A JP 2022523854 A JP2022523854 A JP 2022523854A JP WO2021081225 A5 JPWO2021081225 A5 JP WO2021081225A5
- Authority
- JP
- Japan
- Prior art keywords
- polynucleotide construct
- composition
- sequence
- seq
- expression cassette
- 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
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962924567P | 2019-10-22 | 2019-10-22 | |
| US62/924,567 | 2019-10-22 | ||
| PCT/US2020/056890 WO2021081225A1 (en) | 2019-10-22 | 2020-10-22 | Ornithine transcarbamylase (otc) constructs and methods of using the same |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022553375A JP2022553375A (ja) | 2022-12-22 |
| JP2022553375A5 JP2022553375A5 (https=) | 2023-10-26 |
| JPWO2021081225A5 true JPWO2021081225A5 (https=) | 2023-10-26 |
| JP7826197B2 JP7826197B2 (ja) | 2026-03-09 |
Family
ID=75620841
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022523854A Active JP7826197B2 (ja) | 2019-10-22 | 2020-10-22 | オルニチントランスカルバミラーゼ（ｏｔｃ）構築物およびそれを使用する方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20230001021A1 (https=) |
| EP (1) | EP4048317A4 (https=) |
| JP (1) | JP7826197B2 (https=) |
| KR (1) | KR20240130161A (https=) |
| CN (1) | CN116096428A (https=) |
| AU (1) | AU2020371697A1 (https=) |
| CA (1) | CA3158626A1 (https=) |
| IL (1) | IL292420A (https=) |
| WO (1) | WO2021081225A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN113166783B (zh) | 2018-10-09 | 2024-10-11 | 不列颠哥伦比亚大学 | 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法 |
| WO2022229903A1 (en) * | 2021-04-28 | 2022-11-03 | Genevant Sciences Gmbh | Mrna delivery constructs and methods of using the same |
| KR20230083893A (ko) * | 2021-12-03 | 2023-06-12 | 고려대학교 산학협력단 | 번역 효율이 향상된 5'-utr, 이를 포함하는 합성 핵산 분자 및 이를 포함하는 백신 또는 치료제 조성물 |
| KR20250169641A (ko) * | 2023-03-06 | 2025-12-03 | 제네럴 메디신스 엘엘씨 | 코돈 최적화 및 이의 사용 방법 |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11201405157PA (en) | 2012-02-24 | 2014-10-30 | Protiva Biotherapeutics Inc | Trialkyl cationic lipids and methods of use thereof |
| WO2015011633A1 (en) * | 2013-07-23 | 2015-01-29 | Protiva Biotherapeutics, Inc. | Compositions and methods for delivering messenger rna |
| EP3027221A1 (en) * | 2013-07-30 | 2016-06-08 | PhaseRx, Inc. | Block copolymers and their conjugates or complexes with oligonucleotides |
| US9765477B2 (en) * | 2013-08-23 | 2017-09-19 | Dow Global Technologies Llc | Low density attached polyurethane foams made using a combination of frothing and blowing methods |
| US11219634B2 (en) * | 2015-01-21 | 2022-01-11 | Genevant Sciences Gmbh | Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells |
| CA3027312A1 (en) * | 2016-06-13 | 2017-12-21 | Translate Bio, Inc. | Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency |
| FI3568483T3 (fi) * | 2017-01-10 | 2025-11-21 | Childrens Medical Res Institute | Polynukleotideja ja vektoreita siirtogeenien ilmentämiseen |
| EP3714048B1 (en) * | 2017-11-22 | 2025-04-09 | ModernaTX, Inc. | Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders |
-
2020
- 2020-10-22 AU AU2020371697A patent/AU2020371697A1/en active Pending
- 2020-10-22 CA CA3158626A patent/CA3158626A1/en active Pending
- 2020-10-22 WO PCT/US2020/056890 patent/WO2021081225A1/en not_active Ceased
- 2020-10-22 KR KR1020227017024A patent/KR20240130161A/ko active Pending
- 2020-10-22 US US17/771,421 patent/US20230001021A1/en active Pending
- 2020-10-22 CN CN202080089266.1A patent/CN116096428A/zh active Pending
- 2020-10-22 IL IL292420A patent/IL292420A/en unknown
- 2020-10-22 JP JP2022523854A patent/JP7826197B2/ja active Active
- 2020-10-22 EP EP20878326.6A patent/EP4048317A4/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US11873505B2 (en) | Compositions and methods for regulating production of a precursor protein | |
| AU2013287353B2 (en) | Oligonucleotides for making a change in the sequence of a target RNA molecule present in a living cell | |
| US12569572B2 (en) | Messenger RNA therapy for the treatment of Friedreich's ataxia | |
| CN115515559B (zh) | 信使rna的直肠递送 | |
| AU2021232818A1 (en) | Mrna therapy for pompe disease | |
| CN112334489A (zh) | 密码子优化的酸性α-葡萄糖苷酶表达盒及其使用方法 | |
| JPWO2021183563A5 (https=) | ||
| JP2022524434A (ja) | Ｆｖｉｉｉ治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用 | |
| US6989374B1 (en) | Gene therapy for cardiomyopathy | |
| US20230226147A1 (en) | Composition and methods for treatment of primary ciliary dyskinesia | |
| CN114788876B (zh) | 治疗糖尿病的mRNA药物制剂及其制备方法与应用 | |
| JPWO2021222801A5 (https=) | ||
| CN111617265A (zh) | 一种二级肝细胞靶向递送基因药物的纳米给药系统及用途 | |
| WO2021042944A1 (zh) | 肌肉靶向的微环dna基因治疗 | |
| JP2025028860A5 (https=) | ||
| US5962429A (en) | Complexes of adenovirus with cationic molecules | |
| JPWO2022235935A5 (https=) | ||
| JP3877148B2 (ja) | 糖尿病性虚血性疾患遺伝子治療 | |
| JP2022060514A (ja) | ガバペンチノイドからの干渉が減少し、ｈｇｆ異型体を発現するｄｎａコンストラクトを用いる神経病症の治療 | |
| JPWO2021081225A5 (https=) | ||
| JPWO2023079507A5 (https=) | ||
| CA3229998A1 (en) | Aav particles comprising a liver-tropic capsid protein and alpha-galactosidase and their use to treat fabry disease | |
| CN120754284A (zh) | 一种用于肝脏靶向联合递送CRISPR-Cas9系统和单链DNA修复模板的脂质纳米颗粒 | |
| EP4709859A1 (en) | Engineered liver-specific core promoters and their applications | |
| CN115998905B (zh) | 治疗心衰的mRNA药物制剂及其制备方法与应用 |