JP2022545378A5 - - Google Patents
Info
- Publication number
- JP2022545378A5 JP2022545378A5 JP2022509153A JP2022509153A JP2022545378A5 JP 2022545378 A5 JP2022545378 A5 JP 2022545378A5 JP 2022509153 A JP2022509153 A JP 2022509153A JP 2022509153 A JP2022509153 A JP 2022509153A JP 2022545378 A5 JP2022545378 A5 JP 2022545378A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- acid molecule
- promoter
- expression cassette
- molecule according
- 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.)
- Pending
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962887209P | 2019-08-15 | 2019-08-15 | |
| US62/887,209 | 2019-08-15 | ||
| PCT/US2020/046499 WO2021030745A1 (en) | 2019-08-15 | 2020-08-14 | Combined transgene and intron-derived mirna therapy for treatment of sca1 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022545378A JP2022545378A (ja) | 2022-10-27 |
| JP2022545378A5 true JP2022545378A5 (https=) | 2023-08-15 |
Family
ID=74570777
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022509153A Pending JP2022545378A (ja) | 2019-08-15 | 2020-08-14 | ＳＣＡ１の治療のための、導入遺伝子およびイントロン由来ｍｉＲＮＡを組み合わせた治療法 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220288101A1 (https=) |
| EP (1) | EP4013414A4 (https=) |
| JP (1) | JP2022545378A (https=) |
| CN (1) | CN114555084A (https=) |
| AU (1) | AU2020330108A1 (https=) |
| BR (1) | BR112022002794A2 (https=) |
| CA (1) | CA3151122A1 (https=) |
| MX (1) | MX2022001984A (https=) |
| WO (1) | WO2021030745A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11008627B2 (en) | 2019-08-15 | 2021-05-18 | Talis Biomedical Corporation | Diagnostic system |
| WO2024044469A1 (en) * | 2022-08-26 | 2024-02-29 | The Children's Hospital Of Philadelphia | Mirnas targeting atnx2 for the treatment of als and sca2 |
| EP4651883A1 (en) * | 2023-01-19 | 2025-11-26 | uniQure biopharma B.V. | Pharmaceutical formulations of gene delivery vehicles |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7001733B1 (en) * | 1998-05-12 | 2006-02-21 | Rigel Pharmaceuticals, Inc. | Methods and compositions for screening for modulations of IgE synthesis, secretion and switch rearrangement |
| US20080274989A1 (en) * | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
| US7605249B2 (en) * | 2002-11-26 | 2009-10-20 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| DK2164967T3 (en) * | 2007-05-31 | 2015-10-19 | Univ Iowa Res Found | Reduction of off-target rna interferenstoksicitet |
| US8865881B2 (en) * | 2011-02-22 | 2014-10-21 | California Institute Of Technology | Delivery of proteins using adeno-associated virus (AAV) vectors |
| PL3628334T3 (pl) * | 2014-03-21 | 2023-12-18 | Genzyme Corporation | Terapia genowa w retinopatii barwnikowej |
| EP3146051B8 (en) * | 2014-05-20 | 2019-11-27 | University of Iowa Research Foundation | Huntington's disease therapeutic compounds |
| MA40782A (fr) * | 2014-10-03 | 2017-08-08 | Hocuslocus Llc | Procédés et compositions utilisant un arn non codant en vue de la culture et de la sélection de cellules |
| EP3261582B1 (en) * | 2015-02-26 | 2021-01-06 | Remodeless CV Ltd. | Methods and compositions relating to leptin antagonists |
| WO2016196507A1 (en) * | 2015-05-29 | 2016-12-08 | University Of Iowa Research Foundation | Methods of delivery of transgenes for treating brain diseases |
| CN115896106A (zh) * | 2016-03-01 | 2023-04-04 | 佛罗里达大学研究基金会有限公司 | 用于治疗显性视网膜色素变性的aav载体 |
| CA3075168A1 (en) * | 2017-09-08 | 2019-03-14 | Generation Bio Co. | Modified closed-ended dna (cedna) |
| KR20200119782A (ko) * | 2017-12-06 | 2020-10-20 | 오비드 테라퓨틱스 인크. | 발작 장애 치료 시 mir101 또는 mir128의 용도 |
| US20210355509A1 (en) * | 2018-08-07 | 2021-11-18 | The Children's Hospital Of Philadelphia | Alternative splicing regulation of gene expression and therapeutic methods |
-
2020
- 2020-08-14 EP EP20851939.7A patent/EP4013414A4/en active Pending
- 2020-08-14 MX MX2022001984A patent/MX2022001984A/es unknown
- 2020-08-14 CN CN202080072237.4A patent/CN114555084A/zh active Pending
- 2020-08-14 WO PCT/US2020/046499 patent/WO2021030745A1/en not_active Ceased
- 2020-08-14 BR BR112022002794A patent/BR112022002794A2/pt unknown
- 2020-08-14 JP JP2022509153A patent/JP2022545378A/ja active Pending
- 2020-08-14 CA CA3151122A patent/CA3151122A1/en active Pending
- 2020-08-14 US US17/632,587 patent/US20220288101A1/en active Pending
- 2020-08-14 AU AU2020330108A patent/AU2020330108A1/en active Pending
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