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 (enExample) | 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 (enExample) |
| EP (1) | EP4013414A4 (enExample) |
| JP (1) | JP2022545378A (enExample) |
| CN (1) | CN114555084A (enExample) |
| AU (1) | AU2020330108A1 (enExample) |
| BR (1) | BR112022002794A2 (enExample) |
| CA (1) | CA3151122A1 (enExample) |
| MX (1) | MX2022001984A (enExample) |
| WO (1) | WO2021030745A1 (enExample) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10820847B1 (en) | 2019-08-15 | 2020-11-03 | Talis Biomedical Corporation | Diagnostic system |
| US20260043024A1 (en) * | 2022-08-26 | 2026-02-12 | The Children's Hospital Of Philadelphia | Mirnas targeting atnx2 for the treatment of als and sca2 |
| CN120603600A (zh) * | 2023-01-19 | 2025-09-05 | 优尼科生物制药有限公司 | 基因递送媒介物的药物配制品 |
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 |
| JP6091435B2 (ja) * | 2011-02-22 | 2017-03-08 | カリフォルニア インスティチュート オブ テクノロジー | アデノ随伴ウイルス(aav)ベクターを用いたタンパク質の送達 |
| PT3628334T (pt) * | 2014-03-21 | 2023-09-26 | Genzyme Corp | Terapia genética para retinopatia pigmentar |
| AU2015264263B2 (en) * | 2014-05-20 | 2021-08-05 | 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 |
| CA2977607A1 (en) * | 2015-02-26 | 2016-09-01 | Jacob Schneiderman | 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 |
| IL302455A (en) * | 2016-03-01 | 2023-06-01 | Univ Florida | Adeno-associated virus vectors for the treatment of dominant retinitis pigmentosa |
| 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 WO PCT/US2020/046499 patent/WO2021030745A1/en not_active Ceased
- 2020-08-14 MX MX2022001984A patent/MX2022001984A/es unknown
- 2020-08-14 CA CA3151122A patent/CA3151122A1/en active Pending
- 2020-08-14 JP JP2022509153A patent/JP2022545378A/ja active Pending
- 2020-08-14 EP EP20851939.7A patent/EP4013414A4/en active Pending
- 2020-08-14 CN CN202080072237.4A patent/CN114555084A/zh active Pending
- 2020-08-14 US US17/632,587 patent/US20220288101A1/en active Pending
- 2020-08-14 BR BR112022002794A patent/BR112022002794A2/pt unknown
- 2020-08-14 AU AU2020330108A patent/AU2020330108A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2023514242A5 (enExample) | ||
| JP2021532812A5 (enExample) | ||
| JP2020533959A5 (enExample) | ||
| CN109069672B (zh) | 腺相关病毒载体传递微肌营养不良蛋白以治疗肌营养不良症 | |
| JP2022551986A5 (enExample) | ||
| JP2024045131A (ja) | 神経変性疾患の遺伝子治療 | |
| JP2025162557A5 (enExample) | ||
| JP2020513811A5 (enExample) | ||
| JP2021512072A5 (enExample) | ||
| US20210128749A1 (en) | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy | |
| JP2022535749A5 (enExample) | ||
| JP2022547197A5 (enExample) | ||
| JP2023501897A5 (enExample) | ||
| US20230416757A1 (en) | Sod1 dual expression vectors and uses thereof | |
| JP2020519629A5 (enExample) | ||
| JP2022551744A5 (enExample) | ||
| CA3158518A1 (en) | Triple function adeno-associated virus (aav)vectors for the treatment of c9orf72 associated diseases | |
| JP2023500793A5 (enExample) | ||
| JP2023537358A5 (enExample) | ||
| JP2021534766A5 (enExample) | ||
| JP2024539123A5 (enExample) | ||
| JP2023059858A5 (enExample) | ||
| CN120548368A (zh) | 用于正确包装重复元件的腺相关病毒载体 | |
| CN121925426A (zh) | 肝脏去靶向衣壳 | |
| JP2023542131A5 (enExample) |