JP2022522756A5 - - Google Patents
Info
- Publication number
- JP2022522756A5 JP2022522756A5 JP2021551562A JP2021551562A JP2022522756A5 JP 2022522756 A5 JP2022522756 A5 JP 2022522756A5 JP 2021551562 A JP2021551562 A JP 2021551562A JP 2021551562 A JP2021551562 A JP 2021551562A JP 2022522756 A5 JP2022522756 A5 JP 2022522756A5
- Authority
- JP
- Japan
- Prior art keywords
- protein
- composition according
- dystrophy
- eye
- corneal
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024218892A JP2025061656A (ja) | 2019-02-28 | 2024-12-13 | 治療薬の送達のためのアデノ随伴ウイルスベクター |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962812017P | 2019-02-28 | 2019-02-28 | |
| US62/812,017 | 2019-02-28 | ||
| US201962831838P | 2019-04-10 | 2019-04-10 | |
| US62/831,838 | 2019-04-10 | ||
| US201962878865P | 2019-07-26 | 2019-07-26 | |
| US62/878,865 | 2019-07-26 | ||
| PCT/US2020/020134 WO2020176747A1 (en) | 2019-02-28 | 2020-02-27 | Adeno-associated virus vectors for the delivery of therapeutics |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024218892A Division JP2025061656A (ja) | 2019-02-28 | 2024-12-13 | 治療薬の送達のためのアデノ随伴ウイルスベクター |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022522756A JP2022522756A (ja) | 2022-04-20 |
| JP2022522756A5 true JP2022522756A5 (enExample) | 2023-03-06 |
Family
ID=70155318
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021551562A Pending JP2022522756A (ja) | 2019-02-28 | 2020-02-27 | 治療薬の送達のためのアデノ随伴ウイルスベクター |
| JP2024218892A Pending JP2025061656A (ja) | 2019-02-28 | 2024-12-13 | 治療薬の送達のためのアデノ随伴ウイルスベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024218892A Pending JP2025061656A (ja) | 2019-02-28 | 2024-12-13 | 治療薬の送達のためのアデノ随伴ウイルスベクター |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20220047618A1 (enExample) |
| EP (1) | EP3930694A1 (enExample) |
| JP (2) | JP2022522756A (enExample) |
| CN (1) | CN113557010A (enExample) |
| AU (2) | AU2020229871B2 (enExample) |
| CA (1) | CA3131919A1 (enExample) |
| IL (1) | IL285639A (enExample) |
| WO (1) | WO2020176747A1 (enExample) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2022216614B2 (en) | 2021-02-05 | 2026-03-05 | Christiana Care Gene Editing Institute, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| CN115505593B (zh) * | 2021-06-23 | 2025-10-10 | 武汉纽福斯生物科技有限公司 | 寡核苷酸、病毒载体及其应用和RNAi药物制剂 |
| US20250319202A1 (en) * | 2021-11-09 | 2025-10-16 | Prime Medicine, Inc. | Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy |
| WO2023111351A1 (en) * | 2021-12-17 | 2023-06-22 | Christian Kupatt | Aav vectors for gene therapy in endothelial cells |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| CN103502458B (zh) | 2011-02-17 | 2016-11-16 | 宾夕法尼亚大学托管会 | 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法 |
| DK3693025T3 (da) | 2011-04-22 | 2022-01-10 | Univ California | Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf |
| CN105899658B (zh) * | 2013-12-12 | 2020-02-18 | 布罗德研究所有限公司 | 针对hbv和病毒性疾病以及障碍的crispr-cas系统和组合物的递送、用途和治疗应用 |
| CN111206032B (zh) * | 2013-12-12 | 2024-07-19 | 布罗德研究所有限公司 | 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用 |
| IL251178B (en) | 2014-09-16 | 2022-07-01 | Genzyme Corp | Adeno-associated viral vector for the treatment of glaucoma myocilin (myoc) |
| PL3298134T3 (pl) * | 2015-05-16 | 2023-09-18 | Genzyme Corporation | Edycja genów mutacji głęboko intronowych |
| AU2016335032B2 (en) * | 2015-10-05 | 2022-04-14 | Proqr Therapeutics Ii B.V. | Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion |
| WO2017178590A1 (en) * | 2016-04-14 | 2017-10-19 | Université de Lausanne | Treatment and/or prevention of dna-triplet repeat diseases or disorders |
| JP2019515914A (ja) * | 2016-04-22 | 2019-06-13 | インテリア セラピューティクス，インコーポレイテッド | 転写因子４内のトリヌクレオチドリピートと関連する疾患の治療のための組成物および方法 |
| EP3342868B1 (en) * | 2016-12-30 | 2019-12-25 | Systasy Bioscience GmbH | Constructs and screening methods |
| EP3570888A1 (en) * | 2017-03-03 | 2019-11-27 | MacRegen, Inc. | Treatment of age-related macular degeneration and other eye diseases with one or more therapeutic agents |
| WO2018165541A1 (en) * | 2017-03-10 | 2018-09-13 | The Board Of Regents Of The University Of Texas System | Treatment of fuchs' endothelial corneal dystrophy |
| CA3071769A1 (en) * | 2017-07-31 | 2019-02-07 | Reflection Biotechnologies Limited | Cellular models of and therapies for ocular diseases |
-
2020
- 2020-02-27 EP EP20716596.0A patent/EP3930694A1/en active Pending
- 2020-02-27 CA CA3131919A patent/CA3131919A1/en active Pending
- 2020-02-27 CN CN202080017179.5A patent/CN113557010A/zh active Pending
- 2020-02-27 AU AU2020229871A patent/AU2020229871B2/en active Active
- 2020-02-27 WO PCT/US2020/020134 patent/WO2020176747A1/en not_active Ceased
- 2020-02-27 JP JP2021551562A patent/JP2022522756A/ja active Pending
- 2020-02-27 US US17/433,887 patent/US20220047618A1/en active Pending
-
2021
- 2021-08-16 IL IL285639A patent/IL285639A/en unknown
-
2023
- 2023-08-17 AU AU2023216849A patent/AU2023216849A1/en not_active Abandoned
-
2024
- 2024-12-13 JP JP2024218892A patent/JP2025061656A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7469538B2 (ja) | 調節性ポリヌクレオチド | |
| US20260117224A1 (en) | Modulatory polynucleotides | |
| US20240226203A9 (en) | Compositions and methods of treating huntington's disease | |
| US11697825B2 (en) | Compositions and methods for the production of scAAV | |
| US20210214749A1 (en) | Directed evolution | |
| US20180245073A1 (en) | Regulatable expression using adeno-associated virus (aav) | |
| JP2020503869A (ja) | トリプルガイド配列と共にｃｒｉｓｐｒ／ｃａｓ９を用いるエクソンスキッピング改変のための最適化戦略 | |
| JP2019530463A (ja) | Ａａｖカプシド設計 | |
| BR112018073472A2 (pt) | composições e métodos de tratamento da doença de huntington | |
| TW201905200A (zh) | 調節多核苷酸 | |
| JP2018506980A5 (enExample) | ||
| US20220168450A1 (en) | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord | |
| US20200377887A1 (en) | Compositions and methods of treating huntington's disease | |
| JP2021529513A (ja) | 筋萎縮性側索硬化症および脊髄に関連する障害の治療 | |
| JP2018509154A5 (enExample) | ||
| JP2025061656A (ja) | 治療薬の送達のためのアデノ随伴ウイルスベクター | |
| CN116218849B (zh) | 针对utrophin激活治疗杜氏肌营养不良的sgRNA和应用 | |
| US20220333133A1 (en) | Vectorized editing of nucleic acids to correct overt mutations | |
| CN111705069A (zh) | 一种多神经营养因子联合表达载体及其应用 | |
| WO2023078099A1 (zh) | 一种用于治疗神经损伤疾病的基因药物 | |
| CA3239289A1 (en) | Functional aav capsids for intravitreal administration | |
| CN117721194A (zh) | Ap2a1基因在制备高血压防治产品中的用途 | |
| CN120718885A (zh) | 一种用于治疗青光眼的gRNA、AAV表达载体、CRISPR-CasRx系统 | |
| CN117045791A (zh) | 肝星状细胞neu1的靶向过表达剂在制备治疗肝纤维化的药物中的应用 | |
| EA051485B1 (ru) | Генная терапия kcnv2 |