JPWO2020198737A5 - - Google Patents

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Publication number
JPWO2020198737A5
JPWO2020198737A5 JP2021557657A JP2021557657A JPWO2020198737A5 JP WO2020198737 A5 JPWO2020198737 A5 JP WO2020198737A5 JP 2021557657 A JP2021557657 A JP 2021557657A JP 2021557657 A JP2021557657 A JP 2021557657A JP WO2020198737 A5 JPWO2020198737 A5 JP WO2020198737A5
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JP
Japan
Prior art keywords
aav
cells
library
sequence
heptamer
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
Application number
JP2021557657A
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English (en)
Japanese (ja)
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JP2022527917A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/025720 external-priority patent/WO2020198737A1/en
Publication of JP2022527917A publication Critical patent/JP2022527917A/ja
Publication of JPWO2020198737A5 publication Critical patent/JPWO2020198737A5/ja
Pending legal-status Critical Current

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JP2021557657A 2019-03-28 2020-03-30 導入遺伝子発現のための操作されたアデノ随伴（ａａｖ）ベクター Pending JP2022527917A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962825703P 2019-03-28 2019-03-28
US62/825,703 2019-03-28
PCT/US2020/025720 WO2020198737A1 (en) 2019-03-28 2020-03-30 Engineered adeno-associated (aav) vectors for transgene expression

Publications (2)

Publication Number Publication Date
JP2022527917A JP2022527917A (ja) 2022-06-07
JPWO2020198737A5 true JPWO2020198737A5 (de) 2023-04-06

Family

ID=72609527

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021557657A Pending JP2022527917A (ja) 2019-03-28 2020-03-30 導入遺伝子発現のための操作されたアデノ随伴（ａａｖ）ベクター

Country Status (12)

Country Link
US (1) US20220195458A1 (de)
EP (1) EP3947422A4 (de)
JP (1) JP2022527917A (de)
KR (1) KR20210143869A (de)
CN (1) CN113874387A (de)
AU (1) AU2020248116A1 (de)
BR (1) BR112021019436A2 (de)
CA (1) CA3135292A1 (de)
IL (1) IL286725A (de)
MX (1) MX2021011701A (de)
SG (1) SG11202110165XA (de)
WO (1) WO2020198737A1 (de)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019226516A1 (en) 2018-05-24 2019-11-28 Celanese EVA Performance Polymers Corporation Implantable device for sustained release of a macromolecular drug compound
CN111989068A (zh) 2018-05-24 2020-11-24 塞拉尼斯伊娃高性能聚合物公司 用于持续释放大分子药物化合物的可植入器件
AU2019346447A1 (en) 2018-09-26 2021-04-29 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
CA3171166A1 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
IL309742A (en) 2021-07-08 2024-02-01 Tenaya Therapeutics Inc Optimal expression cassettes for gene therapy
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060021087A1 (en) * 2004-04-09 2006-01-26 Baum James A Compositions and methods for control of insect infestations in plants
EP2058401A1 (de) * 2007-10-05 2009-05-13 Genethon Weitverbreitete Genverabreichung an Motorneuronen unter Verwendung einer peripheren Injektion von AAV-Vektoren
SG10201809075XA (en) * 2013-07-22 2018-11-29 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
ES2739288T3 (es) * 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
CA3234028A1 (en) * 2014-03-11 2015-09-17 Wayne State University A modified mglur6 promoter and methods of use
GB2545763A (en) * 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
US11826433B2 (en) * 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
EP3491008A2 (de) * 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Neuartige adeno-assoziierte virus-kapsid-proteine
EP3662060A2 (de) * 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur aav-freisetzung
CN110709511A (zh) * 2017-08-28 2020-01-17 加利福尼亚大学董事会 腺相关病毒衣壳变体及其使用方法
CN109207520B (zh) * 2018-09-25 2022-05-10 北京锦篮基因科技有限公司 Leber遗传性视神经病变的基因药物

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