JP2023545830A - 組換えアデノ随伴ウイルス組成物及びその生産方法 - Google Patents

組換えアデノ随伴ウイルス組成物及びその生産方法 Download PDF

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JP2023545830A
JP2023545830A JP2023523244A JP2023523244A JP2023545830A JP 2023545830 A JP2023545830 A JP 2023545830A JP 2023523244 A JP2023523244 A JP 2023523244A JP 2023523244 A JP2023523244 A JP 2023523244A JP 2023545830 A JP2023545830 A JP 2023545830A
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therapeutic composition
raav
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cells
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JPWO2022082017A5 (https=
JP2023545830A5 (https=
Inventor
ダイ，ヨン
ジョウ，ジンミン
ダニエルズ，ギャレット
チャン，ジョナサン
ヘラー，ジョージ
ネルソン，スチュアート
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Prevail Therapeutics Inc
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023523244A 2020-10-15 2021-10-15 組換えアデノ随伴ウイルス組成物及びその生産方法 Pending JP2023545830A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063092179P 2020-10-15 2020-10-15
US63/092,179 2020-10-15
PCT/US2021/055243 WO2022082017A2 (en) 2020-10-15 2021-10-15 Recombinant adeno-associated virus compositions and methods for producing same

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JP2023545830A true JP2023545830A (ja) 2023-10-31
JPWO2022082017A5 JPWO2022082017A5 (https=) 2024-10-24
JP2023545830A5 JP2023545830A5 (https=) 2024-10-24

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US (1) US20220119843A1 (https=)
EP (1) EP4229206A2 (https=)
JP (1) JP2023545830A (https=)
KR (1) KR20230088393A (https=)
CN (1) CN116710567A (https=)
AU (1) AU2021359852A1 (https=)
CA (1) CA3198675A1 (https=)
IL (1) IL302109A (https=)
MX (1) MX2023004420A (https=)
WO (1) WO2022082017A2 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4598940A1 (en) 2022-10-04 2025-08-13 Eli Lilly and Company Gene therapy for trem2-associated diseases and disorders
EP4615981A1 (en) 2022-11-11 2025-09-17 Eli Lilly and Company Synthetic nucleic acids including astrocyte-directed promoter constructs and methods of using the same
CN116350801A (zh) * 2022-11-22 2023-06-30 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途
TW202432839A (zh) * 2022-12-06 2024-08-16 美商再生元醫藥公司 用於確定病毒載體基因體滴度之液滴式數位ｐｃｒ分析
WO2025190864A1 (en) * 2024-03-11 2025-09-18 Sartorius Stedim Biotech Gmbh Improved cell lysis methods, reagents, and kits for viral vector release and production
WO2026043765A1 (en) 2024-08-21 2026-02-26 Eli Lilly And Company Engineered regulatory splice elements for gene therapies

Citations (6)

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Publication number Priority date Publication date Assignee Title
WO2007084773A2 (en) * 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
US20180155697A1 (en) * 2015-12-24 2018-06-07 Wuhan Institute Of Physics And Mathematics, Chinese Academy Of Sciences Method of preparing recombinant adeno-associated virus and recombinant baculovirus
WO2019070894A1 (en) * 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMIAL DISORDERS
WO2019094253A1 (en) * 2017-11-08 2019-05-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
US20200231954A1 (en) * 2017-10-03 2020-07-23 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US20200316221A1 (en) * 2016-10-13 2020-10-08 University Of Massachusetts Aav capsid designs

Family Cites Families (4)

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Publication number Priority date Publication date Assignee Title
AU2002359284A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
EP3387138B1 (en) * 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
AU2018354195A1 (en) 2017-10-23 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007084773A2 (en) * 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
US20180155697A1 (en) * 2015-12-24 2018-06-07 Wuhan Institute Of Physics And Mathematics, Chinese Academy Of Sciences Method of preparing recombinant adeno-associated virus and recombinant baculovirus
US20200316221A1 (en) * 2016-10-13 2020-10-08 University Of Massachusetts Aav capsid designs
WO2019070894A1 (en) * 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMIAL DISORDERS
US20200231954A1 (en) * 2017-10-03 2020-07-23 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
WO2019094253A1 (en) * 2017-11-08 2019-05-16 Avexis Inc. Means and method for preparing viral vectors and uses of same

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
NOVEMBER, MOLECULAR THERAPY, vol. Volume 17 Issue 11, JPN6025031839, 2009, pages 1888 - 1896, ISSN: 0005753905 *

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US20220119843A1 (en) 2022-04-21
AU2021359852A9 (en) 2025-03-27
IL302109A (en) 2023-06-01
WO2022082017A3 (en) 2022-07-21
WO2022082017A9 (en) 2022-10-13
CN116710567A (zh) 2023-09-05
MX2023004420A (es) 2023-07-05
KR20230088393A (ko) 2023-06-19
WO2022082017A2 (en) 2022-04-21
AU2021359852A1 (en) 2023-06-08
CA3198675A1 (en) 2022-04-21
EP4229206A2 (en) 2023-08-23

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