JP2019509748A - 組換えタンパク質及び／又はウイルスベクター製造のための細胞株 - Google Patents

組換えタンパク質及び／又はウイルスベクター製造のための細胞株 Download PDF

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JP2019509748A
JP2019509748A JP2018550831A JP2018550831A JP2019509748A JP 2019509748 A JP2019509748 A JP 2019509748A JP 2018550831 A JP2018550831 A JP 2018550831A JP 2018550831 A JP2018550831 A JP 2018550831A JP 2019509748 A JP2019509748 A JP 2019509748A
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cell
hek
nucleic acid
cell line
factor
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JP2019509748A5 (enExample
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ジンミン ジョウ，
ジンミン ジョウ，
グワン チィ，
グワン チィ，
ジョン フレーザー ライト，
ジョン フレーザー ライト，
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Spark Therapeutics Inc
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Spark Therapeutics Inc
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Priority to JP2022070121A priority Critical patent/JP2022101642A/ja
Priority to JP2025019374A priority patent/JP2025087704A/ja
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JP2018550831A 2016-03-30 2017-03-30 組換えタンパク質及び／又はウイルスベクター製造のための細胞株 Withdrawn JP2019509748A (ja)

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JP2022070121A JP2022101642A (ja) 2016-03-30 2022-04-21 組換えタンパク質及び／又はウイルスベクター製造のための細胞株
JP2025019374A JP2025087704A (ja) 2016-03-30 2025-02-07 組換えタンパク質及び／又はウイルスベクター製造のための細胞株

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US201662315480P 2016-03-30 2016-03-30
US62/315,480 2016-03-30
PCT/US2017/024951 WO2017173043A1 (en) 2016-03-30 2017-03-30 Cell line for recombinant protein and/or viral vector production

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JP2022070121A Pending JP2022101642A (ja) 2016-03-30 2022-04-21 組換えタンパク質及び／又はウイルスベクター製造のための細胞株
JP2025019374A Pending JP2025087704A (ja) 2016-03-30 2025-02-07 組換えタンパク質及び／又はウイルスベクター製造のための細胞株

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JP2025019374A Pending JP2025087704A (ja) 2016-03-30 2025-02-07 組換えタンパク質及び／又はウイルスベクター製造のための細胞株

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US (1) US20190078099A1 (enExample)
EP (1) EP3436576A4 (enExample)
JP (3) JP2019509748A (enExample)
KR (1) KR102479894B1 (enExample)
CN (1) CN109563496B (enExample)
AU (2) AU2017244133A1 (enExample)
BR (1) BR112018070250A2 (enExample)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022138869A1 (ja) * 2020-12-25 2022-06-30 Ａｇｃ株式会社 ベクター産生能力が向上したウイルスベクター産生細胞及びその製造方法及びその選択方法

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
KR20200018488A (ko) 2017-05-24 2020-02-19 토리스 게엠베하 고암모니아혈증을 치료하기 위한 글루타민 합성효소의 용도
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP2020525003A (ja) * 2017-06-21 2020-08-27 バートナム，ティモシー，エー． 腎臓病治療のための免疫特権を有する生物活性腎細胞
MX2020004035A (es) 2017-10-20 2020-11-09 Res Inst Nationwide Childrens Hospital Métodos y materiales para terapia génica con nt-3.
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
WO2020012446A1 (en) * 2018-07-13 2020-01-16 Enzene Biosciences Limited Double knock-out cho cell line method of its generation and producing therapeutic proteins therefrom
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
AU2019354296A1 (en) * 2018-10-01 2021-04-29 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
US12686874B2 (en) 2020-03-31 2026-07-21 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
US12054738B2 (en) 2020-07-30 2024-08-06 Shape Therapeutics Inc. Stable cell lines for inducible production of rAAV virions
EP4301861A1 (en) 2021-03-03 2024-01-10 Shape Therapeutics Inc. Auxotrophic cells for virus production and compositions and methods of making
AR127674A1 (es) 2021-10-18 2024-02-21 Regeneron Pharma Células de mamífero que comprenden genes cas9 integrados para producir sitios de integración estables, y células de mamífero que comprenden sitios de integración estables y otros sitios
JP2024537896A (ja) 2021-10-18 2024-10-16 リジェネロン・ファーマシューティカルズ・インコーポレイテッド アデノウイルス随伴ウイルスポリヌクレオチドを含む真核細胞
WO2023150506A2 (en) * 2022-02-01 2023-08-10 Shape Therapeutics Inc. STABLE CELL LINES FOR INDUCIBLE PRODUCTION OF rAAV VIRIONS
EP4514844A1 (en) 2022-04-29 2025-03-05 Broadwing Bio LLC Complement factor h related 4-specific antibodies and uses thereof
CN119487063A (zh) 2022-04-29 2025-02-18 博闻生物有限责任公司 双特异性抗体以及治疗眼部疾病的方法
AU2023259251A1 (en) 2022-04-29 2024-11-14 Broadwing Bio, Inc. Angiopoietin-related protein 7-specific antibodies and uses thereof
WO2024227154A1 (en) 2023-04-28 2024-10-31 Broadwing Bio Llc Complement component 3 (c3)-specific antibodies and uses thereof
CN122349567A (zh) 2023-12-11 2026-07-07 济世易为生物有限公司 利用反式剪接和拆分型选择标记的蛋白质表达

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2009537140A (ja) * 2006-05-19 2009-10-29 サンガモ バイオサイエンシーズ， インコーポレイテッド ジヒドロ葉酸還元酵素の不活性化のための方法及び組成物
JP2012507282A (ja) * 2008-10-29 2012-03-29 サンガモ バイオサイエンシーズ， インコーポレイテッド グルタミンシンセターゼ遺伝子の発現を不活性化する方法及び組成物
WO2012167192A2 (en) * 2011-06-01 2012-12-06 Precision Biosciences, Inc. Methods and products for producing engineered mammalian cell lines with amplified transgenes
AU2015201300A1 (en) * 2008-10-29 2015-04-02 Sangamo Therapeutics, Inc. Methods and Compositions For Inactivating Glutamine Synthetase Gene Expression

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080001001A1 (en) * 2006-04-17 2008-01-03 Pevnick Stephen H Pneumatic Activated Fountain
BR112014020325A2 (pt) * 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
PT2970920T (pt) * 2013-03-15 2018-07-30 Childrens Hospital Philadelphia Processo de fabrico escalável para produzir vetores lentivirais recombinantes em sistema de cultura de células de suspensão livres de soro
NZ716102A (en) * 2013-07-22 2021-12-24 The Children’S Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
KR20170081784A (ko) * 2016-01-04 2017-07-13 한국과학기술원 Gs 유전자가 결핍된 신규한 hek293 세포주 및 상기 형질전환된 hek293 숙주세포를 이용한 목적 단백질의 생산 방법

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2009537140A (ja) * 2006-05-19 2009-10-29 サンガモ バイオサイエンシーズ， インコーポレイテッド ジヒドロ葉酸還元酵素の不活性化のための方法及び組成物
JP2012507282A (ja) * 2008-10-29 2012-03-29 サンガモ バイオサイエンシーズ， インコーポレイテッド グルタミンシンセターゼ遺伝子の発現を不活性化する方法及び組成物
AU2015201300A1 (en) * 2008-10-29 2015-04-02 Sangamo Therapeutics, Inc. Methods and Compositions For Inactivating Glutamine Synthetase Gene Expression
WO2012167192A2 (en) * 2011-06-01 2012-12-06 Precision Biosciences, Inc. Methods and products for producing engineered mammalian cell lines with amplified transgenes

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
APPL MICROBIOL BIOTECHNOL, vol. No.99, p.10117-10126, JPN7021000329, 2015, ISSN: 0004439937 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022138869A1 (ja) * 2020-12-25 2022-06-30 Ａｇｃ株式会社 ベクター産生能力が向上したウイルスベクター産生細胞及びその製造方法及びその選択方法

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