JP7560475B2 - 操作された産生細胞株ならびにそれを作製および使用する方法 - Google Patents

操作された産生細胞株ならびにそれを作製および使用する方法 Download PDF

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JP7560475B2
JP7560475B2 JP2021559793A JP2021559793A JP7560475B2 JP 7560475 B2 JP7560475 B2 JP 7560475B2 JP 2021559793 A JP2021559793 A JP 2021559793A JP 2021559793 A JP2021559793 A JP 2021559793A JP 7560475 B2 JP7560475 B2 JP 7560475B2
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raav
sirna
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オーブリー アール． ティアナン，
ニコラス リチャーズ，
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ウルトラジェニックス ファーマシューティカル インコーポレイテッド
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JP2021559793A 2019-04-12 2020-04-09 操作された産生細胞株ならびにそれを作製および使用する方法 Active JP7560475B2 (ja)

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US201962833548P 2019-04-12 2019-04-12
US62/833,548 2019-04-12
US201962839207P 2019-04-26 2019-04-26
US62/839,207 2019-04-26
US202062979483P 2020-02-21 2020-02-21
US62/979,483 2020-02-21
PCT/US2020/027489 WO2020210507A1 (en) 2019-04-12 2020-04-09 Engineered producer cell lines and methods of making and using the same

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CN (1) CN113966395B (https=)
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MX (1) MX2021012489A (https=)
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Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023172491A1 (en) 2022-03-07 2023-09-14 Ultragenyx Pharmaceutical Inc. Modified batch aav production systems and methods
US12054738B2 (en) 2020-07-30 2024-08-06 Shape Therapeutics Inc. Stable cell lines for inducible production of rAAV virions
US20250019721A1 (en) 2021-08-11 2025-01-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
TW202323529A (zh) 2021-10-18 2023-06-16 美商再生元醫藥公司 包括腺病毒相關病毒聚核苷酸的真核細胞
US20250019668A1 (en) 2021-10-29 2025-01-16 Ultragenyx Pharmaceutical Inc. Engineered cell lines for increased production of recombinant adeno-associated virus (raav)
WO2025078616A1 (en) * 2023-10-12 2025-04-17 Cevec Pharmaceuticals Gmbh Cellular factors affecting recombinant aav production
WO2025226558A1 (en) 2024-04-22 2025-10-30 Ultragenyx Pharmaceutical Inc. Multi-step filtration and clarification methods for recombinant adeno-associated virus purification
WO2026078664A1 (en) 2024-10-10 2026-04-16 Instituto De Biologia Experimental E Tecnológica (Ibet) Transcription factor enhancing recombinant adeno-associated virus production, products, methods and uses thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008545402A (ja) 2005-05-27 2008-12-18 エボテツク・ニユーロサイエンシーズ・ゲー・エム・ベー・ハー 神経変性疾患の診断及び治療ターゲットとしてのｋｃｎｎ３
JP2017506885A (ja) 2014-01-31 2017-03-16 セクレタリー オブ ステート フォー ヘルスＳｅｃｒｅｔａｒｙ Ｏｆ Ｓｔａｔｅ Ｆｏｒ Ｈｅａｌｔｈ アデノ随伴ウイルスベクターの高力価産生

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5387484A (en) 1992-07-07 1995-02-07 International Business Machines Corporation Two-sided mask for patterning of materials with electromagnetic radiation
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US5688676A (en) 1995-06-07 1997-11-18 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
JP2001506133A (ja) 1996-12-18 2001-05-15 ターゲティッド ジェネティクス コーポレイション 組換えａａｖベクターの産生における使用のための、ａａｖスプリット−パッケージング遺伝子およびこのような遺伝子を含む細胞株
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
DK1009808T3 (da) * 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
EP1124976A1 (en) 1998-10-27 2001-08-22 Crucell Holland B.V. Improved aav vector production
DE19905501B4 (de) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
EP2311966A3 (en) 2005-10-20 2012-09-05 Amsterdam Molecular Therapeutics (AMT) B.V. Improved AAV vectors produced in insect cells
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
BR112015018493A2 (pt) 2013-02-05 2017-08-22 Univ Georgia Linhagens celulares para produção de vírus e métodos de uso
CN104592364B (zh) * 2013-10-30 2018-05-01 北京大学 定点突变和定点修饰的腺相关病毒、其制备方法及应用
US20160319277A1 (en) 2015-01-28 2016-11-03 The Johns Hopkins University METHODS OF IMPROVED PROTEIN PRODUCTION USING MIRNAs AND SIRNAs
FI3436593T3 (fi) 2016-03-28 2023-03-16 Ultragenyx Pharmaceutical Inc Adenoviruksen lämpöinaktivointimenetelmiä
WO2018013705A1 (en) 2016-07-12 2018-01-18 Dimension Therapeutics Use of glucocorticoid analogs to enhance recombinant adeno-associated virus yield
PL3526321T3 (pl) 2016-10-14 2025-04-28 Ultragenyx Pharmaceutical Inc. Zastosowanie środków regulujących ciśnienie osmotyczne w celu zwiększenia wydajności rekombinacyjnego wirusa związanego z adenowirusami
US20200048641A1 (en) 2017-03-22 2020-02-13 Ultragenyx Pharmaceutical Inc. Methods for enhancing yield of recombinant adeno-associated virus
WO2018175775A1 (en) 2017-03-22 2018-09-27 Dimension Therapeutics Cell culture methods involving hdac inhibitors or rep proteins
WO2018208960A1 (en) 2017-05-09 2018-11-15 Dimension Therapeutics, Inc. Scalable method for producing transfection reagents
EP3740572A4 (en) 2018-01-19 2022-01-05 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR INHIBITION OF THE INHIBITAL IMMUNE-REACTION RELATED TO AAV TRANSDUCTION
WO2020219543A1 (en) 2019-04-26 2020-10-29 The Regents Of The University Of California Cells for enhanced production of adeno-associated virus
US20250019668A1 (en) * 2021-10-29 2025-01-16 Ultragenyx Pharmaceutical Inc. Engineered cell lines for increased production of recombinant adeno-associated virus (raav)

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008545402A (ja) 2005-05-27 2008-12-18 エボテツク・ニユーロサイエンシーズ・ゲー・エム・ベー・ハー 神経変性疾患の診断及び治療ターゲットとしてのｋｃｎｎ３
JP2017506885A (ja) 2014-01-31 2017-03-16 セクレタリー オブ ステート フォー ヘルスＳｅｃｒｅｔａｒｙ Ｏｆ Ｓｔａｔｅ Ｆｏｒ Ｈｅａｌｔｈ アデノ随伴ウイルスベクターの高力価産生

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
The Journal of Biological Chemistry，2013年，Vol.288, No.15，pp.10792-10804

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US20200325455A1 (en) 2020-10-15
US12018288B2 (en) 2024-06-25
CO2021013699A2 (es) 2021-10-29
EP4603579A3 (en) 2025-12-10
MX2021012489A (es) 2021-11-12
EP4603579A2 (en) 2025-08-20
US20240376440A1 (en) 2024-11-14
TW202104593A (zh) 2021-02-01
KR20210152514A (ko) 2021-12-15
CA3136246A1 (en) 2020-10-15
CN113966395A (zh) 2022-01-21
IL287106A (en) 2021-12-01
CN113966395B (zh) 2025-07-04
JP7706607B2 (ja) 2025-07-11
PH12021552577A1 (en) 2022-04-04
AU2020270960A1 (en) 2021-11-04
JP2022526646A (ja) 2022-05-25
JP2024107295A (ja) 2024-08-08
EP3953458A1 (en) 2022-02-16
AU2020270960B2 (en) 2026-01-29
SG11202110904PA (en) 2021-10-28
WO2020210507A1 (en) 2020-10-15
TWI859216B (zh) 2024-10-21
BR112021020391A2 (pt) 2022-02-08

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