JP2021513355A - 非ウイルス性dnaベクター、ならびに抗体および融合タンパク質の産生のためのその使用 - Google Patents

非ウイルス性dnaベクター、ならびに抗体および融合タンパク質の産生のためのその使用 Download PDF

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JP2021513355A
JP2021513355A JP2020543344A JP2020543344A JP2021513355A JP 2021513355 A JP2021513355 A JP 2021513355A JP 2020543344 A JP2020543344 A JP 2020543344A JP 2020543344 A JP2020543344 A JP 2020543344A JP 2021513355 A JP2021513355 A JP 2021513355A
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antibody
itr
cedna vector
cedna
sequence
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Inventor
オザン アルカン,
オザン アルカン,
ダグラス アンソニー カー,
ダグラス アンソニー カー,
ロバート マイケル コーティン,
ロバート マイケル コーティン,
デブラ クラット,
デブラ クラット,
リア リウ,
リア リウ,
ナサニエル シルバー,
ナサニエル シルバー,
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ジェネレーション バイオ カンパニー
ジェネレーション バイオ カンパニー
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Publication of JP2021513355A publication Critical patent/JP2021513355A/ja
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JP2020543344A 2018-02-14 2019-02-14 非ウイルス性dnaベクター、ならびに抗体および融合タンパク質の産生のためのその使用 Pending JP2021513355A (ja)

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JP2024141968A JP2024167281A (ja) 2018-02-14 2024-08-23 非ウイルス性dnaベクター、ならびに抗体および融合タンパク質の産生のためのその使用

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US201862630670P 2018-02-14 2018-02-14
US201862630676P 2018-02-14 2018-02-14
US62/630,676 2018-02-14
US62/630,670 2018-02-14
US201862680087P 2018-06-04 2018-06-04
US201862680092P 2018-06-04 2018-06-04
US62/680,087 2018-06-04
US62/680,092 2018-06-04
PCT/US2019/018016 WO2019161059A1 (en) 2018-02-14 2019-02-14 Non-viral dna vectors and uses thereof for antibody and fusion protein production

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EP (1) EP3752191A4 (https=)
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KR (1) KR20200120649A (https=)
CN (1) CN111818942A (https=)
AU (1) AU2019221642A1 (https=)
BR (1) BR112020016288A2 (https=)
CA (1) CA3091250A1 (https=)
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MA (1) MA51842A (https=)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023517940A (ja) * 2020-03-11 2023-04-27 イマティクス ユーエス,アイエヌシー. Wpre変異体コンストラクト、組成物、およびその方法

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
US12442015B2 (en) * 2018-01-19 2025-10-14 Generation Bio Co. Closed-ended DNA vectors obtainable from cell-free synthesis and process for obtaining ceDNA vectors
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
WO2021046265A1 (en) * 2019-09-06 2021-03-11 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof
WO2021169167A1 (en) * 2020-02-29 2021-09-02 Nanjing GenScript Biotech Co., Ltd. Method for treating coronavirus infections
JP2023520763A (ja) * 2020-03-24 2023-05-19 ジェネレーション バイオ カンパニー ゴーシェ治療薬を発現するための非ウイルス性dnaベクター及びその使用
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Dna分子の組成物、その作製方法、及びその使用方法
US20240066149A1 (en) * 2021-01-12 2024-02-29 Jcr Pharmaceuticals Co., Ltd. Nucleic Acid Molecule Containing Incorporated Gene Encoding Fused Protein of Ligand and Protein Having Physiological Activity
CA3206107A1 (en) * 2021-01-26 2022-08-04 Brian Furmanski Vector constructs for delivery of nucleic acids encoding therapeutic anti-tnf antibodies and methods of using the same
AU2022264509A1 (en) * 2021-04-27 2023-12-14 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
CA3218126A1 (en) * 2021-05-07 2022-11-10 Matthew MANGANIELLO Lyophilized non-viral dna vector compositions and uses thereof
WO2023091708A1 (en) * 2021-11-18 2023-05-25 The Brigham And Women's Hospital, Inc. Induced proteinopathy models
CN120282800A (zh) * 2022-12-12 2025-07-08 苏州荷光科汇生物科技有限公司 一种感染视网膜的aav载体、阿达木单抗及其应用
US11993783B1 (en) * 2023-03-27 2024-05-28 Genecraft Inc. Nucleic acid molecule comprising asymmetrically modified ITR for improving expression rate of inserted gene, and use thereof
CN116549632B (zh) * 2023-03-28 2024-09-10 中国人民解放军军事科学院军事医学研究院 抗Lingo-1抗体Opicinumab的新用途及针对新用途的药物
EP4713372A1 (en) * 2023-05-15 2026-03-25 Immunecare Biopharmaceuticals (Shanghai) Co., Ltd Recombinant fusion protein for treatment of pulmonary hypertension
WO2025092951A1 (en) * 2023-11-03 2025-05-08 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating fabry disease

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005517385A (ja) * 2001-08-27 2005-06-16 ジェネンテック・インコーポレーテッド 抗体発現系とその構築法
WO2010097437A1 (en) * 2009-02-25 2010-09-02 Ucb Pharma, S.A. Method for producing antibodies
WO2017152149A1 (en) * 2016-03-03 2017-09-08 University Of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE318923T1 (de) * 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
US8865881B2 (en) * 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
EP2500434A1 (en) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
WO2014143932A1 (en) * 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
BR112016017817A2 (pt) * 2014-02-06 2017-10-10 Genzyme Corp composições e métodos para o tratamento e a prevenção de degeneração macular
EP3151866B1 (en) * 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
US11549125B2 (en) * 2017-08-09 2023-01-10 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Closed-ended, linear, duplex adenoassociated virus DNA, and uses thereof
KR102696307B1 (ko) * 2017-09-08 2024-08-16 제너레이션 바이오 컴퍼니 비-바이러스성 캡시드-비함유 dna 벡터의 지질 나노입자 제형
CA3075168A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. Modified closed-ended dna (cedna)
MX2020003888A (es) * 2017-10-18 2020-11-06 Regenxbio Inc Anticuerpos terapeuticos postraduccionalmente modificados completamente humanos.
CA3084185A1 (en) * 2017-12-06 2019-06-13 Generation Bio Co. Gene editing using a modified closed-ended dna (cedna)
US12442015B2 (en) * 2018-01-19 2025-10-14 Generation Bio Co. Closed-ended DNA vectors obtainable from cell-free synthesis and process for obtaining ceDNA vectors
MA52116A (fr) * 2018-03-02 2021-01-06 Generation Bio Co Vecteurs d'adn à extrémité fermée (cedna) pour l'insertion de transgènes au niveau de havres génomiques sécuritaires (gsh) dans des génomes humains et murins

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005517385A (ja) * 2001-08-27 2005-06-16 ジェネンテック・インコーポレーテッド 抗体発現系とその構築法
WO2010097437A1 (en) * 2009-02-25 2010-09-02 Ucb Pharma, S.A. Method for producing antibodies
WO2017152149A1 (en) * 2016-03-03 2017-09-08 University Of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
INT. J. RETIN. VITR., vol. Vol. 2:3, JPN6023013132, 2016, pages 1 - 9, ISSN: 0005027976 *
MICROBIOL. SPECTR., vol. Vol. 2(4), JPN6024015509, 2014, pages 1 - 11, ISSN: 0005509353 *
PREP. BIOCHEM. BIOTECHNOL., vol. 48, JPN6023040904, February 2018 (2018-02-01), pages 160 - 164, ISSN: 0005309229 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023517940A (ja) * 2020-03-11 2023-04-27 イマティクス ユーエス,アイエヌシー. Wpre変異体コンストラクト、組成物、およびその方法
JP7739319B2 (ja) 2020-03-11 2025-09-16 イマティクス ユーエス,アイエヌシー. Wpre変異体コンストラクト、組成物、およびその方法

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CA3091250A1 (en) 2019-08-22
AU2019221642A1 (en) 2020-07-09
US20220042035A1 (en) 2022-02-10
SG11202006431WA (en) 2020-08-28
MX2020008470A (es) 2020-09-25
IL276469A (en) 2020-09-30
JP2024167281A (ja) 2024-12-03
KR20200120649A (ko) 2020-10-21
PH12020551039A1 (en) 2021-08-23
MA51842A (fr) 2020-12-23
CN111818942A (zh) 2020-10-23
EP3752191A4 (en) 2021-12-22
RU2020130010A (ru) 2022-03-14
WO2019161059A1 (en) 2019-08-22
EP3752191A1 (en) 2020-12-23

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