JP2020532981A - 修飾型閉端ｄｎａ（ｃｅｄｎａ） - Google Patents

修飾型閉端ｄｎａ（ｃｅｄｎａ） Download PDF

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JP2020532981A
JP2020532981A JP2020512843A JP2020512843A JP2020532981A JP 2020532981 A JP2020532981 A JP 2020532981A JP 2020512843 A JP2020512843 A JP 2020512843A JP 2020512843 A JP2020512843 A JP 2020512843A JP 2020532981 A JP2020532981 A JP 2020532981A
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itr
cedna
cedna vector
seq
sequence
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JP2020532981A5 (enExample
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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，
オザン アルカン，
オザン アルカン，
アンナリーゼ ジョーンズ，
アンナリーゼ ジョーンズ，
ダグラス アンソニー カー，
ダグラス アンソニー カー，
アラ カール マラキアン，
アラ カール マラキアン，
マシュー ジョン シモンズ，
マシュー ジョン シモンズ，
テレサ エル． ライト，
テレサ エル． ライト，
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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 JP2020532981A publication Critical patent/JP2020532981A/ja
Publication of JP2020532981A5 publication Critical patent/JP2020532981A5/ja
Priority to JP2022176371A priority Critical patent/JP2022190081A/ja
Priority to JP2024189111A priority patent/JP2025010253A/ja
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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
    • C12N15/66—General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/60—Vectors comprising a special translation-regulating system from viruses

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
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JP2020512843A 2017-09-08 2018-09-07 修飾型閉端ｄｎａ（ｃｅｄｎａ） Pending JP2020532981A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022176371A JP2022190081A (ja) 2017-09-08 2022-11-02 修飾型閉端ｄｎａ（ｃｅｄｎａ）
JP2024189111A JP2025010253A (ja) 2017-09-08 2024-10-28 修飾型閉端ｄｎａ（ｃｅｄｎａ）

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US201762556281P 2017-09-08 2017-09-08
US201762556335P 2017-09-08 2017-09-08
US201762556324P 2017-09-08 2017-09-08
US201762556319P 2017-09-08 2017-09-08
US201762556329P 2017-09-08 2017-09-08
US201762556331P 2017-09-08 2017-09-08
US62/556,329 2017-09-08
US62/556,335 2017-09-08
US62/556,319 2017-09-08
US62/556,331 2017-09-08
US62/556,324 2017-09-08
US62/556,281 2017-09-08
PCT/US2018/049996 WO2019051255A1 (en) 2017-09-08 2018-09-07 CLOSED-ENDED DNA (CEDNA) MODIFIED

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JP2022176371A Division JP2022190081A (ja) 2017-09-08 2022-11-02 修飾型閉端ｄｎａ（ｃｅｄｎａ）

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JP2020532981A true JP2020532981A (ja) 2020-11-19
JP2020532981A5 JP2020532981A5 (enExample) 2021-10-21

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JP2020512843A Pending JP2020532981A (ja) 2017-09-08 2018-09-07 修飾型閉端ｄｎａ（ｃｅｄｎａ）
JP2022176371A Pending JP2022190081A (ja) 2017-09-08 2022-11-02 修飾型閉端ｄｎａ（ｃｅｄｎａ）
JP2024189111A Pending JP2025010253A (ja) 2017-09-08 2024-10-28 修飾型閉端ｄｎａ（ｃｅｄｎａ）

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JP2022176371A Pending JP2022190081A (ja) 2017-09-08 2022-11-02 修飾型閉端ｄｎａ（ｃｅｄｎａ）
JP2024189111A Pending JP2025010253A (ja) 2017-09-08 2024-10-28 修飾型閉端ｄｎａ（ｃｅｄｎａ）

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US (1) US20200283794A1 (enExample)
EP (1) EP3678710A4 (enExample)
JP (3) JP2020532981A (enExample)
KR (1) KR20200051011A (enExample)
CN (1) CN111132699A (enExample)
AU (1) AU2018327348A1 (enExample)
BR (1) BR112020004151A2 (enExample)
CA (1) CA3075168A1 (enExample)
IL (1) IL272797A (enExample)
MA (1) MA50100A (enExample)
MX (1) MX2020002500A (enExample)
PH (1) PH12020500465A1 (enExample)
SG (1) SG11202000698SA (enExample)
UA (1) UA129010C2 (enExample)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022506771A (ja) * 2018-11-09 2022-01-17 ジェネレーション バイオ カンパニー 対称的な修飾型逆位末端反復配列を含む修飾型閉端ｄｎａ（ｃｅｄｎａ）

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
CA2935960C (en) 2014-01-08 2023-01-10 Bart Lipkens Acoustophoresis device with dual acoustophoretic chamber
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
JP7374883B2 (ja) 2017-08-09 2023-11-07 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子およびその使用
US20220290186A1 (en) * 2017-12-06 2022-09-15 Generation Bio Co. Gene editing using a modified closed-ended dna (cedna)
KR20220066413A (ko) 2017-12-14 2022-05-24 프로디자인 소닉스, 인크. 음향 트랜스듀서 구동기 및 제어기
KR20200120649A (ko) * 2018-02-14 2020-10-21 제너레이션 바이오 컴퍼니 비-바이러스 dna 벡터 및 항체 및 융합 단백질 생산을 위한 이의 용도
CN119955796A (zh) 2018-08-09 2025-05-09 比奥维拉迪维治疗股份有限公司 核酸分子及其用于非病毒基因疗法的用途
GB201905651D0 (en) * 2019-04-24 2019-06-05 Lightbio Ltd Nucleic acid constructs and methods for their manufacture
US20220220488A1 (en) * 2019-07-17 2022-07-14 Generation Bio Co. Synthetic production of single-stranded adeno associated viral dna vectors
WO2021011840A1 (en) * 2019-07-17 2021-01-21 Generation Bio Co. Compositions and production of nicked closed-ended dna vectors
WO2021030745A1 (en) * 2019-08-15 2021-02-18 The Children's Hospital Of Philadelphia Combined transgene and intron-derived mirna therapy for treatment of sca1
EP4025196A4 (en) * 2019-09-06 2023-07-12 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof
JP2022549138A (ja) 2019-09-18 2022-11-24 インターガラクティック セラピューティクス インコーポレイテッド 合成ｄｎａベクターおよびその使用法
WO2021072031A1 (en) * 2019-10-11 2021-04-15 Insideoutbio, Inc. Methods and compositions for the manufacture and use of circular dna encoded therapeutics for genetic disorders and other diseases
WO2021169167A1 (en) * 2020-02-29 2021-09-02 Nanjing GenScript Biotech Co., Ltd. Method for treating coronavirus infections
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
JP2023520764A (ja) * 2020-03-24 2023-05-19 ジェネレーション バイオ カンパニー 第ｉｘ因子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
MX2022011806A (es) * 2020-03-24 2023-01-11 Generation Bio Co Vectores de adn no virales y usos de estos para expresar agentes terapéuticos contra gaucher.
CN115968280A (zh) * 2020-05-18 2023-04-14 世代生物公司 新型脂质及其纳米颗粒组合物
WO2022023284A1 (en) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
US20220243201A1 (en) * 2020-08-23 2022-08-04 Bioverativ Therapeutics Inc. Engineered itr sequences and methods of use
CA3189673A1 (en) 2020-08-23 2022-03-03 Ajay MAGHODIA Modified baculovirus system for improved production of closed-ended dna (cedna)
US20230383311A1 (en) * 2020-09-16 2023-11-30 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing fviii therapeutics
KR20230066615A (ko) * 2020-09-16 2023-05-16 제너레이션 바이오 컴퍼니 폐쇄형 dna 벡터 및 페닐알라닌 히드록실라아제(pah) 발현을 위한 이의 용도
GB202014751D0 (en) * 2020-09-18 2020-11-04 Lightbio Ltd Targeting vector
CA3211687A1 (en) * 2021-03-19 2022-09-22 Ozan ALKAN Non-viral dna vectors and uses thereof for expressing pfic therapeutics
WO2022232029A2 (en) * 2021-04-26 2022-11-03 University Of Florida Research Foundation, Incorporated Synthetic aav vectors for repeated delivery of therapeutic genes
AU2022264509A1 (en) * 2021-04-27 2023-12-14 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
EP4329884A1 (en) * 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
EP4333886A4 (en) * 2021-05-07 2025-11-12 Generation Bio Co NON-VIRAL DNA VECTORS FOR VACCINE ADMINISTRATION
US20240261395A1 (en) * 2021-05-07 2024-08-08 Generation Bio Co. Lyophilized non-viral dna vector compositions and uses thereof
EP4419677A4 (en) * 2021-10-18 2025-09-24 Flagship Pioneering Innovations Vii Llc DNA COMPOSITIONS AND RELATED METHODS
KR20240161965A (ko) 2022-03-14 2024-11-13 제너레이션 바이오 컴퍼니 이종 프라임 부스트 백신 조성물 및 사용 방법
CN117802161A (zh) * 2022-06-30 2024-04-02 苏州吉恒基因科技有限公司 精准重组腺相关病毒载体及其用途
WO2024040222A1 (en) * 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
WO2024182707A1 (en) 2023-03-02 2024-09-06 Krystal Biotech, Inc. Interleukin-2 and interleukin-12 for cancer therapy
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
US11767541B1 (en) * 2023-03-27 2023-09-26 Genecraft, Inc. Adeno-associated virus complex with improved expression of RUNX3 gene and uses for preventing or treating KRAS mutated lung cancer
US11891617B1 (en) * 2023-03-27 2024-02-06 Genecraft Inc. Modified complex platform of adeno-associated virus with improved rate of expression of loaded genes and reduced genotoxicity
WO2024220969A2 (en) * 2023-04-21 2024-10-24 Arizona Board Of Regents On Behalf Of Arizona State University Novel transmembrane sensors and method of characterization for lysis-free detection of intracellular targets
WO2025114932A1 (en) * 2023-11-30 2025-06-05 Sanofi Purification of closed-ended dna molecules

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004508041A (ja) * 2000-09-08 2004-03-18 メディジーン・アクチェンゲゼルシャフト 組換え型アデノ随伴ウイルス（ｒＡＡＶ）をパッケージングする親細胞、それらの生成方法、およびその使用
WO2014003553A1 (en) * 2012-06-27 2014-01-03 Arthrogen B.V. Combination for treating an inflammatory disorder
JP2014513928A (ja) * 2011-03-11 2014-06-19 アソシアシオン・アンスティテュ・ドゥ・ミオロジー 無カプシドａａｖベクター、組成物ならびにベクター製造および遺伝子運搬のための方法

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050019824A1 (en) * 1994-03-08 2005-01-27 Human Genome Sciences, Inc. Fibroblast Growth Factor-10
US20050026838A1 (en) * 1995-06-05 2005-02-03 Human Genome Sciences, Inc. Fibroblast Growth Factor-13
US20060166363A1 (en) * 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
EP1979485A2 (en) * 2006-01-31 2008-10-15 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
CN104087613B (zh) * 2014-06-30 2017-08-29 中国科学院苏州生物医学工程技术研究所 基于aav‑itr的基因表达微载体及其构建方法和应用
CA3011529A1 (en) * 2016-01-15 2017-07-20 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta t-cells
PT3423110T (pt) * 2016-03-03 2021-11-09 Univ Massachusetts Adn em dúplex linear de extremidades fechadas para transferência génica não viral

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004508041A (ja) * 2000-09-08 2004-03-18 メディジーン・アクチェンゲゼルシャフト 組換え型アデノ随伴ウイルス（ｒＡＡＶ）をパッケージングする親細胞、それらの生成方法、およびその使用
JP2014513928A (ja) * 2011-03-11 2014-06-19 アソシアシオン・アンスティテュ・ドゥ・ミオロジー 無カプシドａａｖベクター、組成物ならびにベクター製造および遺伝子運搬のための方法
WO2014003553A1 (en) * 2012-06-27 2014-01-03 Arthrogen B.V. Combination for treating an inflammatory disorder

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
J. VIROL., vol. 89, no. 2, JPN7023000695, 2015, pages 952 - 961, ISSN: 0005155521 *
MOL. THER., vol. Vol. 19, Supplement 1, S51, 128, JPN7023000696, 2011, ISSN: 0005155520 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022506771A (ja) * 2018-11-09 2022-01-17 ジェネレーション バイオ カンパニー 対称的な修飾型逆位末端反復配列を含む修飾型閉端ｄｎａ（ｃｅｄｎａ）
JP7590963B2 (ja) 2018-11-09 2024-11-27 ジェネレーション バイオ カンパニー 対称的な修飾型逆位末端反復配列を含む修飾型閉端ｄｎａ（ｃｅｄｎａ）

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