JP2021513999A5 - - Google Patents

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Publication number
JP2021513999A5
JP2021513999A5 JP2020544445A JP2020544445A JP2021513999A5 JP 2021513999 A5 JP2021513999 A5 JP 2021513999A5 JP 2020544445 A JP2020544445 A JP 2020544445A JP 2020544445 A JP2020544445 A JP 2020544445A JP 2021513999 A5 JP2021513999 A5 JP 2021513999A5
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JP
Japan
Prior art keywords
pharmaceutical composition
dose
composition according
itrs
adjacent
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2020544445A
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English (en)
Japanese (ja)
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JP2021513999A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/018927 external-priority patent/WO2019165050A1/en
Publication of JP2021513999A publication Critical patent/JP2021513999A/ja
Publication of JP2021513999A5 publication Critical patent/JP2021513999A5/ja
Priority to JP2023207938A priority Critical patent/JP2024028931A/ja
Pending legal-status Critical Current

Links

JP2020544445A 2018-02-22 2019-02-21 閉端dna(cedna)ベクターを使用した導入遺伝子の制御された発現 Pending JP2021513999A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023207938A JP2024028931A (ja) 2018-02-22 2023-12-08 閉端dna(cedna)ベクターを使用した導入遺伝子の制御された発現

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862633882P 2018-02-22 2018-02-22
US201862633795P 2018-02-22 2018-02-22
US201862633757P 2018-02-22 2018-02-22
US62/633,882 2018-02-22
US62/633,795 2018-02-22
US62/633,757 2018-02-22
US201862746762P 2018-10-17 2018-10-17
US62/746,762 2018-10-17
PCT/US2019/018927 WO2019165050A1 (en) 2018-02-22 2019-02-21 Controlled expression of transgenes using close-ended dna (cedna) vectors

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023207938A Division JP2024028931A (ja) 2018-02-22 2023-12-08 閉端dna(cedna)ベクターを使用した導入遺伝子の制御された発現

Publications (2)

Publication Number Publication Date
JP2021513999A JP2021513999A (ja) 2021-06-03
JP2021513999A5 true JP2021513999A5 (https=) 2022-03-01

Family

ID=67687406

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020544445A Pending JP2021513999A (ja) 2018-02-22 2019-02-21 閉端dna(cedna)ベクターを使用した導入遺伝子の制御された発現
JP2023207938A Pending JP2024028931A (ja) 2018-02-22 2023-12-08 閉端dna(cedna)ベクターを使用した導入遺伝子の制御された発現

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023207938A Pending JP2024028931A (ja) 2018-02-22 2023-12-08 閉端dna(cedna)ベクターを使用した導入遺伝子の制御された発現

Country Status (14)

Country Link
US (1) US20220175970A1 (https=)
EP (1) EP3755803A4 (https=)
JP (2) JP2021513999A (https=)
KR (1) KR20200124250A (https=)
CN (1) CN111886343A (https=)
AU (1) AU2019225937A1 (https=)
BR (1) BR112020017060A2 (https=)
CA (1) CA3092059A1 (https=)
IL (1) IL276658A (https=)
MA (1) MA51915A (https=)
MX (1) MX2020008676A (https=)
PH (1) PH12020551235A1 (https=)
SG (1) SG11202007621TA (https=)
WO (1) WO2019165050A1 (https=)

Families Citing this family (14)

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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
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
KR20210090619A (ko) * 2018-11-09 2021-07-20 제너레이션 바이오 컴퍼니 대칭인 변형된 역말단반복을 포함하는 변형된 폐쇄형 DNA(ceDNA)
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
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
US20230138409A1 (en) * 2020-03-24 2023-05-04 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
EP4142758A4 (en) * 2020-04-28 2025-02-26 President and Fellows of Harvard College High efficiency gene delivery system
IL299422A (en) * 2020-06-26 2023-02-01 Univ Leland Stanford Junior Targeting the human ccr5 locus as a safe harbor for the expression of therapeutic proteins
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
WO2022046665A1 (en) * 2020-08-23 2022-03-03 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
EP4214309A4 (en) * 2020-09-16 2025-07-16 Generation Bio Co CLOSED-END DNA VECTORS AND RELATED USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH)
CA3211687A1 (en) * 2021-03-19 2022-09-22 Ozan ALKAN Non-viral dna vectors and uses thereof for expressing pfic therapeutics
WO2025114932A1 (en) * 2023-11-30 2025-06-05 Sanofi Purification of closed-ended dna molecules
CN121662213B (zh) * 2026-02-06 2026-04-21 湖南博极生命科技有限公司 基于注意力权重追溯的中药活性成分可解释性分析方法及系统

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003237805A1 (en) * 2002-04-09 2003-10-27 Cornell Research Foundation, Inc. Use of aav integration efficiency element for mediating site-specific integration of a transcription unit
HUE041875T2 (hu) * 2005-05-31 2019-06-28 Univ Colorado Regents Eljárás génbejuttatásra
ES2426091T3 (es) * 2007-09-19 2013-10-21 Uniqure Ip B.V. Uso de maquinaria de replicación de AAV para producción de proteína mejorada
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
EP3151866B1 (en) * 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
JP7242180B2 (ja) * 2014-09-07 2023-03-20 セレクタ バイオサイエンシーズ インコーポレーテッド 抗ウイルス導入ベクター免疫応答を減弱化するための方法および組成物
EP3889260A1 (en) * 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
MX2018010633A (es) * 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.

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