JPWO2020186207A5 - - Google Patents

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Publication number
JPWO2020186207A5
JPWO2020186207A5 JP2021554696A JP2021554696A JPWO2020186207A5 JP WO2020186207 A5 JPWO2020186207 A5 JP WO2020186207A5 JP 2021554696 A JP2021554696 A JP 2021554696A JP 2021554696 A JP2021554696 A JP 2021554696A JP WO2020186207 A5 JPWO2020186207 A5 JP WO2020186207A5
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JP
Japan
Prior art keywords
cedna vector
itrs
stem
pharmaceutical composition
regions
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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.)
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Application number
JP2021554696A
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English (en)
Japanese (ja)
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JP2022524434A (ja
JP7795357B2 (ja
Publication date
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Priority claimed from PCT/US2020/022738 external-priority patent/WO2020186207A2/en
Publication of JP2022524434A publication Critical patent/JP2022524434A/ja
Publication of JPWO2020186207A5 publication Critical patent/JPWO2020186207A5/ja
Priority to JP2025016352A priority Critical patent/JP2025073123A/ja
Priority to JP2025172911A priority patent/JP2026002894A/ja
Application granted granted Critical
Publication of JP7795357B2 publication Critical patent/JP7795357B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2021554696A 2019-03-13 2020-03-13 Fviii治療薬を発現するための非ウイルス性dnaベクターおよびその使用 Active JP7795357B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2025016352A JP2025073123A (ja) 2019-03-13 2025-02-03 Fviii治療薬を発現するための非ウイルス性dnaベクターおよびその使用
JP2025172911A JP2026002894A (ja) 2019-03-13 2025-10-14 Fviii治療薬を発現するための非ウイルス性dnaベクターおよびその使用

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962817904P 2019-03-13 2019-03-13
US62/817,904 2019-03-13
US201962856432P 2019-06-03 2019-06-03
US62/856,432 2019-06-03
PCT/US2020/022738 WO2020186207A2 (en) 2019-03-13 2020-03-13 Non-viral dna vectors and uses thereof for expressing fviii therapeutics

Related Child Applications (2)

Application Number Title Priority Date Filing Date
JP2025016352A Division JP2025073123A (ja) 2019-03-13 2025-02-03 Fviii治療薬を発現するための非ウイルス性dnaベクターおよびその使用
JP2025172911A Division JP2026002894A (ja) 2019-03-13 2025-10-14 Fviii治療薬を発現するための非ウイルス性dnaベクターおよびその使用

Publications (3)

Publication Number Publication Date
JP2022524434A JP2022524434A (ja) 2022-05-02
JPWO2020186207A5 true JPWO2020186207A5 (enExample) 2023-03-22
JP7795357B2 JP7795357B2 (ja) 2026-01-07

Family

ID=72426821

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2021554696A Active JP7795357B2 (ja) 2019-03-13 2020-03-13 Fviii治療薬を発現するための非ウイルス性dnaベクターおよびその使用
JP2025016352A Pending JP2025073123A (ja) 2019-03-13 2025-02-03 Fviii治療薬を発現するための非ウイルス性dnaベクターおよびその使用
JP2025172911A Pending JP2026002894A (ja) 2019-03-13 2025-10-14 Fviii治療薬を発現するための非ウイルス性dnaベクターおよびその使用

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2025016352A Pending JP2025073123A (ja) 2019-03-13 2025-02-03 Fviii治療薬を発現するための非ウイルス性dnaベクターおよびその使用
JP2025172911A Pending JP2026002894A (ja) 2019-03-13 2025-10-14 Fviii治療薬を発現するための非ウイルス性dnaベクターおよびその使用

Country Status (13)

Country Link
US (2) US12338275B2 (enExample)
EP (1) EP3938523A4 (enExample)
JP (3) JP7795357B2 (enExample)
KR (1) KR20210151785A (enExample)
CN (1) CN113874513A (enExample)
AU (1) AU2020234713A1 (enExample)
BR (1) BR112021017853A2 (enExample)
CA (1) CA3133255A1 (enExample)
IL (1) IL286284A (enExample)
MA (1) MA55313A (enExample)
MX (1) MX2021011039A (enExample)
SG (1) SG11202109850SA (enExample)
WO (1) WO2020186207A2 (enExample)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI904068B (zh) 2017-08-09 2025-11-11 美商生物化學醫療公司 核酸分子及其用途
WO2020033863A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
EP4189098A1 (en) 2020-07-27 2023-06-07 Anjarium Biosciences AG Compositions of dna molecules, methods of making therefor, and methods of use thereof
US20220356490A1 (en) * 2020-08-23 2022-11-10 Bioverativ Therapeutics Inc. Baculovirus expression system
WO2023069948A1 (en) * 2021-10-18 2023-04-27 Flagship Pioneering Innovations Vii, Llc Dna compositions and related methods
US20240335564A1 (en) * 2021-11-02 2024-10-10 Gritgen Therapeutics Limited Isolated nucleic acid molecule and use thereof
JP2023089774A (ja) * 2021-12-16 2023-06-28 株式会社三洋物産 遊技機
WO2023135273A2 (en) * 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
WO2025110984A1 (en) * 2023-11-21 2025-05-30 Hydrogene Therapeutics, Inc. Compositions and methods for treating wilson's disease
KR20260011950A (ko) 2024-07-17 2026-01-26 이민형 수질 시료 채취 드론

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009067548A1 (en) * 2007-11-19 2009-05-28 The Regents Of The University Of California Novel assay for inhibitors of egfr
CN102573915B (zh) * 2009-04-30 2015-02-18 赛托斯生物技术公司 流感血凝素组合物及其用途
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
DK2956477T4 (da) * 2013-02-15 2024-04-15 Bioverativ Therapeutics Inc Optimeret faktor viii-gen
ES2813698T3 (es) * 2013-09-12 2021-03-24 Biomarin Pharm Inc Vectores de AAV que comprenden un gen que codifica el factor VIII
GB2526339A (en) * 2014-05-21 2015-11-25 Imp Innovations Ltd Lentiviral vectors
CN108136048A (zh) * 2015-08-03 2018-06-08 米奥多巴有限公司 左旋多巴的系统合成和调节
BR112018006074A2 (pt) 2015-09-24 2018-10-09 Biomarin Pharm Inc vetores de fator viii de vírus adeno-associado, partículas virais associadas e formulações terapêuticas compreendendo as mesmas
KR102766526B1 (ko) 2015-10-28 2025-02-13 상가모 테라퓨틱스, 인코포레이티드 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
EP3423110B1 (en) 2016-03-03 2021-08-11 University of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer
WO2017180857A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia a
BR112020001979A2 (pt) * 2017-08-01 2020-08-18 Spark Therapeutics, Inc. métodos de terapia genética do fator viii (fviii)
TWI904068B (zh) * 2017-08-09 2025-11-11 美商生物化學醫療公司 核酸分子及其用途

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