JP2022524434A5 - - Google Patents

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Publication number
JP2022524434A5
JP2022524434A5 JP2021554696A JP2021554696A JP2022524434A5 JP 2022524434 A5 JP2022524434 A5 JP 2022524434A5 JP 2021554696 A JP2021554696 A JP 2021554696A JP 2021554696 A JP2021554696 A JP 2021554696A JP 2022524434 A5 JP2022524434 A5 JP 2022524434A5
Authority
JP
Japan
Prior art keywords
cedna vector
itrs
prior
adjacent
vector according
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.)
Granted
Application number
JP2021554696A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022524434A (ja
JP7795357B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/022738 external-priority patent/WO2020186207A2/en
Publication of JP2022524434A publication Critical patent/JP2022524434A/ja
Publication of JP2022524434A5 publication Critical patent/JP2022524434A5/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

Links

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
JP2022524434A5 true JP2022524434A5 (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 Withdrawn 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 Withdrawn 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
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
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
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
EP4428237A4 (en) * 2021-11-02 2026-02-25 Gritgen Therapeutics Ltd ISOLATED NUCLEIC ACID MOLECULE AND ITS USES
JP2023089774A (ja) * 2021-12-16 2023-06-28 株式会社三洋物産 遊技機
JP2025504404A (ja) * 2022-01-14 2025-02-12 アンジャリウム バイオサイエンシズ エージー Viii因子をコードするdna分子の組成物、それを作製する方法、及びその使用方法
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 (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100323957A1 (en) * 2007-11-19 2010-12-23 The Regents Of The University Of California The Office Of The President Novel assay for inhibitors of egfr
NZ596058A (en) * 2009-04-30 2013-08-30 Cytos Biotechnology Ag Influenza hemagglutinin compositions and uses thereof
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
WO2013093760A2 (en) * 2011-12-19 2013-06-27 Grifols, S.A. Compositions, methods, and kits for preparing sialylated recombinant proteins
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
WO2014127215A1 (en) 2013-02-15 2014-08-21 Biogen Idec Ma Inc. Optimized factor viii gene
EP3044231B1 (en) * 2013-09-12 2020-08-05 BioMarin Pharmaceutical Inc. Aav vectors comprising a gene encoding factor viii
GB2526339A (en) * 2014-05-21 2015-11-25 Imp Innovations Ltd Lentiviral vectors
US20190032079A1 (en) * 2015-08-03 2019-01-31 Myodopa Limited Systemic synthesis and regulation of l-dopa
TW202446416A (zh) 2015-09-24 2024-12-01 美商拜奧馬林製藥公司 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物
MY189674A (en) 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
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
MX2018010633A (es) * 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.
MX2018012537A (es) 2016-04-15 2019-02-25 Univ Pennsylvania Terapia de genes para tratar hemofilia a.
JP7235511B2 (ja) * 2016-06-24 2023-03-08 モガム・インスティテュート・フォー・バイオメディカル・リサーチ 組換え型一本鎖fviiiおよびその化学コンジュゲート
PE20200722A1 (es) 2017-08-01 2020-07-21 Spark Therapeutics Inc Metodos de terapia genica del factor viii (fviii)
NZ762100A (en) * 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof

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