JP2025073123A5 - - Google Patents

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Publication number
JP2025073123A5
JP2025073123A5 JP2025016352A JP2025016352A JP2025073123A5 JP 2025073123 A5 JP2025073123 A5 JP 2025073123A5 JP 2025016352 A JP2025016352 A JP 2025016352A JP 2025016352 A JP2025016352 A JP 2025016352A JP 2025073123 A5 JP2025073123 A5 JP 2025073123A5
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JP
Japan
Prior art keywords
seq
nucleotide
pharmaceutical composition
sequence
group
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
JP2025016352A
Other languages
English (en)
Japanese (ja)
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JP2025073123A (ja
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Publication date
Priority claimed from PCT/US2020/022738 external-priority patent/WO2020186207A2/en
Application filed filed Critical
Publication of JP2025073123A publication Critical patent/JP2025073123A/ja
Publication of JP2025073123A5 publication Critical patent/JP2025073123A5/ja
Pending legal-status Critical Current

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JP2025016352A 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 Pending JP2025073123A (ja)

Applications Claiming Priority (6)

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
JP2021554696A 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

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2021554696A Division 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

Publications (2)

Publication Number Publication Date
JP2025073123A JP2025073123A (ja) 2025-05-12
JP2025073123A5 true JP2025073123A5 (https=) 2025-08-06

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JP2025172911A Withdrawn JP2026002894A (ja) 2019-03-13 2025-10-14 Fviii治療薬を発現するための非ウイルス性dnaベクターおよびその使用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 Before (1)

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

Family Applications After (1)

Application Number Title Priority Date Filing Date
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 (https=)
EP (1) EP3938523A4 (https=)
JP (3) JP7795357B2 (https=)
KR (1) KR20210151785A (https=)
CN (1) CN113874513A (https=)
AU (1) AU2020234713A1 (https=)
BR (1) BR112021017853A2 (https=)
CA (1) CA3133255A1 (https=)
IL (1) IL286284A (https=)
MA (1) MA55313A (https=)
MX (1) MX2021011039A (https=)
SG (1) SG11202109850SA (https=)
WO (1) WO2020186207A2 (https=)

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Publication number Priority date Publication date Assignee Title
TW202545984A (zh) 2017-08-09 2025-12-01 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
CN116234917A (zh) 2020-07-27 2023-06-06 安杰瑞姆生物科学公司 Dna分子组合物及其制备方法和使用方法
US20220356490A1 (en) * 2020-08-23 2022-11-10 Bioverativ Therapeutics Inc. Baculovirus expression system
US20250027107A1 (en) 2021-10-18 2025-01-23 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
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
WO2009067548A1 (en) * 2007-11-19 2009-05-28 The Regents Of The University Of California Novel assay for inhibitors of egfr
JP2012525134A (ja) * 2009-04-30 2012-10-22 サイトス バイオテクノロジー アーゲー インフルエンザ赤血球凝集素の組成物とその使用
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
AU2013243948A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
SI3889173T1 (sl) 2013-02-15 2023-11-30 Bioverativ Therapeutics Inc. Optimiran gen dejavnika VIII
RS61039B1 (sr) * 2013-09-12 2020-12-31 Biomarin Pharm Inc Aav vektori koji sadrže gen koji kodira faktor viii
GB2526339A (en) 2014-05-21 2015-11-25 Imp Innovations Ltd Lentiviral vectors
KR20180034467A (ko) * 2015-08-03 2018-04-04 마이오도파 리미티드 L-dopa의 전신 합성 및 조절
RU2742352C2 (ru) 2015-09-24 2021-02-05 Байомарин Фармасьютикал Инк. Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции
CA3002524A1 (en) * 2015-10-28 2017-05-04 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
WO2017152149A1 (en) * 2016-03-03 2017-09-08 University Of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer
KR102450833B1 (ko) * 2016-04-15 2022-10-05 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 혈우병 a 치료용 유전자 요법
KR102219859B1 (ko) * 2016-06-24 2021-02-25 재단법인 목암생명과학연구소 재조합 단쇄 fviii 및 그 화학 접합물
AU2018312565A1 (en) * 2017-08-01 2020-02-27 Spark Therapeutics, Inc. Factor VIII (FVIII) gene therapy methods
TW202545984A (zh) * 2017-08-09 2025-12-01 美商生物化學醫療公司 核酸分子及其用途

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