JP2023542132A5 - - Google Patents

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Publication number
JP2023542132A5
JP2023542132A5 JP2023517284A JP2023517284A JP2023542132A5 JP 2023542132 A5 JP2023542132 A5 JP 2023542132A5 JP 2023517284 A JP2023517284 A JP 2023517284A JP 2023517284 A JP2023517284 A JP 2023517284A JP 2023542132 A5 JP2023542132 A5 JP 2023542132A5
Authority
JP
Japan
Prior art keywords
sequence
nucleic acid
group
vector according
acid sequence
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
JP2023517284A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023542132A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/050715 external-priority patent/WO2022061014A1/en
Publication of JP2023542132A publication Critical patent/JP2023542132A/ja
Publication of JP2023542132A5 publication Critical patent/JP2023542132A5/ja
Pending legal-status Critical Current

Links

JP2023517284A 2020-09-16 2021-09-16 Fviii治療薬を発現させるための非ウイルス性dnaベクター及びその使用 Pending JP2023542132A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063079349P 2020-09-16 2020-09-16
US63/079,349 2020-09-16
US202063132838P 2020-12-31 2020-12-31
US63/132,838 2020-12-31
PCT/US2021/050715 WO2022061014A1 (en) 2020-09-16 2021-09-16 Non-viral dna vectors and uses thereof for expressing fviii therapeutics

Publications (2)

Publication Number Publication Date
JP2023542132A JP2023542132A (ja) 2023-10-05
JP2023542132A5 true JP2023542132A5 (https=) 2024-09-26

Family

ID=80776405

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023517284A Pending JP2023542132A (ja) 2020-09-16 2021-09-16 Fviii治療薬を発現させるための非ウイルス性dnaベクター及びその使用

Country Status (9)

Country Link
US (1) US20230383311A1 (https=)
EP (1) EP4214315A4 (https=)
JP (1) JP2023542132A (https=)
KR (1) KR20230066453A (https=)
AU (1) AU2021342503A1 (https=)
CA (1) CA3191743A1 (https=)
IL (1) IL301197A (https=)
MX (1) MX2023003021A (https=)
WO (1) WO2022061014A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202541849A (zh) * 2023-12-14 2025-11-01 美商聲納席拉公司 用於經由超音波改良核酸遞送之方法及系統

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2656558A1 (en) * 2006-06-30 2008-01-10 The Regents Of The University Of Michigan Method of producing factor viii proteins by recombinant methods
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
AU2013336601B2 (en) * 2012-10-26 2018-01-25 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
GB2526339A (en) * 2014-05-21 2015-11-25 Imp Innovations Ltd Lentiviral vectors
PL231230B1 (pl) * 2015-03-11 2019-02-28 Inst Biochemii I Biofizyki Polskiej Akademii Nauk Czynnik zwiększający efektywność szczepionki DNA skierowanej przeciw wirusowi, preparat plazmidowy DNA, szczepionka DNA, sposób otrzymywania zmodyfikowanego wektora ekspresyjnego oraz zastosowanie sekwencji nukleotydowej rozpoznawanej przez czynnik NF kappa B
MX2018010633A (es) * 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.
NZ762100A (en) * 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
CA3075168A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. Modified closed-ended dna (cedna)
CA3084185A1 (en) * 2017-12-06 2019-06-13 Generation Bio Co. Gene editing using a modified closed-ended dna (cedna)
MA52116A (fr) * 2018-03-02 2021-01-06 Generation Bio Co Vecteurs d'adn à extrémité fermée (cedna) pour l'insertion de transgènes au niveau de havres génomiques sécuritaires (gsh) dans des génomes humains et murins
SG11202108357PA (en) * 2019-02-15 2021-08-30 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression

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