KR20210151785A - 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도 - Google Patents

비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도 Download PDF

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KR20210151785A
KR20210151785A KR1020217029545A KR20217029545A KR20210151785A KR 20210151785 A KR20210151785 A KR 20210151785A KR 1020217029545 A KR1020217029545 A KR 1020217029545A KR 20217029545 A KR20217029545 A KR 20217029545A KR 20210151785 A KR20210151785 A KR 20210151785A
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itr
cedna vector
sequence
cedna
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더글라스 앤써니 케르
데브라 클라떼
필립 사마요아
나다니엘 실버
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제너레이션 바이오 컴퍼니
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    • A—HUMAN NECESSITIES
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    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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KR1020217029545A 2019-03-13 2020-03-13 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도 Pending KR20210151785A (ko)

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

Publications (1)

Publication Number Publication Date
KR20210151785A true KR20210151785A (ko) 2021-12-14

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KR1020217029545A Pending KR20210151785A (ko) 2019-03-13 2020-03-13 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도

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=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20260011950A (ko) 2024-07-17 2026-01-26 이민형 수질 시료 채취 드론

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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

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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
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20260011950A (ko) 2024-07-17 2026-01-26 이민형 수질 시료 채취 드론

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WO2020186207A2 (en) 2020-09-17
JP2026002894A (ja) 2026-01-08
US20250282848A1 (en) 2025-09-11
JP2022524434A (ja) 2022-05-02
JP2025073123A (ja) 2025-05-12
EP3938523A4 (en) 2023-06-21
US20220177545A1 (en) 2022-06-09
SG11202109850SA (en) 2021-10-28
JP7795357B2 (ja) 2026-01-07
IL286284A (en) 2021-10-31
MA55313A (fr) 2022-01-19
US12338275B2 (en) 2025-06-24
EP3938523A2 (en) 2022-01-19
CA3133255A1 (en) 2020-09-17
MX2021011039A (es) 2021-12-15
BR112021017853A2 (pt) 2021-11-30
CN113874513A (zh) 2021-12-31
WO2020186207A8 (en) 2020-11-12
AU2020234713A1 (en) 2021-11-04
WO2020186207A3 (en) 2020-12-17

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