JP7795357B2 - 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 - Google Patents

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

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Publication number
JP7795357B2
JP7795357B2 JP2021554696A JP2021554696A JP7795357B2 JP 7795357 B2 JP7795357 B2 JP 7795357B2 JP 2021554696 A JP2021554696 A JP 2021554696A JP 2021554696 A JP2021554696 A JP 2021554696A JP 7795357 B2 JP7795357 B2 JP 7795357B2
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itr
cedna
seq
cedna vector
itrs
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JPWO2020186207A5 (https=
JP2022524434A5 (https=
JP2022524434A (ja
Inventor
ダグラス アンソニー カー，
デブラ クラット，
フィリップ サマヨア，
ナサニエル シルバー，
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ジェネレーション バイオ カンパニー
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Publication of JP2022524434A5 publication Critical patent/JP2022524434A5/ja
Priority to JP2025016352A priority Critical patent/JP2025073123A/ja
Priority to JP2025172911A priority patent/JP2026002894A/ja
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K48/0025—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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2820/00—Vectors comprising a special origin of replication system
    • C12N2820/60—Vectors comprising a special origin of replication system from viruses

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
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 (4)

Publication Number Publication Date
JP2022524434A JP2022524434A (ja) 2022-05-02
JPWO2020186207A5 JPWO2020186207A5 (https=) 2023-03-22
JP2022524434A5 JP2022524434A5 (https=) 2023-03-22
JP7795357B2 true JP7795357B2 (ja) 2026-01-07

Family

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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)

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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 (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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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

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JP2014513928A (ja) 2011-03-11 2014-06-19 アソシアシオン・アンスティテュ・ドゥ・ミオロジー 無カプシドａａｖベクター、組成物ならびにベクター製造および遺伝子運搬のための方法
WO2014127215A1 (en) 2013-02-15 2014-08-21 Biogen Idec Ma Inc. Optimized factor viii gene
WO2015038625A1 (en) 2013-09-12 2015-03-19 Biomarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors
WO2017075619A1 (en) 2015-10-30 2017-05-04 Spark Therapeutics, Inc. Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders
WO2017152149A1 (en) 2016-03-03 2017-09-08 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
WO2019032898A1 (en) 2017-08-09 2019-02-14 Bioverativ Therapeutics Inc. NUCLEIC ACID MOLECULES AND USES THEREOF

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JP2014513928A (ja) 2011-03-11 2014-06-19 アソシアシオン・アンスティテュ・ドゥ・ミオロジー 無カプシドａａｖベクター、組成物ならびにベクター製造および遺伝子運搬のための方法
WO2014127215A1 (en) 2013-02-15 2014-08-21 Biogen Idec Ma Inc. Optimized factor viii gene
WO2015038625A1 (en) 2013-09-12 2015-03-19 Biomarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors
WO2017075619A1 (en) 2015-10-30 2017-05-04 Spark Therapeutics, Inc. Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders
WO2017152149A1 (en) 2016-03-03 2017-09-08 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
WO2019032898A1 (en) 2017-08-09 2019-02-14 Bioverativ Therapeutics Inc. NUCLEIC ACID MOLECULES AND USES THEREOF

Also Published As

Publication number Publication date
WO2020186207A2 (en) 2020-09-17
JP2026002894A (ja) 2026-01-08
US20250282848A1 (en) 2025-09-11
KR20210151785A (ko) 2021-12-14
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
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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