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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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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JP2022524434A5 (enExample
JP2022524434A (ja
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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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    • 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 (3)

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

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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

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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 (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)

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MX2020001593A (es) 2017-08-09 2020-07-13 Bioverativ Therapeutics Inc Moleculas de acido nucleico y usos de las mismas.
JP7602454B2 (ja) 2018-08-09 2024-12-18 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
MX2023001109A (es) 2020-07-27 2023-04-24 Anjarium Biosciences Ag Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.
US20220356490A1 (en) * 2020-08-23 2022-11-10 Bioverativ Therapeutics Inc. Baculovirus expression system
AU2022334714A1 (en) * 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Closed-end dna production with inverted terminal repeat sequences
WO2023028440A2 (en) * 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Baculovirus expression system
WO2023069948A1 (en) 2021-10-18 2023-04-27 Flagship Pioneering Innovations Vii, Llc Dna compositions and related methods
WO2023078220A1 (zh) * 2021-11-02 2023-05-11 苏州华毅乐健生物科技有限公司 分离的核酸分子及其应用
JP2023089774A (ja) * 2021-12-16 2023-06-28 株式会社三洋物産 遊技機
WO2023135273A2 (en) * 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
EP4598590A4 (en) * 2022-10-08 2026-05-27 Lingyi Biotech Co Ltd CONSTRUCTION TO IMPROVE GENE EXPRESSION
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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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
WO2020186207A8 (en) 2020-11-12
AU2020234713A1 (en) 2021-11-04
KR20210151785A (ko) 2021-12-14
US20220177545A1 (en) 2022-06-09
IL286284A (en) 2021-10-31
MX2021011039A (es) 2021-12-15
CN113874513A (zh) 2021-12-31
JP2025073123A (ja) 2025-05-12
MA55313A (fr) 2022-01-19
US20250282848A1 (en) 2025-09-11
US12338275B2 (en) 2025-06-24
WO2020186207A2 (en) 2020-09-17
JP2026002894A (ja) 2026-01-08
BR112021017853A2 (pt) 2021-11-30
CA3133255A1 (en) 2020-09-17
WO2020186207A3 (en) 2020-12-17
EP3938523A4 (en) 2023-06-21
EP3938523A2 (en) 2022-01-19
SG11202109850SA (en) 2021-10-28
JP2022524434A (ja) 2022-05-02

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