JP2022524434A - 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 Download PDF

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JP2022524434A
JP2022524434A JP2021554696A JP2021554696A JP2022524434A JP 2022524434 A JP2022524434 A JP 2022524434A JP 2021554696 A JP2021554696 A JP 2021554696A JP 2021554696 A JP2021554696 A JP 2021554696A JP 2022524434 A JP2022524434 A JP 2022524434A
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cedna vector
cedna
sequence
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JPWO2020186207A5 (enExample
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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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    • 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
    • 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)
    • 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
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    • 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
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    • 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)
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  • Genetics & Genomics (AREA)
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  • Biotechnology (AREA)
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  • Biochemistry (AREA)
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  • Wood Science & Technology (AREA)
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  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Epidemiology (AREA)
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  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (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 Pending JP2022524434A (ja)

Priority Applications (1)

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

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

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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

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JP2022524434A true JP2022524434A (ja) 2022-05-02
JPWO2020186207A5 JPWO2020186207A5 (enExample) 2023-03-22

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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

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US (2) US12338275B2 (enExample)
EP (1) EP3938523A4 (enExample)
JP (2) JP2022524434A (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)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111247251B (zh) 2017-08-09 2025-06-27 比奥维拉迪维治疗股份有限公司 核酸分子及其用途
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Ｄｎａ分子の組成物、その作製方法、及びその使用方法
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
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
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
WO2025110984A1 (en) * 2023-11-21 2025-05-30 Hydrogene Therapeutics, Inc. Compositions and methods for treating wilson's disease

Citations (7)

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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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US20100323957A1 (en) 2007-11-19 2010-12-23 The Regents Of The University Of California The Office Of The President 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
GB2526339A (en) * 2014-05-21 2015-11-25 Imp Innovations Ltd Lentiviral vectors
WO2017021359A1 (en) 2015-08-03 2017-02-09 Myodopa Limited Systemic synthesis and regulation of l-dopa
RU2020126034A (ru) 2015-09-24 2020-09-02 Байомарин Фармасьютикал Инк. Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции
EP3368664A4 (en) 2015-10-28 2019-08-28 Sangamo Therapeutics, Inc. LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF
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Publication number Priority date Publication date Assignee Title
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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CN113874513A (zh) 2021-12-31
SG11202109850SA (en) 2021-10-28
IL286284A (en) 2021-10-31
MA55313A (fr) 2022-01-19
CA3133255A1 (en) 2020-09-17
WO2020186207A8 (en) 2020-11-12
US20250282848A1 (en) 2025-09-11
MX2021011039A (es) 2021-12-15
US20220177545A1 (en) 2022-06-09
JP2025073123A (ja) 2025-05-12
EP3938523A2 (en) 2022-01-19
WO2020186207A2 (en) 2020-09-17
KR20210151785A (ko) 2021-12-14
US12338275B2 (en) 2025-06-24
AU2020234713A1 (en) 2021-11-04
BR112021017853A2 (pt) 2021-11-30
WO2020186207A3 (en) 2020-12-17
EP3938523A4 (en) 2023-06-21

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