CN113874513A - 非病毒dna载体及其用于表达fviii治疗剂的用途 - Google Patents

非病毒dna载体及其用于表达fviii治疗剂的用途 Download PDF

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CN113874513A
CN113874513A CN202080036209.7A CN202080036209A CN113874513A CN 113874513 A CN113874513 A CN 113874513A CN 202080036209 A CN202080036209 A CN 202080036209A CN 113874513 A CN113874513 A CN 113874513A
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itrs
itr
cedna
vector
sequence
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Chinese (zh)
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D·A·科尔
D·克拉特
P·萨马约亚
N·西尔弗
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Generational Biology Co
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Generational Biology Co
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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
    • 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
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    • 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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  • General Engineering & Computer Science (AREA)
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  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
CN202080036209.7A 2019-03-13 2020-03-13 非病毒dna载体及其用于表达fviii治疗剂的用途 Pending CN113874513A (zh)

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)

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CN113874513A true CN113874513A (zh) 2021-12-31

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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
WO2019032898A1 (en) 2017-08-09 2019-02-14 Bioverativ Therapeutics Inc. NUCLEIC ACID MOLECULES AND USES THEREOF
SG11202101157VA (en) 2018-08-09 2021-03-30 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof for non-viral gene therapy
WO2022023284A1 (en) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
US20220356490A1 (en) * 2020-08-23 2022-11-10 Bioverativ Therapeutics Inc. Baculovirus expression system
JP2024538168A (ja) * 2021-10-18 2024-10-18 フラッグシップ パイオニアリング イノベーションズ セブン，エルエルシー Ｄｎａ組成物及び関連方法
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
EP4463475A2 (en) * 2022-01-14 2024-11-20 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 (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010125202A1 (en) * 2009-04-30 2010-11-04 Cytos Biotechnology Ag Influenza hemagglutinin compositions and uses thereof
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
WO2015038625A1 (en) * 2013-09-12 2015-03-19 Biomarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors
WO2017021359A1 (en) * 2015-08-03 2017-02-09 Myodopa Limited Systemic synthesis and regulation of l-dopa
US20170096684A1 (en) * 2014-05-21 2017-04-06 Imperial Innovations Limited Lentiviral 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
US20170119906A1 (en) * 2015-10-28 2017-05-04 Sangamo Biosciences, Inc. Liver-specific constructs, factor viii expression cassettes and methods of use thereof
CN109195636A (zh) * 2016-03-03 2019-01-11 马萨诸塞大学 用于非病毒基因转移的末端封闭型线性双链体dna
WO2019032898A1 (en) * 2017-08-09 2019-02-14 Bioverativ Therapeutics Inc. NUCLEIC ACID MOLECULES AND USES THEREOF

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
PL2956477T5 (pl) * 2013-02-15 2024-05-27 Bioverativ Therapeutics Inc. Zoptymalizowany gen czynnika viii
MX2018003702A (es) 2015-09-24 2018-04-30 Biomarin Pharm Inc Vectores del factor viii del virus adenoasociado, particulas viricas asociadas y formulaciones terapeuticas que las comprenden.
KR102450833B1 (ko) * 2016-04-15 2022-10-05 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 혈우병 a 치료용 유전자 요법
BR112020001979A2 (pt) 2017-08-01 2020-08-18 Spark Therapeutics, Inc. métodos de terapia genética do fator viii (fviii)

Patent Citations (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN102573915A (zh) * 2009-04-30 2012-07-11 赛托斯生物技术公司 流感血凝素组合物及其用途
WO2010125202A1 (en) * 2009-04-30 2010-11-04 Cytos Biotechnology Ag Influenza hemagglutinin compositions and uses thereof
WO2015038625A1 (en) * 2013-09-12 2015-03-19 Biomarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors
US20170096684A1 (en) * 2014-05-21 2017-04-06 Imperial Innovations Limited Lentiviral vectors
WO2017021359A1 (en) * 2015-08-03 2017-02-09 Myodopa Limited Systemic synthesis and regulation of l-dopa
CN108136048A (zh) * 2015-08-03 2018-06-08 米奥多巴有限公司 左旋多巴的系统合成和调节
CN108473976A (zh) * 2015-10-28 2018-08-31 桑格摩生物治疗股份有限公司 肝特异性构建体、因子viii表达盒及其使用方法
US20170119906A1 (en) * 2015-10-28 2017-05-04 Sangamo Biosciences, Inc. Liver-specific constructs, factor viii expression cassettes and methods of use thereof
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
CN108368164A (zh) * 2015-10-30 2018-08-03 星火治疗有限公司 CpG减少的因子VIII变体、组合物和方法以及用于治疗止血障碍的用途
CN109195636A (zh) * 2016-03-03 2019-01-11 马萨诸塞大学 用于非病毒基因转移的末端封闭型线性双链体dna
US20190032083A1 (en) * 2016-03-03 2019-01-31 University Of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer
WO2019032898A1 (en) * 2017-08-09 2019-02-14 Bioverativ Therapeutics Inc. NUCLEIC ACID MOLECULES AND USES THEREOF

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IL286284A (en) 2021-10-31
WO2020186207A8 (en) 2020-11-12
AU2020234713A1 (en) 2021-11-04
SG11202109850SA (en) 2021-10-28
EP3938523A2 (en) 2022-01-19
KR20210151785A (ko) 2021-12-14
US12338275B2 (en) 2025-06-24
US20250282848A1 (en) 2025-09-11
MX2021011039A (es) 2021-12-15
CA3133255A1 (en) 2020-09-17
JP2025073123A (ja) 2025-05-12
EP3938523A4 (en) 2023-06-21
WO2020186207A2 (en) 2020-09-17
BR112021017853A2 (pt) 2021-11-30
JP2022524434A (ja) 2022-05-02
MA55313A (fr) 2022-01-19
WO2020186207A3 (en) 2020-12-17
US20220177545A1 (en) 2022-06-09

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