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MA55313A - Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii - Google Patents

Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii

Info

Publication number
MA55313A
MA55313A MA055313A MA55313A MA55313A MA 55313 A MA55313 A MA 55313A MA 055313 A MA055313 A MA 055313A MA 55313 A MA55313 A MA 55313A MA 55313 A MA55313 A MA 55313A
Authority
MA
Morocco
Prior art keywords
therapeutic agents
factor viii
viral dna
dna vectors
expressing factor
Prior art date
Application number
MA055313A
Other languages
English (en)
Inventor
Douglas Anthony Kerr
Debra Klatte
Phillip Samayoa
Nathaniel Silver
Original Assignee
Generation Bio Co
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Generation Bio Co filed Critical Generation Bio Co
Publication of MA55313A publication Critical patent/MA55313A/fr

Links

Classifications

    • 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
    • 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
    • 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
    • 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

Landscapes

  • 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)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Hematology (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)
MA055313A 2019-03-13 2020-03-13 Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii MA55313A (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962817904P 2019-03-13 2019-03-13
US201962856432P 2019-06-03 2019-06-03

Publications (1)

Publication Number Publication Date
MA55313A true MA55313A (fr) 2022-01-19

Family

ID=72426821

Family Applications (1)

Application Number Title Priority Date Filing Date
MA055313A MA55313A (fr) 2019-03-13 2020-03-13 Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii

Country Status (13)

Country Link
US (2) US12338275B2 (fr)
EP (1) EP3938523A4 (fr)
JP (2) JP2022524434A (fr)
KR (1) KR20210151785A (fr)
CN (1) CN113874513A (fr)
AU (1) AU2020234713A1 (fr)
BR (1) BR112021017853A2 (fr)
CA (1) CA3133255A1 (fr)
IL (1) IL286284A (fr)
MA (1) MA55313A (fr)
MX (1) MX2021011039A (fr)
SG (1) SG11202109850SA (fr)
WO (1) WO2020186207A2 (fr)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7374883B2 (ja) 2017-08-09 2023-11-07 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 核酸分子およびその使用
CN119955796A (zh) 2018-08-09 2025-05-09 比奥维拉迪维治疗股份有限公司 核酸分子及其用于非病毒基因疗法的用途
WO2022023284A1 (fr) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions de molécules d'adn, leurs procédés de fabrication et leurs procédés d'utilisation
US20220356490A1 (en) * 2020-08-23 2022-11-10 Bioverativ Therapeutics Inc. Baculovirus expression system
EP4419677A4 (fr) * 2021-10-18 2025-09-24 Flagship Pioneering Innovations Vii Llc Compositions d'adn et procédés associés
EP4428237A1 (fr) * 2021-11-02 2024-09-11 Gritgen Therapeutics Limited Molécule d'acide nucléique isolée et son utilisation
WO2023135273A2 (fr) * 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions de molécules d'adn codant pour le facteur viii, leurs procédés de préparation et leurs méthodes d'utilisation
WO2025110984A1 (fr) * 2023-11-21 2025-05-30 Hydrogene Therapeutics, Inc. Compositions et méthodes pour le traitement de la maladie de wilson

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2220222A4 (fr) 2007-11-19 2011-10-12 Univ California Nouvelle analyse pour des inhibiteurs d'egfr
US20120263743A1 (en) 2009-04-30 2012-10-18 Cytos Biotechnology Ag Influenza hemagglutinin compositions and uses thereof
EP2500434A1 (fr) * 2011-03-12 2012-09-19 Association Institut de Myologie Vecteurs AAV sans capside, compositions et procédés pour la production des vecteurs et la thérapie génique
LT2956477T (lt) 2013-02-15 2021-02-10 Bioverativ Therapeutics Inc. Optimizuotas faktoriaus viii genas
EP3044231B1 (fr) 2013-09-12 2020-08-05 BioMarin Pharmaceutical Inc. Vecteurs de virus adeno-associes comprentants un gène du facteur viii
GB2526339A (en) * 2014-05-21 2015-11-25 Imp Innovations Ltd Lentiviral vectors
RU2018104098A (ru) 2015-08-03 2019-09-06 Майодопа Лимитед Системный синтез и регуляция l-дофа
MA42934A (fr) 2015-09-24 2018-08-01 Biomarin Pharm Inc Vecteurs de facteur viii à virus adéno-associé, particules virales associées et formulations thérapeutiques comprenant ceux-ci
MY189674A (en) * 2015-10-28 2022-02-24 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
PT3423110T (pt) * 2016-03-03 2021-11-09 Univ Massachusetts Adn em dúplex linear de extremidades fechadas para transferência génica não viral
AU2017248659B2 (en) * 2016-04-15 2022-08-11 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia A
WO2019028192A1 (fr) * 2017-08-01 2019-02-07 Spark Therapeutics, Inc. Méthodes de thérapie génique ciblant le facteur viii (fviii)
JP7374883B2 (ja) * 2017-08-09 2023-11-07 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 核酸分子およびその使用

Also Published As

Publication number Publication date
CN113874513A (zh) 2021-12-31
KR20210151785A (ko) 2021-12-14
CA3133255A1 (fr) 2020-09-17
WO2020186207A8 (fr) 2020-11-12
BR112021017853A2 (pt) 2021-11-30
JP2022524434A (ja) 2022-05-02
MX2021011039A (es) 2021-12-15
JP2025073123A (ja) 2025-05-12
AU2020234713A1 (en) 2021-11-04
SG11202109850SA (en) 2021-10-28
US20220177545A1 (en) 2022-06-09
IL286284A (en) 2021-10-31
WO2020186207A3 (fr) 2020-12-17
US12338275B2 (en) 2025-06-24
WO2020186207A2 (fr) 2020-09-17
US20250282848A1 (en) 2025-09-11
EP3938523A2 (fr) 2022-01-19
EP3938523A4 (fr) 2023-06-21

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