MA51915A - Expression régulée de transgènes à l'aide de vecteurs adn à extrémités fermées (cedna) - Google Patents

Expression régulée de transgènes à l'aide de vecteurs adn à extrémités fermées (cedna)

Info

Publication number
MA51915A
MA51915A MA051915A MA51915A MA51915A MA 51915 A MA51915 A MA 51915A MA 051915 A MA051915 A MA 051915A MA 51915 A MA51915 A MA 51915A MA 51915 A MA51915 A MA 51915A
Authority
MA
Morocco
Prior art keywords
cedna
transgenes
closed
regulated expression
dna vectors
Prior art date
Application number
MA051915A
Other languages
English (en)
Inventor
Mark D Angelino
Matt Chiocco
Douglas A Kerr
Robert M Kotin
Phillip Samayoa
Matthew G Stanton
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 MA51915A publication Critical patent/MA51915A/fr

Links

Classifications

    • 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
    • A61K48/0083—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 administration regime
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/06—Antipsoriatics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C12N15/86—Viral vectors
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/50—Vectors for producing vectors
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0069—Oxidoreductases (1.) acting on single donors with incorporation of molecular oxygen, i.e. oxygenases (1.13)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Dermatology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Hospice & Palliative Care (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Immunology (AREA)
  • Psychiatry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
MA051915A 2018-02-22 2019-02-21 Expression régulée de transgènes à l'aide de vecteurs adn à extrémités fermées (cedna) MA51915A (fr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862633795P 2018-02-22 2018-02-22
US201862633757P 2018-02-22 2018-02-22
US201862633882P 2018-02-22 2018-02-22
US201862746762P 2018-10-17 2018-10-17

Publications (1)

Publication Number Publication Date
MA51915A true MA51915A (fr) 2020-12-30

Family

ID=67687406

Family Applications (1)

Application Number Title Priority Date Filing Date
MA051915A MA51915A (fr) 2018-02-22 2019-02-21 Expression régulée de transgènes à l'aide de vecteurs adn à extrémités fermées (cedna)

Country Status (14)

Country Link
US (1) US20220175970A1 (fr)
EP (1) EP3755803A4 (fr)
JP (2) JP2021513999A (fr)
KR (1) KR20200124250A (fr)
CN (1) CN111886343A (fr)
AU (1) AU2019225937A1 (fr)
BR (1) BR112020017060A2 (fr)
CA (1) CA3092059A1 (fr)
IL (1) IL276658A (fr)
MA (1) MA51915A (fr)
MX (1) MX2020008676A (fr)
PH (1) PH12020551235A1 (fr)
SG (1) SG11202007621TA (fr)
WO (1) WO2019165050A1 (fr)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI904068B (zh) 2017-08-09 2025-11-11 美商生物化學醫療公司 核酸分子及其用途
WO2020033863A1 (fr) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Molécules d'acide nucléique et leurs utilisations pour une thérapie génique non virale
EP3877528A4 (fr) * 2018-11-09 2022-11-30 Generation Bio Co. Adn à extrémité fermée modifié (cedna) comprenant des répétitions terminales inversées modifiées symétriques
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
CA3172591A1 (fr) * 2020-03-24 2021-09-30 Douglas Anthony KERR Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents therapeutiques de la maladie de gaucher
IL296660A (en) * 2020-03-24 2022-11-01 Generation Bio Co Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
WO2021222476A2 (fr) * 2020-04-28 2021-11-04 President And Fellows Of Harvard College Système d'administration de gènes à haut rendement
US20230265440A1 (en) * 2020-06-26 2023-08-24 The Board Of Trustees Of The Leland Stanford Junior University Targeting the human ccr5 locus as a safe harbor for the expression of therapeutic proteins
EP4189098A1 (fr) 2020-07-27 2023-06-07 Anjarium Biosciences AG Compositions de molécules d'adn, leurs procédés de fabrication et leurs procédés d'utilisation
BR112023002395A2 (pt) * 2020-08-23 2023-03-21 Bioverativ Therapeutics Inc Sistema de baculovírus modificado para produção aprimorada de dna com extremidades fechadas (cedna)
IL301196A (en) * 2020-09-16 2023-05-01 Generation Bio Co Closed-end DNA vectors and their uses for phenylalanine hydroxylase (pah) expression
US20240181085A1 (en) * 2021-03-19 2024-06-06 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing pfic therapeutics
WO2025114932A1 (fr) * 2023-11-30 2025-06-05 Sanofi Purification de molécules d'adn à extrémité fermée

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003087334A2 (fr) * 2002-04-09 2003-10-23 Cornell Research Foundation, Inc. Utilisation d'element d'efficacite d'integration de virus adeno-associe (aav) permettant d'assurer la mediation d'une integration specifique de site d'une unite de transcription
LT2816118T (lt) * 2005-05-31 2018-12-10 The Regents Of The University Of Colorado, A Body Corporate Genų pristatymo būdai
WO2009038462A1 (fr) * 2007-09-19 2009-03-26 Amsterdam Molecular Therapeutics B.V. Utilisation de la machinerie de réplication d'un virus adéno-associé (aav) pour une production protéique améliorée
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
WO2015191508A1 (fr) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Capsides chimériques
WO2016037161A2 (fr) * 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Procédés et compositions pour atténuer des réponses immunitaires contre des vecteurs de transfert viraux pour l'édition génique
EP3889260A1 (fr) * 2014-12-12 2021-10-06 The Broad Institute, Inc. Arn guides protégés (pgrnas)
EP3423110B1 (fr) * 2016-03-03 2021-08-11 University of Massachusetts Adn double hélice linéaire à extrémité fermée pour transfert de gène non viral

Also Published As

Publication number Publication date
JP2021513999A (ja) 2021-06-03
PH12020551235A1 (en) 2021-04-19
KR20200124250A (ko) 2020-11-02
JP2024028931A (ja) 2024-03-05
EP3755803A1 (fr) 2020-12-30
CN111886343A (zh) 2020-11-03
IL276658A (en) 2020-09-30
CA3092059A1 (fr) 2019-08-29
EP3755803A4 (fr) 2022-01-19
MX2020008676A (es) 2020-09-25
US20220175970A1 (en) 2022-06-09
WO2019165050A1 (fr) 2019-08-29
AU2019225937A1 (en) 2020-08-13
BR112020017060A2 (pt) 2020-12-15
SG11202007621TA (en) 2020-09-29
RU2020131041A (ru) 2022-03-22

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