MX2020008676A - Expresion controlada de transgenes mediante el uso de vectores de adn de extremo cerrado (ceadn). - Google Patents

Expresion controlada de transgenes mediante el uso de vectores de adn de extremo cerrado (ceadn).

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Publication number
MX2020008676A
MX2020008676A MX2020008676A MX2020008676A MX2020008676A MX 2020008676 A MX2020008676 A MX 2020008676A MX 2020008676 A MX2020008676 A MX 2020008676A MX 2020008676 A MX2020008676 A MX 2020008676A MX 2020008676 A MX2020008676 A MX 2020008676A
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MX
Mexico
Prior art keywords
level
expression
transgene
cedna
close
Prior art date
Application number
MX2020008676A
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English (en)
Inventor
Phillip Samayoa
Robert M Kotin
Matthew G Stanton
Kerr Douglas A
Matt Chiocco
Mark D Angelino
Original Assignee
Generation Bio Co
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Filing date
Publication date
Application filed by Generation Bio Co filed Critical Generation Bio Co
Publication of MX2020008676A publication Critical patent/MX2020008676A/es

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    • A—HUMAN NECESSITIES
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    • 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
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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
    • 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
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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
    • C12N15/86—Viral vectors
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

En la presente se proporcionan métodos y constructos que comprenden ADN de extremo cerrado (vectores de ceADN) para mantener o sostener un nivel de expresión transgénica en un nivel o rango predeterminado durante un tiempo predefinido, o aumentar el nivel de expresión transgénica en una célula o un sujeto, donde el nivel de expresión transgénica se puede modular (por ejemplo, aumentar) con una o más administraciones posteriores (por ejemplo, una nueva dosis o una administración de refuerzo) después de una administración de cebado inicial. Se proporcionan métodos para personalizar el tratamiento génico a lo largo de la vida de un individuo para expresar un transgén a un nivel que satisfaga las necesidades de un individuo, al modular los niveles de expresión de un transgén expresado por el vector de ceADN de manera gradual, o por etapas, con una o más administraciones después de una administración de cebado inicial (por ejemplo, en el momento 0), lo que permite la titulación del nivel de expresión del transgén a un nivel de expresión predeterminado deseado o a un rango de nivel de expresión deseado.
MX2020008676A 2018-02-22 2019-02-21 Expresion controlada de transgenes mediante el uso de vectores de adn de extremo cerrado (ceadn). MX2020008676A (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862633882P 2018-02-22 2018-02-22
US201862633757P 2018-02-22 2018-02-22
US201862633795P 2018-02-22 2018-02-22
US201862746762P 2018-10-17 2018-10-17
PCT/US2019/018927 WO2019165050A1 (en) 2018-02-22 2019-02-21 Controlled expression of transgenes using close-ended dna (cedna) vectors

Publications (1)

Publication Number Publication Date
MX2020008676A true MX2020008676A (es) 2020-09-25

Family

ID=67687406

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020008676A MX2020008676A (es) 2018-02-22 2019-02-21 Expresion controlada de transgenes mediante el uso de vectores de adn de extremo cerrado (ceadn).

Country Status (13)

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

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020097417A1 (en) * 2018-11-09 2020-05-14 Generation Bio Co. Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats
US20230138409A1 (en) * 2020-03-24 2023-05-04 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
CN115667531A (zh) * 2020-03-24 2023-01-31 世代生物公司 非病毒DNA载体和其用于表达戈谢（Gaucher）治疗剂的用途
EP4142758A2 (en) * 2020-04-28 2023-03-08 President and Fellows of Harvard College High efficiency gene delivery system
CA3177612A1 (en) * 2020-06-26 2021-12-30 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
BR112023001648A2 (pt) 2020-07-27 2023-04-04 Anjarium Biosciences Ag Moléculas de dna de fita dupla, veículo de entrega e método para preparar uma molécula de dna com extremidade em grampo
EP4199971A1 (en) * 2020-08-23 2023-06-28 Bioverativ Therapeutics Inc. Modified baculovirus system for improved production of closed-ended dna (cedna)

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003237805A1 (en) * 2002-04-09 2003-10-27 Cornell Research Foundation, Inc. Use of aav integration efficiency element for mediating site-specific integration of a transcription unit
ES2696824T3 (es) * 2005-05-31 2019-01-18 Univ Colorado Regents Métodos para suministrar genes
US9115373B2 (en) * 2007-09-19 2015-08-25 Uniqure Ip B.V. Use of AAV replication machinery for improved protein production
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
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
SG11201806663TA (en) * 2016-03-03 2018-09-27 Univ Massachusetts Closed-ended linear duplex dna for non-viral gene transfer

Also Published As

Publication number Publication date
MA51915A (fr) 2020-12-30
WO2019165050A1 (en) 2019-08-29
CN111886343A (zh) 2020-11-03
IL276658A (en) 2020-09-30
RU2020131041A (ru) 2022-03-22
US20220175970A1 (en) 2022-06-09
BR112020017060A2 (pt) 2020-12-15
EP3755803A4 (en) 2022-01-19
JP2021513999A (ja) 2021-06-03
JP2024028931A (ja) 2024-03-05
CA3092059A1 (en) 2019-08-29
KR20200124250A (ko) 2020-11-02
SG11202007621TA (en) 2020-09-29
AU2019225937A1 (en) 2020-08-13
EP3755803A1 (en) 2020-12-30

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