MX2021011037A - Vectores de adn no virales y usos de estos para expresar agentes terapéuticos de fenilalanina hidroxilasa (pah). - Google Patents

Vectores de adn no virales y usos de estos para expresar agentes terapéuticos de fenilalanina hidroxilasa (pah).

Info

Publication number
MX2021011037A
MX2021011037A MX2021011037A MX2021011037A MX2021011037A MX 2021011037 A MX2021011037 A MX 2021011037A MX 2021011037 A MX2021011037 A MX 2021011037A MX 2021011037 A MX2021011037 A MX 2021011037A MX 2021011037 A MX2021011037 A MX 2021011037A
Authority
MX
Mexico
Prior art keywords
cedna vectors
pah
pah protein
expression
therapeutics
Prior art date
Application number
MX2021011037A
Other languages
English (en)
Inventor
Phillip Samayoa
Douglas Anthony Kerr
Nathaniel Silver
Matthew Chiocco
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 MX2021011037A publication Critical patent/MX2021011037A/es

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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
    • A61K48/005—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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K48/0016—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 nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • 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
    • 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
    • A61K48/005—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • 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
    • 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.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Urology & Nephrology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

La solicitud describe vectores de ceADN que tienen una estructura lineal y continua para la administración y expresión de un transgén. Los vectores de ceADN comprenden un casete de expresión flanqueado por dos secuencias de ITR, donde el casete de expresión codifica un transgén que codifica una proteína PAH. Algunos vectores de ceADN comprenden además elementos reguladores cis, que incluyen interruptores reguladores. En la presente se proporcionan además métodos y líneas celulares para una expresión génica confiable de la proteína PAH in vitro, ex vivo e in vivo mediante el uso de los vectores de ceADN. En la presente se proporcionan métodos y composiciones que comprenden vectores de ceADN útiles para la expresión de la proteína PAH en una célula, tejido o sujeto, y métodos de tratamiento de enfermedades con dichos vectores de ceADN que expresan la proteína PAH. Tal proteína PAH se puede expresar para tratar enfermedades, por ejemplo.
MX2021011037A 2019-03-13 2020-03-13 Vectores de adn no virales y usos de estos para expresar agentes terapéuticos de fenilalanina hidroxilasa (pah). MX2021011037A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962817771P 2019-03-13 2019-03-13
US201962857514P 2019-06-05 2019-06-05
PCT/US2020/022595 WO2020186150A2 (en) 2019-03-13 2020-03-13 Non-viral dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) therapeutics

Publications (1)

Publication Number Publication Date
MX2021011037A true MX2021011037A (es) 2021-12-15

Family

ID=72426457

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021011037A MX2021011037A (es) 2019-03-13 2020-03-13 Vectores de adn no virales y usos de estos para expresar agentes terapéuticos de fenilalanina hidroxilasa (pah).

Country Status (12)

Country Link
US (1) US20230024354A1 (es)
EP (1) EP3938515A4 (es)
JP (1) JP2022525302A (es)
KR (1) KR20210149702A (es)
CN (1) CN113874508A (es)
AU (1) AU2020235121A1 (es)
BR (1) BR112021017852A2 (es)
CA (1) CA3133330A1 (es)
IL (1) IL286283A (es)
MX (1) MX2021011037A (es)
SG (1) SG11202109830TA (es)
WO (1) WO2020186150A2 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116234917A (zh) 2020-07-27 2023-06-06 安杰瑞姆生物科学公司 Dna分子组合物及其制备方法和使用方法
CN116507730A (zh) * 2020-09-16 2023-07-28 世代生物公司 封闭端dna载体及其用于表达苯丙氨酸羟化酶（pah）的用途
CN115772503B (zh) * 2022-08-16 2023-10-17 广东省南山医药创新研究院 一种表达pah的基因修饰细胞药物及其制备方法与应用
CN116656740A (zh) * 2023-05-30 2023-08-29 浙江大学 一种腺相关病毒基因治疗载体及其在制备治疗苯丙酮尿症的药物中的应用

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2037892B1 (en) * 2006-06-19 2015-03-18 Asklepios Biopharmaceutical, Inc. Modified factor viii and factor ix genes and vectors for gene therapy
LT3423110T (lt) * 2016-03-03 2021-12-27 University Of Massachusetts Linijinis dnr dupleksas su uždarais galais, skirtas nevirusiniam geno pernešimui
CN110325199A (zh) * 2016-12-30 2019-10-11 宾夕法尼亚州立大学托管会 用于治疗苯丙酮尿症的基因疗法
US20200056190A1 (en) * 2017-03-16 2020-02-20 Pfizer Inc. Tyrosine prototrophy
CA3057142A1 (en) * 2017-04-03 2018-10-11 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
EP3630200A4 (en) * 2017-05-31 2021-02-24 Arcturus Therapeutics, Inc. THERAPEUTICS FOR PHENYLKETONURIA
MX2020001593A (es) * 2017-08-09 2020-07-13 Bioverativ Therapeutics Inc Moleculas de acido nucleico y usos de las mismas.

Also Published As

Publication number Publication date
BR112021017852A2 (pt) 2021-11-30
CA3133330A1 (en) 2020-09-17
JP2022525302A (ja) 2022-05-12
US20230024354A1 (en) 2023-01-26
EP3938515A2 (en) 2022-01-19
CN113874508A (zh) 2021-12-31
WO2020186150A2 (en) 2020-09-17
IL286283A (en) 2021-10-31
SG11202109830TA (en) 2021-10-28
EP3938515A4 (en) 2022-12-28
AU2020235121A1 (en) 2021-11-04
WO2020186150A3 (en) 2020-10-29
KR20210149702A (ko) 2021-12-09

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