BR112020006672A2 - vetores com combinações de promotor e acentuador para tratar fenilcetonúria - Google Patents

vetores com combinações de promotor e acentuador para tratar fenilcetonúria Download PDF

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Publication number
BR112020006672A2
BR112020006672A2 BR112020006672-9A BR112020006672A BR112020006672A2 BR 112020006672 A2 BR112020006672 A2 BR 112020006672A2 BR 112020006672 A BR112020006672 A BR 112020006672A BR 112020006672 A2 BR112020006672 A2 BR 112020006672A2
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Brazil
Prior art keywords
pah
sequence
seq
promoter
viral vector
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BR112020006672-9A
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English (en)
Portuguese (pt)
Inventor
Tyler Lahusen
Charles David Pauza
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American Gene Technologies International Inc.
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Publication of BR112020006672A2 publication Critical patent/BR112020006672A2/pt

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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
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    • 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)
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
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    • C12N2740/00—Reverse transcribing RNA viruses
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    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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  • 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)
  • Medicinal Preparation (AREA)
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  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
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BR112020006672-9A 2017-10-02 2018-10-02 vetores com combinações de promotor e acentuador para tratar fenilcetonúria BR112020006672A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762566979P 2017-10-02 2017-10-02
US62/566,979 2017-10-02
PCT/US2018/053919 WO2019070674A2 (en) 2017-10-02 2018-10-02 VECTORS COMPRISING PROMOTER AND ACTIVATOR COMBINATIONS FOR THE TREATMENT OF PHENYLCETONURIA

Publications (1)

Publication Number Publication Date
BR112020006672A2 true BR112020006672A2 (pt) 2020-09-24

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BR112020006672-9A BR112020006672A2 (pt) 2017-10-02 2018-10-02 vetores com combinações de promotor e acentuador para tratar fenilcetonúria

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Country Link
US (1) US20200318081A1 (https=)
EP (1) EP3692157A4 (https=)
JP (2) JP7838779B2 (https=)
KR (1) KR20200057766A (https=)
CN (1) CN111433368A (https=)
AU (1) AU2018345745A1 (https=)
BR (1) BR112020006672A2 (https=)
CA (1) CA3077355A1 (https=)
IL (1) IL273670B2 (https=)
WO (1) WO2019070674A2 (https=)

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CN116064678A (zh) 2016-01-15 2023-05-05 美国基因技术国际有限公司 用于活化γ-δT细胞的方法和组合物
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
WO2017139065A1 (en) 2016-02-08 2017-08-17 American Gene Technologies International Inc. Hiv vaccination and immunotherapy
WO2017156311A2 (en) 2016-03-09 2017-09-14 American Gene Technologies International Inc. Combination vectors and methods for treating cancer
EP3468617A4 (en) 2016-06-08 2020-01-22 American Gene Technologies International Inc. SYSTEM FOR THE NON-INTEGRAL VIRAL ISSUE AND PROCEDURE RELATING TO IT
AU2017292582C1 (en) 2016-07-08 2021-11-11 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
EP3487507A4 (en) 2016-07-21 2020-04-08 American Gene Technologies International, Inc. VIRAL VECTORS FOR THE TREATMENT OF PARKINSON'S DISEASE
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
JP2020524996A (ja) 2017-06-16 2020-08-27 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド ヒトガンマデルタｔ細胞を介する腫瘍細胞傷害性の活性化のための方法および組成物
BR112021024124A2 (pt) * 2019-05-31 2022-01-11 American Gene Tech Int Inc Expressão otimizada de fenilalanina hidroxilase
JP2023510392A (ja) * 2020-01-16 2023-03-13 武田薬品工業株式会社 フェニルケトン尿症に対するアデノ随伴ウイルスベースの遺伝子治療
TW202208632A (zh) * 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
MX2023003805A (es) 2020-10-01 2023-04-12 Genzyme Corp Casete de expresion de pah humana para el tratamiento de la pku mediante terapia de reemplazo genico dirigida al higado.
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