KR20210149702A - 비바이러스성 dna 벡터 및 페닐알라닌 히드록실라아제(pah) 치료제 발현을 위한 이의 용도 - Google Patents

비바이러스성 dna 벡터 및 페닐알라닌 히드록실라아제(pah) 치료제 발현을 위한 이의 용도 Download PDF

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KR20210149702A
KR20210149702A KR1020217029544A KR20217029544A KR20210149702A KR 20210149702 A KR20210149702 A KR 20210149702A KR 1020217029544 A KR1020217029544 A KR 1020217029544A KR 20217029544 A KR20217029544 A KR 20217029544A KR 20210149702 A KR20210149702 A KR 20210149702A
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seq
itr
cedna vector
cedna
pah
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더글라스 앤써니 케르
필립 사마요아
나다니엘 실버
매튜 치오꼬
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제너레이션 바이오 컴퍼니
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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
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    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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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
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    • 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)
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    • 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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    • 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)

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  • 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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020217029544A 2019-03-13 2020-03-13 비바이러스성 dna 벡터 및 페닐알라닌 히드록실라아제(pah) 치료제 발현을 위한 이의 용도 Pending KR20210149702A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962817771P 2019-03-13 2019-03-13
US62/817,771 2019-03-13
US201962857514P 2019-06-05 2019-06-05
US62/857,514 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
KR20210149702A true KR20210149702A (ko) 2021-12-09

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KR1020217029544A Pending KR20210149702A (ko) 2019-03-13 2020-03-13 비바이러스성 dna 벡터 및 페닐알라닌 히드록실라아제(pah) 치료제 발현을 위한 이의 용도

Country Status (12)

Country Link
US (1) US12415002B2 (https=)
EP (1) EP3938515A4 (https=)
JP (2) JP7583732B2 (https=)
KR (1) KR20210149702A (https=)
CN (1) CN113874508A (https=)
AU (1) AU2020235121A1 (https=)
BR (1) BR112021017852A2 (https=)
CA (1) CA3133330A1 (https=)
IL (1) IL286283A (https=)
MX (1) MX2021011037A (https=)
SG (1) SG11202109830TA (https=)
WO (1) WO2020186150A2 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4165196A1 (en) * 2020-06-11 2023-04-19 Sangamo Therapeutics, Inc. Methods and compositions for expressing phenylalanine hydroxylase
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Ｄｎａ分子の組成物、その作製方法、及びその使用方法
EP4214309A4 (en) * 2020-09-16 2025-07-16 Generation Bio Co CLOSED-END DNA VECTORS AND RELATED USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH)
GB202014751D0 (en) * 2020-09-18 2020-11-04 Lightbio Ltd Targeting vector
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.
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
AU2024257493A1 (en) * 2023-04-18 2025-12-04 Siren Biotechnology, Inc. Adeno-associated viral vector compositions and methods of use
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
CN119876192B (zh) * 2024-07-02 2025-11-07 浙江大学滨江研究院 用于治疗苯丙酮尿症的多核苷酸

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2535877T3 (es) 2006-06-19 2015-05-18 Asklepios Biopharmaceutical, Inc. Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica
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
MX2018010633A (es) 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.
KR102738752B1 (ko) 2016-12-30 2024-12-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 페닐케톤뇨증을 치료하기 위한 유전자 치료
WO2018167621A1 (en) 2017-03-16 2018-09-20 Pfizer Inc. Tyrosine prototrophy
US11820999B2 (en) * 2017-04-03 2023-11-21 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
NZ762100A (en) * 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof

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Publication number Publication date
EP3938515A2 (en) 2022-01-19
JP2022525302A (ja) 2022-05-12
CN113874508A (zh) 2021-12-31
CA3133330A1 (en) 2020-09-17
WO2020186150A3 (en) 2020-10-29
JP2025016685A (ja) 2025-02-04
EP3938515A4 (en) 2022-12-28
BR112021017852A2 (pt) 2021-11-30
JP7583732B2 (ja) 2024-11-14
IL286283A (en) 2021-10-31
SG11202109830TA (en) 2021-10-28
WO2020186150A2 (en) 2020-09-17
MX2021011037A (es) 2021-12-15
AU2020235121A1 (en) 2021-11-04
US12415002B2 (en) 2025-09-16
US20230024354A1 (en) 2023-01-26

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