JP7583732B2 - フェニルアラニンヒドロキシラーゼ（ｐａｈ）治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用 - Google Patents

フェニルアラニンヒドロキシラーゼ（ｐａｈ）治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用 Download PDF

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JP7583732B2
JP7583732B2 JP2021554695A JP2021554695A JP7583732B2 JP 7583732 B2 JP7583732 B2 JP 7583732B2 JP 2021554695 A JP2021554695 A JP 2021554695A JP 2021554695 A JP2021554695 A JP 2021554695A JP 7583732 B2 JP7583732 B2 JP 7583732B2
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itr
pah
cedna
sequence
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JP2022525302A (ja
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ダグラス アンソニー カー，
フィリップ サマヨア，
ナサニエル シルバー，
マシュー チオッコ，
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ジェネレーション バイオ カンパニー
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    • 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
    • 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
    • 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
    • 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
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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)
    • 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
    • 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)

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • 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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021554695A 2019-03-13 2020-03-13 フェニルアラニンヒドロキシラーゼ（ｐａｈ）治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用 Active JP7583732B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024193194A JP2025016685A (ja) 2019-03-13 2024-11-01 フェニルアラニンヒドロキシラーゼ（ｐａｈ）治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用

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

Related Child Applications (1)

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JP2024193194A Division JP2025016685A (ja) 2019-03-13 2024-11-01 フェニルアラニンヒドロキシラーゼ（ｐａｈ）治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用

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JP2022525302A JP2022525302A (ja) 2022-05-12
JP2022525302A5 JP2022525302A5 (https=) 2023-03-22
JP7583732B2 true JP7583732B2 (ja) 2024-11-14

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JP2021554695A Active JP7583732B2 (ja) 2019-03-13 2020-03-13 フェニルアラニンヒドロキシラーゼ（ｐａｈ）治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用
JP2024193194A Pending JP2025016685A (ja) 2019-03-13 2024-11-01 フェニルアラニンヒドロキシラーゼ（ｐａｈ）治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用

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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

Citations (5)

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JP2014513928A (ja) 2011-03-11 2014-06-19 アソシアシオン・アンスティテュ・ドゥ・ミオロジー 無カプシドａａｖベクター、組成物ならびにベクター製造および遺伝子運搬のための方法
WO2017152149A1 (en) 2016-03-03 2017-09-08 University Of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer
WO2018126112A1 (en) 2016-12-30 2018-07-05 The Trustees Of The University Of Pennsylvania Gene therapy for treating phenylketonuria
WO2018222925A1 (en) 2017-05-31 2018-12-06 Ultragenyx Pharmaceutical Inc. Therapeutics for phenylketonuria
WO2019032898A1 (en) 2017-08-09 2019-02-14 Bioverativ Therapeutics Inc. NUCLEIC ACID MOLECULES AND USES THEREOF

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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
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

Patent Citations (5)

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Publication number Priority date Publication date Assignee Title
JP2014513928A (ja) 2011-03-11 2014-06-19 アソシアシオン・アンスティテュ・ドゥ・ミオロジー 無カプシドａａｖベクター、組成物ならびにベクター製造および遺伝子運搬のための方法
WO2017152149A1 (en) 2016-03-03 2017-09-08 University Of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer
WO2018126112A1 (en) 2016-12-30 2018-07-05 The Trustees Of The University Of Pennsylvania Gene therapy for treating phenylketonuria
WO2018222925A1 (en) 2017-05-31 2018-12-06 Ultragenyx Pharmaceutical Inc. Therapeutics for phenylketonuria
WO2019032898A1 (en) 2017-08-09 2019-02-14 Bioverativ Therapeutics Inc. NUCLEIC ACID MOLECULES AND USES THEREOF

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KR20210149702A (ko) 2021-12-09
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
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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