JP2023528371A - Ｐａｈ遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用方法 - Google Patents

Ｐａｈ遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用方法 Download PDF

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JP2023528371A
JP2023528371A JP2022572768A JP2022572768A JP2023528371A JP 2023528371 A JP2023528371 A JP 2023528371A JP 2022572768 A JP2022572768 A JP 2022572768A JP 2022572768 A JP2022572768 A JP 2022572768A JP 2023528371 A JP2023528371 A JP 2023528371A
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amino acid
seq
capsid protein
raav
protein corresponding
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ジェーソン・ボーク・ライト
ダニエル・ローレン・ソッキアシアン
ティア・バボヴァル・セント・マーティン
オマー・フランコーネ
アルバート・バーンズ・シーモア
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ホモロジー・メディシンズ・インコーポレイテッド
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    • 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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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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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  • Gastroenterology & Hepatology (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2022572768A 2020-05-27 2021-05-27 Ｐａｈ遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用方法 Pending JP2023528371A (ja)

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US202063030341P 2020-05-27 2020-05-27
US63/030,341 2020-05-27
US202063117252P 2020-11-23 2020-11-23
US63/117,252 2020-11-23
PCT/US2021/070615 WO2021243364A1 (en) 2020-05-27 2021-05-27 Adeno-associated virus compositions for restoring pah gene function and methods of use thereof

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JP2023528371A true JP2023528371A (ja) 2023-07-04
JP2023528371A5 JP2023528371A5 (https=) 2024-04-03
JPWO2021243364A5 JPWO2021243364A5 (https=) 2024-04-03

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US (2) US12076420B2 (https=)
EP (1) EP4158040A4 (https=)
JP (1) JP2023528371A (https=)
KR (1) KR20230029650A (https=)
CN (1) CN115803441A (https=)
AU (1) AU2021279048A1 (https=)
BR (1) BR112022023908A2 (https=)
CA (1) CA3180219A1 (https=)
CO (1) CO2022018370A2 (https=)
MX (1) MX2022014881A (https=)
PE (1) PE20230433A1 (https=)
TW (1) TW202208632A (https=)
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Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023544165A (ja) 2020-10-01 2023-10-20 ジェンザイム・コーポレーション 肝臓に向けられた遺伝子補充療法によってｐｋｕを処置するためのヒトｐａｈ発現カセット
KR20240099164A (ko) * 2021-09-08 2024-06-28 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 Pah-조절 조성물 및 방법
US20230399657A1 (en) * 2022-05-17 2023-12-14 Homology Medicines, Inc. Liver de-targeted recombinant aav capsid proteins
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
WO2025064507A1 (en) * 2023-09-19 2025-03-27 Poseida Therapeutics, Inc. Compositions and methods for integration of viral vectors

Citations (1)

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Publication number Priority date Publication date Assignee Title
US20190256867A1 (en) * 2018-02-01 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof

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