KR20230029650A - Pah 유전자 기능을 복구하기 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 - Google Patents

Pah 유전자 기능을 복구하기 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 Download PDF

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KR20230029650A
KR20230029650A KR1020227044583A KR20227044583A KR20230029650A KR 20230029650 A KR20230029650 A KR 20230029650A KR 1020227044583 A KR1020227044583 A KR 1020227044583A KR 20227044583 A KR20227044583 A KR 20227044583A KR 20230029650 A KR20230029650 A KR 20230029650A
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amino acid
seq
gly
capsid protein
asn
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제이슨 보크 라이트
다니엘 로렌 수키아시안
마틴 티아 바보발 세인트
오마르 프랑콘
앨버트 반즈 세이무어
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호몰로지 메디슨, 인크.
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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
    • 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
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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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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KR1020227044583A 2020-05-27 2021-05-27 Pah 유전자 기능을 복구하기 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 Withdrawn KR20230029650A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
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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KR20230029650A true KR20230029650A (ko) 2023-03-03

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KR1020227044583A Withdrawn KR20230029650A (ko) 2020-05-27 2021-05-27 Pah 유전자 기능을 복구하기 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법

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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=)
WO (1) WO2021243364A1 (https=)

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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.
CA3231712A1 (en) * 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Pah-modulating compositions and methods
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

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