CN111886021A - 用于pah基因转移的腺伴随病毒组合物及其使用方法 - Google Patents

用于pah基因转移的腺伴随病毒组合物及其使用方法 Download PDF

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Publication number
CN111886021A
CN111886021A CN201980019659.2A CN201980019659A CN111886021A CN 111886021 A CN111886021 A CN 111886021A CN 201980019659 A CN201980019659 A CN 201980019659A CN 111886021 A CN111886021 A CN 111886021A
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amino acid
seq
capsid protein
gly
asn
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Chinese (zh)
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A.B.西摩
S.S.阿梅德
J.B.怀特
S.N.多利夫
H.鲁宾
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Cognate3 LLC
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Cognate3 LLC
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    • 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
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

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CN201980019659.2A 2018-02-01 2019-02-01 用于pah基因转移的腺伴随病毒组合物及其使用方法 Pending CN111886021A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862625150P 2018-02-01 2018-02-01
US62/625,150 2018-02-01
PCT/US2019/016351 WO2019152841A1 (en) 2018-02-01 2019-02-01 Adeno-associated virus compositions for pah gene transfer and methods of use thereof

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CN111886021A true CN111886021A (zh) 2020-11-03

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EP (1) EP3746113A4 (https=)
JP (2) JP7066861B2 (https=)
KR (2) KR102528641B1 (https=)
CN (1) CN111886021A (https=)
AU (1) AU2019215150A1 (https=)
BR (1) BR112020015511A2 (https=)
CA (1) CA3089824A1 (https=)
CL (1) CL2020001992A1 (https=)
CO (1) CO2020010783A2 (https=)
EA (1) EA202091844A1 (https=)
IL (2) IL295830A (https=)
MX (1) MX2020008085A (https=)
PE (1) PE20210341A1 (https=)
WO (1) WO2019152841A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN119876192A (zh) * 2024-07-02 2025-04-25 浙江大学滨江研究院 用于治疗苯丙酮尿症的多核苷酸

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PE20190401A1 (es) 2016-07-26 2019-03-13 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado
BR112020022722A8 (pt) 2018-05-09 2022-01-18 Biomarin Pharm Inc Genoma e seu uso, vetor, partícula, molécula de ácido nucleico isolada, ácido nucleico, composição, tratamento, método
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
EP3980447A4 (en) * 2019-06-10 2023-07-26 Homology Medicines, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR ARSA GENE TRANSFER AND METHODS OF USE THEREOF
TW202140791A (zh) * 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
IL294713A (en) * 2020-01-16 2022-09-01 Takeda Pharmaceuticals Co Adeno associated virus based gene therapy for phenylketonuria
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

Citations (2)

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US20040142416A1 (en) * 2002-04-30 2004-07-22 Laipis Philip J. Treatment for phenylketonuria
CN107295802A (zh) * 2014-09-24 2017-10-24 希望之城 用于高效基因组编辑的腺相关病毒载体变异体和其方法

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US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
WO2010124180A1 (en) * 2009-04-23 2010-10-28 Phasebio Pharmaceuticals, Inc. Phenylalanine hydroxylase fusion protein and methods for treating phenylketonuria
US9839696B2 (en) * 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
DK2675902T3 (da) * 2011-02-17 2019-06-03 Univ Pennsylvania Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel
EA034103B1 (ru) * 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
KR102738752B1 (ko) * 2016-12-30 2024-12-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 페닐케톤뇨증을 치료하기 위한 유전자 치료

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040142416A1 (en) * 2002-04-30 2004-07-22 Laipis Philip J. Treatment for phenylketonuria
CN107295802A (zh) * 2014-09-24 2017-10-24 希望之城 用于高效基因组编辑的腺相关病毒载体变异体和其方法

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN119876192A (zh) * 2024-07-02 2025-04-25 浙江大学滨江研究院 用于治疗苯丙酮尿症的多核苷酸

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EP3746113A1 (en) 2020-12-09
JP2022105105A (ja) 2022-07-12
WO2019152841A1 (en) 2019-08-08
IL276215B (en) 2022-10-01
JP7066861B2 (ja) 2022-05-13
JP2021512601A (ja) 2021-05-20
JP7433360B2 (ja) 2024-02-19
KR20200127187A (ko) 2020-11-10
IL276215B2 (en) 2023-02-01
CA3089824A1 (en) 2019-08-08
MX2020008085A (es) 2020-12-10
IL295830A (en) 2022-10-01
BR112020015511A2 (pt) 2021-01-26
PE20210341A1 (es) 2021-02-23
EP3746113A4 (en) 2022-03-09
KR20230065367A (ko) 2023-05-11
IL276215A (en) 2020-09-30
AU2019215150A1 (en) 2020-09-24
KR102528641B1 (ko) 2023-05-03
CL2020001992A1 (es) 2021-02-12
EA202091844A1 (ru) 2021-02-03
CO2020010783A2 (es) 2020-11-20

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