EA202091844A1 - Композиции аденоассоциированных вирусов для переноса гена pah и способы их применения - Google Patents

Композиции аденоассоциированных вирусов для переноса гена pah и способы их применения

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Publication number
EA202091844A1
EA202091844A1 EA202091844A EA202091844A EA202091844A1 EA 202091844 A1 EA202091844 A1 EA 202091844A1 EA 202091844 A EA202091844 A EA 202091844A EA 202091844 A EA202091844 A EA 202091844A EA 202091844 A1 EA202091844 A1 EA 202091844A1
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Eurasian Patent Office
Prior art keywords
compositions
adeno
methods
associated viruses
application
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Application number
EA202091844A
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English (en)
Inventor
Альберт Барнс Сеймур
Симин Сехер Ахмед
Джейсон Боук Райт
Серена Николь Доллив
Хиллард Рубин
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Хомолоджи Медисинс, Инк.
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Application filed by Хомолоджи Медисинс, Инк. filed Critical Хомолоджи Медисинс, Инк.
Publication of EA202091844A1 publication Critical patent/EA202091844A1/ru

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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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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  • Health & Medical Sciences (AREA)
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Abstract

Изобретение относится к композициям аденоассоциированных вирусов (AAV), которые могут экспрессировать в клетке полипептид фенилаланин-гидроксилазу (PAH), тем самым восстанавливая функцию гена PAH. Изобретение также относится к способам применения композиций AAV и систем упаковки для получения композиций AAV.
EA202091844A 2018-02-01 2019-02-01 Композиции аденоассоциированных вирусов для переноса гена pah и способы их применения EA202091844A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862625150P 2018-02-01 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

Publications (1)

Publication Number Publication Date
EA202091844A1 true EA202091844A1 (ru) 2021-02-03

Family

ID=67478560

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202091844A EA202091844A1 (ru) 2018-02-01 2019-02-01 Композиции аденоассоциированных вирусов для переноса гена pah и способы их применения

Country Status (14)

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

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6994018B2 (ja) 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質
US11739345B2 (en) 2018-05-09 2023-08-29 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
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
CN114502575A (zh) * 2019-06-10 2022-05-13 同源药物公司 用于arsa基因转移的腺相关病毒组合物和其使用方法
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
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

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030198620A1 (en) * 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
AU2003237159A1 (en) * 2002-04-30 2003-11-17 University Of Florida Treatment for phenylketonuria
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
CN103502458B (zh) * 2011-02-17 2016-11-16 宾夕法尼亚大学托管会 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法
US9522176B2 (en) * 2013-10-22 2016-12-20 Shire Human Genetic Therapies, Inc. MRNA therapy for phenylketonuria
CN113667696A (zh) * 2014-09-24 2021-11-19 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
CN110325199A (zh) * 2016-12-30 2019-10-11 宾夕法尼亚州立大学托管会 用于治疗苯丙酮尿症的基因疗法

Also Published As

Publication number Publication date
JP2022105105A (ja) 2022-07-12
IL276215B2 (en) 2023-02-01
KR20230065367A (ko) 2023-05-11
AU2019215150A1 (en) 2020-09-24
CO2020010783A2 (es) 2020-11-20
EP3746113A1 (en) 2020-12-09
JP2021512601A (ja) 2021-05-20
JP7066861B2 (ja) 2022-05-13
KR102528641B1 (ko) 2023-05-03
MX2020008085A (es) 2020-12-10
CL2020001992A1 (es) 2021-02-12
IL276215B (en) 2022-10-01
BR112020015511A2 (pt) 2021-01-26
WO2019152841A1 (en) 2019-08-08
JP7433360B2 (ja) 2024-02-19
CA3089824A1 (en) 2019-08-08
CN111886021A (zh) 2020-11-03
PE20210341A1 (es) 2021-02-23
IL276215A (en) 2020-09-30
IL295830A (en) 2022-10-01
EP3746113A4 (en) 2022-03-09
KR20200127187A (ko) 2020-11-10

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