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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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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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

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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)

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Publication number Priority date Publication date Assignee Title
KR20230161535A (ko) 2016-07-26 2023-11-27 바이오마린 파머수티컬 인크. 신규 아데노-관련 바이러스 캡시드 단백질
JP2021522811A (ja) 2018-05-09 2021-09-02 ビオマリン プハルマセウトイカル インコーポレイテッド フェニルケトン尿症の治療方法
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
TW202112807A (zh) * 2019-06-10 2021-04-01 美商霍蒙拉奇醫藥公司 用於ａｒｓａ基因轉移之腺相關病毒組成物及其使用方法
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

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ES2219011T3 (es) * 1998-04-24 2004-11-16 University Of Florida Vector viral adeno-asociado recombinante que codifica alfa-1 antitripsina para terapia genica.
US20030198620A1 (en) * 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
WO2003093436A2 (en) * 2002-04-30 2003-11-13 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
JP6224459B2 (ja) * 2011-02-17 2017-11-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法
EA201992208A1 (ru) * 2013-10-22 2020-07-31 Транслейт Био, Инк. ЛЕЧЕНИЕ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
BR112017005892A2 (pt) * 2014-09-24 2017-12-12 Hope City variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma
JP7229161B2 (ja) * 2016-12-30 2023-02-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア フェニルケトン尿症を処置するための遺伝子療法

Also Published As

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

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