JP7066861B2 - Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法 - Google Patents

Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法 Download PDF

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JP7066861B2
JP7066861B2 JP2020541862A JP2020541862A JP7066861B2 JP 7066861 B2 JP7066861 B2 JP 7066861B2 JP 2020541862 A JP2020541862 A JP 2020541862A JP 2020541862 A JP2020541862 A JP 2020541862A JP 7066861 B2 JP7066861 B2 JP 7066861B2
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amino acid
capsid protein
aav
sequence
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JP2021512601A (ja
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アルバート・バーンズ・シーモア
シーミン・セヘル・アーメド
ジェーソン・ボーク・ライト
セリーナ・ニコル・ドリヴ
ヒラード・ルビン
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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
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    • 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
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    • 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
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    • 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
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
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    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/94—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving narcotics or drugs or pharmaceuticals, neurotransmitters or associated receptors
    • G01N33/9406—Neurotransmitters
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    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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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
    • 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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JP2020541862A 2018-02-01 2019-02-01 Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法 Expired - Fee Related JP7066861B2 (ja)

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JP2022073374A JP7433360B2 (ja) 2018-02-01 2022-04-27 Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法

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US201862625150P 2018-02-01 2018-02-01
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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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EP (1) EP3746113A4 (https=)
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CN (1) CN111886021A (https=)
AU (1) AU2019215150A1 (https=)
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CA (1) CA3089824A1 (https=)
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Families Citing this family (9)

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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
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
CN119876192B (zh) * 2024-07-02 2025-11-07 浙江大学滨江研究院 用于治疗苯丙酮尿症的多核苷酸

Citations (6)

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WO2003093436A2 (en) 2002-04-30 2003-11-13 University Of Florida Treatment for phenylketonuria
JP2005522517A (ja) 2002-04-16 2005-07-28 敬也 小澤 組み換えアデノ随伴ウイルスビリオンを使用したアミノ酸代謝に関与する酵素を搬送するための組成物、およびそのようなアデノ随伴ウイルスビリオンを用いてアミノ酸代謝疾患を治療するための使用方法
JP2014507154A (ja) 2011-02-17 2014-03-27 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 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
JP2016535729A (ja) 2013-10-22 2016-11-17 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド フェニルケトン尿症のためのｍＲＮＡ療法
US20170211094A1 (en) 2010-04-30 2017-07-27 City Of Hope Recombinant adeno-associated vectors for targeted treatment
JP2017529854A (ja) 2014-09-24 2017-10-12 シティ・オブ・ホープＣｉｔｙ ｏｆ Ｈｏｐｅ 高効率ゲノム編集のためのアデノ随伴ウイルスベクターバリアント及びその方法

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WO2010124180A1 (en) * 2009-04-23 2010-10-28 Phasebio Pharmaceuticals, Inc. Phenylalanine hydroxylase fusion protein and methods for treating phenylketonuria
KR102738752B1 (ko) * 2016-12-30 2024-12-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 페닐케톤뇨증을 치료하기 위한 유전자 치료

Patent Citations (6)

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JP2005522517A (ja) 2002-04-16 2005-07-28 敬也 小澤 組み換えアデノ随伴ウイルスビリオンを使用したアミノ酸代謝に関与する酵素を搬送するための組成物、およびそのようなアデノ随伴ウイルスビリオンを用いてアミノ酸代謝疾患を治療するための使用方法
WO2003093436A2 (en) 2002-04-30 2003-11-13 University Of Florida Treatment for phenylketonuria
US20170211094A1 (en) 2010-04-30 2017-07-27 City Of Hope Recombinant adeno-associated vectors for targeted treatment
JP2014507154A (ja) 2011-02-17 2014-03-27 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 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
JP2016535729A (ja) 2013-10-22 2016-11-17 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド フェニルケトン尿症のためのｍＲＮＡ療法
JP2017529854A (ja) 2014-09-24 2017-10-12 シティ・オブ・ホープＣｉｔｙ ｏｆ Ｈｏｐｅ 高効率ゲノム編集のためのアデノ随伴ウイルスベクターバリアント及びその方法

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Molecular Therapy: Nucleic Acids，2017年，Vol.7，pp.339-349

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EP3746113A1 (en) 2020-12-09
JP2022105105A (ja) 2022-07-12
WO2019152841A1 (en) 2019-08-08
IL276215B (en) 2022-10-01
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
CN111886021A (zh) 2020-11-03
CL2020001992A1 (es) 2021-02-12
EA202091844A1 (ru) 2021-02-03
CO2020010783A2 (es) 2020-11-20

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