CN112424345A - Aav载体在青少年对象中的稳定表达 - Google Patents

Aav载体在青少年对象中的稳定表达 Download PDF

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Publication number
CN112424345A
CN112424345A CN201980047182.9A CN201980047182A CN112424345A CN 112424345 A CN112424345 A CN 112424345A CN 201980047182 A CN201980047182 A CN 201980047182A CN 112424345 A CN112424345 A CN 112424345A
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composition
therapeutic
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aav
aav virus
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Chinese (zh)
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S·邦廷
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Biomarin Pharmaceutical Inc
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Biomarin Pharmaceutical Inc
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    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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 delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36Blood coagulation or fibrinolysis factors
    • A61K38/37Factors VIII
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44Oxidoreductases (1)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482Serine endopeptidases (3.4.21)
    • A61K38/4846Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
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    • C07KPEPTIDES
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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745Blood coagulation or fibrinolysis factors
    • C07K14/755Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
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    • C12N9/0004Oxidoreductases (1.)
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    • C12Y114/16Oxidoreductases 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)
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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CN201980047182.9A 2018-05-14 2019-05-14 Aav载体在青少年对象中的稳定表达 Pending CN112424345A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862671271P 2018-05-14 2018-05-14
US62/671,271 2018-05-14
PCT/US2019/032092 WO2019222132A1 (en) 2018-05-14 2019-05-14 Stable expression of aav vectors in juvenile subjects

Publications (1)

Publication Number Publication Date
CN112424345A true CN112424345A (zh) 2021-02-26

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CN201980047182.9A Pending CN112424345A (zh) 2018-05-14 2019-05-14 Aav载体在青少年对象中的稳定表达

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US (2) US20200069819A1 (https=)
EP (1) EP3794112A1 (https=)
JP (2) JP2021523198A (https=)
KR (1) KR20210008491A (https=)
CN (1) CN112424345A (https=)
AR (1) AR117427A1 (https=)
AU (1) AU2019270972A1 (https=)
BR (1) BR112020023159A2 (https=)
CA (1) CA3100000A1 (https=)
MX (1) MX2020012167A (https=)
SG (1) SG11202010832YA (https=)
TW (1) TW202016298A (https=)
WO (1) WO2019222132A1 (https=)

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CN115554418A (zh) * 2022-11-22 2023-01-03 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途
WO2024074143A1 (en) * 2022-10-08 2024-04-11 Lingyi Biotech Co., Ltd. Construct for enhancing gene expression

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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
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
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
US20230407328A1 (en) 2020-11-02 2023-12-21 Biomarin Pharmaceutical Inc. Process for enriching adeno-associated virus
US20240376496A1 (en) 2021-09-03 2024-11-14 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034990A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
US20240384242A1 (en) 2021-09-03 2024-11-21 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034980A1 (en) 2021-09-03 2023-03-09 Bomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034994A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
EP4396203A1 (en) 2021-09-03 2024-07-10 BioMarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
KR20250108100A (ko) 2022-09-22 2025-07-15 디나코 아게 Aav 유전자 치료법 벡터를 이용한 심근병증의 치료
UY40442A (es) 2022-09-22 2024-02-15 Biomarin Pharm Inc Tratamiento de la miocardiopatía arritmogénica con vectores de genoterapia con aav
WO2025214477A1 (en) 2024-04-12 2025-10-16 Skyline Therapeutics (Shanghai) Co., Ltd. Treatment of genetic cardiomyopathies with aav gene therapy vectors

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WO2017053677A1 (en) * 2015-09-24 2017-03-30 Biomarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024074143A1 (en) * 2022-10-08 2024-04-11 Lingyi Biotech Co., Ltd. Construct for enhancing gene expression
CN115554418A (zh) * 2022-11-22 2023-01-03 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途
CN115554418B (zh) * 2022-11-22 2023-04-14 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途

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EP3794112A1 (en) 2021-03-24
TW202016298A (zh) 2020-05-01
US20240285806A1 (en) 2024-08-29
MX2020012167A (es) 2022-07-13
SG11202010832YA (en) 2020-11-27
JP2021523198A (ja) 2021-09-02
JP2024028696A (ja) 2024-03-05
CA3100000A1 (en) 2019-11-21
BR112020023159A2 (pt) 2021-02-02
WO2019222132A8 (en) 2020-12-03
US20200069819A1 (en) 2020-03-05
AU2019270972A1 (en) 2020-12-03
AR117427A1 (es) 2021-08-04
KR20210008491A (ko) 2021-01-22
WO2019222132A1 (en) 2019-11-21

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Application publication date: 20210226