CN117531025A - 使用aav2变体和阿柏西普治疗amd - Google Patents

使用aav2变体和阿柏西普治疗amd Download PDF

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Publication number
CN117531025A
CN117531025A CN202311530329.4A CN202311530329A CN117531025A CN 117531025 A CN117531025 A CN 117531025A CN 202311530329 A CN202311530329 A CN 202311530329A CN 117531025 A CN117531025 A CN 117531025A
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fold
aflibercept
pharmaceutical composition
nucleic acid
acid sequence
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CN202311530329.4A
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Chinese (zh)
Inventor
M·布鲁门克兰兹
M·盖斯米
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Adverum Biotechnologies Inc
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Adverum Biotechnologies Inc
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Publication of CN117531025A publication Critical patent/CN117531025A/zh
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    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/179—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M5/00—Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular way; Accessories therefor, e.g. filling or cleaning devices, arm-rests
    • A61M5/178—Syringes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dermatology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Vascular Medicine (AREA)
  • Dispersion Chemistry (AREA)
  • Hematology (AREA)
CN202311530329.4A 2016-06-16 2017-06-16 使用aav2变体和阿柏西普治疗amd Pending CN117531025A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662351234P 2016-06-16 2016-06-16
US62/351,234 2016-06-16
PCT/US2017/038003 WO2017218974A2 (en) 2016-06-16 2017-06-16 Treatment of amd using aav2 variant with aflibercept
CN201780050283.2A CN109641065A (zh) 2016-06-16 2017-06-16 使用aav2变体和阿柏西普治疗amd

Related Parent Applications (1)

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CN201780050283.2A Division CN109641065A (zh) 2016-06-16 2017-06-16 使用aav2变体和阿柏西普治疗amd

Publications (1)

Publication Number Publication Date
CN117531025A true CN117531025A (zh) 2024-02-09

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CN202311530329.4A Pending CN117531025A (zh) 2016-06-16 2017-06-16 使用aav2变体和阿柏西普治疗amd
CN201780050283.2A Pending CN109641065A (zh) 2016-06-16 2017-06-16 使用aav2变体和阿柏西普治疗amd

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US (2) US20190151409A1 (https=)
EP (3) EP3795181B1 (https=)
JP (5) JP6814822B2 (https=)
KR (2) KR102218265B1 (https=)
CN (2) CN117531025A (https=)
AU (3) AU2017286673B2 (https=)
BR (1) BR112018076124A2 (https=)
CA (1) CA3027737A1 (https=)
CY (1) CY1123697T1 (https=)
DK (2) DK3471780T3 (https=)
ES (2) ES2840059T3 (https=)
FI (1) FI3795181T3 (https=)
HR (2) HRP20251248T1 (https=)
HU (1) HUE051953T2 (https=)
IL (3) IL279919B2 (https=)
LT (2) LT3471780T (https=)
MX (2) MX391779B (https=)
PL (1) PL3795181T3 (https=)
PT (2) PT3471780T (https=)
RS (2) RS61311B1 (https=)
SG (1) SG11201811232XA (https=)
SI (2) SI3795181T1 (https=)
SM (2) SMT202500400T1 (https=)
WO (1) WO2017218974A2 (https=)
ZA (1) ZA201808538B (https=)

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MY187898A (en) 2015-03-02 2021-10-27 Adverum Biotechnologies Inc Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
DK3471780T3 (da) * 2016-06-16 2020-11-23 Adverum Biotechnologies Inc Behandling af amd med aav2-variant med aflibercept
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CN107661492A (zh) * 2017-08-31 2018-02-06 中山大学中山眼科中心 Vegf‑b的新用途
CN107537026A (zh) * 2017-08-31 2018-01-05 中山大学中山眼科中心 Vegf‑b的应用
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CN113952473A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
JP7842082B2 (ja) * 2020-07-21 2026-04-07 エフティージェン コーポレーション 眼疾患を処置するための組成物および方法
CN113952474A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
CN113817775B (zh) * 2020-08-25 2023-02-17 南京吉迈生物技术有限公司 修饰的阿柏西普、组合物、方法及其在基因治疗中的应用
BR112023003548A2 (pt) * 2020-09-03 2023-04-04 Univ Massachusetts Vírus adenoassociado para liberação de kh902 (conbercept) e usos do mesmo
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
JP2021049374A (ja) * 2020-12-09 2021-04-01 株式会社三洋物産 遊技機
JP2024515459A (ja) * 2021-04-27 2024-04-10 アドヴェラム バイオテクノロジーズ， インコーポレイテッド アフリベルセプトをコードするａａｖ２バリアントを使用する眼疾患の治療方法
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DK3471780T3 (da) * 2016-06-16 2020-11-23 Adverum Biotechnologies Inc Behandling af amd med aav2-variant med aflibercept

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SG11201811232XA (en) 2019-01-30
ES3049818T3 (en) 2025-12-18
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MX2022004786A (es) 2022-05-16
CY1123697T1 (el) 2022-03-24
HRP20201842T1 (hr) 2021-04-16
KR20210021113A (ko) 2021-02-24
IL314568B2 (en) 2025-09-01
WO2017218974A3 (en) 2018-01-25
IL279919A (en) 2021-03-01
SMT202500400T1 (it) 2025-11-10
JP2022137244A (ja) 2022-09-21
JP2019521990A (ja) 2019-08-08
CA3027737A1 (en) 2017-12-21
FI3795181T3 (fi) 2025-10-14
AU2017286673A1 (en) 2019-01-31
BR112018076124A2 (pt) 2019-03-26
JP2025186377A (ja) 2025-12-23
HRP20251248T1 (hr) 2026-01-16
IL279919B2 (en) 2025-01-01
SI3795181T1 (sl) 2025-11-28
EP3471780A4 (en) 2019-04-24
AU2025202930A1 (en) 2025-05-15
DK3471780T3 (da) 2020-11-23
US20220265740A1 (en) 2022-08-25
EP4671369A2 (en) 2025-12-31
ZA201808538B (en) 2021-04-28
IL279919B1 (en) 2024-09-01
SI3471780T1 (sl) 2021-03-31
JP6814822B2 (ja) 2021-01-20
MX2018015512A (es) 2019-04-24
DK3795181T3 (da) 2025-10-13
IL314568A (en) 2024-09-01
EP3471780B1 (en) 2020-10-28
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EP3795181A1 (en) 2021-03-24
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JP2024113193A (ja) 2024-08-21
KR102218265B1 (ko) 2021-02-25
PT3471780T (pt) 2020-12-17
AU2021225178B2 (en) 2025-01-30
CN109641065A (zh) 2019-04-16
WO2017218974A2 (en) 2017-12-21
HUE051953T2 (hu) 2021-04-28
PT3795181T (pt) 2025-11-05
IL263686A (en) 2019-01-31
LT3471780T (lt) 2021-01-25
IL263686B (en) 2021-02-28
ES2840059T3 (es) 2021-07-06
RS67328B1 (sr) 2025-11-28
KR20190037228A (ko) 2019-04-05
AU2021225178A1 (en) 2021-09-30
EP4671369A3 (en) 2026-03-18
AU2017286673B2 (en) 2021-06-24
PL3795181T3 (pl) 2025-12-22
IL314568B1 (en) 2025-05-01
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US20190151409A1 (en) 2019-05-23
LT3795181T (lt) 2025-12-29
MX391779B (es) 2025-03-21

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