CN114728049A - 腺相关病毒载体药物组合物和方法 - Google Patents

腺相关病毒载体药物组合物和方法 Download PDF

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Publication number
CN114728049A
CN114728049A CN202080079668.3A CN202080079668A CN114728049A CN 114728049 A CN114728049 A CN 114728049A CN 202080079668 A CN202080079668 A CN 202080079668A CN 114728049 A CN114728049 A CN 114728049A
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months
pharmaceutical composition
aav
time
period
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T·马歇尔
J·比
K·欧贝里
Y·张
R·德帕斯
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Bio Regeneration Co ltd
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Bio Regeneration Co ltd
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A61K47/183—Amino acids, e.g. glycine, EDTA or aspartame
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • A—HUMAN NECESSITIES
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    • 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
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    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61K9/0048—Eye, e.g. artificial tears
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    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
    • C—CHEMISTRY; METALLURGY
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/525—Virus
    • A61K2039/5256—Virus expressing foreign proteins
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    • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
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  • Epidemiology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
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  • Oil, Petroleum & Natural Gas (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Inorganic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CN202080079668.3A 2019-10-07 2020-10-06 腺相关病毒载体药物组合物和方法 Pending CN114728049A (zh)

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US201962911968P 2019-10-07 2019-10-07
US62/911,968 2019-10-07
PCT/US2020/054400 WO2021071835A1 (en) 2019-10-07 2020-10-06 Adeno-associated virus vector pharmaceutical composition and methods

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US (1) US20240108669A1 (https=)
EP (1) EP4041292A1 (https=)
JP (1) JP2022552262A (https=)
KR (1) KR20220081365A (https=)
CN (1) CN114728049A (https=)
AR (1) AR120171A1 (https=)
AU (1) AU2020362119A1 (https=)
BR (1) BR112022006718A2 (https=)
CA (1) CA3156984A1 (https=)
IL (1) IL291930A (https=)
MX (1) MX2022004146A (https=)
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CN115869425A (zh) * 2022-12-12 2023-03-31 北京生物制品研究所有限责任公司 一种aav眼用注射液及其制备方法和应用
WO2025040166A1 (zh) * 2023-08-23 2025-02-27 上海瑞宏迪医药有限公司 药物组合物及其用途
WO2025045082A1 (zh) * 2023-09-01 2025-03-06 康霖生物科技（杭州）有限公司 一种缓冲系统及其相应液体制剂及其用途
TWI877805B (zh) * 2022-11-22 2025-03-21 大陸商四川至善唯新生物科技有限公司 一種重組腺相關病毒載體之醫藥組合物及其用途
CN119769462A (zh) * 2023-10-07 2025-04-08 陈国韬 一种非器质性（心理性）勃起功能障碍大鼠模型的构建方法

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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
US20240066146A1 (en) * 2020-12-18 2024-02-29 Sangamo Therapeutics, Inc. Improved pharmaceutical compositions containing adeno-associated viral vector
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TW202404651A (zh) * 2022-04-06 2024-02-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如形成聚集體之調配物
IL319873A (en) 2022-09-30 2025-05-01 Regenxbio Inc Treatment of eye diseases with recombinant viral vectors containing anti-VEGF FAB
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Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI877805B (zh) * 2022-11-22 2025-03-21 大陸商四川至善唯新生物科技有限公司 一種重組腺相關病毒載體之醫藥組合物及其用途
US12390500B2 (en) 2022-11-22 2025-08-19 Sichuan Real & Best Biotech Co., Ltd. Pharmaceutical composition of a recombinant adeno-associated virus vector and its application
CN115869425A (zh) * 2022-12-12 2023-03-31 北京生物制品研究所有限责任公司 一种aav眼用注射液及其制备方法和应用
CN115869425B (zh) * 2022-12-12 2024-08-20 北京生物制品研究所有限责任公司 一种aav眼用注射液及其制备方法和应用
WO2025040166A1 (zh) * 2023-08-23 2025-02-27 上海瑞宏迪医药有限公司 药物组合物及其用途
WO2025045082A1 (zh) * 2023-09-01 2025-03-06 康霖生物科技（杭州）有限公司 一种缓冲系统及其相应液体制剂及其用途
CN119769462A (zh) * 2023-10-07 2025-04-08 陈国韬 一种非器质性（心理性）勃起功能障碍大鼠模型的构建方法

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JP2022552262A (ja) 2022-12-15
US20240108669A1 (en) 2024-04-04
CA3156984A1 (en) 2021-04-15
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