JP2022552262A - アデノ随伴ウイルスベクター医薬組成物および方法 - Google Patents

アデノ随伴ウイルスベクター医薬組成物および方法 Download PDF

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JP2022552262A
JP2022552262A JP2022521243A JP2022521243A JP2022552262A JP 2022552262 A JP2022552262 A JP 2022552262A JP 2022521243 A JP2022521243 A JP 2022521243A JP 2022521243 A JP2022521243 A JP 2022521243A JP 2022552262 A JP2022552262 A JP 2022552262A
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aav
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JP2022552262A5 (OSRAM
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トリスタン マーシャル
ジャレッド ビー
クリスティン オベリー
ユ チャン
ロベルト デパズ
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リジェネックスバイオ インコーポレイテッド
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    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • 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
    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • 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
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    • 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
    • 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)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Wood Science & Technology (AREA)
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  • Biophysics (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • 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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2022521243A 2019-10-07 2020-10-06 アデノ随伴ウイルスベクター医薬組成物および方法 Pending JP2022552262A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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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JP2022552262A true JP2022552262A (ja) 2022-12-15
JP2022552262A5 JP2022552262A5 (OSRAM) 2023-10-17

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US (1) US20240108669A1 (OSRAM)
EP (1) EP4041292A1 (OSRAM)
JP (1) JP2022552262A (OSRAM)
KR (1) KR20220081365A (OSRAM)
CN (3) CN114728049A (OSRAM)
AR (1) AR120171A1 (OSRAM)
AU (1) AU2020362119A1 (OSRAM)
BR (1) BR112022006718A2 (OSRAM)
CA (1) CA3156984A1 (OSRAM)
IL (1) IL291930A (OSRAM)
MX (1) MX2022004146A (OSRAM)
TW (2) TW202525312A (OSRAM)
WO (1) WO2021071835A1 (OSRAM)

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MX2023006471A (es) * 2020-12-18 2023-08-11 Sangamo Therapeutics Inc Composiciones farmaceuticas mejoradas que contienen vector viral adenoasociado.
JP2025509566A (ja) * 2022-03-14 2025-04-11 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ａａｖ医薬製品の凍結乾燥製剤
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
EP4593955A1 (en) 2022-09-30 2025-08-06 RegenxBio Inc. Treatment of ocular diseases with recombinant viral vectors encoding anti-vegf fab
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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
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AU2024271004A1 (en) 2023-05-16 2026-01-15 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
TW202508610A (zh) * 2023-08-23 2025-03-01 大陸商上海瑞宏迪醫藥有限公司 醫藥組成物及其用途
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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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US20240108669A1 (en) 2024-04-04
CN114728049A (zh) 2022-07-08
CA3156984A1 (en) 2021-04-15
BR112022006718A2 (pt) 2022-07-12
AR120171A1 (es) 2022-02-02
IL291930A (en) 2022-06-01
CN122005863A (zh) 2026-05-12
MX2022004146A (es) 2022-09-19
WO2021071835A1 (en) 2021-04-15
TWI879814B (zh) 2025-04-11
AU2020362119A1 (en) 2022-05-26
TW202126319A (zh) 2021-07-16
TW202525312A (zh) 2025-07-01
EP4041292A1 (en) 2022-08-17
CN122005864A (zh) 2026-05-12
KR20220081365A (ko) 2022-06-15

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