JP2021516671A - 滲出型加齢性黄斑変性の治療のための組成物 - Google Patents

滲出型加齢性黄斑変性の治療のための組成物 Download PDF

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JP2021516671A
JP2021516671A JP2020543914A JP2020543914A JP2021516671A JP 2021516671 A JP2021516671 A JP 2021516671A JP 2020543914 A JP2020543914 A JP 2020543914A JP 2020543914 A JP2020543914 A JP 2020543914A JP 2021516671 A JP2021516671 A JP 2021516671A
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itr
vector
eye
vegf
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JPWO2019164854A5 (enExample
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ウイルソン，ジェームス・エム
ユー，スティーブン
バン・エベレン，シェリ
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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
リジェネックスバイオ・インコーポレーテッド
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Publication of JP2021516671A publication Critical patent/JP2021516671A/ja
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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
    • A61K48/005—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
    • A61K48/0075—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
    • 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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    • 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
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07—ORGANIC CHEMISTRY
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    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
    • 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
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/203—Animal model comprising inducible/conditional expression system, e.g. hormones, tet
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/55—Fab or Fab'
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Dermatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dispersion Chemistry (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2020543914A 2018-02-20 2019-02-19 滲出型加齢性黄斑変性の治療のための組成物 Pending JP2021516671A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023191314A JP2024016207A (ja) 2018-02-20 2023-11-09 滲出型加齢性黄斑変性の治療のための組成物

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862632775P 2018-02-20 2018-02-20
US62/632,775 2018-02-20
US201862663532P 2018-04-27 2018-04-27
US62/663,532 2018-04-27
PCT/US2019/018640 WO2019164854A1 (en) 2018-02-20 2019-02-19 Compositions for treatment of wet age-related macular degeneration

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JP2023191314A Division JP2024016207A (ja) 2018-02-20 2023-11-09 滲出型加齢性黄斑変性の治療のための組成物

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JP2021516671A true JP2021516671A (ja) 2021-07-08
JPWO2019164854A5 JPWO2019164854A5 (enExample) 2022-02-24

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JP2020543914A Pending JP2021516671A (ja) 2018-02-20 2019-02-19 滲出型加齢性黄斑変性の治療のための組成物
JP2023191314A Pending JP2024016207A (ja) 2018-02-20 2023-11-09 滲出型加齢性黄斑変性の治療のための組成物

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US (1) US20210093734A1 (enExample)
EP (1) EP3755804A4 (enExample)
JP (2) JP2021516671A (enExample)
KR (1) KR20210022524A (enExample)
AU (2) AU2019223963A1 (enExample)
CA (1) CA3090293A1 (enExample)
IL (1) IL276777B1 (enExample)
MA (1) MA51916A (enExample)
SG (1) SG11202007252VA (enExample)
WO (1) WO2019164854A1 (enExample)

Families Citing this family (17)

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EP3568774A1 (en) * 2017-01-12 2019-11-20 Telefonaktiebolaget LM Ericsson (PUBL) Anomaly detection of media event sequences
KR20200060456A (ko) * 2017-09-27 2020-05-29 리젠엑스바이오 인크. 완전-인간 번역후 변형된 항-VEGF Fab를 이용한 안구 질환의 치료
JP2021506861A (ja) 2017-12-19 2021-02-22 アコーオス インコーポレイテッド 内耳への治療用抗体のａａｖ媒介送達
US11360969B2 (en) * 2019-03-20 2022-06-14 Promethium, Inc. Natural language based processing of data stored across heterogeneous data sources
WO2021046245A1 (en) * 2019-09-04 2021-03-11 University Of Massachusetts Adeno-associated virus vector platform for delivery of kh902 (conbercept) and uses thereof
US20230139443A1 (en) * 2020-02-28 2023-05-04 The Trustees Of The University Of Pennsylvania Treating autosomal dominant bestrophinopathies and methods for evaluating same
US20230152336A1 (en) * 2020-04-24 2023-05-18 Siemens Healthcare Diagnostics Inc. Calibration and quality control reagents for use with immunoassays for antibodies and methods of production and use thereof
WO2021255590A1 (en) * 2020-06-16 2021-12-23 Intas Pharmaceuticals Ltd. An anti-vegf scfv adeno-associated virus (aav) vector and uses thereof
WO2021255589A1 (en) * 2020-06-16 2021-12-23 Intas Pharmaceuticals Ltd. An anti-vegf scfab adeno-associated virus (aav) vector and uses thereof
AU2021362770A1 (en) 2020-10-16 2022-12-08 Gyroscope Therapeutics Limited Nucleic acid encoding an anti-VEGF entity and a negative complement regulator and uses thereof for the treatment of age-related macular degeneration
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
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
US20240335562A1 (en) * 2021-08-04 2024-10-10 Alpine Biotherapeutics Corporation Sustainable ocular cell-mediated intraocular delivery of cellular therapeutics for treatment of ocular diseases or disorders
CN114181972A (zh) * 2021-11-23 2022-03-15 上海本导基因技术有限公司 适用于难治性血管新生性眼疾病基因治疗的慢病毒载体
KR102789747B1 (ko) * 2021-12-29 2025-04-03 한국과학기술원 Prox1 단백질에 중화 활성을 갖는 결합 분자
AU2023215253A1 (en) * 2022-02-02 2024-09-19 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
WO2024238859A1 (en) * 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof

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WO2017180936A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration

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TWI775096B (zh) * 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
AU2017250797A1 (en) * 2016-04-15 2018-10-25 Regenxbio Inc. Treatment of ocular diseases with fully-human post-translationally modified anti-VEGF fab

Patent Citations (1)

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WO2017180936A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration

Non-Patent Citations (1)

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MOLECULAR THERAPY, 2018.02.07, VOL.26, NO.2, PP.542-549, JPN6022048791, ISSN: 0005126565 *

Also Published As

Publication number Publication date
IL276777B1 (en) 2025-12-01
MA51916A (fr) 2021-05-26
SG11202007252VA (en) 2020-09-29
EP3755804A1 (en) 2020-12-30
AU2019223963A1 (en) 2020-08-13
KR20210022524A (ko) 2021-03-03
WO2019164854A1 (en) 2019-08-29
US20210093734A1 (en) 2021-04-01
JP2024016207A (ja) 2024-02-06
IL276777A (en) 2020-10-29
AU2025204780A1 (en) 2025-07-17
CA3090293A1 (en) 2019-08-29
EP3755804A4 (en) 2021-12-15

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