JP7597380B2 - 対側眼へのａａｖ遺伝子治療の逐次的硝子体内投与 - Google Patents

対側眼へのａａｖ遺伝子治療の逐次的硝子体内投与 Download PDF

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JP7597380B2
JP7597380B2 JP2021551770A JP2021551770A JP7597380B2 JP 7597380 B2 JP7597380 B2 JP 7597380B2 JP 2021551770 A JP2021551770 A JP 2021551770A JP 2021551770 A JP2021551770 A JP 2021551770A JP 7597380 B2 JP7597380 B2 JP 7597380B2
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ダイアナ セペダ，
メヒディ ガスミ，
アナヒタ ケラヴァラ，
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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
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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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083—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 administration regime
    • 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
    • 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
    • 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
    • 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
    • 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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  • Animal Behavior & Ethology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
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  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2021551770A 2019-03-04 2020-03-04 対側眼へのａａｖ遺伝子治療の逐次的硝子体内投与 Active JP7597380B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962813597P 2019-03-04 2019-03-04
US62/813,597 2019-03-04
US201962839457P 2019-04-26 2019-04-26
US62/839,457 2019-04-26
PCT/US2020/020929 WO2020180951A1 (en) 2019-03-04 2020-03-04 Sequential intravitreal administration of aav gene therapy to contralateral eyes

Publications (3)

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JP2022522776A JP2022522776A (ja) 2022-04-20
JP2022522776A5 JP2022522776A5 (enExample) 2023-03-13
JP7597380B2 true JP7597380B2 (ja) 2024-12-10

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JP2021551770A Active JP7597380B2 (ja) 2019-03-04 2020-03-04 対側眼へのａａｖ遺伝子治療の逐次的硝子体内投与

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US (1) US20200297869A1 (enExample)
EP (1) EP3934698A1 (enExample)
JP (1) JP7597380B2 (enExample)
KR (1) KR20210135267A (enExample)
AU (1) AU2020231505A1 (enExample)
WO (1) WO2020180951A1 (enExample)

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PE20240329A1 (es) 2019-06-05 2024-02-27 Regeneron Pharma Dispositivos y metodos para administracion de dosis de precision
CN114250227B (zh) * 2020-09-25 2026-04-03 北京安龙生物医药有限公司 用于高水平表达外源基因的表达载体
AU2021378270A1 (en) * 2020-11-10 2023-06-15 Avirmax Biopharma Inc. Engineered viral capsids and methods of use
AU2022253065A1 (en) * 2021-04-09 2023-09-28 Avirmax Biopharma Inc. Compositions and methods for ocular transgene expression
CN117222400A (zh) * 2021-04-27 2023-12-12 阿德夫拉姆生物技术股份有限公司 使用编码阿柏西普的aav2变体治疗眼部疾病的方法
US12649031B2 (en) 2021-09-08 2026-06-09 Regeneron Pharmaceuticals, Inc. Methods for delivering agents with pre-filled syringes to minimize intraocular inflammation
KR20240152351A (ko) * 2022-02-17 2024-10-21 스카이라인 테라퓨틱스 리미티드 변형된 aav 캡시드 폴리펩티드를 갖는 재조합 아데노-연관 바이러스
CA3252767A1 (en) 2022-06-07 2023-12-14 Adverum Biotechnologies, Inc. VARIANTS OF MELANOPSINE FOR VISION RESTORATION
USD1120314S1 (en) 2022-11-30 2026-03-24 Regeneron Pharmaceuticals, Inc. Dose delivery device
WO2024168252A2 (en) * 2023-02-10 2024-08-15 Yale University Use of inhibitors of vegf-c signaling for inhibiting immune responses and increasing gene therapy efficacy

Citations (1)

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WO2017190125A1 (en) 2016-04-29 2017-11-02 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus

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WO2002053703A2 (en) * 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9725485B2 (en) * 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
TWI698240B (zh) 2012-05-15 2020-07-11 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)
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
FI3265178T3 (fi) * 2015-03-02 2026-08-03 Adverum Biotechnologies Inc Koostumuksia ja menetelmiä polynukleotidien viemiseksi intravitreaalisesti retinaalisiin tappisoluihin
CN117531025A (zh) * 2016-06-16 2024-02-09 阿德夫拉姆生物技术股份有限公司 使用aav2变体和阿柏西普治疗amd
AU2017345470B2 (en) * 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
JP2020510424A (ja) * 2017-02-28 2020-04-09 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 改変ａａｖカプシドおよびその使用
KR102616820B1 (ko) 2017-03-17 2023-12-21 애드베룸 바이오테크놀로지스, 인코포레이티드 향상된 유전자 발현을 위한 조성물 및 방법

Patent Citations (1)

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Publication number Priority date Publication date Assignee Title
WO2017190125A1 (en) 2016-04-29 2017-11-02 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus

Non-Patent Citations (1)

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GRISHANIN, R. et al.，Mol Ther，2019年01月，Vol. 27, No. 1，pp. 118-129

Also Published As

Publication number Publication date
JP2022522776A (ja) 2022-04-20
EP3934698A1 (en) 2022-01-12
WO2020180951A1 (en) 2020-09-10
AU2020231505A1 (en) 2021-08-19
KR20210135267A (ko) 2021-11-12
US20200297869A1 (en) 2020-09-24
AU2020231505A8 (en) 2021-09-02

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