JP2019515027A - 翻訳後修飾された完全ヒト抗ＶＥＧＦ Ｆａｂを用いる眼疾患の治療 - Google Patents

翻訳後修飾された完全ヒト抗ＶＥＧＦ Ｆａｂを用いる眼疾患の治療 Download PDF

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JP2019515027A
JP2019515027A JP2019505138A JP2019505138A JP2019515027A JP 2019515027 A JP2019515027 A JP 2019515027A JP 2019505138 A JP2019505138 A JP 2019505138A JP 2019505138 A JP2019505138 A JP 2019505138A JP 2019515027 A JP2019515027 A JP 2019515027A
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binding fragment
antigen binding
vegf
vector
human
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JP2019515027A5 (enExample
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マシュー シンプソン クラン
マシュー シンプソン クラン
ヨー ステファン
ヨー ステファン
フラン コザルスキー カレン
フラン コザルスキー カレン
ロバート ラインハルト リッキー
ロバート ラインハルト リッキー
エー． コラジー ローラ
エー． コラジー ローラ
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レジェンクスバイオ インコーポレーテッド
レジェンクスバイオ インコーポレーテッド
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Publication of JP2019515027A publication Critical patent/JP2019515027A/ja
Publication of JP2019515027A5 publication Critical patent/JP2019515027A5/ja
Priority to JP2022108815A priority Critical patent/JP2022153418A/ja
Priority to JP2024152809A priority patent/JP2025000642A/ja
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    • 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
    • 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
    • A61K9/0048—Eye, e.g. artificial tears
    • 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
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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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/40—Immunoglobulins specific features characterized by post-translational modification
    • C07K2317/41—Glycosylation, sialylation, or fucosylation
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622—Single chain antibody (scFv)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

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  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2019505138A 2016-04-15 2017-04-14 翻訳後修飾された完全ヒト抗ＶＥＧＦ Ｆａｂを用いる眼疾患の治療 Pending JP2019515027A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022108815A JP2022153418A (ja) 2016-04-15 2022-07-06 翻訳後修飾された完全ヒト抗ＶＥＧＦ Ｆａｂを用いる眼疾患の治療
JP2024152809A JP2025000642A (ja) 2016-04-15 2024-09-05 翻訳後修飾された完全ヒト抗ＶＥＧＦ Ｆａｂを用いる眼疾患の治療

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201662323285P 2016-04-15 2016-04-15
US62/323,285 2016-04-15
US201762442802P 2017-01-05 2017-01-05
US62/442,802 2017-01-05
US201762450438P 2017-01-25 2017-01-25
US62/450,438 2017-01-25
US201762460428P 2017-02-17 2017-02-17
US62/460,428 2017-02-17
PCT/US2017/027650 WO2017181021A1 (en) 2016-04-15 2017-04-14 Treatment of ocular diseases with fully-human post-translationally modified anti-vegf fab

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JP2022108815A Division JP2022153418A (ja) 2016-04-15 2022-07-06 翻訳後修飾された完全ヒト抗ＶＥＧＦ Ｆａｂを用いる眼疾患の治療

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JP2019515027A true JP2019515027A (ja) 2019-06-06
JP2019515027A5 JP2019515027A5 (enExample) 2020-05-28

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JP2019505138A Pending JP2019515027A (ja) 2016-04-15 2017-04-14 翻訳後修飾された完全ヒト抗ＶＥＧＦ Ｆａｂを用いる眼疾患の治療
JP2022108815A Pending JP2022153418A (ja) 2016-04-15 2022-07-06 翻訳後修飾された完全ヒト抗ＶＥＧＦ Ｆａｂを用いる眼疾患の治療
JP2024152809A Pending JP2025000642A (ja) 2016-04-15 2024-09-05 翻訳後修飾された完全ヒト抗ＶＥＧＦ Ｆａｂを用いる眼疾患の治療

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JP2022108815A Pending JP2022153418A (ja) 2016-04-15 2022-07-06 翻訳後修飾された完全ヒト抗ＶＥＧＦ Ｆａｂを用いる眼疾患の治療
JP2024152809A Pending JP2025000642A (ja) 2016-04-15 2024-09-05 翻訳後修飾された完全ヒト抗ＶＥＧＦ Ｆａｂを用いる眼疾患の治療

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US (3) US20190127455A1 (enExample)
EP (1) EP3442577A4 (enExample)
JP (3) JP2019515027A (enExample)
KR (2) KR20240005973A (enExample)
AU (2) AU2017250797A1 (enExample)
CA (1) CA3019665A1 (enExample)
IL (1) IL262277A (enExample)
SG (2) SG10202008378UA (enExample)
WO (1) WO2017181021A1 (enExample)

Cited By (3)

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JP2022545184A (ja) * 2019-08-22 2022-10-26 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アンジェルマン症候群の処置のためのｕｂｅ３ａ
JP2022545967A (ja) * 2019-08-26 2022-11-01 リジェネックスバイオ インコーポレイテッド 完全ヒト翻訳後修飾抗ＶＥＧＦ Ｆａｂを用いる糖尿病性網膜症の処置
JP2024513205A (ja) * 2021-03-31 2024-03-22 ウーシー バイオロジクス アイルランド リミテッド 二重特異性抗ｐｄ－ｌ１／ｖｅｇｆ抗体およびその使用

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IL262207B2 (en) 2016-04-15 2026-01-01 Univ Pennsylvania Preparations for the treatment of wet age-related macular degeneration
CA3076905A1 (en) * 2017-09-27 2019-04-04 The Johns Hopkins University Treatment of ocular diseases with fully-human post-translationally modified anti-vegf fab
CA3086045A1 (en) * 2017-12-19 2019-06-27 Akouos, Inc. Aav-mediated delivery of therapeutic antibodies to the inner ear
GB201800620D0 (en) 2018-01-15 2018-02-28 Univ Manchester C3b Binding Polypeptide
US12558434B2 (en) * 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
CA3092588A1 (en) 2018-03-02 2019-09-06 Kodiak Sciences Inc. Il-6 antibodies and fusion constructs and conjugates thereof
IL313246B2 (en) * 2018-06-28 2025-10-01 Univ North Carolina Chapel Hill Optimal cln5 expression genes and cassettes and their use
CN113853437A (zh) * 2018-11-07 2021-12-28 阿库斯股份有限公司 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途
EP4667578A3 (en) * 2019-04-03 2026-04-01 REGENXBIO Inc. Gene therapy for eye pathologies
US20220195462A1 (en) * 2019-04-24 2022-06-23 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
WO2021071835A1 (en) 2019-10-07 2021-04-15 Regenxbio Inc. Adeno-associated virus vector pharmaceutical composition and methods
CA3157509A1 (en) 2019-10-10 2021-04-15 Kodiak Sciences Inc. Methods of treating an eye disorder
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
EP4225381A1 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
TW202228648A (zh) 2020-10-07 2022-08-01 美商銳進科斯生物股份有限公司 諸如高黏度調配物之用於脈絡膜上投與之調配物
JP2023544803A (ja) 2020-10-07 2023-10-25 レジェンクスバイオ インコーポレーテッド Ｃｌｎ２疾患の眼症状に対する遺伝子療法
TW202228646A (zh) 2020-10-07 2022-08-01 美商銳進科斯生物股份有限公司 諸如凝膠調配物之用於脈絡膜上投與之調配物
AU2021371307A1 (en) * 2020-10-28 2023-06-01 Regenxbio, Inc. VECTORIZED ANTI-TNF-α ANTIBODIES FOR OCULAR INDICATIONS
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
JP2024500115A (ja) * 2020-12-18 2024-01-04 エイシー イミューン ソシエテ アノニム 抗体デリバリー
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
US20260043046A1 (en) * 2022-03-17 2026-02-12 Adverum Biotechnologies, Inc. Cassettes of anti-complement component 3 antibody, vectorization and theraputic application
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
TW202345913A (zh) 2022-04-06 2023-12-01 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凝膠調配物
AU2023250660A1 (en) 2022-04-06 2024-10-24 Regenxbio Inc. Pharmaceutical composition comprising a recombinant adenoassociated virus vector with an expression cassette encoding a transgene for suprachoroidal administration
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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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022545184A (ja) * 2019-08-22 2022-10-26 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アンジェルマン症候群の処置のためのｕｂｅ３ａ
JP2022545967A (ja) * 2019-08-26 2022-11-01 リジェネックスバイオ インコーポレイテッド 完全ヒト翻訳後修飾抗ＶＥＧＦ Ｆａｂを用いる糖尿病性網膜症の処置
JP2024513205A (ja) * 2021-03-31 2024-03-22 ウーシー バイオロジクス アイルランド リミテッド 二重特異性抗ｐｄ－ｌ１／ｖｅｇｆ抗体およびその使用

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CA3019665A1 (en) 2017-10-19
JP2025000642A (ja) 2025-01-07
SG10202008378UA (en) 2020-10-29
AU2017250797A1 (en) 2018-10-25
US20190127455A1 (en) 2019-05-02
KR20240005973A (ko) 2024-01-12
KR20190003556A (ko) 2019-01-09
IL262277A (en) 2018-11-29
JP2022153418A (ja) 2022-10-12
NZ746655A (en) 2025-05-02
EP3442577A4 (en) 2020-03-25
AU2024205641A1 (en) 2024-09-05
WO2017181021A1 (en) 2017-10-19
EP3442577A1 (en) 2019-02-20
US20230057519A1 (en) 2023-02-23
US20190211091A1 (en) 2019-07-11

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