AU2017250797A1 - Treatment of ocular diseases with fully-human post-translationally modified anti-VEGF fab - Google Patents

Treatment of ocular diseases with fully-human post-translationally modified anti-VEGF fab Download PDF

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Publication number
AU2017250797A1
AU2017250797A1 AU2017250797A AU2017250797A AU2017250797A1 AU 2017250797 A1 AU2017250797 A1 AU 2017250797A1 AU 2017250797 A AU2017250797 A AU 2017250797A AU 2017250797 A AU2017250797 A AU 2017250797A AU 2017250797 A1 AU2017250797 A1 AU 2017250797A1
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AU
Australia
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ser
leu
thr
gly
val
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Abandoned
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AU2017250797A
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English (en)
Inventor
Laura A. CORUZZI
Karen Fran Kozarsky
Rickey Robert REINHARDT
Curran Matthew Simpson
Stephen YOO
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Regenxbio Inc
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Regenxbio Inc
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Publication of AU2017250797A1 publication Critical patent/AU2017250797A1/en
Priority to AU2024205641A priority Critical patent/AU2024205641A1/en
Abandoned legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (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)
AU2017250797A 2016-04-15 2017-04-14 Treatment of ocular diseases with fully-human post-translationally modified anti-VEGF fab Abandoned AU2017250797A1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2024205641A AU2024205641A1 (en) 2016-04-15 2024-08-09 Treatment of ocular diseases with fully-human post-translationally modified anti-VEGF fab

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2024205641A Division AU2024205641A1 (en) 2016-04-15 2024-08-09 Treatment of ocular diseases with fully-human post-translationally modified anti-VEGF fab

Publications (1)

Publication Number Publication Date
AU2017250797A1 true AU2017250797A1 (en) 2018-10-25

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
AU2017250797A Abandoned AU2017250797A1 (en) 2016-04-15 2017-04-14 Treatment of ocular diseases with fully-human post-translationally modified anti-VEGF fab
AU2024205641A Pending AU2024205641A1 (en) 2016-04-15 2024-08-09 Treatment of ocular diseases with fully-human post-translationally modified anti-VEGF fab

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2024205641A Pending AU2024205641A1 (en) 2016-04-15 2024-08-09 Treatment of ocular diseases with fully-human post-translationally modified anti-VEGF fab

Country Status (9)

Country Link
US (3) US20190127455A1 (cg-RX-API-DMAC7.html)
EP (1) EP3442577A4 (cg-RX-API-DMAC7.html)
JP (3) JP2019515027A (cg-RX-API-DMAC7.html)
KR (2) KR20190003556A (cg-RX-API-DMAC7.html)
AU (2) AU2017250797A1 (cg-RX-API-DMAC7.html)
CA (1) CA3019665A1 (cg-RX-API-DMAC7.html)
IL (1) IL262277A (cg-RX-API-DMAC7.html)
SG (2) SG11201808440YA (cg-RX-API-DMAC7.html)
WO (1) WO2017181021A1 (cg-RX-API-DMAC7.html)

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US9840553B2 (en) 2014-06-28 2017-12-12 Kodiak Sciences Inc. Dual PDGF/VEGF antagonists
AU2016381964B2 (en) 2015-12-30 2024-02-15 Kodiak Sciences Inc. Antibodies and conjugates thereof
AU2017248731B2 (en) 2016-04-15 2024-08-29 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
JP7685833B2 (ja) * 2017-09-27 2025-05-30 レジェンクスバイオ インコーポレーテッド 翻訳後修飾された完全ヒト抗ＶＥＧＦ Ｆａｂによる眼疾患の治療
MX2020006435A (es) 2017-12-19 2021-02-09 Akouos Inc Administracion de anticuerpos terapeuticos mediada por vector de virus adenoasociado (aav) en el oido interno.
US20210093734A1 (en) * 2018-02-20 2021-04-01 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-realted macular degeneration
WO2019169341A1 (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
CN118497276A (zh) * 2018-11-07 2024-08-16 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
EP3946467A1 (en) * 2019-04-03 2022-02-09 REGENXBIO Inc. Gene therapy for eye pathologies
CA3137284A1 (en) * 2019-04-24 2020-10-29 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
EP4017523A4 (en) * 2019-08-22 2024-01-17 The Regents Of The University Of California UBE3A FOR THE TREATMENT OF ANGELMAN SYNDROME
CN114502197A (zh) 2019-08-26 2022-05-13 再生生物股份有限公司 用全人经翻译后修饰的抗VEGF Fab治疗糖尿病性视网膜病变
EP4041292A1 (en) 2019-10-07 2022-08-17 RegenxBio Inc. Adeno-associated virus vector pharmaceutical composition and methods
CN114786731A (zh) 2019-10-10 2022-07-22 科达制药股份有限公司 治疗眼部病症的方法
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
MX2023004035A (es) 2020-10-07 2023-07-05 Regenxbio Inc Formulaciones para la administración supracoroidea tales como formulaciones de viscosidad alta.
CA3197342A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Gene therapy for ocular manifestations of cln2 disease
WO2022076595A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as gel formulations
CA3195967A1 (en) * 2020-10-28 2022-05-05 Xu Wang Vectorized anti-tnf-? antibodies for ocular indications
CA3197936A1 (en) 2020-12-01 2022-06-09 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
TW202242118A (zh) * 2020-12-18 2022-11-01 瑞士商Ａｃ 免疫有限公司 抗體遞送
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
EP4314081A4 (en) * 2021-03-31 2025-02-26 Wuxi Biologics Ireland Limited BISPECIFIC ANTI-PD-L1/VEGF ANTIBODY AND USES THEREOF
JP2025512962A (ja) 2022-04-06 2025-04-22 リジェネックスバイオ インコーポレイテッド 上脈絡膜投与のための導入遺伝子をコードする発現カセットを有する組換えアデノ随伴ウイルスベクターを含む医薬組成物
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
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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BR0014945A (pt) * 1999-10-21 2004-08-31 Monsanto Co Modificação pós-traducional de proteìnas recombinantes produzidas em plantas
CN1461342A (zh) * 2000-07-26 2003-12-10 路德维格癌症研究所 糖基化vegf－b和增加可溶性vegf－b量的方法
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US20130090375A1 (en) * 2011-10-06 2013-04-11 Cornell University Virus-mediated delivery of bevacizumab for therapeutic applications
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)
MX365658B (es) * 2014-05-13 2019-06-10 Univ Pennsylvania Composiciones que comprenden virus adenoasociado (aav) que expresa constructos de anticuerpos duales y sus usos.

Also Published As

Publication number Publication date
CA3019665A1 (en) 2017-10-19
US20230057519A1 (en) 2023-02-23
KR20240005973A (ko) 2024-01-12
SG10202008378UA (en) 2020-10-29
EP3442577A4 (en) 2020-03-25
IL262277A (en) 2018-11-29
US20190211091A1 (en) 2019-07-11
WO2017181021A1 (en) 2017-10-19
AU2024205641A1 (en) 2024-09-05
KR20190003556A (ko) 2019-01-09
EP3442577A1 (en) 2019-02-20
US20190127455A1 (en) 2019-05-02
JP2022153418A (ja) 2022-10-12
SG11201808440YA (en) 2018-10-30
NZ746655A (en) 2025-05-02
JP2019515027A (ja) 2019-06-06
JP2025000642A (ja) 2025-01-07

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