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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thr
gly
val
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Abandoned
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AU2017250797A
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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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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/505Medicinal preparations containing antigens or antibodies comprising antibodies
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/20Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/40Immunoglobulins specific features characterized by post-translational modification
    • C07K2317/41Glycosylation, sialylation, or fucosylation
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/50Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/55Fab or Fab'
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/60Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622Single chain antibody (scFv)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/42Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/20Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203Vectors 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)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (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 (https=)
EP (1) EP3442577A4 (https=)
JP (3) JP2019515027A (https=)
KR (2) KR20240005973A (https=)
AU (2) AU2017250797A1 (https=)
CA (1) CA3019665A1 (https=)
IL (1) IL262277A (https=)
SG (2) SG10202008378UA (https=)
WO (1) WO2017181021A1 (https=)

Cited By (1)

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Publication number Priority date Publication date Assignee Title
EP4493596A4 (en) * 2022-03-17 2026-04-01 Adverum Biotechnologies Inc Antibody Cassettes of Constituent 3 Anti-Complement, Vectorization and Therapeutic Application

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US9840553B2 (en) 2014-06-28 2017-12-12 Kodiak Sciences Inc. Dual PDGF/VEGF antagonists
KR20250057128A (ko) 2015-12-30 2025-04-28 코디악 사이언시스 인코포레이티드 항체 및 이의 접합체
SG11201808426XA (en) 2016-04-15 2018-10-30 Univ Pennsylvania Compositions for treatment of wet age-related macular degeneration
EP3687464A4 (en) * 2017-09-27 2021-09-29 REGENXBIO Inc. TREATMENT OF EYE DISEASES WITH A TOTALLY HUMAN POST-TRANSLATION MODIFIED ANTI-VEGF FAB
EP3727468A4 (en) * 2017-12-19 2021-09-22 Akouos, Inc. AAV-MEDIATED THERAPEUTIC ANTIBODIES DELIVERY TO THE INTERNAL 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
EP3758737A4 (en) 2018-03-02 2022-10-12 Kodiak Sciences Inc. IL-6 ANTIBODIES AND FUSION CONSTRUCTS AND CONJUGATES THEREOF
BR112020025268A2 (pt) * 2018-06-28 2021-03-09 The University Of North Carolina At Chapel Hill Cassetes de expressão e genes cln5 otimizados e seu uso
EP4063510A1 (en) * 2018-11-07 2022-09-28 Akouos, Inc. Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear
WO2020206098A1 (en) * 2019-04-03 2020-10-08 Regenxbio Inc. Gene therapy for eye pathologies
WO2020219868A1 (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
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
CA3198368A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
CA3198372A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
KR20230106598A (ko) 2020-10-07 2023-07-13 리젠엑스바이오 인크. 겔 제형과 같은 맥락막위 투여용 제형
AU2021358781A1 (en) 2020-10-07 2023-05-18 Tern Therapeutics, Llc Gene therapy for ocular manifestations of cln2 disease
CA3195967A1 (en) * 2020-10-28 2022-05-05 Xu Wang Vectorized anti-tnf-? 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
CN116917491A (zh) * 2020-12-18 2023-10-20 Ac免疫有限公司 抗体递送
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
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
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
US20260034243A1 (en) 2022-04-06 2026-02-05 Regenxbio Inc. Pharmaceutical composition comprising a recombinant adeno-associated virus vector with an expression cassette encoding a transgene for suprachoroidal administration
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
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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Cited By (1)

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Publication number Priority date Publication date Assignee Title
EP4493596A4 (en) * 2022-03-17 2026-04-01 Adverum Biotechnologies Inc Antibody Cassettes of Constituent 3 Anti-Complement, Vectorization and Therapeutic Application

Also Published As

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

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