CA3019665A1 - 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
CA3019665A1
CA3019665A1 CA3019665A CA3019665A CA3019665A1 CA 3019665 A1 CA3019665 A1 CA 3019665A1 CA 3019665 A CA3019665 A CA 3019665A CA 3019665 A CA3019665 A CA 3019665A CA 3019665 A1 CA3019665 A1 CA 3019665A1
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CA
Canada
Prior art keywords
antigen
binding fragment
vegf
certain embodiments
human
Prior art date
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Pending
Application number
CA3019665A
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English (en)
French (fr)
Inventor
Curran Matthew Simpson
Stephen YOO
Karen Fran Kozarsky
Rickey Robert REINHARDT
Laura A. CORUZZI
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Regenxbio Inc
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Regenxbio Inc
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Application filed by Regenxbio Inc filed Critical Regenxbio Inc
Publication of CA3019665A1 publication Critical patent/CA3019665A1/en
Pending legal-status Critical Current

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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
    • 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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Virology (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)
CA3019665A 2016-04-15 2017-04-14 Treatment of ocular diseases with fully-human post-translationally modified anti-vegf fab Pending CA3019665A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3019665A1 true CA3019665A1 (en) 2017-10-19

Family

ID=60041925

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3019665A Pending CA3019665A1 (en) 2016-04-15 2017-04-14 Treatment of ocular diseases with fully-human post-translationally modified anti-vegf fab

Country Status (9)

Country Link
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) SG11201808440YA (enExample)
WO (1) WO2017181021A1 (enExample)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2017180936A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Compositions for 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
BR112020012336A2 (pt) * 2017-12-19 2021-03-30 Akouos, Inc. Administração de anticorpos terapêuticos mediada por aav para o ouvido interno
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
US12071476B2 (en) 2018-03-02 2024-08-27 Kodiak Sciences Inc. IL-6 antibodies and fusion constructs and conjugates thereof
IL279131B2 (en) * 2018-06-28 2025-06-01 Univ North Carolina Chapel Hill Optimized cln5 genes and expression cassettes and their use
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
MX2021011889A (es) 2019-04-03 2021-10-26 Regenxbio Inc Tratamiento genico para patologias oculares.
TW202106708A (zh) * 2019-04-24 2021-02-16 美商銳進科斯生物股份有限公司 全人類轉譯後修飾之抗體治療劑
US20220305098A1 (en) * 2019-08-22 2022-09-29 The Regents Of The University Of California Ube3a for the treatment of angelman syndrome
BR112022003811A2 (pt) * 2019-08-26 2022-11-16 Regenxbio Inc Tratamento de retinopatia diabética com fab anti vegf totalmente humano modificado pós tradução
CN114728049A (zh) 2019-10-07 2022-07-08 再生生物股份有限公司 腺相关病毒载体药物组合物和方法
AU2020364071A1 (en) 2019-10-10 2022-05-26 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
IL301947A (en) 2020-10-07 2023-06-01 Regenxbio Inc Formulations for suprachoroidal administration such as gel formulations
CA3198368A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
US20230364206A1 (en) 2020-10-07 2023-11-16 Regenxbio Inc. Gene therapy for ocular manifestations of cln2 disease
AU2021358048A1 (en) 2020-10-07 2023-05-25 Regenxbio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
JP2023547832A (ja) * 2020-10-28 2023-11-14 リジェネックスバイオ インコーポレイテッド 眼の適応症に対するベクター化抗ＴＮＦ－α抗体
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
WO2022129609A2 (en) * 2020-12-18 2022-06-23 Ac Immune Sa Antibody delivery
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2022206843A1 (en) * 2021-03-31 2022-10-06 Wuxi Biologics (Shanghai) Co., Ltd. A bispecific anti-pd-l1/vegf antibody and uses thereof
EP4493596A4 (en) * 2022-03-17 2026-04-01 Adverum Biotechnologies Inc Antibody Cassettes of Constituent 3 Anti-Complement, Vectorization and Therapeutic Application
CA3247507A1 (en) 2022-04-06 2023-10-12 Regenxbio Inc. PHARMACEUTICAL COMPOSITION COMPRISING A RECOMMENDED ADENO-ASSOCIATED VIRUS (AAV) VECTOR WITH AN EXPRESSION CASSETTE ENCODED BY A TRANSGENE FOR SUPRACHOROID ADMINISTRATION
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
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
EP4593955A1 (en) 2022-09-30 2025-08-06 RegenxBio Inc. Treatment of ocular diseases with recombinant viral vectors encoding anti-vegf fab

Family Cites Families (6)

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Publication number Priority date Publication date Assignee Title
CA2388432A1 (en) * 1999-10-21 2001-04-26 Monsanto Company Post-translational modification of recombinant proteins produced in plants
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
BRPI1012321A8 (pt) * 2009-06-17 2016-09-27 Abbott Biotherapeutics Corp Anticorpos anti-vegf e seus usos
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)
MY186389A (en) * 2014-05-13 2021-07-22 Univ Pennsylvania Compositions comprising aav expressing dual antibody constructs and uses thereof

Also Published As

Publication number Publication date
SG11201808440YA (en) 2018-10-30
WO2017181021A1 (en) 2017-10-19
US20190211091A1 (en) 2019-07-11
SG10202008378UA (en) 2020-10-29
EP3442577A1 (en) 2019-02-20
IL262277A (en) 2018-11-29
AU2017250797A1 (en) 2018-10-25
NZ746655A (en) 2025-05-02
AU2024205641A1 (en) 2024-09-05
KR20240005973A (ko) 2024-01-12
KR20190003556A (ko) 2019-01-09
JP2019515027A (ja) 2019-06-06
US20190127455A1 (en) 2019-05-02
JP2022153418A (ja) 2022-10-12
JP2025000642A (ja) 2025-01-07
EP3442577A4 (en) 2020-03-25
US20230057519A1 (en) 2023-02-23

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