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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Canada
Prior art keywords
antigen
binding fragment
vegf
certain embodiments
human
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Pending
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CA3019665A
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English (en)
French (fr)
Inventor
Curran Matthew Simpson
Stephen YOO
Karen Fran Kozarsky
Rickey Robert REINHARDT
Laura A. CORUZZI
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Regenxbio Inc
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Regenxbio Inc
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Publication of CA3019665A1 publication Critical patent/CA3019665A1/en
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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22Immunoglobulins [IGs], 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)
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 (OSRAM)
EP (1) EP3442577A4 (OSRAM)
JP (3) JP2019515027A (OSRAM)
KR (2) KR20240005973A (OSRAM)
AU (2) AU2017250797A1 (OSRAM)
CA (1) CA3019665A1 (OSRAM)
IL (1) IL262277A (OSRAM)
SG (2) SG10202008378UA (OSRAM)
WO (1) WO2017181021A1 (OSRAM)

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US9840553B2 (en) 2014-06-28 2017-12-12 Kodiak Sciences Inc. Dual PDGF/VEGF antagonists
CA3010056A1 (en) 2015-12-30 2017-07-06 Kodiak Sciences Inc. Antibodies and conjugates thereof
KR102574810B1 (ko) 2016-04-15 2023-09-08 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 습성 연령 관련 황반 변성의 치료를 위한 조성물
KR20200060456A (ko) * 2017-09-27 2020-05-29 리젠엑스바이오 인크. 완전-인간 번역후 변형된 항-VEGF Fab를 이용한 안구 질환의 치료
EA202091509A1 (ru) * 2017-12-19 2020-09-22 Акуос, Инк. Aav-опосредованная доставка терапевтических антител во внутреннее ухо
US20210093734A1 (en) * 2018-02-20 2021-04-01 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-realted macular degeneration
CN112203679A (zh) 2018-03-02 2021-01-08 科达制药股份有限公司 Il-6抗体及其融合构建体和缀合物
EP3814513B1 (en) * 2018-06-28 2025-04-09 The University of North Carolina at Chapel Hill Optimized cln5 genes and expression cassettes and their use
JP2022506613A (ja) * 2018-11-07 2022-01-17 アコーオス インコーポレイテッド 内耳の有毛細胞及び支持細胞における遺伝子欠陥/発現タンパク質を修正するためのアデノ随伴ウイルスベクターの使用
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
KR20220049568A (ko) * 2019-08-22 2022-04-21 더 리전츠 오브 더 유니버시티 오브 캘리포니아 엔젤만 증후군의 치료를 위한 ube3a
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
JP2022552262A (ja) 2019-10-07 2022-12-15 リジェネックスバイオ インコーポレイテッド アデノ随伴ウイルスベクター医薬組成物および方法
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
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
MX2023004005A (es) 2020-10-07 2023-06-06 Regenxbio Inc Formulaciones para administración supracoroidea tales como formulaciones de gel.
EP4225270A1 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
EP4225381A1 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
US20230391864A1 (en) * 2020-10-28 2023-12-07 Regenxbio, Inc. Vectorized anti-tnf-alpha antibodies for ocular indications
PH12023551518A1 (en) 2020-12-01 2024-05-13 Akouos Inc Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
KR20230121096A (ko) * 2020-12-18 2023-08-17 에이씨 이뮨 에스에이 항체 전달
BR112023015303A2 (pt) 2021-02-01 2023-11-14 Regenxbio Inc Método para tratar doença cln2 devido a deficiência de tpp1 em um sujeito
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
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
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
CA2417508A1 (en) * 2000-07-26 2002-01-31 Ludwig Institute For Cancer Research Glycosylated vegf-b and method for increasing the amount of soluble vegf-b
WO2010148223A2 (en) * 2009-06-17 2010-12-23 Facet Biotech Corporation Anti-vegf antibodies and their uses
US20130090375A1 (en) * 2011-10-06 2013-04-11 Cornell University Virus-mediated delivery of bevacizumab for therapeutic applications
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)
JP6974943B2 (ja) * 2014-05-13 2021-12-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 2種の抗体構築物を発現するaavを含む組成物およびこの使用

Also Published As

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

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