CN116829595A - 用于治疗由失调的血浆激肽释放酶介导的疾病的抗体的腺相关病毒载体递送 - Google Patents

用于治疗由失调的血浆激肽释放酶介导的疾病的抗体的腺相关病毒载体递送 Download PDF

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Publication number
CN116829595A
CN116829595A CN202180093553.4A CN202180093553A CN116829595A CN 116829595 A CN116829595 A CN 116829595A CN 202180093553 A CN202180093553 A CN 202180093553A CN 116829595 A CN116829595 A CN 116829595A
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CN
China
Prior art keywords
vector
plasma kallikrein
raav
raav vector
promoter
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Pending
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CN202180093553.4A
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English (en)
Chinese (zh)
Inventor
J·肯尼斯顿
A·赛瑞金
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Takeda Pharmaceutical Co Ltd
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Takeda Pharmaceutical Co Ltd
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Publication of CN116829595A publication Critical patent/CN116829595A/zh
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/40—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against enzymes
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/76—Antagonist effect on antigen, e.g. neutralization or inhibition of binding
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CN202180093553.4A 2020-12-16 2021-12-15 用于治疗由失调的血浆激肽释放酶介导的疾病的抗体的腺相关病毒载体递送 Pending CN116829595A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063126300P 2020-12-16 2020-12-16
US63/126,300 2020-12-16
PCT/IB2021/000874 WO2022130014A1 (en) 2020-12-16 2021-12-15 Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein

Publications (1)

Publication Number Publication Date
CN116829595A true CN116829595A (zh) 2023-09-29

Family

ID=80461568

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180093553.4A Pending CN116829595A (zh) 2020-12-16 2021-12-15 用于治疗由失调的血浆激肽释放酶介导的疾病的抗体的腺相关病毒载体递送

Country Status (5)

Country Link
US (1) US20220186252A1 (https=)
EP (1) EP4263614A1 (https=)
JP (1) JP2023554066A (https=)
CN (1) CN116829595A (https=)
WO (1) WO2022130014A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116790615B (zh) * 2023-07-11 2024-11-22 康霖生物科技（杭州）有限公司 一种用于过敏性疾病的基因治疗载体核酸构建体及其使用方法
GB202402816D0 (en) * 2024-02-28 2024-04-10 Imperial College Innovations Ltd Self-amplifying RNA molecule

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US6136597A (en) 1997-09-18 2000-10-24 The Salk Institute For Biological Studies RNA export element
US7419829B2 (en) 2000-10-06 2008-09-02 Oxford Biomedica (Uk) Limited Vector system
US7575924B2 (en) 2000-11-13 2009-08-18 Research Development Foundation Methods and compositions relating to improved lentiviral vectors and their applications
EP1438075A4 (en) 2001-10-02 2006-04-19 Inst Clayton De La Rech METHOD AND COMPOSITIONS ASSOCIATED WITH LENTIVIRAL VECTORS WITH LIMITED EXPRESSION AND ITS APPLICATIONS
US7816131B2 (en) 2004-04-02 2010-10-19 Board Of Regents, The University Of Texas System Cancer specific promoters
US8865881B2 (en) 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
GB201206455D0 (en) 2012-04-12 2012-05-30 Royal Holloway & Bedford New College Gene expression
WO2014152232A2 (en) 2013-03-15 2014-09-25 Dyax Corp. Anti-plasma kallikrein antibodies
JP2016517440A (ja) 2013-03-15 2016-06-16 ブイエルピー バイオテック，インコーポレイテッド パリビズマブエピトープベースのウイルス様粒子
CN107249646B (zh) 2014-12-16 2021-06-29 内布拉斯加大学董事会 用于青少年巴滕病的基因疗法
CL2014003590A1 (es) 2014-12-30 2015-07-10 Univ Chile Virus aav/xbp1s-ha, método de tratamiento genético y su uso en la optimizacion y mejoramiento de las capacidades cognitivas, de memoria y de aprendizaje.
MA41346A (fr) 2015-01-12 2017-11-21 Juno Therapeutics Inc Eléments régulateurs post-transcriptionnels d'hépatite modifiée
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
GB2547179A (en) 2015-10-26 2017-08-16 Quethera Ltd Genetic construct
CN108291238A (zh) 2015-10-27 2018-07-17 赛尔希恩公司 嵌合转录后调控元件
CN109415687A (zh) 2016-04-07 2019-03-01 蓝鸟生物公司 嵌合抗原受体t细胞组合物
JP7632835B2 (ja) 2016-05-20 2025-02-19 プレジデント アンド フェローズ オブ ハーバード カレッジ 加齢関連疾患及び症状の遺伝子治療法
CN110662839A (zh) 2017-02-17 2020-01-07 珀杜研究基金会 用于细胞疗法的基于靶向性配体-有效负载的药物递送
KR20220012231A (ko) * 2019-04-24 2022-02-03 리젠엑스바이오 인크. 완전-인간 번역 후 변형된 항체 치료제
EP4048800A1 (en) * 2019-10-23 2022-08-31 Shire Human Genetic Therapies, Inc. Adeno-associated virus vectors based gene therapy for hereditary angioedema
US20230390418A1 (en) * 2020-10-29 2023-12-07 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof

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Publication number Publication date
EP4263614A1 (en) 2023-10-25
WO2022130014A1 (en) 2022-06-23
JP2023554066A (ja) 2023-12-26
US20220186252A1 (en) 2022-06-16

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