CA3120253A1 - Liver-specific regulatory nucleic acid sequences - Google Patents

Liver-specific regulatory nucleic acid sequences Download PDF

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Publication number
CA3120253A1
CA3120253A1 CA3120253A CA3120253A CA3120253A1 CA 3120253 A1 CA3120253 A1 CA 3120253A1 CA 3120253 A CA3120253 A CA 3120253A CA 3120253 A CA3120253 A CA 3120253A CA 3120253 A1 CA3120253 A1 CA 3120253A1
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CA
Canada
Prior art keywords
cre0058
crm
cre0065
cre0051
liver
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3120253A
Other languages
English (en)
French (fr)
Inventor
Michael Roberts
Juan Manuel IGLESIAS
Katie BAKER
Nicolle KIPPEN
Jorge Omar YANEZ-CUNA
Ross Fraser
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.)
AskBio UK Ltd
Original Assignee
Synpromics Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Synpromics Ltd filed Critical Synpromics Ltd
Publication of CA3120253A1 publication Critical patent/CA3120253A1/en
Pending legal-status Critical Current

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/067—Hepatocytes
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Cell Biology (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
CA3120253A 2018-11-19 2019-11-19 Liver-specific regulatory nucleic acid sequences Pending CA3120253A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1818816.9A GB201818816D0 (en) 2018-11-19 2018-11-19 Regulatory nucleic acid sequences
GB1818816.9 2018-11-19
PCT/GB2019/053267 WO2020104782A1 (en) 2018-11-19 2019-11-19 Liver-Specific Regulatory Nucleic Acid Sequences

Publications (1)

Publication Number Publication Date
CA3120253A1 true CA3120253A1 (en) 2020-05-28

Family

ID=64740036

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3120253A Pending CA3120253A1 (en) 2018-11-19 2019-11-19 Liver-specific regulatory nucleic acid sequences

Country Status (9)

Country Link
US (2) US12590129B2 (enExample)
EP (2) EP3883951A1 (enExample)
JP (3) JP7489161B2 (enExample)
CN (1) CN113454104A (enExample)
AU (2) AU2019382955B2 (enExample)
CA (1) CA3120253A1 (enExample)
GB (1) GB201818816D0 (enExample)
IL (1) IL283219A (enExample)
WO (2) WO2020104783A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021207526A1 (en) 2020-04-09 2021-10-14 The Regents Of The University Of California Humanized notch receptors with hinge domain
KR102726511B1 (ko) 2021-01-20 2024-11-07 주식회사 큐로젠 Ssu72 단백질 또는 Ssu72 단백질을 코딩하는 뉴클레오티드를 포함하는 간암 예방 또는 치료용 약학적 조성물
AU2022214501B2 (en) 2021-01-29 2025-05-22 Curogen Technology Co., Ltd. Pharmaceutical composition for treating liver diseases using ssu72
WO2023008925A1 (ko) * 2021-07-29 2023-02-02 주식회사 리보퓨틱스 융합 프로모터, 이를 포함하는 재조합 벡터 및 이를 이용하는 간암 치료용 조성물
JP2026516710A (ja) * 2023-04-20 2026-05-26 アスクバイオ インコーポレイテッド 肝臓特異的調節核酸配列
CN116555266B (zh) * 2023-06-09 2024-11-12 呈诺再生医学科技(北京)有限公司 肝脏特异性表达的重组启动子和载体及其在肝脏相关疾病治疗中的应用

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US5721096A (en) * 1991-01-24 1998-02-24 Children's Medical Center Corporation Methods of screening for compounds with ability to alter apolipoprotein AI gene expression
US5578444A (en) 1991-06-27 1996-11-26 Genelabs Technologies, Inc. Sequence-directed DNA-binding molecules compositions and methods
US6268212B1 (en) 1993-10-18 2001-07-31 Amgen Inc. Tissue specific transgene expression
EP0933997A4 (en) 1996-09-06 2000-08-16 Chiron Corp METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS
US7351813B2 (en) 2000-06-20 2008-04-01 The Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
WO2002092134A1 (en) 2001-05-14 2002-11-21 Cell Genesys, Inc. Lentiviral vectors encoding clotting factors for gene therapy
CN102634515B (zh) 2008-02-14 2013-11-06 财团法人牧岩生命工学研究所 适于表达用于基因治疗的编码序列的表达载体
CN102628041B (zh) 2008-02-14 2013-09-11 财团法人牧岩生命工学研究所 适于表达用于基因治疗的编码序列的表达载体
US9617548B2 (en) 2008-04-22 2017-04-11 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
US8815779B2 (en) 2009-09-16 2014-08-26 SwitchGear Genomics, Inc. Transcription biomarkers of biological responses and methods
EP2479278A1 (en) 2011-01-25 2012-07-25 Synpromics Ltd. Method for the construction of specific promoters
JP6879486B2 (ja) * 2015-03-17 2021-06-02 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ Ｆｖｉｉｉおよびｆｉｘ用の最適化された肝臓特異的発現系
PE20181922A1 (es) 2016-04-15 2018-12-11 Univ Pennsylvania Terapia genica para tratar la hemofilia a
WO2018144709A2 (en) 2017-02-01 2018-08-09 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
BR112019024608A2 (pt) 2017-05-24 2020-06-23 Universitat Autonoma De Barcelona Construto de expressão viral que compreende uma sequência de codificação do fator de crescimento de fibroblastos 21 (fgf21)
JP2020533971A (ja) 2017-08-28 2020-11-26 アルター・バイオサイエンス・コーポレーション Ｉｌ−１５をベースとするｉｌ−７及びｉｌ−２１への融合物
CN108588097B (zh) 2018-04-28 2021-07-13 北京锦篮基因科技有限公司 改造后的hbv基因组和相关组合物及其应用

Also Published As

Publication number Publication date
JP2024096349A (ja) 2024-07-12
JP2022507828A (ja) 2022-01-18
JP7489161B2 (ja) 2024-05-23
CN113454104A (zh) 2021-09-28
AU2019382955B2 (en) 2025-09-25
US12630595B2 (en) 2026-05-19
EP3883951A1 (en) 2021-09-29
US12590129B2 (en) 2026-03-31
JP2026124829A (ja) 2026-07-31
WO2020104782A1 (en) 2020-05-28
AU2025287323A1 (en) 2026-03-26
JP7860164B2 (ja) 2026-05-15
IL283219A (en) 2021-06-30
US20210388347A1 (en) 2021-12-16
EP3883952A1 (en) 2021-09-29
GB201818816D0 (en) 2019-01-02
AU2019382955A1 (en) 2021-06-10
WO2020104783A1 (en) 2020-05-28
US20220009981A1 (en) 2022-01-13

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