AU2019382955B2 - Liver-specific regulatory nucleic acid sequences - Google Patents

Liver-specific regulatory nucleic acid sequences

Info

Publication number
AU2019382955B2
AU2019382955B2 AU2019382955A AU2019382955A AU2019382955B2 AU 2019382955 B2 AU2019382955 B2 AU 2019382955B2 AU 2019382955 A AU2019382955 A AU 2019382955A AU 2019382955 A AU2019382955 A AU 2019382955A AU 2019382955 B2 AU2019382955 B2 AU 2019382955B2
Authority
AU
Australia
Prior art keywords
lvr
cre0058
cre0065
cre0051
cre0066
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.)
Active
Application number
AU2019382955A
Other languages
English (en)
Other versions
AU2019382955A1 (en
Inventor
Katie BAKER
Ross Fraser
Juan Manuel IGLESIAS
Nicolle KIPPEN
Michael Roberts
Jorge Omar YANEZ-CUNA
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
AskBio UK 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 AskBio UK Ltd filed Critical AskBio UK Ltd
Publication of AU2019382955A1 publication Critical patent/AU2019382955A1/en
Assigned to ASKBIO UK LTD reassignment ASKBIO UK LTD Amend patent request/document other than specification (104) Assignors: SYNPROMICS LIMITED
Application granted granted Critical
Publication of AU2019382955B2 publication Critical patent/AU2019382955B2/en
Priority to AU2025287323A priority Critical patent/AU2025287323A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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

Landscapes

  • 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)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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)
AU2019382955A 2018-11-19 2019-11-19 Liver-specific regulatory nucleic acid sequences Active AU2019382955B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025287323A AU2025287323A1 (en) 2018-11-19 2025-12-24 Liver-Specific Regulatory Nucleic Acid Sequences

Applications Claiming Priority (3)

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2025287323A Division AU2025287323A1 (en) 2018-11-19 2025-12-24 Liver-Specific Regulatory Nucleic Acid Sequences

Publications (2)

Publication Number Publication Date
AU2019382955A1 AU2019382955A1 (en) 2021-06-10
AU2019382955B2 true AU2019382955B2 (en) 2025-09-25

Family

ID=64740036

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2019382955A Active AU2019382955B2 (en) 2018-11-19 2019-11-19 Liver-specific regulatory nucleic acid sequences
AU2025287323A Pending AU2025287323A1 (en) 2018-11-19 2025-12-24 Liver-Specific Regulatory Nucleic Acid Sequences

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2025287323A Pending AU2025287323A1 (en) 2018-11-19 2025-12-24 Liver-Specific Regulatory Nucleic Acid Sequences

Country Status (9)

Country Link
US (2) US12590129B2 (https=)
EP (2) EP3883951A1 (https=)
JP (2) JP7489161B2 (https=)
CN (1) CN113454104A (https=)
AU (2) AU2019382955B2 (https=)
CA (1) CA3120253A1 (https=)
GB (1) GB201818816D0 (https=)
IL (1) IL283219A (https=)
WO (2) WO2020104782A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117202925A (zh) 2021-01-20 2023-12-08 科罗根科技有限公司 用于治疗和诊断肝癌的Ssu72蛋白或多核苷酸
CN116744961A (zh) 2021-01-29 2023-09-12 科罗根科技有限公司 使用Ssu72治疗肝病的药物组合物
WO2023008925A1 (ko) * 2021-07-29 2023-02-02 주식회사 리보퓨틱스 융합 프로모터, 이를 포함하는 재조합 벡터 및 이를 이용하는 간암 치료용 조성물
CN121358865A (zh) * 2023-04-20 2026-01-16 阿斯克生物公司 肝脏特异性调控核酸序列
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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016146757A1 (en) * 2015-03-17 2016-09-22 Vrije Universiteit Brussel Optimized liver-specific expression systems for fviii and fix

Family Cites Families (17)

* 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
WO1998009524A1 (en) * 1996-09-06 1998-03-12 Chiron Corporation Methods and compositions for liver specific delivery 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
ATE437221T1 (de) 2001-05-14 2009-08-15 Gbp Ip Llc Lentivirale vektoren kodierend für gerinnungsfaktoren für die gentherapie
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
CA2722238C (en) 2008-04-22 2017-11-28 Life Sciences Research Partners 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
MX2018012537A (es) * 2016-04-15 2019-02-25 Univ Pennsylvania Terapia de genes para tratar hemofilia a.
WO2018144709A2 (en) * 2017-02-01 2018-08-09 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
EP3630160A1 (en) * 2017-05-24 2020-04-08 Universitat Autònoma De Barcelona Viral expression construct comprising a fibroblast growth factor 21 (fgf21) coding sequence
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016146757A1 (en) * 2015-03-17 2016-09-22 Vrije Universiteit Brussel Optimized liver-specific expression systems for fviii and fix

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
DATABASE Geneseq [online] 26 July 2018 (2018-07-26), "HBV enhancer DNA sequence (EnhI).", XP002797160, retrieved from EBI accession no. GSN:BFJ18268 Database accession no. BFJ18268 *
Nair, Nisha, et al. "Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy." Blood, The Journal of the American Society of Hematology 123.20 (2014): 3195-3199. *

Also Published As

Publication number Publication date
AU2019382955A1 (en) 2021-06-10
EP3883952A1 (en) 2021-09-29
US20210388347A1 (en) 2021-12-16
WO2020104782A1 (en) 2020-05-28
CA3120253A1 (en) 2020-05-28
CN113454104A (zh) 2021-09-28
EP3883951A1 (en) 2021-09-29
AU2025287323A1 (en) 2026-03-26
JP2022507828A (ja) 2022-01-18
US12590129B2 (en) 2026-03-31
WO2020104783A1 (en) 2020-05-28
JP7489161B2 (ja) 2024-05-23
JP2024096349A (ja) 2024-07-12
US20220009981A1 (en) 2022-01-13
IL283219A (en) 2021-06-30
GB201818816D0 (en) 2019-01-02

Similar Documents

Publication Publication Date Title
AU2019382955B2 (en) Liver-specific regulatory nucleic acid sequences
US20240110179A1 (en) Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
JP2025118716A (ja) ハプロ不全のための遺伝子治療
JP6272279B2 (ja) 肝特異的核酸調節要素ならびにその方法および用途
JP7383478B2 (ja) アルファ１アンチトリプシン欠乏症を治療するための組成物および方法
TW202028466A (zh) 編輯RNAs的方法以及組成物
TW202028461A (zh) 核酸構築體及使用方法
TW202027797A (zh) 用於治療α-1抗胰蛋白酶不足的組成物及方法
US12472269B2 (en) Liver-specific inducible promoters and methods of use thereof
US20210395774A1 (en) Novel virus vector and methods for producing and using same
WO2024220800A1 (en) Liver-specific regulatory nucleic acid sequences
HK40057846A (en) Compositions and methods for treating alpha-1 antitrypsin deficiencey

Legal Events

Date Code Title Description
HB Alteration of name in register

Owner name: ASKBIO UK LTD

Free format text: FORMER NAME(S): SYNPROMICS LIMITED

FGA Letters patent sealed or granted (standard patent)