CA3161735A1 - Regulatory nucleic acid sequences - Google Patents

Regulatory nucleic acid sequences Download PDF

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Publication number
CA3161735A1
CA3161735A1 CA3161735A CA3161735A CA3161735A1 CA 3161735 A1 CA3161735 A1 CA 3161735A1 CA 3161735 A CA3161735 A CA 3161735A CA 3161735 A CA3161735 A CA 3161735A CA 3161735 A1 CA3161735 A1 CA 3161735A1
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CA
Canada
Prior art keywords
seq
promoter
functional variant
crm
specific
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
CA3161735A
Other languages
English (en)
French (fr)
Inventor
Jorge Omar YANEZ-CUNA
Juan Manuel IGLESIAS
Sinclair COOPER
Katie BAKER
Polyxeni KATSOUPI
Rinku RAJAN
Ileana GUERRINI
Antonia EVRIPIOTI
Kira MOURAO
Michael L. Roberts
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 Inc
Original Assignee
Individual
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
Priority claimed from GBGB1919269.9A external-priority patent/GB201919269D0/en
Priority claimed from GBGB2012192.7A external-priority patent/GB202012192D0/en
Application filed by Individual filed Critical Individual
Publication of CA3161735A1 publication Critical patent/CA3161735A1/en
Pending legal-status Critical Current

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Classifications

    • 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
    • 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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • 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
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0657—Cardiomyocytes; Heart cells

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (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)
CA3161735A 2019-12-24 2020-12-24 Regulatory nucleic acid sequences Pending CA3161735A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GB1919269.9 2019-12-24
GBGB1919269.9A GB201919269D0 (en) 2019-12-24 2019-12-24 Regulatory nucleic acid sequences
GB2012192.7 2020-08-05
GBGB2012192.7A GB202012192D0 (en) 2020-08-05 2020-08-05 Regulatory nucleic acid sequences
PCT/GB2020/053371 WO2021130503A1 (en) 2019-12-24 2020-12-24 Regulatory nucleic acid sequences

Publications (1)

Publication Number Publication Date
CA3161735A1 true CA3161735A1 (en) 2021-07-01

Family

ID=74130280

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3161735A Pending CA3161735A1 (en) 2019-12-24 2020-12-24 Regulatory nucleic acid sequences

Country Status (10)

Country Link
US (1) US20230233710A1 (enExample)
EP (1) EP4081643A1 (enExample)
JP (1) JP2023509118A (enExample)
KR (1) KR20220119703A (enExample)
CN (1) CN115151646A (enExample)
AU (1) AU2020412375A1 (enExample)
CA (1) CA3161735A1 (enExample)
IL (1) IL294238A (enExample)
WO (1) WO2021130503A1 (enExample)
ZA (1) ZA202207849B (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FI4103214T3 (fi) 2020-02-13 2026-07-03 Tenaya Therapeutics Inc Geeniterapian vektorit sydänsairauden hoitoon
CN116406297A (zh) * 2020-08-05 2023-07-07 阿斯克肋匹奥生物制药公司 治疗心脏疾患和充血性心力衰竭以及施用aav载体的方法
CA3221707A1 (en) * 2021-06-23 2022-12-29 Jorge Omar YANEZ-CUNA Regulatory nucleic acid sequences
JP2025512574A (ja) * 2022-04-22 2025-04-17 スペースクラフト セブン リミテッド ライアビリティ カンパニー ＡＡＶベクターを使用するＢ細胞リンパ腫２関連Ａｎｔｈａｎｏｇｅｎｅ ３ （ＢＡＧ３）遺伝子治療
CN115948403B (zh) * 2022-12-30 2023-09-15 广州派真生物技术有限公司 在哺乳动物肌肉中特异性启动基因的启动子序列及其应用
CN122396772A (zh) * 2023-12-18 2026-07-14 豪夫迈·罗氏有限公司 骨骼肌特异性启动子

Family Cites Families (16)

* 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
CN1247785C (zh) 2000-10-13 2006-03-29 希龙公司 巨细胞病毒内含子a片段
US20030074679A1 (en) * 2000-12-12 2003-04-17 Schwartz Robert J. Administration of nucleic acid sequence to female animal to enhance growth in offspring
US8106180B2 (en) 2003-08-07 2012-01-31 Whitehead Institute For Biomedical Research Methods and products for expression of micro RNAs
US20050142581A1 (en) 2003-09-04 2005-06-30 Griffey Richard H. Microrna as ligands and target molecules
WO2005056761A2 (en) 2003-12-05 2005-06-23 Massachusetts Institute Of Technology REAGENTS AND METHODS FOR IDENTIFICATION OF RNAi PATHWAY GENES AND CHEMICAL MODULATORS OF RNAi
KR100614827B1 (ko) 2004-05-06 2006-08-25 재단법인서울대학교산학협력재단 양방향 은닉 마코프 모델을 이용한 완숙한마이크로알엔에이 위치예측방법 및 이를 구현하기 위한컴퓨터 프로그램을 기록한 저장매체
EP2494058B1 (en) * 2009-10-29 2017-04-05 Vib Vzw Cardiac-specific nucleic acid regulatory elements and methods and use thereof
CN103074373A (zh) * 2011-10-26 2013-05-01 南京医科大学 肌肉组织特异性表达Follistatin以提高牛瘦肉率的转基因载体
CN102747082B (zh) * 2011-12-29 2014-06-18 华中农业大学 猪肌肉特异性itgb1bp2启动子及其应用
AU2015208247B2 (en) * 2014-01-21 2021-05-27 Universiteit Gent Muscle-specific nucleic acid regulatory elements and methods and use thereof
WO2017021359A1 (en) * 2015-08-03 2017-02-09 Myodopa Limited Systemic synthesis and regulation of l-dopa
CN105063079A (zh) * 2015-08-15 2015-11-18 内蒙古大学 一种三顺反子肌肉特异双向共表达基因转移体及制备方法
US20170253927A1 (en) * 2016-03-01 2017-09-07 Washington State University Heritable epigenetic modifications as markers of chemotherapy exposure
CN108018311A (zh) * 2016-11-04 2018-05-11 中国科学院上海生命科学研究院 通过基因编辑特异靶向肌肉组织mstn治疗恶病质
US11338045B2 (en) * 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy

Also Published As

Publication number Publication date
WO2021130503A1 (en) 2021-07-01
ZA202207849B (en) 2023-12-20
JP2023509118A (ja) 2023-03-07
IL294238A (en) 2022-08-01
US20230233710A1 (en) 2023-07-27
AU2020412375A1 (en) 2022-06-30
KR20220119703A (ko) 2022-08-30
EP4081643A1 (en) 2022-11-02
CN115151646A (zh) 2022-10-04

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