AU2019417697A1 - Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome - Google Patents

Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome Download PDF

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Publication number
AU2019417697A1
AU2019417697A1 AU2019417697A AU2019417697A AU2019417697A1 AU 2019417697 A1 AU2019417697 A1 AU 2019417697A1 AU 2019417697 A AU2019417697 A AU 2019417697A AU 2019417697 A AU2019417697 A AU 2019417697A AU 2019417697 A1 AU2019417697 A1 AU 2019417697A1
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nucleic acid
acid sequence
seq
identity
expression vector
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Pending
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AU2019417697A
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Inventor
Walid Jhan AZAR
Chao-Guang Chen
Chi-Lin LEE
Cédric Pierre VONARBURG
Ming Yan
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CSL Behring LLC
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CSL Behring LLC
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/02—Pentosyltransferases (2.4.2)
    • C12Y204/02008—Hypoxanthine phosphoribosyltransferase (2.4.2.8)

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Biomedical Technology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2019417697A 2018-12-23 2019-12-23 Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome Pending AU2019417697A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862784508P 2018-12-23 2018-12-23
US62/784,508 2018-12-23
PCT/US2019/068233 WO2020139796A1 (en) 2018-12-23 2019-12-23 Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome

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Publication Number Publication Date
AU2019417697A1 true AU2019417697A1 (en) 2021-07-08

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AU2019417697A Pending AU2019417697A1 (en) 2018-12-23 2019-12-23 Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome

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US (1) US20210316013A1 (https=)
EP (1) EP3897745A1 (https=)
JP (1) JP2022514955A (https=)
KR (1) KR102942447B1 (https=)
CN (1) CN113518825A (https=)
AU (1) AU2019417697A1 (https=)
BR (1) BR112021012240A2 (https=)
WO (1) WO2020139796A1 (https=)

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EP4402264A4 (en) * 2021-09-16 2025-10-15 Agency Science Tech & Res MODULATION OF TJP1 EXPRESSION TO TREAT LIVER DISEASES
CN115838765A (zh) * 2021-09-18 2023-03-24 济南赛尔生物科技股份有限公司 一种治疗异染性脑白质营养不良的慢病毒载体
CN114990163A (zh) * 2022-03-31 2022-09-02 中海峡(福建)细胞生物科技有限公司 用于干细胞基因修饰的慢病毒载体及其构建方法和应用
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WO2025077575A1 (zh) * 2023-10-08 2025-04-17 上海齐鲁制药研究中心有限公司 截短的绝缘子及其用于重组蛋白的瞬时表达的用途
WO2025215591A1 (en) 2024-04-12 2025-10-16 Csl Behring L.L.C. Modified vectors for xla gene therapy
CN120536433A (zh) * 2024-05-20 2025-08-26 上海本导基因技术有限公司 一种能够调节基因表达的调控元件及应用
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Publication number Publication date
WO2020139796A1 (en) 2020-07-02
KR102942447B1 (ko) 2026-03-24
US20210316013A1 (en) 2021-10-14
JP2022514955A (ja) 2022-02-16
CN113518825A (zh) 2021-10-19
BR112021012240A2 (pt) 2022-01-18
EP3897745A1 (en) 2021-10-27
KR20210118402A (ko) 2021-09-30

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