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

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

Info

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
Authority
AU
Australia
Prior art keywords
nucleic acid
acid sequence
seq
identity
expression vector
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
AU2019417697A
Other languages
English (en)
Inventor
Walid Jhan AZAR
Chao-Guang Chen
Chi-Lin LEE
Cédric Pierre VONARBURG
Ming Yan
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.)
CSL Behring LLC
Original Assignee
CSL Behring LLC
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 CSL Behring LLC filed Critical CSL Behring LLC
Publication of AU2019417697A1 publication Critical patent/AU2019417697A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N15/11—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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • 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
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • 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
    • 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
    • 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)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (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

Publications (1)

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

Family

ID=69326699

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2019417697A Pending AU2019417697A1 (en) 2018-12-23 2019-12-23 Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome

Country Status (8)

Country Link
US (1) US20210316013A1 (enExample)
EP (1) EP3897745A1 (enExample)
JP (1) JP2022514955A (enExample)
KR (1) KR102942447B1 (enExample)
CN (1) CN113518825A (enExample)
AU (1) AU2019417697A1 (enExample)
BR (1) BR112021012240A2 (enExample)
WO (1) WO2020139796A1 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3684421A4 (en) * 2017-09-18 2021-08-04 Children's Hospital Medical Center STRONG INSULATOR AND ITS USES IN GENE ADMINISTRATION
CA3217247A1 (en) * 2021-04-26 2022-11-03 Csl Behring L.L.C. Lentiviral vectors useful for the treatment of disease
JP2024535716A (ja) * 2021-09-16 2024-10-02 エージェンシー フォー サイエンス， テクノロジー アンド リサーチ 肝疾患を治療するためのｔｊｐ１発現の調節
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
WO2024171034A2 (en) * 2023-02-13 2024-08-22 Csl Behring Llc Optimised polynucleotides
CN121399150A (zh) 2023-05-25 2026-01-23 美国杰特贝林生物制品有限公司 经修饰载体
AU2024358606A1 (en) * 2023-10-08 2026-04-16 Shanghai Qilu Pharmaceutical Research And Development Centre Ltd. Truncated insulator and use thereof in transient expression of recombinant protein
WO2025215591A1 (en) 2024-04-12 2025-10-16 Csl Behring L.L.C. Modified vectors for xla gene therapy
WO2025241977A1 (zh) * 2024-05-20 2025-11-27 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
WO2026003694A1 (en) * 2024-06-24 2026-01-02 Csl Behring Llc Methods of enveloped virus transduction

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5958928A (en) 1995-03-27 1999-09-28 Chugai Seiyaku Kabushiki Kaisha Pharmaceutical agents containing methotrexate derivative
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
WO2005080581A2 (en) 2004-02-17 2005-09-01 University Of Florida Research Foundation, Inc. Insulated herpesvirus-derived gene expression cassettes for sustained and regulatable gene expression
PL1766010T3 (pl) 2004-06-28 2011-07-29 Univ Western Australia Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania
MX2007010008A (es) * 2005-02-16 2008-01-18 Lentigen Corp Vectores lentivirales y su uso.
WO2007098089A2 (en) 2006-02-17 2007-08-30 Novacea, Inc. Treatment of hyperproliferative diseases with methotrexate n-oxide and analogs
ES2785223T3 (es) 2006-06-21 2020-10-06 Uniqure Ip Bv Células de insecto para la producción de vectores de AAV
CN105779453A (zh) 2008-10-24 2016-07-20 萨雷普塔治疗公司 用于dmd的多外显子跳跃组合物
BR122023023194A2 (pt) 2009-11-12 2023-12-26 The University Of Western Australia Oligonucleotídeo antissentido, composição e respectivo uso
US20110294114A1 (en) 2009-12-04 2011-12-01 Cincinnati Children's Hospital Medical Center Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells
RU2728867C2 (ru) * 2011-04-20 2020-07-31 Де Реджентс Оф Де Юниверсити Оф Калифорния Способ совместного кондиционирования и хемоселекции за один цикл
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
EP2892617B1 (en) 2012-09-06 2018-06-13 The University of Chicago Antisense polynucleotides to induce exon skipping and methods of treating dystrophies
NZ631289A (en) 2013-03-15 2017-08-25 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
US20140363454A1 (en) 2013-06-06 2014-12-11 Igenica Biotherapeutics, Inc. Antibody-Drug Conjugates, Compositions and Methods of Use
CN105793425B (zh) * 2013-06-17 2021-10-26 布罗德研究所有限公司 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
CN111206032B (zh) * 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
CN104805120A (zh) * 2014-01-27 2015-07-29 苟德明 一种shRNA-Ago2共表达慢病毒RNAi载体、重组质粒及其构建方法
IL296691B2 (en) 2014-04-25 2023-11-01 2Seventy Bio Inc MND promoter chimeric antigen receptors
EP4316500A3 (en) 2015-05-13 2024-04-10 CSL Behring Gene Therapy, Inc. Bio-production of lentiviral vectors
WO2016186708A1 (en) 2015-05-18 2016-11-24 Calimmune, Inc. Gene therapeutic for the treatment of hiv and uses thereof
HK1257668A1 (zh) * 2016-02-19 2019-10-25 The Regents Of The University Of California 短发夹rna(shrna734)及其用於阳性选择和消除遗传修饰的细胞的用途
KR20230082055A (ko) 2016-06-17 2023-06-08 마젠타 테라퓨틱스 인코포레이티드 세포의 고갈을 위한 조성물 및 방법
MX2018015684A (es) 2016-06-17 2019-08-29 Magenta Therapeutics Inc Composiciones y metodos para el agotamiento de celulas cd117.
BR112020001059A2 (pt) * 2017-07-18 2020-07-14 Calimmune, Inc. composições e métodos para o tratamento de beta-hemoglobinopatias
KR102944997B1 (ko) * 2018-08-24 2026-03-30 씨에스엘 베링 진 테라피, 인크. 무혈청 배지에서 벡터 제조

Also Published As

Publication number Publication date
CN113518825A (zh) 2021-10-19
EP3897745A1 (en) 2021-10-27
US20210316013A1 (en) 2021-10-14
BR112021012240A2 (pt) 2022-01-18
KR102942447B1 (ko) 2026-03-24
KR20210118402A (ko) 2021-09-30
WO2020139796A1 (en) 2020-07-02
JP2022514955A (ja) 2022-02-16

Similar Documents

Publication Publication Date Title
US20210316013A1 (en) Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome
JP7844400B2 (ja) 異常ヘモグロビン症の治療用組成物および方法
EP3655534B1 (en) Compositions and methods for treating beta-hemoglobinopathies
US20250354166A1 (en) Lentiviral vectors useful for the treatment of disease
US20180119123A1 (en) Crispr/cas-related methods and compositions for treating hiv infection and aids
WO2020097049A1 (en) Vector system for expressing regulatory rna
EP3701028B1 (en) Systems and methods for treating hyper-igm syndrome
US20210340563A1 (en) Donor t-cells with kill switch
JP7846007B2 (ja) 異常ヘモグロビン症の治療用組成物及び方法
US20230405116A1 (en) Vectors, systems and methods for eukaryotic gene editing
US20230355674A1 (en) Donor t-cells with kill switch
KR20260018055A (ko) 변형된 벡터
CN118103076A (zh) 可用于疾病治疗的慢病毒载体
WO2023143539A1 (en) Engineered adar-recruiting rnas and methods of use thereof
HK40080440A (en) Compositions and methods for the treatment of hemoglobinopathies