CA3098489A1 - Therapeutic genome editing in x-linked hyper igm syndrome - Google Patents

Therapeutic genome editing in x-linked hyper igm syndrome Download PDF

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Publication number
CA3098489A1
CA3098489A1 CA3098489A CA3098489A CA3098489A1 CA 3098489 A1 CA3098489 A1 CA 3098489A1 CA 3098489 A CA3098489 A CA 3098489A CA 3098489 A CA3098489 A CA 3098489A CA 3098489 A1 CA3098489 A1 CA 3098489A1
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cell
cells
nucleic acid
nuclease
vector
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CA3098489A
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English (en)
French (fr)
Inventor
David J. Rawlings
Daniel THOMSON
Iram F. KHAN
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Seattle Children's Hospital
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Seattle Children's Hospital
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
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    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70575—NGF/TNF-superfamily, e.g. CD70, CD95L, CD153, CD154
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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
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    • 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
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    • C12N15/86—Viral vectors
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0387—Animal model for diseases of the immune system
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
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  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Mycology (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3098489A 2018-04-27 2019-04-24 Therapeutic genome editing in x-linked hyper igm syndrome Pending CA3098489A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862663485P 2018-04-27 2018-04-27
US62/663,485 2018-04-27
PCT/US2019/028858 WO2019209912A2 (en) 2018-04-27 2019-04-24 Therapeutic genome editing in x-linked hyper igm syndrome

Publications (1)

Publication Number Publication Date
CA3098489A1 true CA3098489A1 (en) 2019-10-31

Family

ID=68295746

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3098489A Pending CA3098489A1 (en) 2018-04-27 2019-04-24 Therapeutic genome editing in x-linked hyper igm syndrome

Country Status (8)

Country Link
US (1) US20210324381A1 (enExample)
EP (1) EP3784292A4 (enExample)
JP (1) JP2021521850A (enExample)
KR (1) KR20210005178A (enExample)
CN (1) CN112312931A (enExample)
AU (1) AU2019261385A1 (enExample)
CA (1) CA3098489A1 (enExample)
WO (1) WO2019209912A2 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018195360A1 (en) 2017-04-21 2018-10-25 Seattle Children's Hospital (Dba Seattle Childern's Research Institute) Therapeutic genome editing in wiskott-aldrich syndrome and x-linked thrombocytopenia
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
US12492411B2 (en) 2018-05-30 2025-12-09 The Regents Of The University Of California Gene editing of monogenic disorders in human hematopoietic stem cells—correction of X-linked agammaglobulinemia (XLA)
AU2022352772A1 (en) * 2021-09-24 2024-04-11 Maxcyte, Inc. A method of enhanced viral transduction using electroporation
KR20260082334A (ko) * 2023-10-02 2026-06-04 콘소르시오 센트로 데 인베스티카시온 비오메디카 엔 레드 임상적으로 의미있는 효능을 달성하기 위한 cd34+ 세포의 유전자 편집 방법
WO2026024636A1 (en) * 2024-07-24 2026-01-29 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Methods and compositions for editing a cd40lg gene

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU4102099A (en) * 1998-05-29 1999-12-13 Heska Corporation Canine Interleukin -4 Proteins, Nucleic Acid Molecules, and Uses Thereof
DK3363902T3 (da) * 2012-12-06 2020-01-02 Sigma Aldrich Co Llc Crispr-baseret genommodifikation og -regulering
EP3289080B1 (en) * 2015-04-30 2021-08-25 The Trustees of Columbia University in the City of New York Gene therapy for autosomal dominant diseases
US10179918B2 (en) * 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
AU2016262093B2 (en) * 2015-05-13 2022-08-18 Seattle Children' S Hospital (Dba Seattle Children 's Research Institute) Enhancing endonuclease based gene editing in primary cells
WO2018035387A1 (en) * 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2018073393A2 (en) * 2016-10-19 2018-04-26 Cellectis Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy
GB201618414D0 (en) * 2016-11-01 2016-12-14 Patterson James Regulated cell lines and methods of use thereof
WO2018083606A1 (en) * 2016-11-01 2018-05-11 Novartis Ag Methods and compositions for enhancing gene editing
EP3622056A1 (en) * 2017-05-10 2020-03-18 Aarhus Universitet Interferon primed plasmacytoid dendritic cells
US20220080055A9 (en) * 2017-10-17 2022-03-17 Crispr Therapeutics Ag Compositions and methods for gene editing for hemophilia a
US12214055B2 (en) * 2017-10-24 2025-02-04 Editas Medicine, Inc. Systems and methods for treating hyper-igm syndrome
EP3707266A1 (en) * 2017-11-07 2020-09-16 Editas Medicine, Inc. Targeted integration systems and methods for the treatment of hemoglobinopathies
CA3082611A1 (en) * 2017-11-17 2019-05-23 Memorial Sloan-Kettering Cancer Center Methods and compositions for alleviating cytokine release syndrome

Also Published As

Publication number Publication date
WO2019209912A2 (en) 2019-10-31
WO2019209912A3 (en) 2020-01-16
JP2021521850A (ja) 2021-08-30
CN112312931A (zh) 2021-02-02
KR20210005178A (ko) 2021-01-13
AU2019261385A1 (en) 2020-11-19
EP3784292A4 (en) 2022-01-19
EP3784292A2 (en) 2021-03-03
US20210324381A1 (en) 2021-10-21

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