MA50833A - Compositions et méthodes pour l'édition génique pour l'hémophilie a - Google Patents

Compositions et méthodes pour l'édition génique pour l'hémophilie a

Info

Publication number
MA50833A
MA50833A MA050833A MA50833A MA50833A MA 50833 A MA50833 A MA 50833A MA 050833 A MA050833 A MA 050833A MA 50833 A MA50833 A MA 50833A MA 50833 A MA50833 A MA 50833A
Authority
MA
Morocco
Prior art keywords
hemophilia
compositions
methods
gene editing
editing
Prior art date
Application number
MA050833A
Other languages
English (en)
Inventor
Alan Richard Brooks
Original Assignee
Bayer Healthcare Llc
Crispr Therapeutics Ag
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 Bayer Healthcare Llc, Crispr Therapeutics Ag filed Critical Bayer Healthcare Llc
Publication of MA50833A publication Critical patent/MA50833A/fr

Links

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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • 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
    • 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]
    • 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
    • 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]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Dermatology (AREA)
  • Dispersion Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
MA050833A 2017-10-17 2018-10-17 Compositions et méthodes pour l'édition génique pour l'hémophilie a MA50833A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762573633P 2017-10-17 2017-10-17

Publications (1)

Publication Number Publication Date
MA50833A true MA50833A (fr) 2020-08-26

Family

ID=64267913

Family Applications (1)

Application Number Title Priority Date Filing Date
MA050833A MA50833A (fr) 2017-10-17 2018-10-17 Compositions et méthodes pour l'édition génique pour l'hémophilie a

Country Status (13)

Country Link
US (3) US20220080055A9 (fr)
EP (1) EP3697907A1 (fr)
JP (2) JP7482028B2 (fr)
KR (1) KR102728416B1 (fr)
CN (1) CN111684070A (fr)
AU (1) AU2018353012B2 (fr)
BR (1) BR112020007502A2 (fr)
CA (1) CA3079172A1 (fr)
IL (1) IL273999A (fr)
MA (1) MA50833A (fr)
MX (1) MX2020004043A (fr)
SG (1) SG11202003464VA (fr)
WO (1) WO2019079527A1 (fr)

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WO2019014564A1 (fr) 2017-07-14 2019-01-17 Editas Medicine, Inc. Systèmes et procédés d'intégration ciblée et d'édition du génome et détection de celle-ci à l'aide de sites d'amorçage intégrés
US20210324381A1 (en) * 2018-04-27 2021-10-21 Seattle Children's Hospital (dba Seattle Children's Research Institute) Therapeutic genome editing in x-linked hyper igm syndrome
CA3116885A1 (fr) * 2018-10-17 2020-04-23 Crispr Therapeutics Ag Compositions et methodes d'administration de transgene
BR112021007301A2 (pt) 2018-10-18 2021-07-27 Intellia Therapeutics, Inc. composições e métodos para expressar fator ix
SG11202103733SA (en) * 2018-10-18 2021-05-28 Intellia Therapeutics Inc Compositions and methods for transgene expression from an albumin locus
EP3923992A1 (fr) 2019-02-15 2021-12-22 CRISPR Therapeutics AG Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée
CN113710284A (zh) * 2019-06-05 2021-11-26 克里斯珀医疗股份公司 具有改善的因子viii表达的血友病a基因编辑
CA3137764A1 (fr) 2019-06-07 2020-12-10 Regeneron Pharmaceuticals, Inc. Animaux non humains comprenant un locus d'albumine humanise
WO2021043278A1 (fr) * 2019-09-04 2021-03-11 博雅辑因（北京）生物科技有限公司 Procédé d'évaluation d'une thérapie d'édition génique sur la base d'une évaluation hors cible
WO2021083073A1 (fr) * 2019-10-31 2021-05-06 华东师范大学 Produit pour le traitement d'une maladie à base du gène alb hépatocytaire
US12139705B2 (en) * 2019-11-04 2024-11-12 California Institute Of Technology Deep learning enabled spatial optical barcodes for pooled library screens
JP7756639B2 (ja) 2019-11-08 2025-10-20 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｘ連鎖性若年網膜分離療法のためのｃｒｉｓｐｒおよびａａｖ戦略
EP4110406A4 (fr) * 2020-02-28 2024-02-28 Blueallele, LLC Méthodes de traitement de troubles liés à un gain de fonction associant l'édition de gènes et la thérapie génique
EP4114932A4 (fr) 2020-03-06 2024-03-13 Metagenomi, Inc. Systèmes crispr de classe ii, de type v
CN116200368A (zh) * 2021-11-30 2023-06-02 上海科技大学 一种基于c2c9核酸酶的新型基因组编辑系统及其应用
EP4490299A1 (fr) * 2022-03-07 2025-01-15 Inedita Bio, Inc. Procédés et compositions pour l'expression médiée par intron d'éléments régulateurs pour le développement de caractères
CN114591957B (zh) * 2022-03-22 2024-04-26 吴文书 一种重症a型血友病动物模型的构建方法及其应用
US20250250322A1 (en) * 2022-04-25 2025-08-07 The Children's Hospital Of Philadelphia Compositions and methods for modulating factor viii function
KR20250021327A (ko) 2022-05-25 2025-02-12 메타지노미, 인크. 간 효소 발현의 보충
WO2024254519A2 (fr) * 2023-06-08 2024-12-12 Mammoth Biosciences, Inc. Compositions et procédés pour la modification de gènes humains exprimés par des cellules hépatiques
WO2025117858A1 (fr) * 2023-11-28 2025-06-05 Metagenomi, Inc. Supplémentation d'expression d'enzyme hépatique
EP4574975A1 (fr) * 2023-12-20 2025-06-25 Bayer AG Compositions et procédés d'administration de transgènes
WO2025153529A1 (fr) * 2024-01-16 2025-07-24 Novo Nordisk A/S Séquence de vecteur viral adéno-associé

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