BR112017017812A2 - materiais e métodos para tratamento de hemoglobinopatias - Google Patents

materiais e métodos para tratamento de hemoglobinopatias

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Publication number
BR112017017812A2
BR112017017812A2 BR112017017812A BR112017017812A BR112017017812A2 BR 112017017812 A2 BR112017017812 A2 BR 112017017812A2 BR 112017017812 A BR112017017812 A BR 112017017812A BR 112017017812 A BR112017017812 A BR 112017017812A BR 112017017812 A2 BR112017017812 A2 BR 112017017812A2
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BR
Brazil
Prior art keywords
methods
hemoglobinopathies
materials
cells
hbf
Prior art date
Application number
BR112017017812A
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English (en)
Inventor
Gang Bao
Thomas James Cradick
Ciaran M Lee
Matthew Hebden Porteus
Original Assignee
Crispr Therapeutics Ag
Georgia Tech Res Inst
Univ Rice William M
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.)
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Publication date
Application filed by Crispr Therapeutics Ag, Georgia Tech Res Inst, Univ Rice William M filed Critical Crispr Therapeutics Ag
Publication of BR112017017812A2 publication Critical patent/BR112017017812A2/pt

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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
    • 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
    • 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
    • A61K35/48—Reproductive organs
    • A61K35/54—Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
    • A61K35/545—Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
    • 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
    • A61K38/41—Porphyrin- or corrin-ring-containing peptides
    • A61K38/42—Haemoglobins; Myoglobins
    • 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
    • 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
    • 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
    • A61P7/06—Antianaemics
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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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
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    • 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
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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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 [CRISPRs]
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/70—Enzymes
    • C12N2501/73—Hydrolases (EC 3.)
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    • C12N2510/00—Genetically modified cells

Abstract

a presente invenção refere-se a materiais e métodos para tratamento de hemoglobinopatias. mais especificamente, o pedido refere-se a métodos para produzir células progenitoras que são geneticamente modificadas via edição de genoma para aumentar a produção de hemoglobina fetal (hbf), bem como células progenitoras modificadas (incluindo, por exemplo, células tronco hematopoiéticas cd34+) que produzem níveis aumentados de hbf, e métodos de utilização de tais células para tratamento de hemoglobinopatias tais como anemia de célula falciforme e ß-talassemia.
BR112017017812A 2015-02-23 2016-02-23 materiais e métodos para tratamento de hemoglobinopatias BR112017017812A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562119754P 2015-02-23 2015-02-23
PCT/IB2016/000282 WO2016135558A2 (en) 2015-02-23 2016-02-23 Materials and methods for treatment of hemoglobinopathies

Publications (1)

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BR112017017812A2 true BR112017017812A2 (pt) 2018-04-10

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BR112017017812A BR112017017812A2 (pt) 2015-02-23 2016-02-23 materiais e métodos para tratamento de hemoglobinopatias
BR112017017810A BR112017017810A2 (pt) 2015-02-23 2016-02-23 materiais e métodos para tratamento de hemoglobinopatias

Family Applications After (1)

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US (3) US20180030438A1 (pt)
EP (2) EP3262172A2 (pt)
CN (2) CN107532168A (pt)
AU (2) AU2016225178B2 (pt)
BR (2) BR112017017812A2 (pt)
CA (2) CA2977447A1 (pt)
SG (2) SG11201706767RA (pt)
WO (2) WO2016135557A2 (pt)

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