SG11201706766WA - Materials and methods for treatment of hemoglobinopathies - Google Patents

Materials and methods for treatment of hemoglobinopathies

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Publication number
SG11201706766WA
SG11201706766WA SG11201706766WA SG11201706766WA SG11201706766WA SG 11201706766W A SG11201706766W A SG 11201706766WA SG 11201706766W A SG11201706766W A SG 11201706766WA SG 11201706766W A SG11201706766W A SG 11201706766WA SG 11201706766W A SG11201706766W A SG 11201706766WA
Authority
SG
Singapore
Prior art keywords
hemoglobinopathies
treatment
materials
methods
Prior art date
Application number
SG11201706766WA
Inventor
Matthew Hebden Porteus
Original Assignee
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 Crispr Therapeutics Ag filed Critical Crispr Therapeutics Ag
Publication of SG11201706766WA publication Critical patent/SG11201706766WA/en

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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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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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]
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    • C12N2320/10—Applications; Uses in screening processes
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
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    • C12N2510/00—Genetically modified cells

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  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Hematology (AREA)
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  • Plant Pathology (AREA)
  • Virology (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Transplantation (AREA)
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SG11201706766WA 2015-02-23 2016-02-23 Materials and methods for treatment of hemoglobinopathies SG11201706766WA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562119754P 2015-02-23 2015-02-23
PCT/IB2016/000276 WO2016135557A2 (en) 2015-02-23 2016-02-23 Materials and methods for treatment of hemoglobinopathies

Publications (1)

Publication Number Publication Date
SG11201706766WA true SG11201706766WA (en) 2017-09-28

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SG11201706767RA SG11201706767RA (en) 2015-02-23 2016-02-23 Materials and methods for treatment of hemoglobinopathies
SG11201706766WA SG11201706766WA (en) 2015-02-23 2016-02-23 Materials and methods for treatment of hemoglobinopathies

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SG11201706767RA SG11201706767RA (en) 2015-02-23 2016-02-23 Materials and methods for treatment of hemoglobinopathies

Country Status (8)

Country Link
US (3) US20180030438A1 (en)
EP (2) EP3262171A2 (en)
CN (2) CN107532168A (en)
AU (2) AU2016225178B2 (en)
BR (2) BR112017017812A2 (en)
CA (2) CA2977447A1 (en)
SG (2) SG11201706767RA (en)
WO (2) WO2016135558A2 (en)

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