HRP20211706T1 - Nukleazom posredovana regulacija ekspresije gena - Google Patents

Nukleazom posredovana regulacija ekspresije gena Download PDF

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Publication number
HRP20211706T1
HRP20211706T1 HRP20211706TT HRP20211706T HRP20211706T1 HR P20211706 T1 HRP20211706 T1 HR P20211706T1 HR P20211706T T HRP20211706T T HR P20211706TT HR P20211706 T HRP20211706 T HR P20211706T HR P20211706 T1 HRP20211706 T1 HR P20211706T1
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Croatia
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cell
genetically modified
pharmaceutical preparation
stem cell
hemoglobinopathy
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HRP20211706TT
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English (en)
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Stuart H. Orkin
Andreas Reik
Fyodor Urnov
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The Children's Medical Center Corporation
Sangamo Therapeutics, Inc.
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Application filed by The Children's Medical Center Corporation, Sangamo Therapeutics, Inc. filed Critical The Children's Medical Center Corporation
Publication of HRP20211706T1 publication Critical patent/HRP20211706T1/hr

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    • C12N9/22—Ribonucleases RNAses, DNAses
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    • 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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
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    • 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
    • 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
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • 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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    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • Physics & Mathematics (AREA)
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  • Toxicology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)

Claims (11)

1. Genetski modificirana stanica koja sadrži delecije i/ili insercije u endogenoj sekvenci pojačivača BCL11a koju tvori nukleaza koja sadrži domenu cijepanja i domenu koja veže DNA koja se veže na ciljno mjesto koje sadrži najmanje 9 parova baza od SEQ ID NO:71 do 74.
2. Genetski modificirana stanica prema zahtjevu 1, naznačena time što je stanica matična stanica.
3. Genetski modificirana stanica prema patentnom zahtjevu 2, naznačena time što je matična stanica krvotvorna matična stanica, kao što je CD34+ krvotvorna matična stanica.
4. Genetski modificirana diferencirana stanica koja potječe od matične stanice prema patentnom zahtjevu 2 ili 3.
5. Genetski modificirana diferencirana stanica prema patentnom zahtjevu 4, naznačena time što je stanica crvena krvna stanica (RBC).
6. Genetski modificirana stanica prema bilo kojem od patentnih zahtjeva 1 do 5, naznačena time što domena koja veže DNA sadrži protein cinkovog prsta (ZFP), protein TAL-efektorske domene ili jednu vodeću RNK sustava CRISPR/Cas.
7. Farmaceutski pripravak naznačen time što sadrži genetski modificiranu stanicu prema bilo kojem od patentnih zahtjeva 1 do 6.
8. Farmaceutski pripravak prema patentnom zahtjevu 7 naznačen time što je za uporabu u liječenju ili prevenciji hemoglobinopatije.
9. Farmaceutski pripravak prema patentnom zahtjevu 7 naznačen time što je za uporabu u postupku liječenja ili sprječavanja hemoglobinopatije kod pacijenata kojima je potrebno povećanje ekspresije gena globina, pri čemu se postupak sastoji u davanju pacijentu farmaceutskog pripravka u količini dovoljnoj za povećanje ekspresije gena globina kod pacijenata.
10. Farmaceutski pripravak za uporabu u skladu s patentnim zahtjevom 9, naznačen time što je ekspresija gena globina razina proizvedene globinske mRNA ili globinskog proteina.
11. Farmaceutski pripravak za uporabu prema bilo kojem od patentnih zahtjeva 8-10, naznačen time što je hemoglobinopatija, talasemija ili bolest srpastih stanica, kao što je β-talasemija.
HRP20211706TT 2013-11-13 2014-11-13 Nukleazom posredovana regulacija ekspresije gena HRP20211706T1 (hr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361903823P 2013-11-13 2013-11-13
US201462042075P 2014-08-26 2014-08-26
EP18214711.6A EP3492593B1 (en) 2013-11-13 2014-11-13 Nuclease-mediated regulation of gene expression

Publications (1)

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HRP20211706T1 true HRP20211706T1 (hr) 2022-02-04

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Application Number Title Priority Date Filing Date
HRP20211706TT HRP20211706T1 (hr) 2013-11-13 2014-11-13 Nukleazom posredovana regulacija ekspresije gena
HRP20190825TT HRP20190825T1 (hr) 2013-11-13 2019-05-02 Regulacija genske ekspresije posredovana nukleazama

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HRP20190825TT HRP20190825T1 (hr) 2013-11-13 2019-05-02 Regulacija genske ekspresije posredovana nukleazama

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US (2) US11021696B2 (hr)
EP (3) EP3960856A1 (hr)
CY (1) CY1121630T1 (hr)
DK (2) DK3068881T3 (hr)
ES (2) ES2721936T3 (hr)
HR (2) HRP20211706T1 (hr)
HU (2) HUE044540T2 (hr)
LT (2) LT3068881T (hr)
PL (2) PL3492593T3 (hr)
PT (2) PT3068881T (hr)
RS (2) RS58671B1 (hr)
SI (2) SI3068881T1 (hr)
WO (1) WO2015073683A2 (hr)

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