SG10201710487VA - Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Targeting Disorders and Diseases Using Viral Components - Google Patents

Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Targeting Disorders and Diseases Using Viral Components

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Publication number
SG10201710487VA
SG10201710487VA SG10201710487VA SG10201710487VA SG10201710487VA SG 10201710487V A SG10201710487V A SG 10201710487VA SG 10201710487V A SG10201710487V A SG 10201710487VA SG 10201710487V A SG10201710487V A SG 10201710487VA SG 10201710487V A SG10201710487V A SG 10201710487VA
Authority
SG
Singapore
Prior art keywords
crispr
diseases
delivery
compositions
therapeutic applications
Prior art date
Application number
SG10201710487VA
Other languages
English (en)
Inventor
Zhang Feng
Cong Le
Ran Fei
HEIDENREICH Matthias
Swiech Lukasz
Original Assignee
Broad Inst Inc
Massachusetts Inst Technology
Harvard College
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=52105124&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=SG10201710487V(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from PCT/US2013/074667 external-priority patent/WO2014093622A2/en
Application filed by Broad Inst Inc, Massachusetts Inst Technology, Harvard College filed Critical Broad Inst Inc
Publication of SG10201710487VA publication Critical patent/SG10201710487VA/en

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    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1082—Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, 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
    • C12N15/11—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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    • C12N15/00—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
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    • C12N15/09—Recombinant DNA-technology
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    • 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
    • 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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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
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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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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)

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  • Chemical & Material Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Biodiversity & Conservation Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
SG10201710487VA 2013-06-17 2014-06-11 Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Targeting Disorders and Diseases Using Viral Components SG10201710487VA (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201361836123P 2013-06-17 2013-06-17
US201361847537P 2013-07-17 2013-07-17
US201361862355P 2013-08-05 2013-08-05
US201361871301P 2013-08-28 2013-08-28
US201361915225P 2013-12-12 2013-12-12
PCT/US2013/074667 WO2014093622A2 (en) 2012-12-12 2013-12-12 Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US201461979879P 2014-04-15 2014-04-15

Publications (1)

Publication Number Publication Date
SG10201710487VA true SG10201710487VA (en) 2018-01-30

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SG11201510286QA SG11201510286QA (en) 2013-06-17 2014-06-11 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
SG10201710487VA SG10201710487VA (en) 2013-06-17 2014-06-11 Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Targeting Disorders and Diseases Using Viral Components

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US (2) US10946108B2 (OSRAM)
EP (2) EP3011034B1 (OSRAM)
JP (1) JP6702858B2 (OSRAM)
KR (2) KR20160056869A (OSRAM)
CN (2) CN114015726B (OSRAM)
AU (1) AU2014281031B2 (OSRAM)
BR (1) BR122021009076B1 (OSRAM)
CA (1) CA2915795C (OSRAM)
IL (1) IL243195B (OSRAM)
MX (1) MX374532B (OSRAM)
RU (1) RU2716421C2 (OSRAM)
SG (2) SG11201510286QA (OSRAM)
WO (1) WO2014204729A1 (OSRAM)
ZA (1) ZA201508961B (OSRAM)

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US11643652B2 (en) 2019-03-19 2023-05-09 The Broad Institute, Inc. Methods and compositions for prime editing nucleotide sequences
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
US11932884B2 (en) 2017-08-30 2024-03-19 President And Fellows Of Harvard College High efficiency base editors comprising Gam
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US12473573B2 (en) 2013-09-06 2025-11-18 President And Fellows Of Harvard College Switchable Cas9 nucleases and uses thereof

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KR102479178B1 (ko) 2012-12-06 2022-12-19 시그마-알드리치 컴퍼니., 엘엘씨 Crispr-기초된 유전체 변형과 조절
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EP3597755A1 (en) 2020-01-22
KR20160056869A (ko) 2016-05-20
AU2014281031B2 (en) 2020-05-21
CN105793425B (zh) 2021-10-26
SG11201510286QA (en) 2016-01-28
EP3011034B1 (en) 2019-08-07
US20160175462A1 (en) 2016-06-23
BR112015031610A2 (pt) 2017-08-22
JP2016521995A (ja) 2016-07-28
CN114015726A (zh) 2022-02-08
IL243195B (en) 2020-06-30
ZA201508961B (en) 2025-08-27
RU2716421C2 (ru) 2020-03-11
MX374532B (es) 2025-03-06
BR122021009076B1 (pt) 2024-02-15
EP3011034A1 (en) 2016-04-27

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