IL274526B2 - Targeted crispr delivery platforms - Google Patents

Targeted crispr delivery platforms

Info

Publication number
IL274526B2
IL274526B2 IL274526A IL27452620A IL274526B2 IL 274526 B2 IL274526 B2 IL 274526B2 IL 274526 A IL274526 A IL 274526A IL 27452620 A IL27452620 A IL 27452620A IL 274526 B2 IL274526 B2 IL 274526B2
Authority
IL
Israel
Prior art keywords
nucleotides
sgrna
product
truncated
tracrrna
Prior art date
Application number
IL274526A
Other languages
English (en)
Hebrew (he)
Other versions
IL274526A (en
IL274526B1 (en
Inventor
Erik Joseph Sontheimer
Raed Ibraheim
Wen Xue
Aamir Mir
Alireza Edraki
Gainetdinov Ildar
Original Assignee
Univ Massachusetts
Erik Joseph Sontheimer
Raed Ibraheim
Wen Xue
Aamir Mir
Alireza Edraki
Gainetdinov Ildar
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 Univ Massachusetts, Erik Joseph Sontheimer, Raed Ibraheim, Wen Xue, Aamir Mir, Alireza Edraki, Gainetdinov Ildar filed Critical Univ Massachusetts
Publication of IL274526A publication Critical patent/IL274526A/en
Publication of IL274526B1 publication Critical patent/IL274526B1/en
Publication of IL274526B2 publication Critical patent/IL274526B2/en

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • C12N15/86—Viral vectors
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • 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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Manufacturing & Machinery (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
IL274526A 2017-11-10 2018-11-09 Targeted crispr delivery platforms IL274526B2 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762584310P 2017-11-10 2017-11-10
US201762596375P 2017-12-08 2017-12-08
US201862667084P 2018-05-04 2018-05-04
PCT/US2018/060126 WO2019094791A2 (en) 2017-11-10 2018-11-09 Targeted crispr delivery platforms

Publications (3)

Publication Number Publication Date
IL274526A IL274526A (en) 2020-06-30
IL274526B1 IL274526B1 (en) 2025-01-01
IL274526B2 true IL274526B2 (en) 2025-05-01

Family

ID=66438139

Family Applications (2)

Application Number Title Priority Date Filing Date
IL274526A IL274526B2 (en) 2017-11-10 2018-11-09 Targeted crispr delivery platforms
IL317505A IL317505A (en) 2017-11-10 2018-11-09 CRISPR-directed delivery platforms

Family Applications After (1)

Application Number Title Priority Date Filing Date
IL317505A IL317505A (en) 2017-11-10 2018-11-09 CRISPR-directed delivery platforms

Country Status (13)

Country Link
US (3) US20220389447A9 (enExample)
EP (1) EP3707254A4 (enExample)
JP (2) JP2021502097A (enExample)
KR (1) KR20200080314A (enExample)
CN (1) CN111868240A (enExample)
AU (2) AU2018364993B2 (enExample)
BR (1) BR112020009268A2 (enExample)
CA (1) CA3082370A1 (enExample)
CO (1) CO2020007046A2 (enExample)
IL (2) IL274526B2 (enExample)
MX (1) MX2020004777A (enExample)
SG (1) SG11202005103RA (enExample)
WO (1) WO2019094791A2 (enExample)

Families Citing this family (18)

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EP3868887A4 (en) * 2018-09-25 2022-12-07 Microbial Chemistry Research Foundation NOVEL VIRAL VECTOR AND METHODS OF PRODUCTION AND USE THEREOF
CA3129055A1 (en) 2019-02-05 2020-08-13 AMMR Joint Venture Enhanced selection of efficient targeted genome manipulating agents
EP3924477A4 (en) * 2019-02-14 2023-03-29 Metagenomi, Inc. ENZYMES WITH RUVC DOMAINS
CN110257406B (zh) * 2019-06-21 2022-07-22 安徽省农业科学院水稻研究所 密码子植物化改造的Plant Nme2Cas9基因及其应用
US12297426B2 (en) 2019-10-01 2025-05-13 The Broad Institute, Inc. DNA damage response signature guided rational design of CRISPR-based systems and therapies
US20230054569A1 (en) 2019-12-18 2023-02-23 Alia Therapeutics Srl Compositions and methods for treating retinitis pigmentosa
CN113652411B (zh) * 2021-07-30 2025-01-10 复旦大学 Cas9蛋白、含有Cas9蛋白的基因编辑系统及应用
WO2023028348A1 (en) 2021-08-27 2023-03-02 Metagenomi, Inc. Enzymes with ruvc domains
US20230183687A1 (en) * 2021-10-13 2023-06-15 University Of Massachusetts Modified guide rnas for neisseria meningitidis cas9
MX2024005241A (es) * 2021-11-03 2024-07-02 Intellia Therapeutics Inc Arn guia modificados para la edicion genica.
CA3237303A1 (en) 2021-11-03 2023-05-11 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing
KR20250124819A (ko) 2022-12-21 2025-08-20 인텔리아 테라퓨틱스, 인크. 프로단백질 전환효소 서브틸리신 켁신 9(pcsk9) 편집을 위한 조성물 및 방법
TW202436622A (zh) 2023-03-06 2024-09-16 美商英特利亞醫療公司 用於ｂ型肝炎病毒（ｈｂｖ）基因體編輯之組合物及方法
WO2025038648A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
WO2025038646A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Cd70 car-t compositions and methods for cell-based therapy
WO2025038637A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
WO2025038642A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying cd70
WO2025255308A1 (en) 2024-06-07 2025-12-11 Intellia Therapeutics, Inc. Cd8 co-receptor chimeric polypeptides in tcr cell therapy

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US20160177278A1 (en) * 2014-12-22 2016-06-23 University Of Massachusetts Cas9-DNA Targeting Unit Chimeras
US20160346359A1 (en) * 2015-05-01 2016-12-01 Spark Therapeutics, Inc. Adeno-associated Virus-Mediated CRISPR-Cas9 Treatment of Ocular Disease

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JP6718813B2 (ja) * 2013-07-10 2020-07-08 プレジデント アンド フェローズ オブ ハーバード カレッジ Ｒｎａ誘導性遺伝子制御および編集のための直交性ｃａｓ９タンパク質
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US20160177278A1 (en) * 2014-12-22 2016-06-23 University Of Massachusetts Cas9-DNA Targeting Unit Chimeras
US20160346359A1 (en) * 2015-05-01 2016-12-01 Spark Therapeutics, Inc. Adeno-associated Virus-Mediated CRISPR-Cas9 Treatment of Ocular Disease

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Also Published As

Publication number Publication date
BR112020009268A2 (pt) 2020-11-17
EP3707254A2 (en) 2020-09-16
WO2019094791A3 (en) 2019-06-20
MX2020004777A (es) 2020-10-08
US20250223611A1 (en) 2025-07-10
AU2023200084B2 (en) 2025-10-16
JP2024019727A (ja) 2024-02-09
WO2019094791A2 (en) 2019-05-16
CO2020007046A2 (es) 2020-08-31
AU2023200084A1 (en) 2023-02-09
US20220389447A9 (en) 2022-12-08
AU2018364993A1 (en) 2020-06-11
EP3707254A4 (en) 2021-08-18
KR20200080314A (ko) 2020-07-06
CN111868240A (zh) 2020-10-30
IL274526A (en) 2020-06-30
IL317505A (en) 2025-02-01
IL274526B1 (en) 2025-01-01
JP2021502097A (ja) 2021-01-28
SG11202005103RA (en) 2020-06-29
AU2018364993B2 (en) 2022-10-06
CA3082370A1 (en) 2019-05-16
US20190338308A1 (en) 2019-11-07
US20250197889A1 (en) 2025-06-19

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