MX2021000343A - Vesículas para la administración sin trazas de moléculas de arn guía y/o complejos de nucleasa guiado por arn/molécula de arn guía y un método de producción de las mismas. - Google Patents

Vesículas para la administración sin trazas de moléculas de arn guía y/o complejos de nucleasa guiado por arn/molécula de arn guía y un método de producción de las mismas.

Info

Publication number
MX2021000343A
MX2021000343A MX2021000343A MX2021000343A MX2021000343A MX 2021000343 A MX2021000343 A MX 2021000343A MX 2021000343 A MX2021000343 A MX 2021000343A MX 2021000343 A MX2021000343 A MX 2021000343A MX 2021000343 A MX2021000343 A MX 2021000343A
Authority
MX
Mexico
Prior art keywords
guide rna
traceless
vesicles
delivery
production method
Prior art date
Application number
MX2021000343A
Other languages
English (en)
Inventor
Anna Cereseto
Antonio Casini
Gianluca Petris
Original Assignee
Alia Therapeutics S R L
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 Alia Therapeutics S R L filed Critical Alia Therapeutics S R L
Publication of MX2021000343A publication Critical patent/MX2021000343A/es

Links

Classifications

    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • 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 RNAses, DNAses
    • 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 [CRISPRs]
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16023—Virus like particles [VLP]
    • 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
    • C12N2760/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
    • C12N2760/00011—Details
    • C12N2760/20011—Rhabdoviridae
    • C12N2760/20211—Vesiculovirus, e.g. vesicular stomatitis Indiana virus
    • C12N2760/20223—Virus like particles [VLP]
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente invención describe vesículas que llevan ARNg y/o complejos de ribonucleoproteína nucleasa (RNP) guiados por ARN asociado a CRISPR que se administran eficazmente a las células diana a través de proteínas fusogénicas de envoltura.
MX2021000343A 2018-07-10 2019-07-08 Vesículas para la administración sin trazas de moléculas de arn guía y/o complejos de nucleasa guiado por arn/molécula de arn guía y un método de producción de las mismas. MX2021000343A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
IT201800007055 2018-07-10
PCT/IB2019/055805 WO2020012335A1 (en) 2018-07-10 2019-07-08 Vesicles for traceless delivery of guide rna molecules and/or guide rna molecule/rna-guided nuclease complex(es) and a production method thereof

Publications (1)

Publication Number Publication Date
MX2021000343A true MX2021000343A (es) 2021-05-28

Family

ID=63684346

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021000343A MX2021000343A (es) 2018-07-10 2019-07-08 Vesículas para la administración sin trazas de moléculas de arn guía y/o complejos de nucleasa guiado por arn/molécula de arn guía y un método de producción de las mismas.

Country Status (8)

Country Link
US (1) US20210261957A1 (es)
EP (1) EP3820995A1 (es)
JP (1) JP2021524272A (es)
KR (1) KR20210044213A (es)
BR (1) BR112021000408A2 (es)
CA (1) CA3105925A1 (es)
MX (1) MX2021000343A (es)
WO (1) WO2020012335A1 (es)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019051097A1 (en) 2017-09-08 2019-03-14 The Regents Of The University Of California RNA-GUIDED ENDONUCLEASE FUSION POLYPEPTIDES AND METHODS OF USING SAME
WO2020061229A2 (en) 2018-09-18 2020-03-26 Vnv Newco Inc. Arc-based capsids and uses thereof
WO2020206072A1 (en) * 2019-04-03 2020-10-08 University Of Georgia Research Foundation, Inc. Delivery of crispr/mcas9 through extracellular vesicles for genome editing
US20220184225A1 (en) 2019-04-23 2022-06-16 Case Western Reserve University Fusogenic particles and related methods for delivering therapeutic agents to cells
JP2023518395A (ja) 2020-03-19 2023-05-01 インテリア セラピューティクス，インコーポレイテッド 指向性ゲノム編集のための方法及び組成物
US20230365989A1 (en) * 2020-03-20 2023-11-16 The Broad Institute, Inc. Compositions and methods for enhanced lentiviral production
AU2021381397A1 (en) * 2020-11-19 2023-06-15 Wake Forest University Health Sciences Vectors, systems and methods for eukaryotic gene editing
WO2022152746A1 (en) 2021-01-13 2022-07-21 Alia Therapeutics Srl K526d cas9 variants and applications thereof
WO2022261149A2 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
WO2023077148A1 (en) 2021-11-01 2023-05-04 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
WO2023118349A1 (en) 2021-12-21 2023-06-29 Alia Therapeutics Srl Type ii cas proteins and applications thereof
WO2023194359A1 (en) 2022-04-04 2023-10-12 Alia Therapeutics Srl Compositions and methods for treatment of usher syndrome type 2a
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
CN115261340A (zh) * 2022-05-20 2022-11-01 四川大学 一种gh-1噬菌体病毒样颗粒、制备方法及应用
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
WO2024056880A2 (en) 2022-09-16 2024-03-21 Alia Therapeutics Srl Enqp type ii cas proteins and applications thereof
WO2024105162A1 (en) 2022-11-16 2024-05-23 Alia Therapeutics Srl Type ii cas proteins and applications thereof
CN116083488A (zh) * 2023-01-03 2023-05-09 北京镁伽机器人科技有限公司 基因编辑方法、基因编辑方法得到的细胞、基因编辑系统及其应用

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8697359B1 (en) * 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP3008192B1 (en) 2013-06-11 2019-07-17 Takara Bio USA, Inc. Protein enriched microvesicles and methods of making and using the same
WO2015191911A2 (en) * 2014-06-12 2015-12-17 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
US20150376586A1 (en) 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
US9816080B2 (en) * 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
EP3858990A1 (en) 2015-03-03 2021-08-04 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
CA2989830A1 (en) 2015-06-18 2016-12-22 The Broad Institute, Inc. Crispr enzyme mutations reducing off-target effects
WO2017040348A1 (en) 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases
JP7059179B2 (ja) * 2015-10-20 2022-04-25 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 遺伝子操作のための方法及び製品

Also Published As

Publication number Publication date
US20210261957A1 (en) 2021-08-26
EP3820995A1 (en) 2021-05-19
JP2021524272A (ja) 2021-09-13
BR112021000408A2 (pt) 2021-06-29
WO2020012335A1 (en) 2020-01-16
KR20210044213A (ko) 2021-04-22
CA3105925A1 (en) 2020-01-16

Similar Documents

Publication Publication Date Title
MX2021000343A (es) Vesículas para la administración sin trazas de moléculas de arn guía y/o complejos de nucleasa guiado por arn/molécula de arn guía y un método de producción de las mismas.
EP4036079A3 (en) Compounds and compositions for intracellular delivery of agents
MX2019011004A (es) Compuestos y composiciones para la administracion intracelular de agentes terapeuticos.
WO2018209158A3 (en) CRISPR / RNA GUIDED NUCLEASE SYSTEMS AND METHODS
CY1121232T1 (el) Τριαλκυλο κατιονικα λιπιδια και μεθοδοι χρησης αυτων
EP4286012A3 (en) Compounds and compositions for intracellular delivery of therapeutic agents
EA202091486A1 (ru) Способы и композиции для поляризации макрофагов
WO2019006471A3 (en) NOVEL CRISPR RNA TARGETING ENZYMES, SYSTEMS AND USES THEREOF
EP3622062A4 (en) DIRECTED EDITING OF CELLULAR RNA BY NUCLEAR ADMINISTRATION OF CRISPR / CAS9
MX2019005617A (es) Linfocitos infiltrantes de tumores remanentes y metodos de preparacion y uso de los mismos.
MX2021014704A (es) Peptidos y nanoparticulas para suministro intracelular de moleculas editoras de genoma.
MX2017016688A (es) Nuevas enzimas y sistemas crispr.
MX2021005130A (es) Metodos terapeuticos.
WO2017180694A8 (en) Cas9 fusion molecules, gene editing systems, and methods of use thereof
MX2020003413A (es) Preparacion y almacenamiento de formulaciones liposomales de arn adecuadas para terapia.
MX2020006065A (es) Metodos y productos para la produccion y administracion de acido nucleico.
WO2016154127A3 (en) Compositions and methods for treating hypertriglyceridemia
EP4253551A3 (en) Novel crispr dna and rna targeting enzymes and systems
WO2015191693A3 (en) Method for gene editing
MX2018001511A (es) Polimeros furtivos desordenados de forma intrinseca codificados geneticamente para suministro y metodos para usar los mismos.
NZ739133A (en) Cellular targeted active ingredient delivery system
WO2017115128A3 (en) Vector-free delivery of gene editing proteins and compositions to cells and tissues
MX2016013025A (es) Focalizacion de cancer de mama her2+ resistente a trastuzumab con una nanoparticula dirigida de her3.
MX2017009044A (es) Metodo para el tratamiento de malignidades.
ZA202202628B (en) Novel crispr dna targeting enzymes and systems