PE20221123A1 - Endonucleasa de escision dual encapsulada en lipidos para edicion de genes y adn - Google Patents

Endonucleasa de escision dual encapsulada en lipidos para edicion de genes y adn

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Publication number
PE20221123A1
PE20221123A1 PE2021001835A PE2021001835A PE20221123A1 PE 20221123 A1 PE20221123 A1 PE 20221123A1 PE 2021001835 A PE2021001835 A PE 2021001835A PE 2021001835 A PE2021001835 A PE 2021001835A PE 20221123 A1 PE20221123 A1 PE 20221123A1
Authority
PE
Peru
Prior art keywords
endonuclease
excision
lipid
gene
dna editing
Prior art date
Application number
PE2021001835A
Other languages
English (en)
Inventor
David R Edgell
Thomas A Mcmurrough
Brent E Stead
Odisho K Israel
Original Assignee
Specific Biologics Inc
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 Specific Biologics Inc filed Critical Specific Biologics Inc
Publication of PE20221123A1 publication Critical patent/PE20221123A1/es

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    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0078—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N15/87—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0043—Nose
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12R—INDEXING SCHEME ASSOCIATED WITH SUBCLASSES C12C - C12Q, RELATING TO MICROORGANISMS
    • C12R2001/00—Microorganisms ; Processes using microorganisms
    • C12R2001/01—Bacteria or Actinomycetales ; using bacteria or Actinomycetales
    • C12R2001/44—Staphylococcus
    • C12R2001/445—Staphylococcus aureus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12R—INDEXING SCHEME ASSOCIATED WITH SUBCLASSES C12C - C12Q, RELATING TO MICROORGANISMS
    • C12R2001/00—Microorganisms ; Processes using microorganisms
    • C12R2001/01—Bacteria or Actinomycetales ; using bacteria or Actinomycetales
    • C12R2001/46—Streptococcus ; Enterococcus; Lactococcus

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Dispersion Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Pulmonology (AREA)
  • Otolaryngology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Metodos para editar genes mediante la administracion de una nucleasa quimerica a una celula u organismo sin el uso de un vector viral
PE2021001835A 2019-05-03 2020-05-04 Endonucleasa de escision dual encapsulada en lipidos para edicion de genes y adn PE20221123A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962842586P 2019-05-03 2019-05-03
US202063019423P 2020-05-03 2020-05-03
PCT/IB2020/054229 WO2020225719A1 (en) 2019-05-03 2020-05-04 Lipid-encapsulated dual-cleaving endonuclease for dna and gene editing

Publications (1)

Publication Number Publication Date
PE20221123A1 true PE20221123A1 (es) 2022-07-13

Family

ID=73050812

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2021001835A PE20221123A1 (es) 2019-05-03 2020-05-04 Endonucleasa de escision dual encapsulada en lipidos para edicion de genes y adn

Country Status (13)

Country Link
US (3) US20220195404A1 (es)
EP (1) EP3963062A4 (es)
KR (1) KR20220115860A (es)
CN (1) CN114761550A (es)
AU (1) AU2020268959A1 (es)
BR (1) BR112021021979A2 (es)
CA (1) CA3139581A1 (es)
GB (1) GB2600568A (es)
IL (1) IL287788A (es)
MX (1) MX2021013400A (es)
PE (1) PE20221123A1 (es)
SG (1) SG11202112161TA (es)
WO (1) WO2020225719A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3139581A1 (en) * 2019-05-03 2020-11-12 Specific Biologics Inc. Lipid-encapsulated dual-cleaving endonuclease for dna and gene editing
KR20230131178A (ko) * 2020-11-04 2023-09-12 스페시픽 바이오로직스 인크. 변형된 엔도뉴클레아제를 사용한 유전자 편집
WO2022200858A1 (en) * 2021-03-26 2022-09-29 Specific Biologics Inc. Targeting oncogentic mutations with dual-cleaving endonuclease
WO2023154451A1 (en) * 2022-02-10 2023-08-17 Christiana Care Gene Editing Institute, Inc. Methods for lipid nanoparticle delivery of crispr/cas system

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130210151A1 (en) * 2011-11-07 2013-08-15 University Of Western Ontario Endonuclease for genome editing
SG10201912327SA (en) 2012-12-12 2020-02-27 Broad Inst Inc Engineering and Optimization of Improved Systems, Methods and Enzyme Compositions for Sequence Manipulation
RU2721275C2 (ru) * 2012-12-12 2020-05-18 Те Брод Инститьют, Инк. Доставка, конструирование и оптимизация систем, способов и композиций для манипуляции с последовательностями и применения в терапии
WO2014121222A1 (en) * 2013-02-01 2014-08-07 The University Of Western Ontario Endonuclease for genome editing
WO2016125143A1 (en) * 2015-02-02 2016-08-11 Danziger Innovations Ltd. Mdmv based vector for gene expression and silencing
CA2985506A1 (en) * 2015-05-15 2016-11-24 Pioneer Hi-Bred International, Inc. Guide rna/cas endonuclease systems
WO2016205759A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
CA2996982A1 (en) * 2015-09-23 2017-03-30 Universite Laval Modification of the dystrophin gene and uses thereof
US20190225955A1 (en) * 2015-10-23 2019-07-25 President And Fellows Of Harvard College Evolved cas9 proteins for gene editing
RU2018124657A (ru) * 2015-12-09 2020-01-09 Эксижн Биотерапевтикс, Инк. Способы редактирования генов и композиции для устранения риска активации вируса jc и пмл (прогрессирующая мультифокальная лейкоэнцефалопатия) во время иммуносупрессивной терапии
CA3008413A1 (en) * 2015-12-18 2017-06-22 Sangamo Therapeutics, Inc. Targeted disruption of the t cell receptor
WO2017165859A1 (en) * 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
CN114380922A (zh) * 2016-06-15 2022-04-22 中国科学院上海营养与健康研究所 在细胞内产生点突变的融合蛋白、其制备及用途
WO2018136396A2 (en) * 2017-01-18 2018-07-26 Excision Biotherapeutics, Inc. Crisprs
WO2018237369A2 (en) * 2017-06-23 2018-12-27 Vical Incorporated LIPID NANOPARTICLE MEDIA ADMINISTRATION OF PLASMIDIC DNA EXPRESSING CRISPR FOR THE TREATMENT OF CHRONIC INFECTION WITH HEPATITIS B VIRUS
US20190218532A1 (en) * 2017-09-19 2019-07-18 Massachusetts Institute Of Technology Streptococcus Canis Cas9 as a Genome Engineering Platform with Novel PAM Specificity
CN110157727A (zh) * 2017-12-21 2019-08-23 中国科学院遗传与发育生物学研究所 植物碱基编辑方法
CA3139581A1 (en) * 2019-05-03 2020-11-12 Specific Biologics Inc. Lipid-encapsulated dual-cleaving endonuclease for dna and gene editing

Also Published As

Publication number Publication date
CA3139581A1 (en) 2020-11-12
US20220195404A1 (en) 2022-06-23
MX2021013400A (es) 2022-04-20
WO2020225719A1 (en) 2020-11-12
GB2600568A (en) 2022-05-04
GB2600568A8 (en) 2022-05-18
EP3963062A1 (en) 2022-03-09
KR20220115860A (ko) 2022-08-19
US11814658B2 (en) 2023-11-14
IL287788A (en) 2022-01-01
GB202117443D0 (en) 2022-01-19
EP3963062A4 (en) 2023-09-06
SG11202112161TA (en) 2021-12-30
CN114761550A (zh) 2022-07-15
US20230203464A1 (en) 2023-06-29
US20230016280A1 (en) 2023-01-19
AU2020268959A1 (en) 2021-12-09
BR112021021979A2 (pt) 2022-12-06

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