MA55527A - Polynucléotides, compositions et procédés d'expression de polypeptides - Google Patents

Polynucléotides, compositions et procédés d'expression de polypeptides

Info

Publication number
MA55527A
MA55527A MA055527A MA55527A MA55527A MA 55527 A MA55527 A MA 55527A MA 055527 A MA055527 A MA 055527A MA 55527 A MA55527 A MA 55527A MA 55527 A MA55527 A MA 55527A
Authority
MA
Morocco
Prior art keywords
polynucleotides
compositions
methods
expressing polypeptides
polypeptides
Prior art date
Application number
MA055527A
Other languages
English (en)
Inventor
Seth C Alexander
Christian Dombrowski
Bradley Andrew Murray
Original Assignee
Intellia Therapeutics 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 Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Publication of MA55527A publication Critical patent/MA55527A/fr

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
    • 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
    • C12N15/67—General methods for enhancing the expression
    • 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
    • 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
    • C12N15/63—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
    • 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
    • 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
    • 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
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/30—Nucleotides
    • C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/02—Preparation of peptides or proteins having a known sequence of two or more amino acids, e.g. glutathione
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
MA055527A 2019-03-28 2020-03-27 Polynucléotides, compositions et procédés d'expression de polypeptides MA55527A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962825656P 2019-03-28 2019-03-28

Publications (1)

Publication Number Publication Date
MA55527A true MA55527A (fr) 2022-02-09

Family

ID=70416544

Family Applications (1)

Application Number Title Priority Date Filing Date
MA055527A MA55527A (fr) 2019-03-28 2020-03-27 Polynucléotides, compositions et procédés d'expression de polypeptides

Country Status (17)

Country Link
US (1) US20230012687A1 (fr)
EP (1) EP3947670A2 (fr)
JP (1) JP2022527302A (fr)
KR (1) KR20220004649A (fr)
CN (1) CN113993994A (fr)
AU (1) AU2020248470A1 (fr)
BR (1) BR112021019224A2 (fr)
CA (1) CA3135172A1 (fr)
CO (1) CO2021014400A2 (fr)
EA (1) EA202192637A1 (fr)
IL (1) IL286579A (fr)
MA (1) MA55527A (fr)
MX (1) MX2021011757A (fr)
PH (1) PH12021552299A1 (fr)
SG (1) SG11202110135YA (fr)
TW (1) TW202102529A (fr)
WO (1) WO2020198641A2 (fr)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3204496A1 (fr) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions et procédés pour activer une réparation dirigée par homologie
US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
AU2019291918B2 (en) 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
EP4426822A2 (fr) 2021-11-03 2024-09-11 Intellia Therapeutics, Inc. Polynucléotides, compositions et méthodes pour l'édition génomique
WO2023133525A1 (fr) * 2022-01-07 2023-07-13 Precision Biosciences, Inc. Polynucléotides optimisés pour l'expression de protéines
EP4471158A4 (fr) * 2022-01-27 2026-01-14 Sk Bioscience Co Ltd Arnm pour l'expression de protéines et matrice associée
EP4475856A2 (fr) * 2022-02-09 2024-12-18 The Regents of the University of California Traduction de protéines in vitro et in vivo par l'intermédiaire d'arn circularisés in situ
TW202423959A (zh) * 2022-08-24 2024-06-16 美商步行魚醫療公司 用於治療法布瑞氏症(fabry disease)之組成物及方法
AU2024374554A1 (en) 2023-11-10 2026-05-21 Intellia Therapeutics, Inc. Compositions, methods, and systems for genomic editing
WO2025128871A2 (fr) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Nanoparticules lipidiques comprenant des molécules d'arn codant destinées à être utilisées dans l'édition de gènes et en tant que vaccins et agents thérapeutiques
US20260000702A1 (en) 2024-06-26 2026-01-01 Flagship Pioneering Innovations Vii, Llc Therapeutic circular dna forms
WO2026163162A1 (fr) 2025-01-31 2026-08-06 Intellia Therapeutics, Inc. Variants de cas9 améliorés et leurs utilisations

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US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
ATE515510T1 (de) 1991-12-24 2011-07-15 Isis Pharmaceuticals Inc Durch dna-abschnitte unterbrochene modifizierte oligonukleotide
EP0760008A1 (fr) 1994-05-19 1997-03-05 Dako A/S Sondes d'acide nucleique peptidique de detection de neisseria gonorrhoeae et de chlamydia trachomatis
WO2006007712A1 (fr) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methodes permettant de distribuer des agents therapeutiques comprenant des conjugues de lipide-polyethylene glycol
CA2657975A1 (fr) * 2006-06-29 2008-01-03 Dsm Ip Assets B.V. Procede pour obtenir une expression de polypeptides amelioree
EP2668282A2 (fr) * 2011-01-28 2013-12-04 MedImmune, LLC Expression de glycoprotéines de fusion virales solubles dans des cellules de mammifères
PT2931898E (pt) 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais
WO2014093694A1 (fr) 2012-12-12 2014-06-19 The Broad Institute, Inc. Systèmes, procédés et compositions de crispr-nickase cas pour la manipulation de séquences dans les eucaryotes
IL308158A (en) 2012-12-17 2023-12-01 Harvard College Rna-guided human genome engineering
ES2887254T3 (es) 2013-03-08 2021-12-22 Novartis Ag Lípidos y composiciones lipídicas para el suministro de agentes activos
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
US10059655B2 (en) 2013-12-19 2018-08-28 Novartis Ag Lipids and lipid compositions for the delivery of active agents
CN106794141B (zh) 2014-07-16 2021-05-28 诺华股份有限公司 将核酸包封在脂质纳米粒主体中的方法
AU2016226077B2 (en) 2015-03-03 2021-12-23 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
DK4276189T3 (da) 2015-09-21 2026-05-26 Trilink Biotechnologies Llc Initierende oligonukleotidprimere med kappe til syntesering af 5'-ende RNA'er med kappe
SG10201912763QA (en) * 2015-11-05 2020-02-27 Bamboo Therapeutics Inc Modified friedreich ataxia genes and vectors for gene therapy
EP3405579A1 (fr) * 2016-01-22 2018-11-28 Modernatx, Inc. Acides ribonucléiques messagers pour la production de polypeptides de liaison intracellulaires et leurs procédés d'utilisation
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FI3516056T3 (fi) * 2016-09-23 2025-02-28 Dsm Ip Assets Bv Opas-rna:n ilmentämisjärjestelmä isäntäsolulle
WO2018067447A1 (fr) 2016-10-03 2018-04-12 Itellia Therapeutics, Inc. Méthodes améliorées d'identification de sites de rupture de double-brin
IL311278B2 (en) * 2017-09-29 2026-05-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing
TWI904069B (zh) * 2017-09-29 2025-11-11 美商英特利亞醫療公司 用於ttr基因編輯及治療attr澱粉樣變性之組合物及方法

Also Published As

Publication number Publication date
WO2020198641A2 (fr) 2020-10-01
AU2020248470A1 (en) 2021-11-11
CN113993994A (zh) 2022-01-28
IL286579A (en) 2021-10-31
BR112021019224A2 (pt) 2021-11-30
CO2021014400A2 (es) 2021-11-19
TW202102529A (zh) 2021-01-16
KR20220004649A (ko) 2022-01-11
SG11202110135YA (en) 2021-10-28
EA202192637A1 (ru) 2022-03-18
CA3135172A1 (fr) 2020-10-01
JP2022527302A (ja) 2022-06-01
EP3947670A2 (fr) 2022-02-09
US20230012687A1 (en) 2023-01-19
MX2021011757A (es) 2021-12-10
WO2020198641A3 (fr) 2020-11-05
PH12021552299A1 (en) 2022-08-22

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