SG11202110135YA - Polynucleotides, compositions, and methods for polypeptide expression - Google Patents

Polynucleotides, compositions, and methods for polypeptide expression

Info

Publication number
SG11202110135YA
SG11202110135YA SG11202110135YA SG11202110135YA SG11202110135YA SG 11202110135Y A SG11202110135Y A SG 11202110135YA SG 11202110135Y A SG11202110135Y A SG 11202110135YA SG 11202110135Y A SG11202110135Y A SG 11202110135YA SG 11202110135Y A SG11202110135Y A SG 11202110135YA
Authority
SG
Singapore
Prior art keywords
polynucleotides
compositions
methods
polypeptide expression
polypeptide
Prior art date
Application number
SG11202110135YA
Other languages
English (en)
Inventor
Bradley Andrew Murray
Christian Dombrowski
Seth C Alexander
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 SG11202110135YA publication Critical patent/SG11202110135YA/en

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    • 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 RNAses, DNAses
    • 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 [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
    • 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)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
SG11202110135YA 2019-03-28 2020-03-27 Polynucleotides, compositions, and methods for polypeptide expression SG11202110135YA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962825656P 2019-03-28 2019-03-28
PCT/US2020/025372 WO2020198641A2 (fr) 2019-03-28 2020-03-27 Polynucléotides, compositions et procédés d'expression de polypeptides

Publications (1)

Publication Number Publication Date
SG11202110135YA true SG11202110135YA (en) 2021-10-28

Family

ID=70416544

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202110135YA SG11202110135YA (en) 2019-03-28 2020-03-27 Polynucleotides, compositions, and methods for polypeptide expression

Country Status (16)

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)
SG (1) SG11202110135YA (fr)
TW (1) TW202102529A (fr)
WO (1) WO2020198641A2 (fr)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3714047A2 (fr) 2017-11-22 2020-09-30 ModernaTX, Inc. Polynucléotides codant pour la phénylalanine hydroxylase pour le traitement de la phénylcétonurie
CA3237303A1 (fr) 2021-11-03 2023-05-11 Intellia Therapeutics, Inc. Polynucleotides, compositions et methodes pour l'edition genomique
WO2023133525A1 (fr) * 2022-01-07 2023-07-13 Precision Biosciences, Inc. Polynucléotides optimisés pour l'expression de protéines
WO2023154749A2 (fr) * 2022-02-09 2023-08-17 The Regents Of The University Of California Traduction de protéines in vitro et in vivo par l'intermédiaire d'arn circularisés in situ
WO2024044697A2 (fr) * 2022-08-24 2024-02-29 Walking Fish Therapeutics, Inc. Compositions et méthodes de traitement de la maladie de fabry

Family Cites Families (20)

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Publication number Priority date Publication date Assignee Title
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
US6169169B1 (en) 1994-05-19 2001-01-02 Dako A/S PNA probes for detection of Neisseria gonorrhoeae and 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
EP2035561A1 (fr) * 2006-06-29 2009-03-18 DSMIP Assets B.V. Procédé pour obtenir une expression de polypeptides améliorée
US8993233B2 (en) 2012-12-12 2015-03-31 The Broad Institute Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
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
EP3553174A1 (fr) 2012-12-17 2019-10-16 President and Fellows of Harvard College Ingénierie de génome humain guidée par arn
EA030650B1 (ru) 2013-03-08 2018-09-28 Новартис Аг Липиды и липидные композиции для доставки активных агентов
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
US10059655B2 (en) 2013-12-19 2018-08-28 Novartis Ag Lipids and lipid compositions for the delivery of active agents
JP6778175B2 (ja) 2014-07-16 2020-10-28 ノバルティス アーゲー 脂質ナノ粒子ホスト中に核酸を封入する方法
CA2978314A1 (fr) 2015-03-03 2016-09-09 The General Hospital Corporation Nucleases crispr-cas9 genetiquement modifiees presentant une specificite pam modifiee
ES2968391T3 (es) 2015-09-21 2024-05-09 Trilink Biotechnologies Llc Cebadores oligonucleotídicos iniciadores con caperuza para sintetizar ARN con caperuza 5'
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
TWI773666B (zh) 2016-03-30 2022-08-11 美商英特利亞醫療公司 Crispr/cas 組分之脂質奈米粒子調配物
CN109715804A (zh) * 2016-09-23 2019-05-03 帝斯曼知识产权资产管理有限公司 用于宿主细胞的指导rna表达系统
WO2018067447A1 (fr) 2016-10-03 2018-04-12 Itellia Therapeutics, Inc. Méthodes améliorées d'identification de sites de rupture de double-brin
IL311278A (en) * 2017-09-29 2024-05-01 Intellia Therapeutics Inc Polynucleotides, preparations, and methods for genome editing

Also Published As

Publication number Publication date
IL286579A (en) 2021-10-31
EA202192637A1 (ru) 2022-03-18
WO2020198641A3 (fr) 2020-11-05
EP3947670A2 (fr) 2022-02-09
JP2022527302A (ja) 2022-06-01
US20230012687A1 (en) 2023-01-19
WO2020198641A2 (fr) 2020-10-01
KR20220004649A (ko) 2022-01-11
MA55527A (fr) 2022-02-09
AU2020248470A1 (en) 2021-11-11
CN113993994A (zh) 2022-01-28
CO2021014400A2 (es) 2021-11-19
CA3135172A1 (fr) 2020-10-01
TW202102529A (zh) 2021-01-16
MX2021011757A (es) 2021-12-10
BR112021019224A2 (pt) 2021-11-30

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