MX2020002500A - Adn de extremo cerrado modificado (adnec). - Google Patents

Adn de extremo cerrado modificado (adnec).

Info

Publication number
MX2020002500A
MX2020002500A MX2020002500A MX2020002500A MX2020002500A MX 2020002500 A MX2020002500 A MX 2020002500A MX 2020002500 A MX2020002500 A MX 2020002500A MX 2020002500 A MX2020002500 A MX 2020002500A MX 2020002500 A MX2020002500 A MX 2020002500A
Authority
MX
Mexico
Prior art keywords
cedna
cedna vectors
linear
vectors
ended dna
Prior art date
Application number
MX2020002500A
Other languages
English (en)
Inventor
Robert Michael Kotin
Ozan Alkan
Annaliese Jones
Douglas Anthony Kerr
Ara Karl Malakian
Matthew John Simmons
Teresa L Wright
Original Assignee
Generation Bio Co
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 Generation Bio Co filed Critical Generation Bio Co
Publication of MX2020002500A publication Critical patent/MX2020002500A/es

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/64General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/66General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/14011Baculoviridae
    • C12N2710/14111Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141Use of virus, viral particle or viral elements as a vector
    • C12N2710/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00Nucleic acids vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/60Vectors comprising a special translation-regulating system from viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

La presente invención se refiere a vectores de ADNec que tienen estructura lineal y continua pueden ser producidos en altos rendimientos y usados para la transferencia y expresión efectiva de un transgén. Los vectores de ADNec comprenden un casete de expresión y dos diferentes secuencias de ITR derivadas de genomas de AAV en un orden especificado. Algunos vectores de ADNec proporcionados en la presente comprenden además, elementos cis-reguladores y proporcionan eficiencias de alta expresión de gen. Además se proporciona en la presente, métodos y líneas celulares para la producción confiable y eficiente de vectores de ADN lineales, continuos y libres de cápsido.
MX2020002500A 2017-09-08 2018-09-07 Adn de extremo cerrado modificado (adnec). MX2020002500A (es)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762556329P 2017-09-08 2017-09-08
US201762556331P 2017-09-08 2017-09-08
US201762556324P 2017-09-08 2017-09-08
US201762556281P 2017-09-08 2017-09-08
US201762556335P 2017-09-08 2017-09-08
US201762556319P 2017-09-08 2017-09-08
PCT/US2018/049996 WO2019051255A1 (en) 2017-09-08 2018-09-07 CLOSED-ENDED DNA (CEDNA) MODIFIED

Publications (1)

Publication Number Publication Date
MX2020002500A true MX2020002500A (es) 2020-09-17

Family

ID=65635235

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020002500A MX2020002500A (es) 2017-09-08 2018-09-07 Adn de extremo cerrado modificado (adnec).

Country Status (14)

Country Link
US (1) US20200283794A1 (es)
EP (1) EP3678710A4 (es)
JP (2) JP2020532981A (es)
KR (1) KR20200051011A (es)
CN (1) CN111132699A (es)
AU (1) AU2018327348A1 (es)
BR (1) BR112020004151A2 (es)
CA (1) CA3075168A1 (es)
IL (1) IL272797A (es)
MA (1) MA50100A (es)
MX (1) MX2020002500A (es)
PH (1) PH12020500465A1 (es)
SG (1) SG11202000698SA (es)
WO (1) WO2019051255A1 (es)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US9725710B2 (en) 2014-01-08 2017-08-08 Flodesign Sonics, Inc. Acoustophoresis device with dual acoustophoretic chamber
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
AU2018378672A1 (en) * 2017-12-06 2020-07-09 Generation Bio Co. Gene editing using a modified closed-ended dna (ceDNA)
CN114900773A (zh) 2017-12-14 2022-08-12 弗洛设计声能学公司 声泳系统及其操作方法、控制声换能器及声学系统的方法
MA51842A (fr) * 2018-02-14 2020-12-23 Generation Bio Co Vecteurs d'adn non viraux et utilisations associées pour la production d'anticorps et de protéines de fusion
GB201905651D0 (en) 2019-04-24 2019-06-05 Lightbio Ltd Nucleic acid constructs and methods for their manufacture
US20220228171A1 (en) * 2019-07-17 2022-07-21 Generation Bio Co. Compositions and production of nicked closed-ended dna vectors
CA3147414A1 (en) * 2019-07-17 2021-01-21 Generation Bio Co. Synthetic production of single-stranded adeno associated viral dna vectors
EP4025196A4 (en) * 2019-09-06 2023-07-12 Generation Bio Co. LIPID-NANOPARTICLE COMPOSITIONS WITH CLOSED-END DNA AND CLEAVABLE LIPIDS AND METHODS OF USE
EP4031168A4 (en) * 2019-09-18 2023-10-11 Intergalactic Therapeutics, Inc. SYNTHETIC DNA VECTORS AND METHODS OF USE
WO2021072031A1 (en) * 2019-10-11 2021-04-15 Insideoutbio, Inc. Methods and compositions for the manufacture and use of circular dna encoded therapeutics for genetic disorders and other diseases
WO2021169167A1 (en) * 2020-02-29 2021-09-02 Nanjing GenScript Biotech Co., Ltd. Method for treating coronavirus infections
WO2022023284A1 (en) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
BR112023002395A2 (pt) 2020-08-23 2023-03-21 Bioverativ Therapeutics Inc Sistema de baculovírus modificado para produção aprimorada de dna com extremidades fechadas (cedna)
US20240026374A1 (en) * 2020-09-16 2024-01-25 Generation Bio Co. Closed-ended dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah)
GB202014751D0 (en) * 2020-09-18 2020-11-04 Lightbio Ltd Targeting vector
WO2022232029A2 (en) * 2021-04-26 2022-11-03 University Of Florida Research Foundation, Incorporated Synthetic aav vectors for repeated delivery of therapeutic genes
WO2022232289A1 (en) * 2021-04-27 2022-11-03 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
WO2022232286A1 (en) * 2021-04-27 2022-11-03 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
WO2022236014A1 (en) * 2021-05-07 2022-11-10 Generation Bio Co. Non-viral dna vectors for vaccine delivery
AU2022269664A1 (en) * 2021-05-07 2023-11-30 Generation Bio Co. Lyophilized non-viral dna vector compositions and uses thereof
WO2023177655A1 (en) 2022-03-14 2023-09-21 Generation Bio Co. Heterologous prime boost vaccine compositions and methods of use
CN117802161A (zh) * 2022-06-30 2024-04-02 苏州吉恒基因科技有限公司 精准重组腺相关病毒载体及其用途
WO2024040222A1 (en) * 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
US11993783B1 (en) 2023-03-27 2024-05-28 Genecraft Inc. Nucleic acid molecule comprising asymmetrically modified ITR for improving expression rate of inserted gene, and use thereof

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050019824A1 (en) * 1994-03-08 2005-01-27 Human Genome Sciences, Inc. Fibroblast Growth Factor-10
US20050026838A1 (en) * 1995-06-05 2005-02-03 Human Genome Sciences, Inc. Fibroblast Growth Factor-13
DE10066104A1 (de) * 2000-09-08 2003-01-09 Medigene Ag Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung
WO2005072364A2 (en) * 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
EP1979485A2 (en) * 2006-01-31 2008-10-15 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
EP2500434A1 (en) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
AU2013281328B2 (en) * 2012-06-27 2017-11-23 Meiragtx Uk Ii Limited Combination for treating an inflammatory disorder
CN104087613B (zh) * 2014-06-30 2017-08-29 中国科学院苏州生物医学工程技术研究所 基于aav‑itr的基因表达微载体及其构建方法和应用
CN111569090A (zh) * 2016-01-15 2020-08-25 美国基因技术国际有限公司 用于活化γ-δ T细胞的方法和组合物
DK3423110T3 (da) * 2016-03-03 2021-11-15 Univ Massachusetts Lineært duplex-dna med lukket ende til ikke-viral genoverførsel

Also Published As

Publication number Publication date
JP2022190081A (ja) 2022-12-22
IL272797A (en) 2020-04-30
BR112020004151A2 (pt) 2020-09-08
US20200283794A1 (en) 2020-09-10
EP3678710A1 (en) 2020-07-15
EP3678710A4 (en) 2021-06-09
CA3075168A1 (en) 2019-03-14
SG11202000698SA (en) 2020-03-30
MA50100A (fr) 2020-07-15
KR20200051011A (ko) 2020-05-12
JP2020532981A (ja) 2020-11-19
WO2019051255A1 (en) 2019-03-14
RU2020109904A (ru) 2021-10-08
AU2018327348A1 (en) 2020-02-20
PH12020500465A1 (en) 2021-01-25
CN111132699A (zh) 2020-05-08

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