MA50100A - Adn à extrémité fermée (cedna) modifié - Google Patents

Adn à extrémité fermée (cedna) modifié

Info

Publication number
MA50100A
MA50100A MA050100A MA50100A MA50100A MA 50100 A MA50100 A MA 50100A MA 050100 A MA050100 A MA 050100A MA 50100 A MA50100 A MA 50100A MA 50100 A MA50100 A MA 50100A
Authority
MA
Morocco
Prior art keywords
cedna
closed end
end dna
modified closed
modified
Prior art date
Application number
MA050100A
Other languages
English (en)
Inventor
Ozan Alkan
Annaliese Jones
Douglas Anthony Kerr
Robert Michael Kotin
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 MA50100A publication Critical patent/MA50100A/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
    • 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
    • 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
    • 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
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • 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
    • C12N15/66—General 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/60—Vectors 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)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (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)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
MA050100A 2017-09-08 2018-09-07 Adn à extrémité fermée (cedna) modifié MA50100A (fr)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201762556319P 2017-09-08 2017-09-08
US201762556329P 2017-09-08 2017-09-08
US201762556331P 2017-09-08 2017-09-08
US201762556324P 2017-09-08 2017-09-08
US201762556335P 2017-09-08 2017-09-08
US201762556281P 2017-09-08 2017-09-08

Publications (1)

Publication Number Publication Date
MA50100A true MA50100A (fr) 2020-07-15

Family

ID=65635235

Family Applications (1)

Application Number Title Priority Date Filing Date
MA050100A MA50100A (fr) 2017-09-08 2018-09-07 Adn à extrémité fermée (cedna) modifié

Country Status (14)

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

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US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
CN105939767B (zh) 2014-01-08 2018-04-06 弗洛设计声能学公司 具有双声电泳腔的声电泳装置
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
JP2021505159A (ja) * 2017-12-06 2021-02-18 ジェネレーション バイオ カンパニー 修飾型閉端ｄｎａ（ｃｅｄｎａ）を使用する遺伝子編集
BR112020009889A2 (pt) 2017-12-14 2020-11-03 Flodesign Sonics, Inc. acionador e controlador de transdutor acústico
JP2021513355A (ja) * 2018-02-14 2021-05-27 ジェネレーション バイオ カンパニー 非ウイルス性ｄｎａベクター、ならびに抗体および融合タンパク質の産生のためのその使用
KR20210090619A (ko) * 2018-11-09 2021-07-20 제너레이션 바이오 컴퍼니 대칭인 변형된 역말단반복을 포함하는 변형된 폐쇄형 DNA(ceDNA)
GB201905651D0 (en) * 2019-04-24 2019-06-05 Lightbio Ltd Nucleic acid constructs and methods for their manufacture
WO2021011840A1 (fr) * 2019-07-17 2021-01-21 Generation Bio Co. Compositions et production de vecteurs d'adn à extrémités fermées nickelés
CA3147414A1 (fr) * 2019-07-17 2021-01-21 Generation Bio Co. Production synthetique de vecteurs de type adn viraux adeno-associes simple brin
CA3150452A1 (fr) * 2019-09-06 2021-03-11 Generation Bio Co. Compositions de nanoparticules lipidiques comprenant de l'adn a extremites fermees et des lipides clivables et leurs procedes d'utilisation
CN115066255A (zh) * 2019-09-18 2022-09-16 星际治疗有限公司 合成的dna载体和使用方法
WO2021072031A1 (fr) * 2019-10-11 2021-04-15 Insideoutbio, Inc. Procédés et compositions pour la fabrication et l'utilisation d'agents thérapeutiques codés par de l'adn circulaire dans des troubles génétiques et d'autres maladies
WO2021169167A1 (fr) * 2020-02-29 2021-09-02 Nanjing GenScript Biotech Co., Ltd. Méthode de traitement d'infections à coronavirus
MX2023001109A (es) 2020-07-27 2023-04-24 Anjarium Biosciences Ag Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.
MX2023002221A (es) 2020-08-23 2023-03-15 Bioverativ Therapeutics Inc Sistema de baculovirus modificado para produccion mejorada de adn de extremo cerrado (adnce).
WO2022061000A1 (fr) * 2020-09-16 2022-03-24 Generation Bio Co. Vecteurs d'adn à extrémité fermée et utilisations associées pour exprimer de la phénylalanine hydroxylase (pah)
GB202014751D0 (en) * 2020-09-18 2020-11-04 Lightbio Ltd Targeting vector
WO2022232029A2 (fr) * 2021-04-26 2022-11-03 University Of Florida Research Foundation, Incorporated Vecteurs vaa synthétiques pour l'administration répétée de gènes thérapeutiques
US20240216535A1 (en) * 2021-04-27 2024-07-04 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
US20240226324A1 (en) * 2021-04-27 2024-07-11 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
WO2022236016A1 (fr) * 2021-05-07 2022-11-10 Generation Bio Co. Compositions de vecteurs d'adn non viraux lyophilisées et leurs utilisations
KR20240005903A (ko) * 2021-05-07 2024-01-12 제너레이션 바이오 컴퍼니 백신 전달을 위한 비바이러스성 dna 벡터
CA3235493A1 (fr) * 2021-10-18 2023-04-27 Flagship Pioneering Innovations Vii, Llc Compositions d'adn et procedes associes
WO2023177655A1 (fr) 2022-03-14 2023-09-21 Generation Bio Co. Compositions vaccinales prime-boost hétérologues et méthodes d'utilisation
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 (fr) * 2022-08-19 2024-02-22 Generation Bio Co. Adn à extrémités fermées clivable (adnce) et ses procédés d'utilisation
WO2024182707A1 (fr) 2023-03-02 2024-09-06 Krystal Biotech, Inc. Interleukine-2 et interleukine-12 pour le traitement du cancer
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

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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
US20060166363A1 (en) * 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
WO2008016391A2 (fr) * 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Vecteurs parvoviraux auto-complémentaires et leurs procédés de fabrication et d'utilisation
EP2500434A1 (fr) * 2011-03-12 2012-09-19 Association Institut de Myologie Vecteurs AAV sans capside, compositions et procédés pour la production des vecteurs et la thérapie génique
CA2876293C (fr) * 2012-06-27 2023-10-10 Arthrogen B.V. Combinaison de traitement d'un trouble inflammatoire
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
JP7260898B2 (ja) * 2016-01-15 2023-04-19 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド ガンマデルタｔ細胞の活性化のための方法および組成物
CN115287301A (zh) * 2016-03-03 2022-11-04 马萨诸塞大学 用于非病毒基因转移的末端封闭型线性双链体dna

Also Published As

Publication number Publication date
AU2018327348A1 (en) 2020-02-20
MX2020002500A (es) 2020-09-17
KR20200051011A (ko) 2020-05-12
US20200283794A1 (en) 2020-09-10
IL272797A (en) 2020-04-30
EP3678710A4 (fr) 2021-06-09
RU2020109904A (ru) 2021-10-08
JP2022190081A (ja) 2022-12-22
BR112020004151A2 (pt) 2020-09-08
CA3075168A1 (fr) 2019-03-14
SG11202000698SA (en) 2020-03-30
EP3678710A1 (fr) 2020-07-15
PH12020500465A1 (en) 2021-01-25
WO2019051255A1 (fr) 2019-03-14
JP2020532981A (ja) 2020-11-19
CN111132699A (zh) 2020-05-08

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