MA54188A - Adn à extrémité fermée modifié (cedna) comprenant des répétitions terminales inversées modifiées symétriques - Google Patents

Adn à extrémité fermée modifié (cedna) comprenant des répétitions terminales inversées modifiées symétriques

Info

Publication number
MA54188A
MA54188A MA054188A MA54188A MA54188A MA 54188 A MA54188 A MA 54188A MA 054188 A MA054188 A MA 054188A MA 54188 A MA54188 A MA 54188A MA 54188 A MA54188 A MA 54188A
Authority
MA
Morocco
Prior art keywords
modified
cedna
closed end
inverted terminal
terminal repeats
Prior art date
Application number
MA054188A
Other languages
English (en)
Inventor
Ozan Alkan
Annaliese Jones
Robert Michael Kotin
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 MA54188A publication Critical patent/MA54188A/fr

Links

Classifications

    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • 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
    • C12N15/63—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
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Manufacturing & Machinery (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Virology (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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
MA054188A 2018-11-09 2019-11-08 Adn à extrémité fermée modifié (cedna) comprenant des répétitions terminales inversées modifiées symétriques MA54188A (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862757892P 2018-11-09 2018-11-09
US201862757872P 2018-11-09 2018-11-09

Publications (1)

Publication Number Publication Date
MA54188A true MA54188A (fr) 2021-09-15

Family

ID=70612486

Family Applications (1)

Application Number Title Priority Date Filing Date
MA054188A MA54188A (fr) 2018-11-09 2019-11-08 Adn à extrémité fermée modifié (cedna) comprenant des répétitions terminales inversées modifiées symétriques

Country Status (13)

Country Link
US (1) US20210388379A1 (fr)
EP (1) EP3877528A4 (fr)
JP (1) JP2022506771A (fr)
KR (1) KR20210090619A (fr)
CN (1) CN113316640A (fr)
AU (1) AU2019376663A1 (fr)
BR (1) BR112021007102A2 (fr)
CA (1) CA3119310A1 (fr)
IL (1) IL282925A (fr)
MA (1) MA54188A (fr)
MX (1) MX2021004842A (fr)
SG (1) SG11202104743WA (fr)
WO (1) WO2020097417A1 (fr)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3146966A1 (fr) * 2019-07-17 2021-01-21 Generation Bio Co. Compositions et production de vecteurs d'adn a extremites fermees nickeles
CA3147414A1 (fr) * 2019-07-17 2021-01-21 Generation Bio Co. Production synthetique de vecteurs de type adn viraux adeno-associes simple brin
EP4189098A1 (fr) 2020-07-27 2023-06-07 Anjarium Biosciences AG Compositions de molécules d'adn, leurs procédés de fabrication et leurs procédés 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
CN115896135B (zh) * 2022-11-02 2024-03-01 苏州诺洁贝生物技术有限公司 优化的pah基因和表达盒及其用途
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
CN116110602B (zh) * 2023-04-13 2023-06-20 云南医无界医疗网络科技有限公司 一种应用于医共体的信息处理方法及系统

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP6831779B2 (ja) * 2015-06-23 2021-02-17 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用
ES2898337T3 (es) * 2016-03-03 2022-03-07 Univ Massachusetts ADN dúplex lineal de extremos cerrados para la transferencia de genes no víricos
MX2020005790A (es) * 2018-01-19 2020-10-28 Generation Bio Co Vectores de adn de extremo cerrado que pueden obtenerse de síntesis libre de células y un proceso para obtener vectores de ceadn.
BR112020017060A2 (pt) * 2018-02-22 2020-12-15 Generation Bio Co. Expressão controlada de transgenes usando vetores de dna de extremidade fechada (cedna)

Also Published As

Publication number Publication date
IL282925A (en) 2021-06-30
JP2022506771A (ja) 2022-01-17
BR112021007102A2 (pt) 2021-08-03
SG11202104743WA (en) 2021-06-29
CN113316640A (zh) 2021-08-27
MX2021004842A (es) 2021-06-08
EP3877528A4 (fr) 2022-11-30
AU2019376663A1 (en) 2021-06-24
WO2020097417A9 (fr) 2020-06-18
EP3877528A1 (fr) 2021-09-15
KR20210090619A (ko) 2021-07-20
WO2020097417A1 (fr) 2020-05-14
CA3119310A1 (fr) 2020-05-14
US20210388379A1 (en) 2021-12-16

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