SG11202104743WA - Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats - Google Patents

Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats

Info

Publication number
SG11202104743WA
SG11202104743WA SG11202104743WA SG11202104743WA SG11202104743WA SG 11202104743W A SG11202104743W A SG 11202104743WA SG 11202104743W A SG11202104743W A SG 11202104743WA SG 11202104743W A SG11202104743W A SG 11202104743WA SG 11202104743W A SG11202104743W A SG 11202104743WA
Authority
SG
Singapore
Prior art keywords
modified
cedna
inverted terminal
terminal repeats
symmetrical
Prior art date
Application number
SG11202104743WA
Other languages
English (en)
Inventor
Robert Michael Kotin
Ozan Alkan
Annaliese Jones
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 SG11202104743WA publication Critical patent/SG11202104743WA/en

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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)
SG11202104743WA 2018-11-09 2019-11-08 Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats SG11202104743WA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862757892P 2018-11-09 2018-11-09
US201862757872P 2018-11-09 2018-11-09
PCT/US2019/060395 WO2020097417A1 (en) 2018-11-09 2019-11-08 Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats

Publications (1)

Publication Number Publication Date
SG11202104743WA true SG11202104743WA (en) 2021-06-29

Family

ID=70612486

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202104743WA SG11202104743WA (en) 2018-11-09 2019-11-08 Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats

Country Status (13)

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

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3999646A4 (en) * 2019-07-17 2023-08-30 Generation Bio Co. COMPOSITIONS AND PRODUCTION OF NICKEL-CLOSED CLOSED-END DNA VECTOR
EP3999122A4 (en) * 2019-07-17 2023-08-30 Generation Bio Co. SYNTHETIC MANUFACTURE OF SINGLE STRANDED ADENO-ASSOCIATED VIRAL DNA VECTORS
WO2022023284A1 (en) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
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 (en) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
US10799566B2 (en) * 2015-06-23 2020-10-13 The Children's Hospital Of Philadelphia Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues
ES2898337T3 (es) * 2016-03-03 2022-03-07 Univ Massachusetts ADN dúplex lineal de extremos cerrados para la transferencia de genes no víricos
MA51626A (fr) * 2018-01-19 2020-11-25 Generation Bio Co Vecteurs d'adn à extrémité fermée pouvant être obtenus à partir d'une synthèse acellulaire et procédé d'obtention de vecteurs d'adnce
SG11202007621TA (en) * 2018-02-22 2020-09-29 Generation Bio Co Controlled expression of transgenes using close-ended dna (cedna) vectors

Also Published As

Publication number Publication date
CA3119310A1 (en) 2020-05-14
KR20210090619A (ko) 2021-07-20
WO2020097417A1 (en) 2020-05-14
CN113316640A (zh) 2021-08-27
MA54188A (fr) 2021-09-15
IL282925A (en) 2021-06-30
MX2021004842A (es) 2021-06-08
JP2022506771A (ja) 2022-01-17
WO2020097417A9 (en) 2020-06-18
EP3877528A1 (en) 2021-09-15
EP3877528A4 (en) 2022-11-30
US20210388379A1 (en) 2021-12-16
BR112021007102A2 (pt) 2021-08-03
AU2019376663A1 (en) 2021-06-24

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