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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/075—Adenoviridae
    • 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)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Manufacturing & Machinery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (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)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (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
US201862757872P 2018-11-09 2018-11-09
US201862757892P 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) JP7590963B2 (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 (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI904068B (zh) 2017-08-09 2025-11-11 美商生物化學醫療公司 核酸分子及其用途
WO2020033863A1 (fr) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Molécules d'acide nucléique et leurs utilisations pour une thérapie génique non virale
EP3999122A4 (fr) * 2019-07-17 2023-08-30 Generation Bio Co. Production synthétique de vecteurs de type adn viraux adéno-associés simple brin
AU2020314865A1 (en) * 2019-07-17 2021-12-23 Generation Bio Co. Compositions and production of nicked closed-ended DNA vectors
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
US20220243201A1 (en) * 2020-08-23 2022-08-04 Bioverativ Therapeutics Inc. Engineered itr sequences and methods of use
BR112023002395A2 (pt) * 2020-08-23 2023-03-21 Bioverativ Therapeutics Inc Sistema de baculovírus modificado para produção aprimorada de dna com extremidades fechadas (cedna)
US20240181085A1 (en) * 2021-03-19 2024-06-06 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing pfic therapeutics
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
US11891617B1 (en) * 2023-03-27 2024-02-06 Genecraft Inc. Modified complex platform of adeno-associated virus with improved rate of expression of loaded genes and reduced genotoxicity
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 (8)

* 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
US9943574B2 (en) * 2012-06-27 2018-04-17 Arthrogen B.V. Combination for treating an inflammatory disorder
HUE068603T2 (hu) * 2015-06-23 2025-01-28 Childrens Hospital Philadelphia Módosított IX-es faktor, valamint készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
EP3423110B1 (fr) * 2016-03-03 2021-08-11 University of Massachusetts Adn double hélice linéaire à extrémité fermée pour transfert de gène non viral
WO2019051255A1 (fr) * 2017-09-08 2019-03-14 Generation Bio Co. Adn à extrémité fermée (cedna) modifié
US20220290186A1 (en) * 2017-12-06 2022-09-15 Generation Bio Co. Gene editing using a modified closed-ended dna (cedna)
BR112020013319A2 (pt) * 2018-01-19 2020-12-01 Generation Bio Co. vetores de dna de extremidade fechada obteníveis a partir de síntese livre de células e processo para obter vetores de cedna
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
JP2022506771A (ja) 2022-01-17
IL282925A (en) 2021-06-30
MX2021004842A (es) 2021-06-08
WO2020097417A1 (fr) 2020-05-14
AU2019376663A1 (en) 2021-06-24
EP3877528A4 (fr) 2022-11-30
SG11202104743WA (en) 2021-06-29
JP7590963B2 (ja) 2024-11-27
CN113316640A (zh) 2021-08-27
US20210388379A1 (en) 2021-12-16
BR112021007102A2 (pt) 2021-08-03
CA3119310A1 (fr) 2020-05-14
EP3877528A1 (fr) 2021-09-15
KR20210090619A (ko) 2021-07-20
WO2020097417A9 (fr) 2020-06-18

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