SG11202005281XA - Gene editing using a modified closed-ended dna (cedna) - Google Patents

Gene editing using a modified closed-ended dna (cedna)

Info

Publication number
SG11202005281XA
SG11202005281XA SG11202005281XA SG11202005281XA SG11202005281XA SG 11202005281X A SG11202005281X A SG 11202005281XA SG 11202005281X A SG11202005281X A SG 11202005281XA SG 11202005281X A SG11202005281X A SG 11202005281XA SG 11202005281X A SG11202005281X A SG 11202005281XA
Authority
SG
Singapore
Prior art keywords
cedna
gene editing
ended dna
modified closed
modified
Prior art date
Application number
SG11202005281XA
Inventor
Robert M Kotin
Douglas Kerr
Phillip Samayoa
Ozan Alkan
Matthew J Simmons
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 SG11202005281XA publication Critical patent/SG11202005281XA/en

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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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
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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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • 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
    • A61K48/0008Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
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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
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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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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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
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    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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
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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
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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
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases RNAses, DNAses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • CCHEMISTRY; METALLURGY
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    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • CCHEMISTRY; METALLURGY
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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
    • C12N2710/14041Use of virus, viral particle or viral elements as a vector
    • CCHEMISTRY; METALLURGY
    • 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
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (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)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Mycology (AREA)
  • Public Health (AREA)
  • Cell Biology (AREA)
  • Virology (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)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
SG11202005281XA 2017-12-06 2018-12-06 Gene editing using a modified closed-ended dna (cedna) SG11202005281XA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762595328P 2017-12-06 2017-12-06
US201762607069P 2017-12-18 2017-12-18
PCT/US2018/064242 WO2019113310A1 (en) 2017-12-06 2018-12-06 Gene editing using a modified closed-ended dna (cedna)

Publications (1)

Publication Number Publication Date
SG11202005281XA true SG11202005281XA (en) 2020-07-29

Family

ID=66751200

Family Applications (2)

Application Number Title Priority Date Filing Date
SG11202005281XA SG11202005281XA (en) 2017-12-06 2018-12-06 Gene editing using a modified closed-ended dna (cedna)
SG10202012132WA SG10202012132WA (en) 2017-12-06 2018-12-06 Gene editing using a modified closed-ended dna (cedna)

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG10202012132WA SG10202012132WA (en) 2017-12-06 2018-12-06 Gene editing using a modified closed-ended dna (cedna)

Country Status (14)

Country Link
US (1) US20220290186A1 (en)
EP (1) EP3720952A4 (en)
JP (2) JP2021505159A (en)
KR (1) KR20200093635A (en)
CN (1) CN111527200A (en)
AU (1) AU2018378672A1 (en)
BR (1) BR112020009858A2 (en)
CA (1) CA3084185A1 (en)
IL (1) IL274845A (en)
MA (1) MA51113A (en)
MX (1) MX2020005808A (en)
PH (1) PH12020550771A1 (en)
SG (2) SG11202005281XA (en)
WO (1) WO2019113310A1 (en)

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US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
EP3092049A1 (en) 2014-01-08 2016-11-16 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
US20200263204A1 (en) * 2017-09-28 2020-08-20 Toolgen Incorporated Composition for treating hemophilia a by crispr/cas system of reverting fviii gene inversion
MX2020008470A (en) * 2018-02-14 2020-09-25 Generation Bio Co Non-viral dna vectors and uses thereof for antibody and fusion protein production.
CN109022389A (en) * 2018-07-19 2018-12-18 陕西慧康生物科技有限责任公司 A kind of production method of Bacillus coli expression people Iduronate-2-sulfatase
AU2020289581A1 (en) 2019-06-07 2021-11-18 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized albumin locus
EP4022074A4 (en) * 2019-08-27 2023-11-15 The Trustees of Columbia University in the City of New York Engineered exosomes for targeted delivery
US20220275400A1 (en) * 2019-08-30 2022-09-01 The Trustees Of Columbia University In The City Of New York Methods for scalable gene insertions
KR20230003477A (en) * 2020-03-24 2023-01-06 제너레이션 바이오 컴퍼니 Non-viral DNA vectors and their use for expressing Factor IX therapeutics
US20210355506A1 (en) * 2020-05-13 2021-11-18 Lysogene Compositions and methods for treating gm1 gangliosidosis and other disorders
WO2022015856A1 (en) * 2020-07-14 2022-01-20 The Regents Of The University Of California Compositions and methods for treating an inherited retinal disease
EP4189098A1 (en) 2020-07-27 2023-06-07 Anjarium Biosciences AG Compositions of dna molecules, methods of making therefor, and methods of use thereof
KR20230066453A (en) * 2020-09-16 2023-05-15 제너레이션 바이오 컴퍼니 Non-viral DNA vectors and their use for expressing FVIII therapeutics
JP2023543291A (en) 2020-09-29 2023-10-13 アルベルト-ルドビクス-ウニベルジテート フライブルク Rescue of recombinant adenovirus by CRISPR/CAS-mediated in vivo end separation
CN112481262B (en) * 2020-12-04 2022-08-19 中国农业科学院农业基因组研究所 Method for analyzing biological functions of enhancer cells based on CRISPR/Cas9 gene editing technology
CN112852880B (en) * 2021-01-28 2022-12-06 中吉智药(南京)生物技术有限公司 Method for producing AAV gene medicine based on induced insect cell
AU2022237643A1 (en) * 2021-03-19 2023-09-28 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing pfic therapeutics
EP4330416A1 (en) * 2021-04-26 2024-03-06 University of Massachusetts Direct raav-mediated in vivo gene editing of hematopoietic stem cells
EP4337177A1 (en) * 2021-05-11 2024-03-20 Modernatx, Inc. Non-viral delivery of dna for prolonged polypeptide expression in vivo
AU2022280952A1 (en) * 2021-05-28 2023-11-23 Beam Therapeutics Inc. Compositions and methods for the self-inactivation of base editors
WO2023283420A2 (en) * 2021-07-09 2023-01-12 The Board Of Trustees Of The University Of Illinois Therapeutic gene silencing with crispr-cas13
AR126846A1 (en) * 2021-08-23 2023-11-22 Bioverativ Therapeutics Inc OPTIMIZED FACTOR VIII GENES
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
WO2024059699A2 (en) * 2022-09-16 2024-03-21 Joseph Fenton Lawler Trans-splicing methods and compositions for generation of single sex offspring

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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
JP2016518142A (en) * 2013-05-10 2016-06-23 サンガモ バイオサイエンシーズ, インコーポレイテッド Delivery methods and compositions for nuclease-mediated genomic gene manipulation
SG10201804976YA (en) * 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Genome Editing
US20160369258A1 (en) * 2014-03-11 2016-12-22 University Of Washington Restricting nuclear protein to specific phases of the cell cycle
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WO2016205728A1 (en) * 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
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WO2019051255A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. Modified closed-ended dna (cedna)

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Publication number Publication date
IL274845A (en) 2020-07-30
JP2021505159A (en) 2021-02-18
MA51113A (en) 2020-10-14
PH12020550771A1 (en) 2021-05-10
SG10202012132WA (en) 2021-01-28
KR20200093635A (en) 2020-08-05
WO2019113310A1 (en) 2019-06-13
BR112020009858A2 (en) 2020-11-17
EP3720952A1 (en) 2020-10-14
RU2020121128A (en) 2022-01-11
EP3720952A4 (en) 2021-09-01
US20220290186A1 (en) 2022-09-15
CN111527200A (en) 2020-08-11
MX2020005808A (en) 2020-10-28
JP2024003220A (en) 2024-01-11
AU2018378672A1 (en) 2020-07-09
CA3084185A1 (en) 2019-06-13

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