MX2020002500A - Adn de extremo cerrado modificado (adnec). - Google Patents

Adn de extremo cerrado modificado (adnec).

Info

Publication number
MX2020002500A
MX2020002500A MX2020002500A MX2020002500A MX2020002500A MX 2020002500 A MX2020002500 A MX 2020002500A MX 2020002500 A MX2020002500 A MX 2020002500A MX 2020002500 A MX2020002500 A MX 2020002500A MX 2020002500 A MX2020002500 A MX 2020002500A
Authority
MX
Mexico
Prior art keywords
cedna
cedna vectors
linear
vectors
ended dna
Prior art date
Application number
MX2020002500A
Other languages
English (en)
Spanish (es)
Inventor
Robert Michael Kotin
Ozan Alkan
Annaliese Jones
Douglas Anthony Kerr
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 MX2020002500A publication Critical patent/MX2020002500A/es

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
    • 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
    • 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
    • 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
    • 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)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Cell Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
MX2020002500A 2017-09-08 2018-09-07 Adn de extremo cerrado modificado (adnec). MX2020002500A (es)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762556281P 2017-09-08 2017-09-08
US201762556324P 2017-09-08 2017-09-08
US201762556331P 2017-09-08 2017-09-08
US201762556319P 2017-09-08 2017-09-08
US201762556335P 2017-09-08 2017-09-08
US201762556329P 2017-09-08 2017-09-08
PCT/US2018/049996 WO2019051255A1 (en) 2017-09-08 2018-09-07 CLOSED-ENDED DNA (CEDNA) MODIFIED

Publications (1)

Publication Number Publication Date
MX2020002500A true MX2020002500A (es) 2020-09-17

Family

ID=65635235

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020002500A MX2020002500A (es) 2017-09-08 2018-09-07 Adn de extremo cerrado modificado (adnec).

Country Status (15)

Country Link
US (1) US20200283794A1 (enExample)
EP (1) EP3678710A4 (enExample)
JP (3) JP2020532981A (enExample)
KR (1) KR20200051011A (enExample)
CN (1) CN111132699A (enExample)
AU (1) AU2018327348A1 (enExample)
BR (1) BR112020004151A2 (enExample)
CA (1) CA3075168A1 (enExample)
IL (1) IL272797A (enExample)
MA (1) MA50100A (enExample)
MX (1) MX2020002500A (enExample)
PH (1) PH12020500465A1 (enExample)
SG (1) SG11202000698SA (enExample)
UA (1) UA129010C2 (enExample)
WO (1) WO2019051255A1 (enExample)

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US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
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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
CN114900773A (zh) 2017-12-14 2022-08-12 弗洛设计声能学公司 声泳系统及其操作方法、控制声换能器及声学系统的方法
AU2019221642A1 (en) * 2018-02-14 2020-07-09 Generation Bio Co. Non-viral DNA vectors and uses thereof for antibody and fusion protein production
JP7602454B2 (ja) 2018-08-09 2024-12-18 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
WO2020097417A1 (en) * 2018-11-09 2020-05-14 Generation Bio Co. Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats
GB201905651D0 (en) * 2019-04-24 2019-06-05 Lightbio Ltd Nucleic acid constructs and methods for their manufacture
US20220228171A1 (en) * 2019-07-17 2022-07-21 Generation Bio Co. Compositions and production of nicked closed-ended dna vectors
WO2021011842A1 (en) * 2019-07-17 2021-01-21 Generation Bio Co. Synthetic production of single-stranded adeno associated viral dna vectors
WO2021030745A1 (en) * 2019-08-15 2021-02-18 The Children's Hospital Of Philadelphia Combined transgene and intron-derived mirna therapy for treatment of sca1
CN114929205B (zh) * 2019-09-06 2024-09-03 世代生物公司 包括末端封闭式dna和可切割脂质的脂质纳米颗粒组合物及其使用方法
WO2021055760A1 (en) 2019-09-18 2021-03-25 Intergalactic Therapeutics, Inc. Synthetic dna vectors and methods of use
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IL296660A (en) * 2020-03-24 2022-11-01 Generation Bio Co Non-viral DNA vectors and their use for therapeutic expression of factor ix
IL296662A (en) * 2020-03-24 2022-11-01 Generation Bio Co Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
CN115968280A (zh) * 2020-05-18 2023-04-14 世代生物公司 新型脂质及其纳米颗粒组合物
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AU2021330936A1 (en) 2020-08-23 2023-05-04 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
US20220243201A1 (en) * 2020-08-23 2022-08-04 Bioverativ Therapeutics Inc. Engineered itr sequences and methods of use
CA3191743A1 (en) * 2020-09-16 2022-03-24 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing fviii therapeutics
MX2023003019A (es) * 2020-09-16 2023-05-08 Generation Bio Co Vectores de adn de extremo cerrado y usos de estos para expresar fenilalanina hidroxilasa (pah).
GB202014751D0 (en) * 2020-09-18 2020-11-04 Lightbio Ltd Targeting vector
WO2022198025A2 (en) * 2021-03-19 2022-09-22 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing pfic therapeutics
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JP2024515788A (ja) * 2021-04-27 2024-04-10 ジェネレーション バイオ カンパニー 治療抗体を発現する非ウイルスdnaベクター及びその使用
US20240216535A1 (en) * 2021-04-27 2024-07-04 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
EP4333886A4 (en) * 2021-05-07 2025-11-12 Generation Bio Co NON-VIRAL DNA VECTORS FOR VACCINE ADMINISTRATION
WO2022236016A1 (en) * 2021-05-07 2022-11-10 Generation Bio Co. Lyophilized non-viral dna vector compositions and uses thereof
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Also Published As

Publication number Publication date
UA129010C2 (uk) 2024-12-25
MA50100A (fr) 2020-07-15
RU2020109904A (ru) 2021-10-08
CA3075168A1 (en) 2019-03-14
EP3678710A1 (en) 2020-07-15
PH12020500465A1 (en) 2021-01-25
JP2022190081A (ja) 2022-12-22
CN111132699A (zh) 2020-05-08
NZ761178A (en) 2024-05-31
US20200283794A1 (en) 2020-09-10
KR20200051011A (ko) 2020-05-12
JP2025010253A (ja) 2025-01-20
EP3678710A4 (en) 2021-06-09
AU2018327348A1 (en) 2020-02-20
IL272797A (en) 2020-04-30
SG11202000698SA (en) 2020-03-30
BR112020004151A2 (pt) 2020-09-08
JP2020532981A (ja) 2020-11-19
WO2019051255A1 (en) 2019-03-14

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