AU2016260401B2 - Nucleic acid molecules containing spacers and methods of use thereof - Google Patents

Nucleic acid molecules containing spacers and methods of use thereof Download PDF

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Publication number
AU2016260401B2
AU2016260401B2 AU2016260401A AU2016260401A AU2016260401B2 AU 2016260401 B2 AU2016260401 B2 AU 2016260401B2 AU 2016260401 A AU2016260401 A AU 2016260401A AU 2016260401 A AU2016260401 A AU 2016260401A AU 2016260401 B2 AU2016260401 B2 AU 2016260401B2
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nucleic acid
acid molecule
spacer
length
cell
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AU2016260401A1 (en
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John T. Gray
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St Jude Childrens Research Hospital
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St Jude Childrens Research Hospital
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Priority to AU2022215274A priority Critical patent/AU2022215274A1/en
Priority to AU2025203194A priority patent/AU2025203194A1/en
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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • C12N15/86—Viral 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
    • 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
    • C12N15/861—Adenoviral vectors
    • C12N15/8616—Special methods for targeting systems
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/17—Immunomodulatory nucleic acids
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Manufacturing & Machinery (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2016260401A 2015-05-14 2016-05-13 Nucleic acid molecules containing spacers and methods of use thereof Active AU2016260401B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2022215274A AU2022215274A1 (en) 2015-05-14 2022-08-12 Nucleic acid molecules containing spacers and methods of use thereof
AU2025203194A AU2025203194A1 (en) 2015-05-14 2025-05-05 Nucleic acid molecules containing spacers and methods of use thereof

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562161505P 2015-05-14 2015-05-14
US62/161,505 2015-05-14
PCT/US2016/032321 WO2016183422A1 (en) 2015-05-14 2016-05-13 Nucleic acid molecules containing spacers and methods of use thereof

Related Child Applications (1)

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AU2022215274A Division AU2022215274A1 (en) 2015-05-14 2022-08-12 Nucleic acid molecules containing spacers and methods of use thereof

Publications (2)

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AU2016260401A1 AU2016260401A1 (en) 2017-11-02
AU2016260401B2 true AU2016260401B2 (en) 2022-05-19

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AU2022215274A Abandoned AU2022215274A1 (en) 2015-05-14 2022-08-12 Nucleic acid molecules containing spacers and methods of use thereof
AU2025203194A Pending AU2025203194A1 (en) 2015-05-14 2025-05-05 Nucleic acid molecules containing spacers and methods of use thereof

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AU2025203194A Pending AU2025203194A1 (en) 2015-05-14 2025-05-05 Nucleic acid molecules containing spacers and methods of use thereof

Country Status (5)

Country Link
US (2) US11103597B2 (enExample)
EP (1) EP3294309A4 (enExample)
JP (3) JP6929230B2 (enExample)
AU (3) AU2016260401B2 (enExample)
WO (1) WO2016183422A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11103597B2 (en) 2015-05-14 2021-08-31 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers outside ITR
WO2017066579A1 (en) 2015-10-14 2017-04-20 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
WO2018191622A1 (en) * 2017-04-14 2018-10-18 Rhode Island Hospital Vegf gene therapy for tendon and ligament injuries
WO2019032898A1 (en) 2017-08-09 2019-02-14 Bioverativ Therapeutics Inc. NUCLEIC ACID MOLECULES AND USES THEREOF
CA3092088A1 (en) * 2018-03-15 2019-09-19 Igtx, Llc Synthetic dna vectors and methods of use
SG11202101157VA (en) 2018-08-09 2021-03-30 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof for non-viral gene therapy
US12383587B2 (en) 2018-10-25 2025-08-12 Takeda Pharmaceutical Company Limited AAV triple-plasmid system
TW202124722A (zh) 2019-09-18 2021-07-01 美商英特佳樂帝克醫療公司 合成dna載體及其使用方法
CN115197967B (zh) * 2021-04-08 2023-09-15 广州派真生物技术有限公司 制备重组腺相关病毒的辅助质粒及其应用

Citations (5)

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WO2001036623A2 (en) * 1999-11-05 2001-05-25 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
WO2012158757A1 (en) * 2011-05-16 2012-11-22 The Trustees Of The University Of Pennsylvania Proviral plasmids for production of recombinant adeno-associated virus
WO2013173129A2 (en) * 2012-05-15 2013-11-21 Avalanche Australia Pty Ltd. Treatment of amd using aav sflt-1
WO2014144486A2 (en) * 2013-03-15 2014-09-18 The Children's Hospital Of Philadelphia Vectors comprising stuffer/filler polynucleotide sequences and methods of use

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US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
CA2264499A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
US20020182595A1 (en) 2001-04-27 2002-12-05 Weitzman Matthew D. Method of identifying cellular regulators of adeno-associated virus (AAV)
EP1402023A4 (en) 2001-05-31 2005-06-15 Univ Rockefeller PROCESS FOR GENERATING REPLICATION-DEFICIENT VIRUS VECTORS THAT ARE HELPFUL-FREE
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
WO2004029278A2 (en) 2002-09-26 2004-04-08 Greenville Hospital System Aav itr-mediated modulation
DK2438931T3 (da) * 2004-09-22 2013-12-02 St Jude Childrens Res Hospital Forbedret ekspression af faktor ix i genterapivektorer
ES2683695T3 (es) * 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
US9124527B2 (en) * 2012-08-29 2015-09-01 International Business Machines Corporation Sliced routing table management
JP6454643B2 (ja) * 2012-10-26 2019-01-16 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ 血友病の肝臓指向性遺伝子治療のためのベクター並びにその方法及び使用
CN105228615A (zh) 2013-03-15 2016-01-06 鲁美纳医药公司 用于治疗巴雷特食管和胃食管返流疾病的胆汁酸再循环抑制剂
PH12016500162B1 (en) * 2013-07-22 2024-02-21 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene trnsfer to cells, organs, and tissues
CN107427557B (zh) * 2014-08-13 2022-01-04 费城儿童医院 用于包装和表达变体因子viii以治疗血友病的改良表达组件
US11103597B2 (en) 2015-05-14 2021-08-31 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers outside ITR

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
WO2001036623A2 (en) * 1999-11-05 2001-05-25 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
WO2012158757A1 (en) * 2011-05-16 2012-11-22 The Trustees Of The University Of Pennsylvania Proviral plasmids for production of recombinant adeno-associated virus
WO2013173129A2 (en) * 2012-05-15 2013-11-21 Avalanche Australia Pty Ltd. Treatment of amd using aav sflt-1
WO2014144486A2 (en) * 2013-03-15 2014-09-18 The Children's Hospital Of Philadelphia Vectors comprising stuffer/filler polynucleotide sequences and methods of use

Also Published As

Publication number Publication date
US20210369869A1 (en) 2021-12-02
CA2983593A1 (en) 2016-11-17
US11103597B2 (en) 2021-08-31
EP3294309A4 (en) 2019-01-16
AU2022215274A1 (en) 2022-09-01
WO2016183422A1 (en) 2016-11-17
AU2016260401A1 (en) 2017-11-02
US20180126006A1 (en) 2018-05-10
JP2021184734A (ja) 2021-12-09
EP3294309A1 (en) 2018-03-21
JP2018515102A (ja) 2018-06-14
JP2023138527A (ja) 2023-10-02
JP7316326B2 (ja) 2023-07-27
US12453783B2 (en) 2025-10-28
AU2025203194A1 (en) 2025-05-22
JP6929230B2 (ja) 2021-09-01

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