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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AU2016260401A
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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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Preparation Of Compounds By Using 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)

Application Number Title Priority Date Filing Date
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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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

Country Status (5)

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

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3294309A4 (en) 2015-05-14 2019-01-16 St. Jude Children's Research Hospital, Inc. NUCLEIC ACID MOLECULES WITH SPACERS AND METHOD FOR USE THEREOF
CA3001594A1 (en) 2015-10-14 2017-04-20 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
US20200113972A1 (en) * 2017-04-14 2020-04-16 Rhode Island Hospital Vegf gene therapy for tendon and ligament injuries
NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
US20210002667A1 (en) * 2018-03-15 2021-01-07 Intergalactic Therapeutics, Inc. Synthetic dna vectors and methods of use
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
CA3112883A1 (en) 2018-10-25 2020-04-30 Baxalta Incorporated Aav triple-plasmid system
KR20220128607A (ko) 2019-09-18 2022-09-21 인터갈락틱 테라퓨틱스, 인크. 합성 dna 벡터 및 사용 방법
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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
AU722375B2 (en) 1996-09-06 2000-08-03 Trustees Of The University Of Pennsylvania, The 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)
US8067156B2 (en) 2001-05-31 2011-11-29 The Rockefeller University Method for generating replication defective viral vectors that are helper 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
US20040137626A1 (en) * 2002-09-26 2004-07-15 Greenville Hospital System AAV ITR-mediated modulation
WO2006036502A2 (en) * 2004-09-22 2006-04-06 St. Jude Children's Research Hospital Improved expression of factor ix in gene therapy vectors
EP2524037B1 (en) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
US9124527B2 (en) * 2012-08-29 2015-09-01 International Business Machines Corporation Sliced routing table management
AU2013336601B2 (en) 2012-10-26 2018-01-25 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
AU2014229050A1 (en) 2013-03-15 2015-10-22 Lumena Pharmaceuticals Llc Bile acid recycling inhibitors for treatment of Barrett's esophagus and gastroesophageal reflux disease
IL297919A (en) * 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer 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
EP3294309A4 (en) 2015-05-14 2019-01-16 St. Jude Children's Research Hospital, Inc. NUCLEIC ACID MOLECULES WITH SPACERS AND METHOD FOR USE THEREOF

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
CA2983593A1 (en) 2016-11-17
JP7316326B2 (ja) 2023-07-27
JP2021184734A (ja) 2021-12-09
EP3294309A1 (en) 2018-03-21
JP2018515102A (ja) 2018-06-14
AU2025203194A1 (en) 2025-05-22
JP2023138527A (ja) 2023-10-02
WO2016183422A1 (en) 2016-11-17
JP7796702B2 (ja) 2026-01-09
EP3294309A4 (en) 2019-01-16
US12453783B2 (en) 2025-10-28
US20210369869A1 (en) 2021-12-02
US20180126006A1 (en) 2018-05-10
US11103597B2 (en) 2021-08-31
AU2022215274A1 (en) 2022-09-01
AU2016260401A1 (en) 2017-11-02
JP6929230B2 (ja) 2021-09-01

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