CA3014671A1 - Aav vectors for treatment of dominant retinitis pigmentosa - Google Patents

Aav vectors for treatment of dominant retinitis pigmentosa Download PDF

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Publication number
CA3014671A1
CA3014671A1 CA3014671A CA3014671A CA3014671A1 CA 3014671 A1 CA3014671 A1 CA 3014671A1 CA 3014671 A CA3014671 A CA 3014671A CA 3014671 A CA3014671 A CA 3014671A CA 3014671 A1 CA3014671 A1 CA 3014671A1
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CA
Canada
Prior art keywords
rna
sequence
seq
rho
vector
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Legal status (The legal status 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 status listed.)
Pending
Application number
CA3014671A
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English (en)
French (fr)
Inventor
Alfred S. Lewin
William W. Hauswirth
Michael T. Massengill
William BELTRAN
Gustavo D. Aguirre
Artur CIDECIYAN
Samuel Jacobson
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Pennsylvania Penn
University of Florida Research Foundation Inc
Original Assignee
University of Pennsylvania Penn
University of Florida Research Foundation Inc
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Application filed by University of Pennsylvania Penn, University of Florida Research Foundation Inc filed Critical University of Pennsylvania Penn
Publication of CA3014671A1 publication Critical patent/CA3014671A1/en
Pending legal-status Critical Current

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    • 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
    • C12N15/113—Non-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
    • 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
    • C12N15/113—Non-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
    • C12N15/1138—Non-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 against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—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
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pigments, Carbon Blacks, Or Wood Stains (AREA)
  • Inorganic Compounds Of Heavy Metals (AREA)
  • Paper (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
CA3014671A 2016-03-01 2017-03-01 Aav vectors for treatment of dominant retinitis pigmentosa Pending CA3014671A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662302122P 2016-03-01 2016-03-01
US62/302,122 2016-03-01
US201662398451P 2016-09-22 2016-09-22
US62/398,451 2016-09-22
PCT/US2017/020289 WO2017151823A1 (en) 2016-03-01 2017-03-01 Aav vectors for treatment of dominant retinitis pigmentosa

Publications (1)

Publication Number Publication Date
CA3014671A1 true CA3014671A1 (en) 2017-09-08

Family

ID=59743213

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3014671A Pending CA3014671A1 (en) 2016-03-01 2017-03-01 Aav vectors for treatment of dominant retinitis pigmentosa

Country Status (9)

Country Link
US (2) US11118185B2 (enExample)
EP (2) EP3423582B1 (enExample)
JP (3) JP7272795B2 (enExample)
CN (2) CN109154002B (enExample)
AU (2) AU2017227776C1 (enExample)
CA (1) CA3014671A1 (enExample)
ES (1) ES2923877T3 (enExample)
IL (2) IL261014B2 (enExample)
WO (1) WO2017151823A1 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL261014B2 (en) 2016-03-01 2023-10-01 Univ Florida Aav vectors for treatment of dominant retinitis pigmentosa
EP3707257A4 (en) * 2017-11-07 2021-12-08 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR CIRCULAR RNA MOLECULES
EP3793566A4 (en) * 2018-05-15 2022-03-16 University of Washington Compositions and methods for reducing spliceopathy and treating rna dominance disorders
JP7563694B2 (ja) * 2018-06-01 2024-10-08 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 優性網膜色素変性症の処置のための組成物および方法
GB201817469D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy for retinal disease
CN111518813B (zh) * 2019-02-03 2023-04-28 武汉纽福斯生物科技有限公司 视紫红质的编码序列、其表达载体构建及其应用
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
CN113038971B (zh) * 2020-04-21 2022-06-24 北京中因科技有限公司 RHO-adRP基于基因编辑的方法和组合物
CN111926044B (zh) * 2020-10-12 2021-01-22 北京大学第三医院（北京大学第三临床医学院） 结合突变rho基因的核酸分子和试剂盒
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2025231487A1 (en) * 2024-05-03 2025-11-06 The Trustees Of The University Of Pennsylvania Photoreceptor specific promotors for use in gene therapies for treatment of retinal degeneration

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US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
GB9606961D0 (en) 1996-04-02 1996-06-05 Farrar Gwyneth J Genetic strategy III
US8551970B2 (en) 1996-04-02 2013-10-08 Optigen Patents Limited Genetic suppression and replacement
US20040234999A1 (en) 1996-04-02 2004-11-25 Farrar Gwenyth Jane Genetic suppression and replacement
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
WO2004094606A2 (en) 2003-04-18 2004-11-04 The Trustees Of The University Of Pennsylvania COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF ANGIOPOIETIN 1 AND 2 AND THEIR RECEPTOR TIE2
US20070009899A1 (en) 2003-10-02 2007-01-11 Mounts William M Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases
WO2005090572A2 (en) 2004-03-24 2005-09-29 Oncotherapy Science, Inc. Compositions and methods for treating pancreatic cancer
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EP2140004B1 (en) * 2007-04-12 2022-01-12 The Provost, Fellows, Foundation Scholars, & the other members of Board, of the College of the Holy & Undiv. Trinity of Queen Elizabeth near Dublin Genetic suppression and replacement
CA2702701A1 (en) 2007-08-03 2009-03-19 Melba Ketchum Compositions, methods and systems for the simultaneous determination of parentage, identity, sex, genotype and/or phenotype, and breed determination in animals
US8450473B2 (en) * 2009-04-30 2013-05-28 The Research Foundation Of State University Of New York Compositions and methods for therapy of macular degeneration
CA2801523C (en) 2010-07-30 2021-08-03 Curevac Gmbh Complexation of nucleic acids with disulfide-crosslinked cationic components for transfection and immunostimulation
CN102061308B (zh) 2010-10-29 2013-05-08 北京未名凯拓作物设计中心有限公司 一种用百草枯筛选转基因植物的方法
CN102061305B (zh) 2010-12-15 2012-07-18 深圳市百恩维生物科技有限公司 一种多ShRNA高效协同沉默基因方法及载体
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US20170348387A1 (en) 2016-02-29 2017-12-07 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for nphp5 lca-ciliopathy
IL261014B2 (en) 2016-03-01 2023-10-01 Univ Florida Aav vectors for treatment of dominant retinitis pigmentosa

Also Published As

Publication number Publication date
JP2019511216A (ja) 2019-04-25
EP3423582A4 (en) 2019-11-13
EP3423582B1 (en) 2022-05-04
JP2025016451A (ja) 2025-02-04
IL261014B2 (en) 2023-10-01
CN109154002A (zh) 2019-01-04
CN109154002B (zh) 2022-08-30
EP4119668A1 (en) 2023-01-18
JP7642211B2 (ja) 2025-03-10
JP2022185052A (ja) 2022-12-13
US11118185B2 (en) 2021-09-14
AU2017227776B2 (en) 2023-07-06
US20210324387A1 (en) 2021-10-21
AU2017227776A1 (en) 2018-08-23
AU2017227776C1 (en) 2024-01-25
JP7272795B2 (ja) 2023-05-12
AU2023237106B2 (en) 2025-08-28
AU2023237106A1 (en) 2023-10-19
IL302455A (en) 2023-06-01
ES2923877T3 (es) 2022-10-03
EP3423582A1 (en) 2019-01-09
WO2017151823A1 (en) 2017-09-08
IL261014B1 (en) 2023-06-01
IL261014A (en) 2018-10-31
US20190093111A1 (en) 2019-03-28
CN115896106A (zh) 2023-04-04

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