ES2923877T3 - Vectores VAA para el tratamiento de la retinitis pigmentosa dominante - Google Patents

Vectores VAA para el tratamiento de la retinitis pigmentosa dominante Download PDF

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Publication number
ES2923877T3
ES2923877T3 ES17760752T ES17760752T ES2923877T3 ES 2923877 T3 ES2923877 T3 ES 2923877T3 ES 17760752 T ES17760752 T ES 17760752T ES 17760752 T ES17760752 T ES 17760752T ES 2923877 T3 ES2923877 T3 ES 2923877T3
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ES
Spain
Prior art keywords
rna
rho
vector
options
sequence
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Active
Application number
ES17760752T
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English (en)
Spanish (es)
Inventor
Alfred Lewin
William Hauswirth
Michael Massengill
William Beltran
Gustavo 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 Florida
University of Pennsylvania
University of Florida Research Foundation Inc
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University of Florida
University of Pennsylvania
University of Florida Research Foundation Inc
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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
    • 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
    • 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
    • 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)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Paper (AREA)
ES17760752T 2016-03-01 2017-03-01 Vectores VAA para el tratamiento de la retinitis pigmentosa dominante Active ES2923877T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662302122P 2016-03-01 2016-03-01
US201662398451P 2016-09-22 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
ES2923877T3 true ES2923877T3 (es) 2022-10-03

Family

ID=59743213

Family Applications (1)

Application Number Title Priority Date Filing Date
ES17760752T Active ES2923877T3 (es) 2016-03-01 2017-03-01 Vectores VAA para el tratamiento de la retinitis pigmentosa dominante

Country Status (9)

Country Link
US (2) US11118185B2 (enExample)
EP (2) EP3423582B1 (enExample)
JP (3) JP7272795B2 (enExample)
CN (2) CN115896106A (enExample)
AU (2) AU2017227776C1 (enExample)
CA (1) CA3014671A1 (enExample)
ES (1) ES2923877T3 (enExample)
IL (2) IL302455A (enExample)
WO (1) WO2017151823A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL302455A (en) 2016-03-01 2023-06-01 Univ Florida Adeno-associated virus vectors for the treatment of dominant retinitis pigmentosa
JP7385282B2 (ja) * 2017-11-07 2023-11-22 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 環状ｒｎａ分子のための方法及び組成物
JP2021522836A (ja) * 2018-05-15 2021-09-02 ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ スプライスオパシーの軽減、及びｒｎａドミナンス障害の治療のための組成物及び方法
EP3802836A4 (en) * 2018-06-01 2022-10-19 University of Florida Research Foundation, Incorporated COMPOSITIONS AND METHODS FOR THE TREATMENT OF DOMINANT RETINITIS PIGMENTOSA
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
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
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
WO2026087373A1 (en) * 2024-10-21 2026-04-30 MeiraGTx Ocular UK Limited Promoters for driving gene expression in rod photoreceptors and uses thereof

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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
US8551970B2 (en) 1996-04-02 2013-10-08 Optigen Patents Limited Genetic suppression and replacement
GB9606961D0 (en) 1996-04-02 1996-06-05 Farrar Gwyneth J Genetic strategy III
US20040234999A1 (en) 1996-04-02 2004-11-25 Farrar Gwenyth Jane Genetic suppression and replacement
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
AU2004233043A1 (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
GB0403600D0 (en) * 2004-02-18 2004-03-24 Trinity College Dublin Methods and reagents for treating disease
CN1977044A (zh) * 2004-03-24 2007-06-06 肿瘤疗法科学股份有限公司 治疗胰腺癌的组合物和方法
US20080221057A1 (en) 2007-02-16 2008-09-11 Wyeth Secreted protein ccdc80 regulates adipocyte differentiation
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
US8257969B2 (en) * 2007-04-12 2012-09-04 The Provost Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Genetic suppression and replacement
US20110129825A1 (en) * 2007-08-03 2011-06-02 Melba Stinnett 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
HUE041571T2 (hu) * 2010-04-23 2019-05-28 Univ Florida RAAV-guanilát-cikláz-készítmények és ejárások 1-es típusú Leber-féle veleszületett vakság (LCA1) kezelésére
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EP2855684A1 (en) * 2012-05-25 2015-04-08 Commissariat à l'Énergie Atomique et aux Énergies Alternatives Vector for the selective silencing of a gene in astrocytes
US20140289881A1 (en) 2013-03-14 2014-09-25 Commonwealth Scientific And Industrial Research Organisation Double-stranded rna
PT3628334T (pt) * 2014-03-21 2023-09-26 Genzyme Corp Terapia genética para retinopatia pigmentar
MX391217B (es) 2015-02-26 2025-03-21 Ionis Pharmaceuticals Inc Moduladores de rodopsina p23h con especificidad de alelo.
EP3289080B1 (en) 2015-04-30 2021-08-25 The Trustees of Columbia University in the City of New York Gene therapy for autosomal dominant diseases
US11096955B2 (en) 2016-02-09 2021-08-24 Fondazione Telethon Synthetic promoters and uses thereof
US20170348387A1 (en) 2016-02-29 2017-12-07 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for nphp5 lca-ciliopathy
IL302455A (en) 2016-03-01 2023-06-01 Univ Florida Adeno-associated virus vectors for the treatment of dominant retinitis pigmentosa

Also Published As

Publication number Publication date
CA3014671A1 (en) 2017-09-08
IL261014B2 (en) 2023-10-01
EP4119668A1 (en) 2023-01-18
JP2025016451A (ja) 2025-02-04
AU2017227776B2 (en) 2023-07-06
EP3423582B1 (en) 2022-05-04
EP3423582A1 (en) 2019-01-09
JP7872961B2 (ja) 2026-06-11
US20190093111A1 (en) 2019-03-28
JP2022185052A (ja) 2022-12-13
IL261014B1 (en) 2023-06-01
IL302455A (en) 2023-06-01
AU2023237106B2 (en) 2025-08-28
AU2017227776C1 (en) 2024-01-25
CN109154002B (zh) 2022-08-30
AU2023237106A1 (en) 2023-10-19
US20210324387A1 (en) 2021-10-21
CN109154002A (zh) 2019-01-04
AU2017227776A1 (en) 2018-08-23
JP7642211B2 (ja) 2025-03-10
EP3423582A4 (en) 2019-11-13
JP2019511216A (ja) 2019-04-25
WO2017151823A1 (en) 2017-09-08
US11118185B2 (en) 2021-09-14
IL261014A (en) 2018-10-31
JP7272795B2 (ja) 2023-05-12
CN115896106A (zh) 2023-04-04

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