AU2016321244B2 - Treatment of retinitis pigmentosa using engineered meganucleases - Google Patents

Treatment of retinitis pigmentosa using engineered meganucleases Download PDF

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AU2016321244B2
AU2016321244B2 AU2016321244A AU2016321244A AU2016321244B2 AU 2016321244 B2 AU2016321244 B2 AU 2016321244B2 AU 2016321244 A AU2016321244 A AU 2016321244A AU 2016321244 A AU2016321244 A AU 2016321244A AU 2016321244 B2 AU2016321244 B2 AU 2016321244B2
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AU2016321244A1 (en
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Victor Bartsevich
Derek Jantz
Michael G. Nicholson
James Jefferson Smith
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Precision Biosciences Inc
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Precision Biosciences Inc
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Priority to AU2026201608A priority patent/AU2026201608A1/en
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2016321244A 2015-09-08 2016-09-08 Treatment of retinitis pigmentosa using engineered meganucleases Active AU2016321244B2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
AU2020294264A AU2020294264A1 (en) 2015-09-08 2020-12-23 Treatment of retinitis pigmentosa using engineered meganucleases
AU2023210619A AU2023210619A1 (en) 2015-09-08 2023-08-03 Treatment of retinitis pigmentosa using engineered meganucleases
AU2026201608A AU2026201608A1 (en) 2015-09-08 2026-03-03 Treatment of retinitis pigmentosa using engineered meganucleases

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562215460P 2015-09-08 2015-09-08
US62/215,460 2015-09-08
PCT/US2016/050809 WO2017044649A1 (en) 2015-09-08 2016-09-08 Treatment of retinitis pigmentosa using engineered meganucleases

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2020294264A Division AU2020294264A1 (en) 2015-09-08 2020-12-23 Treatment of retinitis pigmentosa using engineered meganucleases

Publications (2)

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AU2016321244A1 AU2016321244A1 (en) 2018-04-12
AU2016321244B2 true AU2016321244B2 (en) 2020-09-24

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Application Number Title Priority Date Filing Date
AU2016321244A Active AU2016321244B2 (en) 2015-09-08 2016-09-08 Treatment of retinitis pigmentosa using engineered meganucleases
AU2020294264A Abandoned AU2020294264A1 (en) 2015-09-08 2020-12-23 Treatment of retinitis pigmentosa using engineered meganucleases
AU2023210619A Abandoned AU2023210619A1 (en) 2015-09-08 2023-08-03 Treatment of retinitis pigmentosa using engineered meganucleases
AU2026201608A Pending AU2026201608A1 (en) 2015-09-08 2026-03-03 Treatment of retinitis pigmentosa using engineered meganucleases

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AU2020294264A Abandoned AU2020294264A1 (en) 2015-09-08 2020-12-23 Treatment of retinitis pigmentosa using engineered meganucleases
AU2023210619A Abandoned AU2023210619A1 (en) 2015-09-08 2023-08-03 Treatment of retinitis pigmentosa using engineered meganucleases
AU2026201608A Pending AU2026201608A1 (en) 2015-09-08 2026-03-03 Treatment of retinitis pigmentosa using engineered meganucleases

Country Status (6)

Country Link
US (6) US10603363B2 (https=)
EP (2) EP3347463B1 (https=)
JP (5) JP6775584B2 (https=)
AU (4) AU2016321244B2 (https=)
CA (1) CA2997909A1 (https=)
WO (1) WO2017044649A1 (https=)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015134812A1 (en) 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US12460231B2 (en) 2014-04-02 2025-11-04 Editas Medicine, Inc. Crispr/CAS-related methods and compositions for treating primary open angle glaucoma
CA2982966C (en) 2015-04-24 2024-02-20 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
US10603363B2 (en) 2015-09-08 2020-03-31 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases
EP3612205B9 (en) * 2017-04-21 2023-10-04 Precision Biosciences, Inc. Engineered meganucleases specific for recognition sequences in the pcsk9 gene
MA50877A (fr) * 2017-11-21 2020-09-30 Bayer Healthcare Llc Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante
CA3137975A1 (en) 2019-05-07 2020-11-12 Precision Biosciences, Inc. Optimization of engineered meganucleases for recognition sequences
CA3159316A1 (en) * 2019-12-06 2021-06-10 Benjamin OAKES Compositions and methods for the targeting of rhodopsin
WO2021212686A1 (zh) * 2020-04-21 2021-10-28 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
US20230183664A1 (en) 2020-05-11 2023-06-15 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
US12595469B2 (en) 2020-05-12 2026-04-07 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using improved engineered meganucleases
MX2024004840A (es) * 2021-10-19 2024-07-09 Prec Biosciences Inc Método de edición de genes para tratar la deficiencia de alfa-1 antitripsina (aat).
WO2025101946A1 (en) 2023-11-08 2025-05-15 Precision Biosciences, Inc. Polypeptide linkers for use in engineered meganucleases

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012167192A2 (en) * 2011-06-01 2012-12-06 Precision Biosciences, Inc. Methods and products for producing engineered mammalian cell lines with amplified transgenes

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4873192A (en) 1987-02-17 1989-10-10 The United States Of America As Represented By The Department Of Health And Human Services Process for site specific mutagenesis without phenotypic selection
US6015832A (en) 1997-12-31 2000-01-18 The Regents Of The University Of Michigan Methods of inactivating bacteria including bacterial spores
US6559189B2 (en) 1999-04-28 2003-05-06 Regents Of The University Of Michigan Non-toxic antimicrobial compositions and methods of use
US7767216B2 (en) 1999-04-28 2010-08-03 The Regents Of The University Of Michigan Antimicrobial compositions and methods of use
US6635676B2 (en) 1999-04-28 2003-10-21 Regents Of The University Of Michigan Non-toxic antimicrobial compositions and methods of use
US6506803B1 (en) 1999-04-28 2003-01-14 Regents Of The University Of Michigan Methods of preventing and treating microbial infections
CA2891996A1 (en) 2005-10-18 2007-04-26 Duke University Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity
EP2215223B1 (en) 2007-10-31 2013-05-01 Precision Biosciences, Inc. Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
EP2531609A1 (en) 2010-02-02 2012-12-12 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the diagnosis and therapy of retinitis pigmentosa
US20130183282A1 (en) 2010-05-12 2013-07-18 Cellectis Meganuclease variants cleaving a DNA target sequence from the rhodopsin gene and uses thereof
WO2012106725A2 (en) 2011-02-04 2012-08-09 Sangamo Biosciences, Inc. Methods and compositions for treating occular disorders
US20140287510A1 (en) 2011-11-08 2014-09-25 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute Rod cell-specific promoter
AU2014361781B2 (en) * 2013-12-12 2021-04-01 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing
CA2942268A1 (en) * 2014-03-12 2015-09-17 Precision Biosciences, Inc. Dystrophin gene exon deletion using engineered nucleases
US10603363B2 (en) * 2015-09-08 2020-03-31 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012167192A2 (en) * 2011-06-01 2012-12-06 Precision Biosciences, Inc. Methods and products for producing engineered mammalian cell lines with amplified transgenes

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JP2018531624A (ja) 2018-11-01
JP2025131592A (ja) 2025-09-09
EP3347463B1 (en) 2024-11-13
JP7068413B2 (ja) 2022-05-16
US10758595B2 (en) 2020-09-01
JP2024023183A (ja) 2024-02-21
US20240238387A1 (en) 2024-07-18
WO2017044649A1 (en) 2017-03-16
AU2026201608A1 (en) 2026-03-26
EP4530354A2 (en) 2025-04-02
US20220143155A1 (en) 2022-05-12
US20250121039A1 (en) 2025-04-17
AU2020294264A1 (en) 2021-01-28
US20180250370A1 (en) 2018-09-06
EP3347463C0 (en) 2024-11-13
CA2997909A1 (en) 2017-03-16
JP6775584B2 (ja) 2020-10-28
AU2023210619A1 (en) 2023-08-24
JP2022110004A (ja) 2022-07-28
US20210000928A1 (en) 2021-01-07
AU2016321244A1 (en) 2018-04-12
EP3347463A1 (en) 2018-07-18
JP2021054824A (ja) 2021-04-08
US10603363B2 (en) 2020-03-31
EP4530354A3 (en) 2025-09-10
US20200179495A1 (en) 2020-06-11

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