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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    • 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)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2016321244A 2015-09-08 2016-09-08 Treatment of retinitis pigmentosa using engineered meganucleases Active AU2016321244B2 (en)

Priority Applications (2)

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

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)

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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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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 Pending AU2023210619A1 (en) 2015-09-08 2023-08-03 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 Pending AU2023210619A1 (en) 2015-09-08 2023-08-03 Treatment of retinitis pigmentosa using engineered meganucleases

Country Status (6)

Country Link
US (6) US10603363B2 (enExample)
EP (2) EP4530354A3 (enExample)
JP (5) JP6775584B2 (enExample)
AU (3) AU2016321244B2 (enExample)
CA (1) CA2997909A1 (enExample)
WO (1) WO2017044649A1 (enExample)

Families Citing this family (13)

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Publication number Priority date Publication date Assignee Title
EP3114227B1 (en) 2014-03-05 2021-07-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
EP3126495A1 (en) 2014-04-02 2017-02-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
US11180793B2 (en) 2015-04-24 2021-11-23 Editas Medicine, Inc. Evaluation of Cas9 molecule/guide RNA molecule complexes
EP4530354A3 (en) * 2015-09-08 2025-09-10 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases
CN110769845B (zh) * 2017-04-21 2023-08-15 精密生物科学公司 对pcsk9基因中的识别序列具有特异性的工程化大范围核酸酶
US10662425B2 (en) 2017-11-21 2020-05-26 Crispr Therapeutics Ag Materials and methods for treatment of autosomal dominant retinitis pigmentosa
JP2022531459A (ja) 2019-05-07 2022-07-06 プレシジョン バイオサイエンシズ，インク． 認識配列に対する、操作されたメガヌクレアーゼの最適化
EP4069845A1 (en) * 2019-12-06 2022-10-12 Scribe Therapeutics Inc. 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
CA3172171A1 (en) * 2020-05-12 2021-11-18 Victor Bartsevich Treatment of retinitis pigmentosa using improved engineered meganucleases
EP4419660A2 (en) * 2021-10-19 2024-08-28 Precision BioSciences, Inc. Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency
WO2025101946A1 (en) 2023-11-08 2025-05-15 Precision Biosciences, Inc. Polypeptide linkers for use in engineered meganucleases

Citations (1)

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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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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
US6506803B1 (en) 1999-04-28 2003-01-14 Regents Of The University Of Michigan Methods of preventing and treating microbial infections
US6559189B2 (en) 1999-04-28 2003-05-06 Regents Of The University Of Michigan Non-toxic 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
US7767216B2 (en) 1999-04-28 2010-08-03 The Regents Of The University Of Michigan Antimicrobial compositions and methods of use
ES2602184T3 (es) 2005-10-18 2017-02-20 Precision Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
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
EP2569435A1 (en) * 2010-05-12 2013-03-20 Cellectis Meganuclease variants cleaving a dna target sequence from the rhodopsin gene and uses thereof
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DK3116533T3 (da) * 2014-03-12 2020-08-24 Prec Biosciences Inc Deletion af dystrophingenexon under anvendelse af genmanipulerede nukleaser
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Patent Citations (1)

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

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