CA2997909A1 - Treatment of retinitis pigmentosa using engineered meganucleases - Google Patents

Treatment of retinitis pigmentosa using engineered meganucleases Download PDF

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Publication number
CA2997909A1
CA2997909A1 CA2997909A CA2997909A CA2997909A1 CA 2997909 A1 CA2997909 A1 CA 2997909A1 CA 2997909 A CA2997909 A CA 2997909A CA 2997909 A CA2997909 A CA 2997909A CA 2997909 A1 CA2997909 A1 CA 2997909A1
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Canada
Prior art keywords
seq
meganuclease
residues
nos
rho
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Pending
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CA2997909A
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English (en)
French (fr)
Inventor
Victor Bartsevich
Derek Jantz
James Jefferson Smith
Michael G. Nicholson
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Precision Biosciences Inc
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Precision Biosciences Inc
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Publication of CA2997909A1 publication Critical patent/CA2997909A1/en
Pending legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52Genes encoding for enzymes or proenzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/861Adenoviral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (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)
CA2997909A 2015-09-08 2016-09-08 Treatment of retinitis pigmentosa using engineered meganucleases Pending CA2997909A1 (en)

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

Publications (1)

Publication Number Publication Date
CA2997909A1 true CA2997909A1 (en) 2017-03-16

Family

ID=57003577

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2997909A Pending CA2997909A1 (en) 2015-09-08 2016-09-08 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)

* 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
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
CA2997909A1 (en) * 2015-09-08 2017-03-16 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases
KR20230170125A (ko) * 2017-04-21 2023-12-18 프리시젼 바이오사이언시스 인코포레이티드 Pcsk9 유전자 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제
AU2018372763A1 (en) * 2017-11-21 2020-05-28 Bayer Healthcare Llc Materials and methods for treatment of autosomal dominant Retinitis Pigmentosa
CN120843482A (zh) 2019-05-07 2025-10-28 精密生物科学公司 用于识别序列的工程化大范围核酸酶的优化
WO2021113763A1 (en) * 2019-12-06 2021-06-10 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
WO2021231495A1 (en) * 2020-05-12 2021-11-18 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using improved engineered meganucleases
US20250228973A1 (en) * 2021-10-19 2025-07-17 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

Family Cites Families (16)

* 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
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
EP2628794B1 (en) 2005-10-18 2016-05-04 Precision Biosciences Rationally designed meganucleases with altered sequence specificity and DNA-binding affinity
WO2009059195A2 (en) 2007-10-31 2009-05-07 Precision Biosciences 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
US20120204282A1 (en) 2011-02-04 2012-08-09 Sangamo Biosciences, Inc. Methods and compositions for treating occular disorders
EP3683319A1 (en) 2011-06-01 2020-07-22 Precision Biosciences, Inc. Methods and products for producing engineered mammalian cell lines with amplified transgenes
US20140287510A1 (en) 2011-11-08 2014-09-25 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute Rod cell-specific promoter
MX374529B (es) * 2013-12-12 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma.
ES3037550T3 (en) * 2014-03-12 2025-10-03 Prec Biosciences Inc Dystrophin gene exon deletion using engineered nucleases
CA2997909A1 (en) 2015-09-08 2017-03-16 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases

Also Published As

Publication number Publication date
AU2016321244A1 (en) 2018-04-12
JP2024023183A (ja) 2024-02-21
JP2018531624A (ja) 2018-11-01
EP3347463C0 (en) 2024-11-13
EP4530354A2 (en) 2025-04-02
AU2016321244B2 (en) 2020-09-24
US20180250370A1 (en) 2018-09-06
US20210000928A1 (en) 2021-01-07
AU2020294264A1 (en) 2021-01-28
US20250121039A1 (en) 2025-04-17
EP4530354A3 (en) 2025-09-10
JP2021054824A (ja) 2021-04-08
US10603363B2 (en) 2020-03-31
US20240238387A1 (en) 2024-07-18
AU2023210619A1 (en) 2023-08-24
EP3347463B1 (en) 2024-11-13
JP2025131592A (ja) 2025-09-09
JP7068413B2 (ja) 2022-05-16
JP6775584B2 (ja) 2020-10-28
US20200179495A1 (en) 2020-06-11
EP3347463A1 (en) 2018-07-18
JP2022110004A (ja) 2022-07-28
US10758595B2 (en) 2020-09-01
WO2017044649A1 (en) 2017-03-16
US20220143155A1 (en) 2022-05-12

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