EP4408534A4 - Methods and systems for modifying the CRUMB homolog 1 gene - Google Patents

Methods and systems for modifying the CRUMB homolog 1 gene

Info

Publication number
EP4408534A4
EP4408534A4 EP22873938.9A EP22873938A EP4408534A4 EP 4408534 A4 EP4408534 A4 EP 4408534A4 EP 22873938 A EP22873938 A EP 22873938A EP 4408534 A4 EP4408534 A4 EP 4408534A4
Authority
EP
European Patent Office
Prior art keywords
crumb
homolog
gene
modifying
systems
Prior art date
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
EP22873938.9A
Other languages
German (de)
French (fr)
Other versions
EP4408534A1 (en
Inventor
Peter M J Quinn
Da Costa Bruna Lopes
Stephen H Tsang
David R Liu
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.)
Columbia University in the City of New York
Broad Institute Inc
Harvard University
Original Assignee
Columbia University in the City of New York
Broad Institute Inc
Harvard University
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Columbia University in the City of New York, Broad Institute Inc, Harvard University filed Critical Columbia University in the City of New York
Publication of EP4408534A1 publication Critical patent/EP4408534A1/en
Publication of EP4408534A4 publication Critical patent/EP4408534A4/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • C12N15/63—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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
EP22873938.9A 2021-09-27 2022-09-27 Methods and systems for modifying the CRUMB homolog 1 gene Pending EP4408534A4 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163248808P 2021-09-27 2021-09-27
PCT/US2022/077100 WO2023049931A1 (en) 2021-09-27 2022-09-27 Methods and systems for modifying the crumbs homologue-1 (crb1) gene

Publications (2)

Publication Number Publication Date
EP4408534A1 EP4408534A1 (en) 2024-08-07
EP4408534A4 true EP4408534A4 (en) 2025-08-27

Family

ID=85721320

Family Applications (1)

Application Number Title Priority Date Filing Date
EP22873938.9A Pending EP4408534A4 (en) 2021-09-27 2022-09-27 Methods and systems for modifying the CRUMB homolog 1 gene

Country Status (2)

Country Link
EP (1) EP4408534A4 (en)
WO (1) WO2023049931A1 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024226986A1 (en) * 2023-04-26 2024-10-31 The Trustees Of Columbia University In The City Of New York Methods and systems for modifying the crumbs homologue-1 (crb1) gene

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015020522A1 (en) * 2013-08-05 2015-02-12 Koninklijke Nederlandse Akademie Van Wetenschappen Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp
WO2021138469A1 (en) * 2019-12-30 2021-07-08 The Broad Institute, Inc. Genome editing using reverse transcriptase enabled and fully active crispr complexes

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9957558B2 (en) * 2011-04-28 2018-05-01 Life Technologies Corporation Methods and compositions for multiplex PCR
ES2697249T3 (en) * 2013-03-11 2019-01-22 Fond Telethon miR-204 and miR-211 and uses thereof
US10113201B2 (en) * 2013-04-05 2018-10-30 The Wistar Institute Of Anatomy And Biology Methods and compositions for diagnosis of glioblastoma or a subtype thereof
JP7657726B2 (en) * 2019-03-19 2025-04-07 ザ ブロード インスティテュート，インコーポレーテッド Editing Methods and compositions for editing nucleotide sequences

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015020522A1 (en) * 2013-08-05 2015-02-12 Koninklijke Nederlandse Akademie Van Wetenschappen Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp
WO2021138469A1 (en) * 2019-12-30 2021-07-08 The Broad Institute, Inc. Genome editing using reverse transcriptase enabled and fully active crispr complexes

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
BRUNA LOPES DA COSTA ET AL: "Development of a Prime Editing Strategy to Treat Mutations in the Crumbs Homologue 1 (CRB1) Gene", MOLECULAR THERAPY, vol. 30, no. 4 suppl 1, 1 April 2022 (2022-04-01), pages 260 - 261, XP093294524 *
GALLEGO CARMEN ET AL: "Novel Therapeutic Approaches for the Treatment of Retinal Degenerative Diseases: Focus on CRISPR/Cas-Based Gene Editing", FRONTIERS IN NEUROSCIENCE, vol. 14, 20 August 2020 (2020-08-20), CH, XP055880788, ISSN: 1662-4548, Retrieved from the Internet <URL:http://frontiersin.org/neuroscience/> DOI: 10.3389/fnins.2020.00838 *
See also references of WO2023049931A1 *

Also Published As

Publication number Publication date
EP4408534A1 (en) 2024-08-07
WO2023049931A1 (en) 2023-03-30

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