CA3219160A1 - Genomic safe harbors - Google Patents

Genomic safe harbors Download PDF

Info

Publication number
CA3219160A1
CA3219160A1 CA3219160A CA3219160A CA3219160A1 CA 3219160 A1 CA3219160 A1 CA 3219160A1 CA 3219160 A CA3219160 A CA 3219160A CA 3219160 A CA3219160 A CA 3219160A CA 3219160 A1 CA3219160 A1 CA 3219160A1
Authority
CA
Canada
Prior art keywords
cell
nucleic acid
gsh
protein
promoter
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
CA3219160A
Other languages
English (en)
French (fr)
Inventor
Robert Kotin
Charlotte Mcguinness
Sebastian AGUIRRE
Shannon LONCAR
Robert Gifford
Matthew A. Campbell
Marco Antonio Quezada Ramirez
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.)
Synteny Therapeutics Inc
University of Massachusetts Amherst
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA3219160A1 publication Critical patent/CA3219160A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B25/00—ICT specially adapted for hybridisation; ICT specially adapted for gene or protein expression
    • G16B25/10—Gene or protein expression profiling; Expression-ratio estimation or normalisation
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • 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
    • 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
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6897—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids involving reporter genes operably linked to promoters
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Environmental Sciences (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Analytical Chemistry (AREA)
  • Evolutionary Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medical Informatics (AREA)
  • Spectroscopy & Molecular Physics (AREA)
  • Theoretical Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Animal Husbandry (AREA)
  • Virology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3219160A 2021-05-20 2022-05-19 Genomic safe harbors Pending CA3219160A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163190996P 2021-05-20 2021-05-20
US63/190,996 2021-05-20
PCT/US2022/030024 WO2022246063A1 (en) 2021-05-20 2022-05-19 Genomic safe harbors

Publications (1)

Publication Number Publication Date
CA3219160A1 true CA3219160A1 (en) 2022-11-24

Family

ID=84141733

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3219160A Pending CA3219160A1 (en) 2021-05-20 2022-05-19 Genomic safe harbors

Country Status (7)

Country Link
US (1) US20250087304A1 (OSRAM)
EP (1) EP4352519A4 (OSRAM)
JP (1) JP2024521679A (OSRAM)
KR (1) KR20240023030A (OSRAM)
AU (1) AU2022277688A1 (OSRAM)
CA (1) CA3219160A1 (OSRAM)
WO (1) WO2022246063A1 (OSRAM)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024334482C1 (en) * 2023-08-31 2026-04-23 Kenai Therapeutics Inc. Methods and compositions for engineered da neuronal cells
CN118256559B (zh) * 2024-01-24 2024-09-17 中国医学科学院阜外医院 制备射血分数保留的心衰的动物模型的方法
WO2026064739A1 (en) * 2024-09-23 2026-03-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2539445B1 (en) * 2010-02-26 2018-03-21 Cellectis Use of endonucleases for inserting transgenes into safe harbor loci
CN110891420B (zh) * 2017-07-31 2022-06-03 瑞泽恩制药公司 Cas转基因小鼠胚胎干细胞和小鼠及其应用
KR20200093635A (ko) * 2017-12-06 2020-08-05 제너레이션 바이오 컴퍼니 변형된 폐쇄된 말단 dna (cedna)를 사용한 유전자 편집
EP3759226A4 (en) * 2018-03-02 2022-06-15 Generation Bio Co. IDENTIFICATION AND CHARACTERIZATION OF GENOMIC SAFE HARBOR (GSH) IN HUMAN AND MURINE GENOMES AND VIRAL AND NON-VIRAL VECTOR COMPOSITIONS FOR TARGETED INTEGRATION AT IDENTIFIED GSH LOCI
WO2019169233A1 (en) * 2018-03-02 2019-09-06 Generation Bio Co. Closed-ended dna (cedna) vectors for insertion of transgenes at genomic safe harbors (gsh) in humans and murine genomes
US20200370067A1 (en) * 2019-05-21 2020-11-26 University Of Washington Method to identify and validate genomic safe harbor sites for targeted genome engineering
AU2020348776A1 (en) * 2019-09-17 2022-05-05 Memorial Sloan-Kettering Cancer Center Genomic safe harbors for transgene integration
WO2021055592A1 (en) * 2019-09-17 2021-03-25 Memorial Sloan-Kettering Cancer Center Methods for identifying genomic safe harbors
EP4034640A4 (en) * 2019-09-23 2023-10-25 Regents Of The University Of Minnesota GENETICALLY EDITED IMMUNE CELLS AND THERAPY METHODS
JP7664913B2 (ja) * 2019-10-17 2025-04-18 フェイト セラピューティクス，インコーポレイテッド 免疫のための増強されたキメラ抗原受容体エフェクター細胞操作およびその使用

Also Published As

Publication number Publication date
US20250087304A1 (en) 2025-03-13
WO2022246063A1 (en) 2022-11-24
EP4352519A1 (en) 2024-04-17
EP4352519A4 (en) 2025-05-14
AU2022277688A1 (en) 2023-12-21
JP2024521679A (ja) 2024-06-04
KR20240023030A (ko) 2024-02-20

Similar Documents

Publication Publication Date Title
US20250000071A1 (en) Identifying and characterizing genomic safe harbors (gsh) in humans and murine genomes, and viral and non-viral vector compositions for targeted integration at an identified gsh loci
JP7858729B2 (ja) 抗体コード配列をセーフハーバー遺伝子座に挿入するための方法および組成物
JP7448953B2 (ja) 眼疾患のための細胞モデル及び治療関連出願への相互参照
US20250087304A1 (en) Genomic safe harbors
CA3080546A1 (en) Hpv-specific binding molecules
AU2019226527A1 (en) Closed-ended DNA (ceDNA) vectors for insertion of transgenes at genomic safe harbors (GSH) in humans and murine genomes
US20240066080A1 (en) Protoparvovirus and tetraparvovirus compositions and methods for gene therapy
JP7406253B2 (ja) 免疫回避性ベクターおよび遺伝子療法のための使用
WO2021108363A1 (en) Crispr/cas-mediated upregulation of humanized ttr allele
US20250302994A1 (en) Erythroparvovirus with a modified genome for gene therapy
JP2023507174A (ja) Ｄｍｄ変異の修正のための方法及び組成物
US20250295810A1 (en) Erythroparvovirus with a modified capsid for gene therapy
HK40102528A (zh) 用於基因疗法的原细小病毒和四细小病毒组合物和方法

Legal Events

Date Code Title Description
MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD

Year of fee payment: 3

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250509

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250509