JP2024529899A - 関節機能を改善する遺伝子編集 - Google Patents

関節機能を改善する遺伝子編集 Download PDF

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JP2024529899A
JP2024529899A JP2024502462A JP2024502462A JP2024529899A JP 2024529899 A JP2024529899 A JP 2024529899A JP 2024502462 A JP2024502462 A JP 2024502462A JP 2024502462 A JP2024502462 A JP 2024502462A JP 2024529899 A JP2024529899 A JP 2024529899A
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exon
gene
nucleotides
composition
guide rna
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JP2024529899A5 (enExample
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ゲンチュ，ジョージ
ミレット，ピーター・ジェイ
ラッセル，イアン・アラスダー
アレン，マシュー・ジェイ
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オーソバイオ・セラピューティクス，インコーポレイテッド
ジョージ・ゲンチュ
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Priority claimed from PCT/US2021/042100 external-priority patent/WO2022016121A2/en
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
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    • 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
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    • C12N15/102—Mutagenizing nucleic acids
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    • 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
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    • 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
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    • 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
    • C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
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    • 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
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    • 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
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    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/316—Phosphonothioates
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    • 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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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dispersion Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
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  • Crystallography & Structural Chemistry (AREA)
  • Endocrinology (AREA)
JP2024502462A 2021-07-16 2022-07-18 関節機能を改善する遺伝子編集 Pending JP2024529899A (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
USPCT/US2021/042100 2021-07-16
PCT/US2021/042100 WO2022016121A2 (en) 2020-07-16 2021-07-16 Gene editing to improve joint function
US202163222972P 2021-07-17 2021-07-17
US63/222,972 2021-07-17
US202263300822P 2022-01-19 2022-01-19
US63/300,822 2022-01-19
US202263326571P 2022-04-01 2022-04-01
US63/326,571 2022-04-01
PCT/US2022/037490 WO2023288135A2 (en) 2021-07-16 2022-07-18 Gene editing to improve joint function

Publications (2)

Publication Number Publication Date
JP2024529899A true JP2024529899A (ja) 2024-08-14
JP2024529899A5 JP2024529899A5 (enExample) 2025-07-24

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JP2024502462A Pending JP2024529899A (ja) 2021-07-16 2022-07-18 関節機能を改善する遺伝子編集

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US (1) US20230235321A1 (enExample)
EP (1) EP4370165A4 (enExample)
JP (1) JP2024529899A (enExample)
KR (1) KR20240095529A (enExample)
CN (1) CN119110732A (enExample)
AU (1) AU2022311936A1 (enExample)
CA (1) CA3226089A1 (enExample)
IL (1) IL310118A (enExample)
MX (1) MX2024000821A (enExample)
WO (1) WO2023288135A2 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024215959A2 (en) * 2023-04-11 2024-10-17 Orthobio Therapeutics, Inc. Lipid nanoparticles for gene editing systems

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020112908A2 (en) * 2018-11-28 2020-06-04 Casebia Therapeutics Limited Liability Partnership OPTIMIZED mRNA ENCODING CAS9 FOR USE IN LNPs
US20200360454A1 (en) * 2019-01-18 2020-11-19 Orthobio Therapeutics, Inc. Gene Editing to Impove Joint Function

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050282198A1 (en) * 1997-05-29 2005-12-22 Interleukin Genetics, Inc. Diagnostics and therapeutics for diseases associated with an IL-1 inflammatory haplotype
EP3230452B1 (en) * 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
EP3374494A4 (en) * 2015-11-11 2019-05-01 Coda Biotherapeutics, Inc. CRISPR COMPOSITIONS AND METHODS OF USE THEREOF FOR GENE THERAPY
ES3030658T3 (en) * 2016-03-30 2025-07-01 Intellia Therapeutics Inc Lipid nanoparticle formulations for crispr/cas components
US11807850B2 (en) * 2017-12-06 2023-11-07 Klogenix Llc Compositions and methods for modulating gene expression
CN112533942A (zh) * 2018-06-01 2021-03-19 华盛顿大学 嵌合抗原受体细胞疗法中细胞因子释放综合征的抑制
CA3144356A1 (en) * 2019-06-21 2020-12-24 Function Oncology, Inc. A genetic pharmacopeia for comprehensive functional profiling of human cancers

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020112908A2 (en) * 2018-11-28 2020-06-04 Casebia Therapeutics Limited Liability Partnership OPTIMIZED mRNA ENCODING CAS9 FOR USE IN LNPs
US20200360454A1 (en) * 2019-01-18 2020-11-19 Orthobio Therapeutics, Inc. Gene Editing to Impove Joint Function

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MX2024000821A (es) 2024-06-04
CN119110732A (zh) 2024-12-10
EP4370165A4 (en) 2025-10-01
WO2023288135A3 (en) 2023-02-23
EP4370165A2 (en) 2024-05-22
AU2022311936A1 (en) 2024-02-29
KR20240095529A (ko) 2024-06-25
CA3226089A1 (en) 2023-01-19
US20230235321A1 (en) 2023-07-27
WO2023288135A2 (en) 2023-01-19
IL310118A (en) 2024-03-01

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