MA50877A - Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante - Google Patents

Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante

Info

Publication number
MA50877A
MA50877A MA050877A MA50877A MA50877A MA 50877 A MA50877 A MA 50877A MA 050877 A MA050877 A MA 050877A MA 50877 A MA50877 A MA 50877A MA 50877 A MA50877 A MA 50877A
Authority
MA
Morocco
Prior art keywords
pigmental
retinitis
treatment
materials
methods
Prior art date
Application number
MA050877A
Other languages
English (en)
Inventor
Albena Kantardzhieva
Akiko Noma
Abraham Scaria
Ryo Takeuchi
Original Assignee
Bayer Healthcare Llc
Crispr Therapeutics Ag
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 Bayer Healthcare Llc, Crispr Therapeutics Ag filed Critical Bayer Healthcare Llc
Publication of MA50877A publication Critical patent/MA50877A/fr

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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • 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
    • 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
    • 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
    • C12N15/11—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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • 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
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
MA050877A 2017-11-21 2018-11-21 Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante MA50877A (fr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762589111P 2017-11-21 2017-11-21
US201862649133P 2018-03-28 2018-03-28
US201862693080P 2018-07-02 2018-07-02
US201862724319P 2018-08-29 2018-08-29

Publications (1)

Publication Number Publication Date
MA50877A true MA50877A (fr) 2020-09-30

Family

ID=64870535

Family Applications (1)

Application Number Title Priority Date Filing Date
MA050877A MA50877A (fr) 2017-11-21 2018-11-21 Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante

Country Status (8)

Country Link
US (3) US10662425B2 (fr)
EP (1) EP3714055A1 (fr)
JP (1) JP7522656B2 (fr)
CN (1) CN111727251B (fr)
AU (1) AU2018372763A1 (fr)
CA (1) CA3082450A1 (fr)
MA (1) MA50877A (fr)
WO (1) WO2019102381A1 (fr)

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MA50579A (fr) * 2017-11-09 2020-09-16 Crispr Therapeutics Ag Systèmes crispr/cas ou crispr/cpf1 à auto-inactivation (sin) et leurs utilisations
EP3714055A1 (fr) 2017-11-21 2020-09-30 CRISPR Therapeutics AG Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante
US20230173105A1 (en) * 2017-11-28 2023-06-08 Emendobio Inc. Differential knockout of an allele of a heterozygous rhodopsin gene
JP7578590B2 (ja) * 2018-10-18 2024-11-06 インテリア セラピューティクス，インコーポレーテッド 第ｉｘ因子を発現するための組成物及び方法
AU2019396622B2 (en) * 2018-12-13 2024-11-07 Edwards Lifesciences Corporation Blood oxygenation treatment methods and devices
US20220133768A1 (en) * 2019-02-25 2022-05-05 Editas Medicine, Inc. Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp)
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US12173290B2 (en) * 2019-06-28 2024-12-24 Crispr Therapeutics Ag Materials and methods for controlling gene editing
CN118813654A (zh) 2019-08-12 2024-10-22 生命编辑制药股份有限公司 Rna指导的核酸酶及其活性片段及变体以及使用方法
WO2021053582A1 (fr) * 2019-09-18 2021-03-25 Crispr Therapeutics Ag Vecteurs crispr à auto-inactivation tout-en-un
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
WO2021113763A1 (fr) * 2019-12-06 2021-06-10 Scribe Therapeutics Inc. Compositions et méthodes pour le ciblage de la rhodopsine
WO2021122944A1 (fr) 2019-12-18 2021-06-24 Alia Therapeutics Srl Compositions et méthodes de traitement de la rétinite pigmentaire
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WO2022152746A1 (fr) 2021-01-13 2022-07-21 Alia Therapeutics Srl Variants de cas9 k526d et applications associées
AU2022216614B2 (en) 2021-02-05 2026-03-05 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
WO2022170216A2 (fr) * 2021-02-08 2022-08-11 Emendobio Inc. Nucléases crispr omni 90-99, 101, 104-110, 114, 116, 118-123, 125, 126, 128, 129 et 131-138
KR20230142740A (ko) 2021-02-08 2023-10-11 에멘도바이오 인코포레이티드 Omni-103 crispr 뉴클레아제
WO2022221741A1 (fr) * 2021-04-16 2022-10-20 Editas Medicine, Inc. Méthodes et compositions associées à la nucléase guidée par crispr/arn pour le traitement de la rétinite pigmentaire autosomique dominante associée à rho (adrp)
CN113549650B (zh) * 2021-07-05 2023-05-09 天津协和生物科技开发有限公司 一种CRISPR-SaCas9基因编辑系统及其应用
WO2023285431A1 (fr) 2021-07-12 2023-01-19 Alia Therapeutics Srl Compositions et procédés de traitement spécifique d'allèle de rétinite pigmentaire
JP2024534894A (ja) * 2021-09-03 2024-09-26 ビオマリン プハルマセウトイカル インコーポレイテッド Ａａｖカプシド組成物及び送達方法
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JP2026502531A (ja) 2023-01-11 2026-01-23 アリア セラピューティクス エスアールエル Ｉｉ型ｃａｓタンパク質およびその適用
CN116716345A (zh) * 2023-05-10 2023-09-08 赛业（苏州）生物科技有限公司 一种用于制备人源化rho和或rho突变体小鼠的方法及其应用
WO2025003344A1 (fr) 2023-06-28 2025-01-02 Alia Therapeutics Srl Protéines cas de type ii et leurs applications
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US20190153441A1 (en) 2019-05-23
JP7522656B2 (ja) 2024-07-25
US20190153440A1 (en) 2019-05-23
AU2018372763A1 (en) 2020-05-28
US12247201B2 (en) 2025-03-11
CA3082450A1 (fr) 2019-05-31
WO2019102381A1 (fr) 2019-05-31
US20200332288A1 (en) 2020-10-22
CN111727251B (zh) 2024-09-20
US10662425B2 (en) 2020-05-26
EP3714055A1 (fr) 2020-09-30

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