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 RNAses, DNAses
    • 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 [CRISPRs]
    • 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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (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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EP3707256A1 (fr) * 2017-11-09 2020-09-16 CRISPR Therapeutics AG Systèmes crispr/cas ou crispr/cpf1 à auto-inactivation (sin) et leurs utilisations
US20230173105A1 (en) * 2017-11-28 2023-06-08 Emendobio Inc. Differential knockout of an allele of a heterozygous rhodopsin gene
JP2022512731A (ja) * 2018-10-18 2022-02-07 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
EP3931326A1 (fr) * 2019-02-25 2022-01-05 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)
SG11202111256XA (en) 2019-06-07 2021-11-29 Regeneron Pharma Non-human animals comprising a humanized albumin locus
EP3990636A1 (fr) * 2019-06-28 2022-05-04 CRISPR Therapeutics AG Matériels et méthodes de régulation de l'édition de gènes
WO2021053582A1 (fr) * 2019-09-18 2021-03-25 Crispr Therapeutics Ag Vecteurs crispr à auto-inactivation tout-en-un
WO2021113763A1 (fr) 2019-12-06 2021-06-10 Scribe Therapeutics Inc. Compositions et méthodes pour le ciblage de la rhodopsine
US20230054569A1 (en) 2019-12-18 2023-02-23 Alia Therapeutics Srl Compositions and methods for treating retinitis pigmentosa
IT202000008014A1 (it) * 2020-04-15 2021-10-15 Fond Telethon RNA guida e loro usi
US20230183664A1 (en) * 2020-05-11 2023-06-15 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
CN111850044A (zh) * 2020-07-16 2020-10-30 中国科学技术大学 基于在体基因敲除的视网膜色素变性猕猴模型构建方法
CN112175909B (zh) * 2020-09-11 2023-04-28 中山大学中山眼科中心 一株vsx2绿色荧光报告基因人诱导多能干细胞系及其构建方法
US10894812B1 (en) 2020-09-30 2021-01-19 Alpine Roads, Inc. Recombinant milk proteins
US10947552B1 (en) 2020-09-30 2021-03-16 Alpine Roads, Inc. Recombinant fusion proteins for producing milk proteins in plants
CA3191387A1 (fr) 2020-09-30 2022-04-07 Nobell Foods, Inc. Proteines de lait recombinantes et compositions les comprenant
CN111926044B (zh) * 2020-10-12 2021-01-22 北京大学第三医院（北京大学第三临床医学院） 结合突变rho基因的核酸分子和试剂盒
WO2022152746A1 (fr) 2021-01-13 2022-07-21 Alia Therapeutics Srl Variants de cas9 k526d et applications associées
US20240207448A1 (en) * 2021-04-16 2024-06-27 Editas Medicine, Inc. Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (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
EP4396202A1 (fr) * 2021-09-03 2024-07-10 BioMarin Pharmaceutical Inc. Compositions capsidiques de vaa et méthodes d'administration
EP4408992A1 (fr) * 2021-09-27 2024-08-07 Proxygen GmbH Procédé pour améliorer le marquage par introns et la reconnaissance automatique des clones
WO2023118349A1 (fr) 2021-12-21 2023-06-29 Alia Therapeutics Srl Protéines cas de type ii et leurs applications
WO2023194359A1 (fr) 2022-04-04 2023-10-12 Alia Therapeutics Srl Compositions et méthodes de traitement du syndrome d'usher de type 2a
WO2023218021A1 (fr) * 2022-05-13 2023-11-16 Integra Therapeutics Utilisation de transposases pour améliorer l'expression transgénique et la localisation nucléaire
CN116064517A (zh) * 2022-07-29 2023-05-05 之江实验室 一种先导编辑gRNA的产生方式及其用途
WO2024036366A1 (fr) * 2022-08-16 2024-02-22 The University Of Adelaide Agent pour le traitement ou la prévention d'une maladie à prédominance héréditaire
WO2024056880A2 (fr) 2022-09-16 2024-03-21 Alia Therapeutics Srl Protéines cas de type ii enqp et leurs applications
WO2024105162A1 (fr) 2022-11-16 2024-05-23 Alia Therapeutics Srl Protéines cas de type ii et leurs applications
WO2024133600A1 (fr) * 2022-12-20 2024-06-27 Nanocell Therapeutics B.V. Vecteurs viraux à intégration et à auto-inactivation et constructions et utilisations de ceux-ci
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