JP7522656B2 - 常染色体優性網膜色素変性の処置のための材料および方法 - Google Patents
常染色体優性網膜色素変性の処置のための材料および方法 Download PDFInfo
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- JP7522656B2 JP7522656B2 JP2020545012A JP2020545012A JP7522656B2 JP 7522656 B2 JP7522656 B2 JP 7522656B2 JP 2020545012 A JP2020545012 A JP 2020545012A JP 2020545012 A JP2020545012 A JP 2020545012A JP 7522656 B2 JP7522656 B2 JP 7522656B2
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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]
-
- 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
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- 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)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762589111P | 2017-11-21 | 2017-11-21 | |
| US62/589,111 | 2017-11-21 | ||
| US201862649133P | 2018-03-28 | 2018-03-28 | |
| US62/649,133 | 2018-03-28 | ||
| US201862693080P | 2018-07-02 | 2018-07-02 | |
| US62/693,080 | 2018-07-02 | ||
| US201862724319P | 2018-08-29 | 2018-08-29 | |
| US62/724,319 | 2018-08-29 | ||
| PCT/IB2018/059190 WO2019102381A1 (en) | 2017-11-21 | 2018-11-21 | Materials and methods for treatment of autosomal dominant retinitis pigmentosa |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021503945A JP2021503945A (ja) | 2021-02-15 |
| JP2021503945A5 JP2021503945A5 (enExample) | 2022-01-04 |
| JP7522656B2 true JP7522656B2 (ja) | 2024-07-25 |
Family
ID=64870535
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020545012A Active JP7522656B2 (ja) | 2017-11-21 | 2018-11-21 | 常染色体優性網膜色素変性の処置のための材料および方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (3) | US20190153440A1 (enExample) |
| EP (1) | EP3714055A1 (enExample) |
| JP (1) | JP7522656B2 (enExample) |
| CN (1) | CN111727251B (enExample) |
| AU (1) | AU2018372763A1 (enExample) |
| CA (1) | CA3082450A1 (enExample) |
| MA (1) | MA50877A (enExample) |
| WO (1) | WO2019102381A1 (enExample) |
Families Citing this family (45)
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| EP3707155A2 (en) * | 2017-11-09 | 2020-09-16 | Vertex Pharmaceuticals Incorporated | Crispr/cas systems for treatment of dmd |
| AU2018372763A1 (en) | 2017-11-21 | 2020-05-28 | Bayer Healthcare Llc | Materials and methods for treatment of autosomal dominant Retinitis Pigmentosa |
| US20230173105A1 (en) * | 2017-11-28 | 2023-06-08 | Emendobio Inc. | Differential knockout of an allele of a heterozygous rhodopsin gene |
| WO2020082046A2 (en) * | 2018-10-18 | 2020-04-23 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor ix |
| AU2019396622B2 (en) * | 2018-12-13 | 2024-11-07 | Nxt Biomedical, Llc | Blood oxygenation treatment methods and devices |
| AU2020227740A1 (en) * | 2019-02-25 | 2021-10-07 | Editas Medicine, Inc. | CRISPR/RNA-guided nuclease-related methods and compositions for treating RHO-associated autosomal-dominant retinitis pigmentosa (adRP) |
| CN113939595A (zh) | 2019-06-07 | 2022-01-14 | 瑞泽恩制药公司 | 包括人源化白蛋白基因座的非人动物 |
| US12173290B2 (en) * | 2019-06-28 | 2024-12-24 | Crispr Therapeutics Ag | Materials and methods for controlling gene editing |
| WO2021053582A1 (en) * | 2019-09-18 | 2021-03-25 | Crispr Therapeutics Ag | All-in-one self inactivating crispr vectors |
| EP4054651A1 (en) | 2019-11-08 | 2022-09-14 | Regeneron Pharmaceuticals, Inc. | Crispr and aav strategies for x-linked juvenile retinoschisis therapy |
| WO2021113763A1 (en) * | 2019-12-06 | 2021-06-10 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of rhodopsin |
| 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 |
| WO2021222148A2 (en) * | 2020-04-27 | 2021-11-04 | 4D Molecular Therapeutics Inc. | Adeno-associated variants, formulations and methods for pulmonary delivery |
| 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 |
| EP4222167A1 (en) | 2020-09-30 | 2023-08-09 | Nobell Foods, Inc. | Recombinant milk proteins and food compositions comprising the same |
| US10947552B1 (en) | 2020-09-30 | 2021-03-16 | Alpine Roads, Inc. | Recombinant fusion proteins for producing milk proteins in plants |
| US10894812B1 (en) | 2020-09-30 | 2021-01-19 | Alpine Roads, Inc. | Recombinant milk proteins |
| 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 (en) | 2021-01-13 | 2022-07-21 | Alia Therapeutics Srl | K526d cas9 variants and applications thereof |
| AU2022216614A1 (en) | 2021-02-05 | 2023-02-23 | Christiana Care Gene Editing Institute, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| AU2022215636A1 (en) * | 2021-02-08 | 2023-09-21 | Emendobio Inc. | Omni 90-99, 101, 104-110, 114, 116, 118-123, 125, 126, 128, 129, and 131-138 crispr nucleases |
| CA3206576A1 (en) * | 2021-02-08 | 2022-08-11 | Lior IZHAR | Omni 103 crispr nuclease |
| EP4323522A1 (en) * | 2021-04-16 | 2024-02-21 | 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 (en) | 2021-07-12 | 2023-01-19 | Alia Therapeutics Srl | Compositions and methods for allele specific treatment of retinitis pigmentosa |
| WO2023034996A1 (en) * | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| EP4408992A1 (en) * | 2021-09-27 | 2024-08-07 | Proxygen GmbH | Method for improved intron tagging and automated clone recognition |
| US20250197854A1 (en) | 2021-12-21 | 2025-06-19 | Alia Therapeutics Srl | Type ii cas proteins and applications thereof |
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| EP4522735A1 (en) * | 2022-05-13 | 2025-03-19 | Integra Therapeutics | Use of transposases for improving transgene expression and nuclear localization |
| 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 |
| CN116334141A (zh) * | 2022-08-04 | 2023-06-27 | 北京中因科技有限公司 | 基于基因编辑的RHO-R135W-adRP基因编辑药物 |
| WO2024036366A1 (en) * | 2022-08-16 | 2024-02-22 | The University Of Adelaide | Agent for treating or preventing a dominantly-inherited disease |
| WO2024056880A2 (en) | 2022-09-16 | 2024-03-21 | Alia Therapeutics Srl | Enqp type ii cas proteins and applications thereof |
| WO2024105162A1 (en) | 2022-11-16 | 2024-05-23 | Alia Therapeutics Srl | Type ii cas proteins and applications thereof |
| JP2025542309A (ja) * | 2022-12-20 | 2025-12-25 | ナノセル セラピューティクス ホールディングス ビー.ブイ. | 組み込み型ウイルスベクターおよび自己不活性化ウイルスベクターならびにその構築物および使用 |
| CN120882867A (zh) | 2023-01-11 | 2025-10-31 | 阿利亚治疗有限公司 | Ii型cas蛋白及其应用 |
| 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 (en) | 2023-06-28 | 2025-01-02 | Alia Therapeutics Srl | Type ii cas proteins and applications thereof |
| WO2025021702A1 (en) | 2023-07-21 | 2025-01-30 | Alia Therapeutics S.R.L. | Cas9 orthologue nuclease and uses thereof |
| WO2025193808A1 (en) * | 2024-03-13 | 2025-09-18 | The Regents Of The University Of California | Cns-specific gene replacement therapy for genetic forms of als/ftd |
| WO2025210147A1 (en) | 2024-04-04 | 2025-10-09 | Alia Therapeutics Srl | Type v cas proteins and applications thereof |
Citations (5)
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| WO2015089462A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
| WO2016176690A2 (en) | 2015-04-30 | 2016-11-03 | The Trustees Of Columbia University In The City Of New York | Gene therapy for autosomal dominant diseases |
| JP2017501699A (ja) | 2013-12-12 | 2017-01-19 | ザ・ブロード・インスティテュート・インコーポレイテッド | 組成物、及びヌクレオチドリピート障害におけるcrispr−cas系の使用方法 |
| WO2017044649A1 (en) | 2015-09-08 | 2017-03-16 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
| WO2017136335A1 (en) | 2016-02-01 | 2017-08-10 | The Regents Of The University Of California | Self-inactivating endonuclease-encoding nucleic acids and methods of using the same |
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- 2018-11-21 WO PCT/IB2018/059190 patent/WO2019102381A1/en not_active Ceased
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| JP2021503945A (ja) | 2021-02-15 |
| US10662425B2 (en) | 2020-05-26 |
| US20190153441A1 (en) | 2019-05-23 |
| US20190153440A1 (en) | 2019-05-23 |
| CN111727251A (zh) | 2020-09-29 |
| US12247201B2 (en) | 2025-03-11 |
| CN111727251B (zh) | 2024-09-20 |
| MA50877A (fr) | 2020-09-30 |
| AU2018372763A1 (en) | 2020-05-28 |
| US20200332288A1 (en) | 2020-10-22 |
| CA3082450A1 (en) | 2019-05-31 |
| EP3714055A1 (en) | 2020-09-30 |
| WO2019102381A1 (en) | 2019-05-31 |
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