JP6932698B2 - アルファ1アンチトリプシン欠乏症の治療のための材料および方法 - Google Patents
アルファ1アンチトリプシン欠乏症の治療のための材料および方法 Download PDFInfo
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- JP6932698B2 JP6932698B2 JP2018529170A JP2018529170A JP6932698B2 JP 6932698 B2 JP6932698 B2 JP 6932698B2 JP 2018529170 A JP2018529170 A JP 2018529170A JP 2018529170 A JP2018529170 A JP 2018529170A JP 6932698 B2 JP6932698 B2 JP 6932698B2
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
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- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
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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]
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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
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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]
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Crystallography & Structural Chemistry (AREA)
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- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Dermatology (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)
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021133299A JP7240456B2 (ja) | 2015-12-01 | 2021-08-18 | アルファ1アンチトリプシン欠乏症の治療のための材料および方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562261661P | 2015-12-01 | 2015-12-01 | |
| US62/261,661 | 2015-12-01 | ||
| US201662324056P | 2016-04-18 | 2016-04-18 | |
| US62/324,056 | 2016-04-18 | ||
| PCT/IB2016/001845 WO2017093804A2 (en) | 2015-12-01 | 2016-12-01 | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021133299A Division JP7240456B2 (ja) | 2015-12-01 | 2021-08-18 | アルファ1アンチトリプシン欠乏症の治療のための材料および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018535691A JP2018535691A (ja) | 2018-12-06 |
| JP2018535691A5 JP2018535691A5 (https=) | 2020-05-14 |
| JP6932698B2 true JP6932698B2 (ja) | 2021-09-08 |
Family
ID=58098656
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018529170A Active JP6932698B2 (ja) | 2015-12-01 | 2016-12-01 | アルファ1アンチトリプシン欠乏症の治療のための材料および方法 |
| JP2021133299A Active JP7240456B2 (ja) | 2015-12-01 | 2021-08-18 | アルファ1アンチトリプシン欠乏症の治療のための材料および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021133299A Active JP7240456B2 (ja) | 2015-12-01 | 2021-08-18 | アルファ1アンチトリプシン欠乏症の治療のための材料および方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US11851653B2 (https=) |
| EP (2) | EP3967758A1 (https=) |
| JP (2) | JP6932698B2 (https=) |
| CN (1) | CN109715801B (https=) |
| AU (1) | AU2016364667A1 (https=) |
| CA (1) | CA3006618A1 (https=) |
| WO (1) | WO2017093804A2 (https=) |
Families Citing this family (43)
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| WO2018002730A1 (en) * | 2016-06-29 | 2018-01-04 | Crispr Therapeutics Ag | Materials and methods for treatment of apolipoprotein c3 (apociii)-related disorders |
| IL308824A (en) * | 2016-08-17 | 2024-01-01 | Factor Bioscience Inc | Nucleic acid products and methods of administration thereof |
| GB201619876D0 (en) | 2016-11-24 | 2017-01-11 | Cambridge Entpr Ltd | Controllable transcription |
| TWI851534B (zh) * | 2016-12-22 | 2024-08-11 | 美商英特利亞醫療公司 | 用於治療α-1抗胰蛋白酶缺乏症之組合物及方法 |
| AU2018224380A1 (en) * | 2017-02-22 | 2019-08-29 | Crispr Therapeutics Ag | Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (PCSK9)-related disorders |
| EP3585895A1 (en) | 2017-02-22 | 2020-01-01 | CRISPR Therapeutics AG | Compositions and methods for gene editing |
| JP7123982B2 (ja) * | 2017-06-15 | 2022-08-23 | ツールゲン インコーポレイテッド | 肝臓において目的のタンパク質を発現するためのプラットフォーム |
| SE1750774A1 (en) * | 2017-06-19 | 2018-12-11 | Hepgene Medical Ab | Novel nucleic acid molecules and their use in therapy |
| US20230140670A1 (en) * | 2017-09-29 | 2023-05-04 | Intellia Therapeutics, Inc. | Formulations |
| EP3688161A1 (en) | 2017-09-29 | 2020-08-05 | Intellia Therapeutics, Inc. | Compositions and methods for ttr gene editing and treating attr amyloidosis |
| MA50833A (fr) * | 2017-10-17 | 2020-08-26 | Bayer Healthcare Llc | Compositions et méthodes pour l'édition génique pour l'hémophilie a |
| CA3088180A1 (en) | 2018-01-12 | 2019-07-18 | Crispr Therapeutics Ag | Compositions and methods for gene editing by targeting transferrin |
| CN119570787A (zh) * | 2018-05-11 | 2025-03-07 | 比姆医疗股份有限公司 | 使用可编程碱基编辑器系统遏止病原性突变的方法 |
| AU2019265019B2 (en) | 2018-05-11 | 2025-11-06 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| KR20250133482A (ko) * | 2018-05-11 | 2025-09-05 | 빔 테라퓨틱스, 인크. | 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일 뉴클레오티드 다형성을 편집하는 방법 |
| AU2019293286A1 (en) * | 2018-06-28 | 2021-01-07 | Crispr Therapeutics Ag | Compositions and methods for genomic editing by insertion of donor polynucleotides |
| WO2020051562A2 (en) | 2018-09-07 | 2020-03-12 | Beam Therapeutics Inc. | Compositions and methods for improving base editing |
| EP3847254A4 (en) | 2018-09-07 | 2022-08-10 | Beam Therapeutics Inc. | COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM |
| JP7520826B2 (ja) * | 2018-10-17 | 2024-07-23 | クリスパー・セラピューティクス・アクチェンゲゼルシャフト | 導入遺伝子を送達するための組成物および方法 |
| AU2019360270B2 (en) | 2018-10-18 | 2025-08-07 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor IX. |
| WO2020082047A1 (en) * | 2018-10-18 | 2020-04-23 | Intellia Therapeutics, Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiencey |
| CN113272428A (zh) | 2018-10-18 | 2021-08-17 | 英特利亚治疗股份有限公司 | 核酸构建体和使用方法 |
| US20200270617A1 (en) * | 2018-10-18 | 2020-08-27 | Intellia Therapeutics, Inc. | Compositions and methods for transgene expression from an albumin locus |
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- 2016-12-01 CN CN201680080398.1A patent/CN109715801B/zh active Active
- 2016-12-01 US US15/779,836 patent/US11851653B2/en active Active
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| EP3384024B1 (en) | 2022-02-02 |
| JP7240456B2 (ja) | 2023-03-15 |
| CN109715801B (zh) | 2022-11-01 |
| WO2017093804A3 (en) | 2017-07-20 |
| US20190010490A1 (en) | 2019-01-10 |
| JP2021182934A (ja) | 2021-12-02 |
| EP3384024A2 (en) | 2018-10-10 |
| US11851653B2 (en) | 2023-12-26 |
| US20240175014A1 (en) | 2024-05-30 |
| JP2018535691A (ja) | 2018-12-06 |
| EP3967758A1 (en) | 2022-03-16 |
| AU2016364667A1 (en) | 2018-06-21 |
| CN109715801A (zh) | 2019-05-03 |
| CA3006618A1 (en) | 2017-06-08 |
| WO2017093804A2 (en) | 2017-06-08 |
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