KR20230010826A - B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 - Google Patents
B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 Download PDFInfo
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- KR20230010826A KR20230010826A KR1020237000425A KR20237000425A KR20230010826A KR 20230010826 A KR20230010826 A KR 20230010826A KR 1020237000425 A KR1020237000425 A KR 1020237000425A KR 20237000425 A KR20237000425 A KR 20237000425A KR 20230010826 A KR20230010826 A KR 20230010826A
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- hbv
- leu
- ser
- lys
- meganuclease
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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]
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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
- 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
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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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
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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
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/20—Antivirals for DNA viruses
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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
- C12N15/52—Genes encoding for enzymes or proenzymes
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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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
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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
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- C12N2730/00—Reverse transcribing DNA viruses
- C12N2730/00011—Details
- C12N2730/10011—Hepadnaviridae
- C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
- C12N2730/10122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2730/00—Reverse transcribing DNA viruses
- C12N2730/00011—Details
- C12N2730/10011—Hepadnaviridae
- C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
- C12N2730/10132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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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
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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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
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- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Communicable Diseases (AREA)
- Oncology (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| KR1020247026203A KR20240123417A (ko) | 2016-10-14 | 2017-10-13 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
Applications Claiming Priority (10)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662408356P | 2016-10-14 | 2016-10-14 | |
| US62/408,356 | 2016-10-14 | ||
| US201762452506P | 2017-01-31 | 2017-01-31 | |
| US62/452,506 | 2017-01-31 | ||
| US201762527196P | 2017-06-30 | 2017-06-30 | |
| US201762527159P | 2017-06-30 | 2017-06-30 | |
| US62/527,196 | 2017-06-30 | ||
| US62/527,159 | 2017-06-30 | ||
| KR1020217029958A KR20210118240A (ko) | 2016-10-14 | 2017-10-13 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
| PCT/US2017/056638 WO2018071849A2 (en) | 2016-10-14 | 2017-10-13 | Engineered meganucleases specific for recognition sequences in the hepatitis b virus genome |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020217029958A Division KR20210118240A (ko) | 2016-10-14 | 2017-10-13 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020247026203A Division KR20240123417A (ko) | 2016-10-14 | 2017-10-13 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20230010826A true KR20230010826A (ko) | 2023-01-19 |
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Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020237000425A Ceased KR20230010826A (ko) | 2016-10-14 | 2017-10-13 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
| KR1020197013312A Active KR102305215B1 (ko) | 2016-10-14 | 2017-10-13 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
| KR1020217029958A Ceased KR20210118240A (ko) | 2016-10-14 | 2017-10-13 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
| KR1020257040268A Pending KR20260004503A (ko) | 2016-10-14 | 2017-10-13 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
| KR1020247026203A Ceased KR20240123417A (ko) | 2016-10-14 | 2017-10-13 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020197013312A Active KR102305215B1 (ko) | 2016-10-14 | 2017-10-13 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
| KR1020217029958A Ceased KR20210118240A (ko) | 2016-10-14 | 2017-10-13 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
| KR1020257040268A Pending KR20260004503A (ko) | 2016-10-14 | 2017-10-13 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
| KR1020247026203A Ceased KR20240123417A (ko) | 2016-10-14 | 2017-10-13 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
Country Status (21)
| Country | Link |
|---|---|
| US (6) | US10662416B2 (https=) |
| EP (2) | EP4234691A3 (https=) |
| JP (5) | JP6811857B2 (https=) |
| KR (5) | KR20230010826A (https=) |
| CN (2) | CN117402852A (https=) |
| AU (3) | AU2017342536A1 (https=) |
| BR (1) | BR112019007450A2 (https=) |
| CA (1) | CA3040143A1 (https=) |
| CL (1) | CL2019000993A1 (https=) |
| CO (1) | CO2019003675A2 (https=) |
| CR (1) | CR20190181A (https=) |
| DK (1) | DK3526323T5 (https=) |
| DO (1) | DOP2019000097A (https=) |
| FI (1) | FI3526323T3 (https=) |
| IL (4) | IL317537A (https=) |
| MA (1) | MA46535A (https=) |
| MX (2) | MX2019004349A (https=) |
| PE (1) | PE20191353A1 (https=) |
| PH (1) | PH12019500788A1 (https=) |
| SG (2) | SG11201903042VA (https=) |
| WO (1) | WO2018071849A2 (https=) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
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| KR20230010826A (ko) * | 2016-10-14 | 2023-01-19 | 프리시젼 바이오사이언시스 인코포레이티드 | B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 |
| US11142750B2 (en) | 2018-04-12 | 2021-10-12 | Precision Biosciences, Inc. | Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome |
| CN114026228A (zh) | 2019-05-07 | 2022-02-08 | 精密生物科学公司 | 用于识别序列的工程化大范围核酸酶的优化 |
| DK4037708T3 (da) | 2019-09-30 | 2024-09-30 | Gilead Sciences Inc | HBV-vacciner og fremgangsmåder til at behandle HBV |
| DK4069729T3 (da) | 2019-12-06 | 2025-04-07 | Prec Biosciences Inc | Optimerede, modificerede meganukleaser med specificitet for en genkendelsessekvens i hepatitis b-virusgenomet |
| WO2021188959A1 (en) | 2020-03-20 | 2021-09-23 | Gilead Sciences, Inc. | Prodrugs of 4'-c-substituted-2-halo-2'-deoxyadenosine nucleosides and methods of making and using the same |
| US20230183664A1 (en) | 2020-05-11 | 2023-06-15 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| TW202406932A (zh) | 2020-10-22 | 2024-02-16 | 美商基利科學股份有限公司 | 介白素2-Fc融合蛋白及使用方法 |
| JP7642809B2 (ja) * | 2020-11-12 | 2025-03-10 | プレシジョン バイオサイエンシズ,インク. | ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ |
| WO2022241134A1 (en) | 2021-05-13 | 2022-11-17 | Gilead Sciences, Inc. | COMBINATION OF A TLR8 MODULATING COMPOUND AND ANTI-HBV siRNA THERAPEUTICS |
| WO2024124044A1 (en) | 2022-12-07 | 2024-06-13 | The Brigham And Women’S Hospital, Inc. | Compositions and methods targeting sat1 for enhancing anti¬ tumor immunity during tumor progression |
| WO2024192141A1 (en) | 2023-03-13 | 2024-09-19 | Dana-Farber Cancer Institute, Inc. | Treatment of cancers having a drug-resistant mesenchymal cell state |
| WO2024226838A2 (en) | 2023-04-25 | 2024-10-31 | The Brigham And Women's Hospital, Inc. | Treatment of autoimmune diseases having a pathogenic t cell state |
| AU2024270764A1 (en) | 2023-05-15 | 2025-12-04 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| WO2025049788A1 (en) | 2023-08-29 | 2025-03-06 | The Broad Institute, Inc. | Optical genetic screens of intracellular and intercellular transcriptional circuits with perturb-fish |
| WO2025096916A1 (en) | 2023-11-03 | 2025-05-08 | The Broad Institute, Inc. | Multi-site editing in living cells |
| WO2025129158A1 (en) | 2023-12-15 | 2025-06-19 | The Broad Institute, Inc. | Engineered arc delivery vesicles and uses thereof |
| WO2025240242A1 (en) | 2024-05-13 | 2025-11-20 | Gilead Sciences, Inc. | Combination therapies with ribavirin |
| WO2025240244A1 (en) | 2024-05-13 | 2025-11-20 | Gilead Sciences, Inc. | Combination therapies comprising bulevirtide and lonafarnib for use in the treatment of hepatitis d virus infection |
| WO2025240243A1 (en) | 2024-05-13 | 2025-11-20 | Gilead Sciences, Inc. | Combination therapies with bulevirtide and an inhibitory nucleic acid targeting hepatitis b virus |
| WO2025240246A1 (en) | 2024-05-13 | 2025-11-20 | Gilead Sciences, Inc. | Combination therapies with ribavirin |
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| US4873192A (en) | 1987-02-17 | 1989-10-10 | The United States Of America As Represented By The Department Of Health And Human Services | Process for site specific mutagenesis without phenotypic selection |
| EP0618924B1 (en) | 1991-12-23 | 2001-02-14 | Bayer Corporation | Hbv amplifier probes for use in solution phase sandwich hybridization assays |
| EP1288296A3 (en) * | 1992-05-11 | 2003-03-12 | Ribozyme Pharmaceuticals, Inc. | Method and reagent for inhibiting HBV viral replication |
| US6015832A (en) | 1997-12-31 | 2000-01-18 | The Regents Of The University Of Michigan | Methods of inactivating bacteria including bacterial spores |
| US7767216B2 (en) | 1999-04-28 | 2010-08-03 | The Regents Of The University Of Michigan | Antimicrobial compositions and methods of use |
| US6635676B2 (en) | 1999-04-28 | 2003-10-21 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| US6559189B2 (en) | 1999-04-28 | 2003-05-06 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| US6506803B1 (en) | 1999-04-28 | 2003-01-14 | Regents Of The University Of Michigan | Methods of preventing and treating microbial infections |
| US6555674B2 (en) | 2000-08-09 | 2003-04-29 | Nsgene A/S | JeT promoter |
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- 2017-10-13 IL IL317537A patent/IL317537A/en unknown
- 2017-10-13 AU AU2017342536A patent/AU2017342536A1/en not_active Abandoned
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