AU2017324462B2 - Modulation of liver genes - Google Patents
Modulation of liver genes Download PDFInfo
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- AU2017324462B2 AU2017324462B2 AU2017324462A AU2017324462A AU2017324462B2 AU 2017324462 B2 AU2017324462 B2 AU 2017324462B2 AU 2017324462 A AU2017324462 A AU 2017324462A AU 2017324462 A AU2017324462 A AU 2017324462A AU 2017324462 B2 AU2017324462 B2 AU 2017324462B2
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/10—Antioedematous agents; Diuretics
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/81—Protease inhibitors
- C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
- C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
- C07K14/8121—Serpins
- C07K14/8125—Alpha-1-antitrypsin
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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
- C12N15/63—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
- C12N15/861—Adenoviral vectors
- C12N15/8616—Special methods for targeting systems
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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
- C12N15/63—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
- C12N15/867—Retroviral vectors
- C12N15/8676—Special methods for targeting systems
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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
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0006—Oxidoreductases (1.) acting on CH-OH groups as donors (1.1)
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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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- 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)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
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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)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
- C12N9/6445—Kallikreins (3.4.21.34; 3.4.21.35)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
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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
- 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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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Toxicology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Epidemiology (AREA)
- Urology & Nephrology (AREA)
- Pulmonology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662384428P | 2016-09-07 | 2016-09-07 | |
| US62/384,428 | 2016-09-07 | ||
| PCT/US2017/050446 WO2018049009A2 (en) | 2016-09-07 | 2017-09-07 | Modulation of liver genes |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2017324462A1 AU2017324462A1 (en) | 2019-03-14 |
| AU2017324462B2 true AU2017324462B2 (en) | 2024-03-21 |
Family
ID=61281860
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2017324462A Expired - Fee Related AU2017324462B2 (en) | 2016-09-07 | 2017-09-07 | Modulation of liver genes |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US10960085B2 (enExample) |
| EP (1) | EP3509645A4 (enExample) |
| JP (2) | JP7256739B2 (enExample) |
| AU (1) | AU2017324462B2 (enExample) |
| CA (1) | CA3035534A1 (enExample) |
| WO (1) | WO2018049009A2 (enExample) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
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| MX2019006426A (es) | 2016-12-01 | 2019-08-14 | Sangamo Therapeutics Inc | Reguladores de tau, y composiciones y metodos para su administracion. |
| US11820728B2 (en) | 2017-04-28 | 2023-11-21 | Acuitas Therapeutics, Inc. | Carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| EP3585162B1 (en) | 2017-09-29 | 2023-08-30 | Regeneron Pharmaceuticals, Inc. | Rodents comprising a humanized ttr locus and methods of use |
| WO2020010186A1 (en) * | 2018-07-06 | 2020-01-09 | Derek Klarin | Pcsk9 variants |
| CA3113190A1 (en) * | 2018-07-31 | 2020-02-06 | Intellia Therapeutics, Inc. | Compositions and methods for hydroxyacid oxidase 1 (hao1) gene editing for treating primary hyperoxaluria type 1 (ph1) |
| EP3852911B1 (en) | 2018-09-21 | 2025-01-22 | Acuitas Therapeutics, Inc. | Systems and methods for manufacturing lipid nanoparticles and liposomes |
| CN113226333A (zh) | 2018-10-02 | 2021-08-06 | 桑格摩生物治疗股份有限公司 | 用于调控Tau蛋白的方法和组合物 |
| EP3861130A4 (en) * | 2018-10-02 | 2022-08-03 | Sangamo Therapeutics, Inc. | GENETIC MODULATORS |
| BR112021007301A2 (pt) | 2018-10-18 | 2021-07-27 | Intellia Therapeutics, Inc. | composições e métodos para expressar fator ix |
| EP3898661A1 (en) * | 2018-12-21 | 2021-10-27 | Precision BioSciences, Inc. | Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria |
| US11453639B2 (en) | 2019-01-11 | 2022-09-27 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| SG11202108568YA (en) * | 2019-02-25 | 2021-09-29 | Univ Massachusetts | Dna-binding domain transactivators and uses thereof |
| WO2020247452A1 (en) | 2019-06-04 | 2020-12-10 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use |
| WO2021158883A1 (en) * | 2020-02-07 | 2021-08-12 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized klkb1 locus and methods of use |
| MX2023000614A (es) | 2020-07-16 | 2023-02-13 | Acuitas Therapeutics Inc | Lipidos cationicos para usarse en nanoparticulas lipidicas. |
| TW202221119A (zh) * | 2020-07-24 | 2022-06-01 | 麻州大學 | Dna結合域轉活化子及其用途 |
| EP4244342A1 (en) | 2020-11-12 | 2023-09-20 | Precision BioSciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
| WO2022256655A2 (en) | 2021-06-04 | 2022-12-08 | Arbor Biotechnologies, Inc. | Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof |
| CA3231594A1 (en) * | 2021-09-08 | 2023-03-16 | Flagship Pioneering Innovations Vi, Llc | Serpina-modulating compositions and methods |
| IL313486A (en) | 2021-12-16 | 2024-08-01 | Acuitas Therapeutics Inc | Lipids for use in lipid nanoparticle formulations |
| US20250360224A1 (en) * | 2022-03-11 | 2025-11-27 | Epicrispr Biotechnologies, Inc. | Systems and methods for genetic modulation to treat liver disease |
| AU2023283464A1 (en) | 2022-06-07 | 2024-12-05 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of pcsk9 |
| TW202444906A (zh) | 2023-03-29 | 2024-11-16 | 美商斯奎柏治療公司 | 用於靶向pcsk9之組合物及方法 |
| WO2024229020A2 (en) * | 2023-05-01 | 2024-11-07 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of pcsk9 expression |
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- 2017-09-07 EP EP17849517.2A patent/EP3509645A4/en not_active Withdrawn
- 2017-09-07 WO PCT/US2017/050446 patent/WO2018049009A2/en not_active Ceased
- 2017-09-07 AU AU2017324462A patent/AU2017324462B2/en not_active Expired - Fee Related
- 2017-09-07 CA CA3035534A patent/CA3035534A1/en active Pending
- 2017-09-07 JP JP2019512750A patent/JP7256739B2/ja active Active
-
2020
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2023
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| Publication number | Publication date |
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| EP3509645A2 (en) | 2019-07-17 |
| EP3509645A4 (en) | 2020-06-17 |
| US20210060179A1 (en) | 2021-03-04 |
| WO2018049009A3 (en) | 2018-04-19 |
| JP7256739B2 (ja) | 2023-04-12 |
| CA3035534A1 (en) | 2018-03-15 |
| JP2019531068A (ja) | 2019-10-31 |
| WO2018049009A2 (en) | 2018-03-15 |
| AU2017324462A1 (en) | 2019-03-14 |
| US10960085B2 (en) | 2021-03-30 |
| US20180064827A1 (en) | 2018-03-08 |
| JP2023082133A (ja) | 2023-06-13 |
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