JP2024521767A - H因子ファミリータンパク質のための阻害性核酸 - Google Patents
H因子ファミリータンパク質のための阻害性核酸 Download PDFInfo
- Publication number
- JP2024521767A JP2024521767A JP2023572717A JP2023572717A JP2024521767A JP 2024521767 A JP2024521767 A JP 2024521767A JP 2023572717 A JP2023572717 A JP 2023572717A JP 2023572717 A JP2023572717 A JP 2023572717A JP 2024521767 A JP2024521767 A JP 2024521767A
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- Prior art keywords
- nucleic acid
- complement
- nucleotide sequence
- agent
- fhr1
- Prior art date
- Legal status (The legal status 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 status listed.)
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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
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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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- 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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- 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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- 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
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
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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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N15/87—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
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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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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6803—General methods of protein analysis not limited to specific proteins or families of proteins
- G01N33/6848—Methods of protein analysis involving mass spectrometry
- G01N33/6851—Methods of protein analysis involving laser desorption ionisation mass spectrometry
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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- C—CHEMISTRY; METALLURGY
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/435—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
- G01N2333/46—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans from vertebrates
- G01N2333/47—Assays involving proteins of known structure or function as defined in the subgroups
- G01N2333/4701—Details
- G01N2333/4716—Complement proteins, e.g. anaphylatoxin, C3a, C5a
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2800/00—Detection or diagnosis of diseases
- G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis
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- Life Sciences & Earth Sciences (AREA)
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- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Urology & Nephrology (AREA)
- Hematology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Plant Pathology (AREA)
- Cell Biology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Bioinformatics & Computational Biology (AREA)
- Food Science & Technology (AREA)
- General Physics & Mathematics (AREA)
- Analytical Chemistry (AREA)
- Pathology (AREA)
- Epidemiology (AREA)
- Spectroscopy & Molecular Physics (AREA)
- Gastroenterology & Hepatology (AREA)
- Optics & Photonics (AREA)
- Mycology (AREA)
- Chemical Kinetics & Catalysis (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GB2107586.6 | 2021-05-27 | ||
| GBGB2107586.6A GB202107586D0 (en) | 2021-05-27 | 2021-05-27 | Inhibitory nucleic acids for Factor H family proteins |
| PCT/EP2022/064376 WO2022248651A2 (en) | 2021-05-27 | 2022-05-26 | Inhibitory nucleic acids for factor h family proteins |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024521767A true JP2024521767A (ja) | 2024-06-04 |
| JP2024521767A5 JP2024521767A5 (enExample) | 2025-06-03 |
Family
ID=76741290
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023572717A Pending JP2024521767A (ja) | 2021-05-27 | 2022-05-26 | H因子ファミリータンパク質のための阻害性核酸 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20240209367A1 (enExample) |
| EP (1) | EP4347030A2 (enExample) |
| JP (1) | JP2024521767A (enExample) |
| CN (1) | CN118632711A (enExample) |
| AU (1) | AU2022279580A1 (enExample) |
| CA (1) | CA3219247A1 (enExample) |
| GB (1) | GB202107586D0 (enExample) |
| WO (1) | WO2022248651A2 (enExample) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2024165568A2 (en) * | 2023-02-06 | 2024-08-15 | E-Therapeutics Plc | Inhibitors of expression and/or function |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7217807B2 (en) * | 2002-11-26 | 2007-05-15 | Rosetta Genomics Ltd | Bioinformatically detectable group of novel HIV regulatory genes and uses thereof |
| US20050118625A1 (en) * | 2003-10-02 | 2005-06-02 | Mounts William M. | Nucleic acid arrays for detecting gene expression associated with human osteoarthritis and human proteases |
| WO2005116204A1 (ja) * | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Rna干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法 |
| US7745389B2 (en) | 2005-02-14 | 2010-06-29 | University Of Iowa Research Foundation | Methods for treatment of age-related macular degeneration |
| GB0611606D0 (en) | 2006-06-13 | 2006-07-19 | Univ Belfast | Protection against and treatment of age related macular degeneration |
| WO2009059321A2 (en) | 2007-11-01 | 2009-05-07 | University Of Iowa Research Foundation | Rca locus analysis to assess susceptibility to amd and mpgnii |
| US9598733B2 (en) | 2011-02-17 | 2017-03-21 | The Trustees Of Columbia University In The City Of New York | Methods for identifying subjects with a genetic risk for developing IgA nephropathy |
| US20150157675A1 (en) | 2012-04-02 | 2015-06-11 | Sung Kyun Biotech Co., Ltd. | Composition comprising eupatorium spp. extract as active ingredient for preventing and treating obesity and metabolic bone disease |
| EP2858679B2 (en) | 2012-06-08 | 2024-06-05 | Translate Bio, Inc. | Pulmonary delivery of mrna to non-lung target cells |
| IL310721B2 (en) | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors and uses thereof |
| GB201709222D0 (en) | 2017-06-09 | 2017-07-26 | Univ Manchester | C3b Inactivating Polypeptide |
| WO2019051443A1 (en) | 2017-09-11 | 2019-03-14 | Insideoutbio, Inc. | METHODS AND COMPOSITIONS FOR IMPROVING IMMUNOGENICITY OF TUMORS |
| GB201800620D0 (en) | 2018-01-15 | 2018-02-28 | Univ Manchester | C3b Binding Polypeptide |
| WO2019215330A1 (en) | 2018-05-10 | 2019-11-14 | The University Of Manchester | Methods for assessing macular degeneration |
| EP4214515A1 (en) | 2020-09-16 | 2023-07-26 | Complement Therapeutics Limited | Complementome assay |
-
2021
- 2021-05-27 GB GBGB2107586.6A patent/GB202107586D0/en not_active Ceased
-
2022
- 2022-05-26 JP JP2023572717A patent/JP2024521767A/ja active Pending
- 2022-05-26 CN CN202280038270.4A patent/CN118632711A/zh active Pending
- 2022-05-26 WO PCT/EP2022/064376 patent/WO2022248651A2/en not_active Ceased
- 2022-05-26 EP EP22731536.3A patent/EP4347030A2/en active Pending
- 2022-05-26 AU AU2022279580A patent/AU2022279580A1/en active Pending
- 2022-05-26 CA CA3219247A patent/CA3219247A1/en active Pending
-
2023
- 2023-11-27 US US18/519,153 patent/US20240209367A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AU2022279580A1 (en) | 2024-01-04 |
| EP4347030A2 (en) | 2024-04-10 |
| US20240209367A1 (en) | 2024-06-27 |
| CN118632711A (zh) | 2024-09-10 |
| WO2022248651A3 (en) | 2023-02-02 |
| CA3219247A1 (en) | 2022-12-01 |
| WO2022248651A2 (en) | 2022-12-01 |
| GB202107586D0 (en) | 2021-07-14 |
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