BR112023000738A2 - Composições farmacêuticas, métodos para o tratamento ou prevenção de uma doença, tratar claudicação canina, tratar claudicação equina, inativar uma il-1a ou il-1ss endógena e reduzir a expressão do gene il-1a ou il-1ss, método de tratamento de um indivíduo com artrite, doença articular e osteoartrite, método mediado por crispr/cas9 para tratamento de doenças articulares, nuclease crisprcas, repetições palindrômicas, sistema vetorial e método de alteração de uma sequência de ácido nucleico - Google Patents
Composições farmacêuticas, métodos para o tratamento ou prevenção de uma doença, tratar claudicação canina, tratar claudicação equina, inativar uma il-1a ou il-1ss endógena e reduzir a expressão do gene il-1a ou il-1ss, método de tratamento de um indivíduo com artrite, doença articular e osteoartrite, método mediado por crispr/cas9 para tratamento de doenças articulares, nuclease crisprcas, repetições palindrômicas, sistema vetorial e método de alteração de uma sequência de ácido nucleicoInfo
- Publication number
- BR112023000738A2 BR112023000738A2 BR112023000738A BR112023000738A BR112023000738A2 BR 112023000738 A2 BR112023000738 A2 BR 112023000738A2 BR 112023000738 A BR112023000738 A BR 112023000738A BR 112023000738 A BR112023000738 A BR 112023000738A BR 112023000738 A2 BR112023000738 A2 BR 112023000738A2
- Authority
- BR
- Brazil
- Prior art keywords
- treatment
- treat
- disease
- arthritis
- osteoarthritis
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 10
- 108091033409 CRISPR Proteins 0.000 title abstract 4
- 206010003246 arthritis Diseases 0.000 title abstract 4
- 201000010099 disease Diseases 0.000 title abstract 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title abstract 4
- 201000008482 osteoarthritis Diseases 0.000 title abstract 4
- 208000012659 Joint disease Diseases 0.000 title abstract 3
- 238000010354 CRISPR gene editing Methods 0.000 title abstract 2
- 241000282465 Canis Species 0.000 title abstract 2
- 206010022562 Intermittent claudication Diseases 0.000 title abstract 2
- 108091028043 Nucleic acid sequence Proteins 0.000 title abstract 2
- 208000024980 claudication Diseases 0.000 title abstract 2
- 230000014509 gene expression Effects 0.000 title abstract 2
- 230000001404 mediated effect Effects 0.000 title abstract 2
- 150000007523 nucleic acids Chemical group 0.000 title abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 title abstract 2
- 230000002265 prevention Effects 0.000 title abstract 2
- 101001002634 Homo sapiens Interleukin-1 alpha Proteins 0.000 title 2
- 102100020881 Interleukin-1 alpha Human genes 0.000 title 2
- 239000000203 mixture Substances 0.000 abstract 2
- 230000002757 inflammatory effect Effects 0.000 abstract 1
Classifications
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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
- C12N15/87—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
-
- 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
- C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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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
-
- 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
-
- 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
- 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
- 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
-
- 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
- C12N2750/14145—Special targeting system for viral vectors
-
- 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
- C12N2750/14171—Demonstrated in vivo effect
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Endocrinology (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Epidemiology (AREA)
- Peptides Or Proteins (AREA)
- Immunology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Rheumatology (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063052881P | 2020-07-16 | 2020-07-16 | |
| US202063055808P | 2020-07-23 | 2020-07-23 | |
| PCT/US2021/042100 WO2022016121A2 (en) | 2020-07-16 | 2021-07-16 | Gene editing to improve joint function |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112023000738A2 true BR112023000738A2 (pt) | 2023-03-21 |
Family
ID=79555038
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112023000738A BR112023000738A2 (pt) | 2020-07-16 | 2021-07-16 | Composições farmacêuticas, métodos para o tratamento ou prevenção de uma doença, tratar claudicação canina, tratar claudicação equina, inativar uma il-1a ou il-1ss endógena e reduzir a expressão do gene il-1a ou il-1ss, método de tratamento de um indivíduo com artrite, doença articular e osteoartrite, método mediado por crispr/cas9 para tratamento de doenças articulares, nuclease crisprcas, repetições palindrômicas, sistema vetorial e método de alteração de uma sequência de ácido nucleico |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230257779A1 (https=) |
| EP (1) | EP4182461A4 (https=) |
| JP (1) | JP2023535351A (https=) |
| KR (1) | KR20230041729A (https=) |
| CN (1) | CN116113696A (https=) |
| AU (1) | AU2021308079A1 (https=) |
| BR (1) | BR112023000738A2 (https=) |
| CA (1) | CA3186119A1 (https=) |
| IL (1) | IL299846A (https=) |
| MX (1) | MX2023000671A (https=) |
| WO (2) | WO2022016100A2 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4694913A2 (en) * | 2023-04-11 | 2026-02-18 | Orthobio Therapeutics, Inc. | Lipid nanoparticles for gene editing systems |
| WO2025024814A2 (en) * | 2023-07-26 | 2025-01-30 | Orthobio Therapeutics, Inc. | Transmembrane receptor gene editing |
| CN117448379A (zh) * | 2023-12-22 | 2024-01-26 | 上海元戊医学技术有限公司 | iPSC来源的IL-10蛋白过表达MSC细胞株的构建方法及其应用 |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB9711040D0 (en) * | 1997-05-29 | 1997-07-23 | Duff Gordon W | Prediction of inflammatory disease |
| WO2005077333A2 (en) * | 2004-02-10 | 2005-08-25 | University Of Florida Research Foundation, Inc. | Gel-based delivery of recombinant adeno-associated virus vectors |
| US20160279202A1 (en) * | 2013-10-31 | 2016-09-29 | Ramot At Tel-Aviv University Ltd. | Interleukin-1 (il-1) inhibitors for treating fertility disorders |
| US20170240901A1 (en) * | 2014-02-26 | 2017-08-24 | The University Of Liverpool | Nucleic acids, and uses therof |
| US20170335331A1 (en) * | 2014-10-31 | 2017-11-23 | The Trustees Of The University Of Pennsylvania | Altering Gene Expression in CART Cells and Uses Thereof |
| WO2017223107A1 (en) * | 2016-06-20 | 2017-12-28 | Unity Biotechnology, Inc. | Genome modifying enzyme therapy for diseases modulated by senescent cells |
| WO2018031950A1 (en) * | 2016-08-12 | 2018-02-15 | Caribou Biosciences, Inc. | Protein engineering methods |
-
2021
- 2021-07-16 EP EP21842178.2A patent/EP4182461A4/en active Pending
- 2021-07-16 CN CN202180054064.8A patent/CN116113696A/zh active Pending
- 2021-07-16 BR BR112023000738A patent/BR112023000738A2/pt unknown
- 2021-07-16 US US18/005,544 patent/US20230257779A1/en active Pending
- 2021-07-16 KR KR1020237004715A patent/KR20230041729A/ko active Pending
- 2021-07-16 JP JP2023502921A patent/JP2023535351A/ja active Pending
- 2021-07-16 CA CA3186119A patent/CA3186119A1/en active Pending
- 2021-07-16 MX MX2023000671A patent/MX2023000671A/es unknown
- 2021-07-16 WO PCT/US2021/042048 patent/WO2022016100A2/en not_active Ceased
- 2021-07-16 WO PCT/US2021/042100 patent/WO2022016121A2/en not_active Ceased
- 2021-07-16 IL IL299846A patent/IL299846A/en unknown
- 2021-07-16 AU AU2021308079A patent/AU2021308079A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| IL299846A (en) | 2023-03-01 |
| WO2022016100A2 (en) | 2022-01-20 |
| JP2023535351A (ja) | 2023-08-17 |
| US20230257779A1 (en) | 2023-08-17 |
| WO2022016100A3 (en) | 2022-03-17 |
| MX2023000671A (es) | 2023-05-16 |
| CN116113696A (zh) | 2023-05-12 |
| AU2021308079A8 (en) | 2023-03-16 |
| WO2022016121A3 (en) | 2022-03-10 |
| EP4182461A4 (en) | 2025-03-19 |
| KR20230041729A (ko) | 2023-03-24 |
| WO2022016121A2 (en) | 2022-01-20 |
| AU2021308079A1 (en) | 2023-03-02 |
| EP4182461A2 (en) | 2023-05-24 |
| CA3186119A1 (en) | 2022-01-20 |
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