BR112023002703A2 - Métodos para tratar angioedema hereditário, para induzir a remissão sustentada de hae, para prevenir ataques de angioedema, para reduzir o risco de ataques de angioedema e para reduzir o risco de desenvolver ataques de angioedema, composições farmacêuticas e kit - Google Patents
Métodos para tratar angioedema hereditário, para induzir a remissão sustentada de hae, para prevenir ataques de angioedema, para reduzir o risco de ataques de angioedema e para reduzir o risco de desenvolver ataques de angioedema, composições farmacêuticas e kitInfo
- Publication number
- BR112023002703A2 BR112023002703A2 BR112023002703A BR112023002703A BR112023002703A2 BR 112023002703 A2 BR112023002703 A2 BR 112023002703A2 BR 112023002703 A BR112023002703 A BR 112023002703A BR 112023002703 A BR112023002703 A BR 112023002703A BR 112023002703 A2 BR112023002703 A2 BR 112023002703A2
- Authority
- BR
- Brazil
- Prior art keywords
- angioedema
- risk
- attacks
- reduce
- methods
- Prior art date
Links
- 208000028185 Angioedema Diseases 0.000 title abstract 6
- 206010019860 Hereditary angioedema Diseases 0.000 title abstract 3
- 239000008194 pharmaceutical composition Substances 0.000 title abstract 2
- 230000002459 sustained effect Effects 0.000 title abstract 2
- 229940009550 c1 esterase inhibitor Drugs 0.000 abstract 3
- 239000000203 mixture Substances 0.000 abstract 3
- 102000055157 Complement C1 Inhibitor Human genes 0.000 abstract 2
- 108050007539 Plasma protease C1 inhibitor Proteins 0.000 abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 230000000694 effects Effects 0.000 abstract 1
Classifications
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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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- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/48—Reproductive organs
- A61K35/54—Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
- A61K35/545—Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
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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
- A61K38/005—Enzyme inhibitors
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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
- A61P7/10—Antioedematous agents; Diuretics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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- 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
- C07K14/4703—Inhibitors; Suppressors
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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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
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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 RNAses, DNAses
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- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16071—Demonstrated in vivo effect
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- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Cell Biology (AREA)
- Developmental Biology & Embryology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Physics & Mathematics (AREA)
- Toxicology (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Hematology (AREA)
- Reproductive Health (AREA)
- Transplantation (AREA)
- Cardiology (AREA)
- Gynecology & Obstetrics (AREA)
Abstract
MÉTODOS PARA TRATAR ANGIOEDEMA HEREDITÁRIO, PARA INDUZIR A REMISSÃO SUSTENTADA DE HAE, PARA PREVENIR ATAQUES DE ANGIOEDEMA, PARA REDUZIR O RISCO DE ATAQUES DE ANGIOEDEMA E PARA REDUZIR O RISCO DE DESENVOLVER ATAQUES DE ANGIOEDEMA, COMPOSIÇÕES FARMACÊUTICAS E KIT. Trata-se de composições e métodos para tratar um sujeito com ou em risco de desenvolver angioedema hereditário. Com o uso das composições e dos métodos da divulgação, um paciente pode receber um ou mais agentes que elevam os níveis de expressão e/ou atividade do inibidor de C1-esterase (C1-INH). Os agentes exemplificativos que podem ser usados em conjunto com as composições e os métodos da divulgação para essa finalidade incluem células, como células pluripotentes, que expressam C1-INH.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063066011P | 2020-08-14 | 2020-08-14 | |
PCT/US2021/045881 WO2022036179A2 (en) | 2020-08-14 | 2021-08-13 | Compositions and methods for treating or preventing hereditary angioedema |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112023002703A2 true BR112023002703A2 (pt) | 2023-04-25 |
Family
ID=80248196
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112023002703A BR112023002703A2 (pt) | 2020-08-14 | 2021-08-13 | Métodos para tratar angioedema hereditário, para induzir a remissão sustentada de hae, para prevenir ataques de angioedema, para reduzir o risco de ataques de angioedema e para reduzir o risco de desenvolver ataques de angioedema, composições farmacêuticas e kit |
Country Status (10)
Country | Link |
---|---|
US (1) | US20230313227A1 (pt) |
EP (1) | EP4196172A2 (pt) |
JP (1) | JP2023540877A (pt) |
KR (1) | KR20230110243A (pt) |
AU (1) | AU2021324891A1 (pt) |
BR (1) | BR112023002703A2 (pt) |
CA (1) | CA3191729A1 (pt) |
IL (1) | IL300568A (pt) |
MX (1) | MX2023001764A (pt) |
WO (1) | WO2022036179A2 (pt) |
Family Cites Families (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20070093443A1 (en) * | 2005-10-21 | 2007-04-26 | Madison Edwin L | Modified proteases that inhibit complement activation |
-
2021
- 2021-08-13 US US18/021,364 patent/US20230313227A1/en active Pending
- 2021-08-13 IL IL300568A patent/IL300568A/en unknown
- 2021-08-13 BR BR112023002703A patent/BR112023002703A2/pt unknown
- 2021-08-13 JP JP2023510350A patent/JP2023540877A/ja active Pending
- 2021-08-13 WO PCT/US2021/045881 patent/WO2022036179A2/en unknown
- 2021-08-13 KR KR1020237008825A patent/KR20230110243A/ko unknown
- 2021-08-13 AU AU2021324891A patent/AU2021324891A1/en active Pending
- 2021-08-13 EP EP21856764.2A patent/EP4196172A2/en active Pending
- 2021-08-13 CA CA3191729A patent/CA3191729A1/en active Pending
- 2021-08-13 MX MX2023001764A patent/MX2023001764A/es unknown
Also Published As
Publication number | Publication date |
---|---|
CA3191729A1 (en) | 2022-02-17 |
AU2021324891A8 (en) | 2023-04-27 |
MX2023001764A (es) | 2023-04-25 |
WO2022036179A2 (en) | 2022-02-17 |
WO2022036179A3 (en) | 2022-03-24 |
IL300568A (en) | 2023-04-01 |
AU2021324891A1 (en) | 2023-04-06 |
EP4196172A2 (en) | 2023-06-21 |
JP2023540877A (ja) | 2023-09-27 |
US20230313227A1 (en) | 2023-10-05 |
KR20230110243A (ko) | 2023-07-21 |
AU2021324891A9 (en) | 2023-04-27 |
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