BRPI0417186A - método de tratamento de um indivìduo tendo uma hemoglobinopatia ou uma anemia, de modulação da diferenciação de uma célula-tronco ou precursora de cd34+ para uma linhagem de eritróides, e, composição farmacêutica - Google Patents
método de tratamento de um indivìduo tendo uma hemoglobinopatia ou uma anemia, de modulação da diferenciação de uma célula-tronco ou precursora de cd34+ para uma linhagem de eritróides, e, composição farmacêuticaInfo
- Publication number
- BRPI0417186A BRPI0417186A BRPI0417186-1A BRPI0417186A BRPI0417186A BR PI0417186 A BRPI0417186 A BR PI0417186A BR PI0417186 A BRPI0417186 A BR PI0417186A BR PI0417186 A BRPI0417186 A BR PI0417186A
- Authority
- BR
- Brazil
- Prior art keywords
- anemia
- compounds
- differentiation
- modulating
- individual
- Prior art date
Links
- 208000007502 anemia Diseases 0.000 title abstract 3
- 230000004069 differentiation Effects 0.000 title abstract 2
- 208000034737 hemoglobinopathy Diseases 0.000 title abstract 2
- 238000000034 method Methods 0.000 title abstract 2
- 210000000130 stem cell Anatomy 0.000 title abstract 2
- 230000000925 erythroid effect Effects 0.000 title 1
- 239000008194 pharmaceutical composition Substances 0.000 title 1
- 239000002243 precursor Substances 0.000 title 1
- 150000001875 compounds Chemical class 0.000 abstract 3
- 108090000623 proteins and genes Proteins 0.000 abstract 3
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 abstract 2
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 abstract 2
- LWDVJEYZVXKVMQ-UHFFFAOYSA-N 3-(4-amino-1,3-dihydroisoindol-2-yl)piperidine-2,6-dione Chemical class C1C=2C(N)=CC=CC=2CN1C1CCC(=O)NC1=O LWDVJEYZVXKVMQ-UHFFFAOYSA-N 0.000 abstract 1
- 108010044495 Fetal Hemoglobin Proteins 0.000 abstract 1
- 108010054147 Hemoglobins Proteins 0.000 abstract 1
- 102000001554 Hemoglobins Human genes 0.000 abstract 1
- 208000018020 Sickle cell-beta-thalassemia disease syndrome Diseases 0.000 abstract 1
- 206010043391 Thalassaemia beta Diseases 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 239000003814 drug Substances 0.000 abstract 1
- 229940079593 drug Drugs 0.000 abstract 1
- 230000037406 food intake Effects 0.000 abstract 1
- 230000002519 immonomodulatory effect Effects 0.000 abstract 1
- 208000018337 inherited hemoglobinopathy Diseases 0.000 abstract 1
- GOTYRUGSSMKFNF-UHFFFAOYSA-N lenalidomide Chemical compound C1C=2C(N)=CC=CC=2C(=O)N1C1CCC(=O)NC1=O GOTYRUGSSMKFNF-UHFFFAOYSA-N 0.000 abstract 1
- 231100000614 poison Toxicity 0.000 abstract 1
- 239000002574 poison Substances 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 208000007056 sickle cell anemia Diseases 0.000 abstract 1
- 230000000087 stabilizing effect Effects 0.000 abstract 1
- 238000001356 surgical procedure Methods 0.000 abstract 1
- 231100000765 toxin Toxicity 0.000 abstract 1
- 239000003053 toxin Substances 0.000 abstract 1
- 108700012359 toxins Proteins 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
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
- A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
-
- 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
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
- A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
- A61K31/454—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
-
- 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
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/18—Growth factors; Growth regulators
-
- 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
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/18—Growth factors; Growth regulators
- A61K38/1816—Erythropoietin [EPO]
-
- 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
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/19—Cytokines; Lymphokines; Interferons
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- A61P7/06—Antianaemics
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Zoology (AREA)
- Gastroenterology & Hepatology (AREA)
- General Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Plural Heterocyclic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Abstract
"MéTODO DE TRATAMENTO DE UM INDIVIDUO TENDO UMA HEMOGLOBINOPATIA OU UMA ANEMIA, DE MODULAçãO DA DIFERENCIAçãO DE UMA CéLULA-TRONCO OU PRECURSORA DE CD34¬ +¬ PARA UMA LINHAGEM DE ERITRóIDES, E, COMPOSIçãO FARMACêUTICA". A presente invenção é dirigida ao uso de compostos imunomodulatórios, particularmente membros da classe de compostos conhecidos como IMiDS¬ TM¬, e mais especificamente os compostos 4-(amino)2-(2,6-dioxo (3-piperidil))-isoindolina-1,3-diona e 3-(4-amino-1-oxo-1,3diidroisoindol-2-il) piperidina 2,6-diona, para induzir a expressão de genes de hemoglobina fetal, genes essenciais para eritropoiese, e genes codificando proteina estabilizando alfa hemoglobina, em uma população de células CD34¬ +¬. Estes compostos são usados para tratar hemoglobinopatias como anemia da célula falciforme ou <225>-talassemia, ou anemias causadas por doença, cirurgia, acidente, ou a introdução ou ingestão de toxinas, venenos ou drogas.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US52691003P | 2003-12-02 | 2003-12-02 | |
| PCT/US2004/040226 WO2005055929A2 (en) | 2003-12-02 | 2004-12-02 | Methods and compositions for the treatment and management of hemoglobinopathy and anemia |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BRPI0417186A true BRPI0417186A (pt) | 2007-03-06 |
Family
ID=34676680
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BRPI0417186-1A BRPI0417186A (pt) | 2003-12-02 | 2004-12-02 | método de tratamento de um indivìduo tendo uma hemoglobinopatia ou uma anemia, de modulação da diferenciação de uma célula-tronco ou precursora de cd34+ para uma linhagem de eritróides, e, composição farmacêutica |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20050143420A1 (pt) |
| EP (1) | EP1694328A4 (pt) |
| JP (2) | JP5089170B2 (pt) |
| KR (3) | KR20060109979A (pt) |
| CN (2) | CN1913896B (pt) |
| AU (2) | AU2004296765B2 (pt) |
| BR (1) | BRPI0417186A (pt) |
| CA (1) | CA2547570A1 (pt) |
| IL (1) | IL176020A0 (pt) |
| MX (1) | MXPA06006061A (pt) |
| NZ (1) | NZ548049A (pt) |
| WO (1) | WO2005055929A2 (pt) |
| ZA (1) | ZA200604815B (pt) |
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| KR20180105266A (ko) | 2005-12-29 | 2018-09-27 | 안트로제네시스 코포레이션 | 태반 줄기 세포 집단 |
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| AU2011202457A1 (en) | 2011-06-16 |
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| HK1100262A1 (en) | 2007-09-14 |
| KR20120039065A (ko) | 2012-04-24 |
| KR20110116225A (ko) | 2011-10-25 |
| WO2005055929A2 (en) | 2005-06-23 |
| EP1694328A2 (en) | 2006-08-30 |
| AU2004296765B2 (en) | 2011-03-24 |
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| IL176020A0 (en) | 2006-10-05 |
| JP2011207906A (ja) | 2011-10-20 |
| CN1913896A (zh) | 2007-02-14 |
| US20050143420A1 (en) | 2005-06-30 |
| CN1913896B (zh) | 2010-12-01 |
| EP1694328A4 (en) | 2010-02-17 |
| WO2005055929A3 (en) | 2006-01-05 |
| ZA200604815B (en) | 2007-10-31 |
| MXPA06006061A (es) | 2006-08-11 |
| KR20060109979A (ko) | 2006-10-23 |
| CA2547570A1 (en) | 2005-06-23 |
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