CO2021014747A2 - Métodos para el tratamiento de βeta-talasemia - Google Patents
Métodos para el tratamiento de βeta-talasemiaInfo
- Publication number
- CO2021014747A2 CO2021014747A2 CONC2021/0014747A CO2021014747A CO2021014747A2 CO 2021014747 A2 CO2021014747 A2 CO 2021014747A2 CO 2021014747 A CO2021014747 A CO 2021014747A CO 2021014747 A2 CO2021014747 A2 CO 2021014747A2
- Authority
- CO
- Colombia
- Prior art keywords
- thalassemia
- methods
- beta
- treatment
- compositions
- Prior art date
Links
- 208000005980 beta thalassemia Diseases 0.000 title abstract 2
- 239000000203 mixture Substances 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
- 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
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K39/46
- A61K2239/31—Indexing codes associated with cellular immunotherapy of group A61K39/46 characterized by the route of administration
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K39/46
- A61K2239/38—Indexing codes associated with cellular immunotherapy of group A61K39/46 characterised by the dose, timing or administration schedule
-
- 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
- A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
- A61K31/255—Esters, e.g. nitroglycerine, selenocyanates of sulfoxy acids or sulfur analogues thereof
-
- 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
-
- 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
- A61K35/14—Blood; Artificial blood
- A61K35/18—Erythrocytes
-
- 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
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/461—Cellular immunotherapy characterised by the cell type used
-
- 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
- A61K39/46—Cellular immunotherapy
- A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
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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
- C07K14/82—Translation products from oncogenes
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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
- 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
- C12N5/0634—Cells from the blood or the immune system
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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
- 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
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0641—Erythrocytes
-
- 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
- A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
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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
- C12N2510/00—Genetically modified cells
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Immunology (AREA)
- Organic Chemistry (AREA)
- Cell Biology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Hematology (AREA)
- Pharmacology & Pharmacy (AREA)
- Genetics & Genomics (AREA)
- Epidemiology (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Developmental Biology & Embryology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Mycology (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Molecular Biology (AREA)
- Gastroenterology & Hepatology (AREA)
- Oncology (AREA)
- Dermatology (AREA)
- Emergency Medicine (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
En la presente memoria se describen métodos y composiciones para tratar una beta-talasemia
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962828182P | 2019-04-02 | 2019-04-02 | |
US201962930846P | 2019-11-05 | 2019-11-05 | |
US201962944626P | 2019-12-06 | 2019-12-06 | |
PCT/US2020/025919 WO2020205838A1 (en) | 2019-04-02 | 2020-03-31 | Methods for the treatment of beta-thalassemia |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2021014747A2 true CO2021014747A2 (es) | 2021-11-19 |
Family
ID=70465393
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2021/0014747A CO2021014747A2 (es) | 2019-04-02 | 2021-11-02 | Métodos para el tratamiento de βeta-talasemia |
Country Status (15)
Country | Link |
---|---|
US (2) | US20200316116A1 (es) |
EP (1) | EP3946384A1 (es) |
JP (1) | JP2022519949A (es) |
KR (1) | KR20210146986A (es) |
CN (2) | CN113939586A (es) |
AU (1) | AU2020253362A1 (es) |
BR (1) | BR112021019448A2 (es) |
CA (1) | CA3132167A1 (es) |
CO (1) | CO2021014747A2 (es) |
IL (1) | IL286857A (es) |
MA (1) | MA55531A (es) |
MX (1) | MX2021012152A (es) |
SG (1) | SG11202110776TA (es) |
TW (1) | TW202102665A (es) |
WO (1) | WO2020205838A1 (es) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2023014839A1 (en) * | 2021-08-03 | 2023-02-09 | Sangamo Therapeutics, Inc. | Methods for the treatment of sickle cell disease |
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CN105940013B (zh) | 2013-12-09 | 2020-03-27 | 桑格摩生物科学股份有限公司 | 用于治疗血友病的方法和组合物 |
PT3102673T (pt) | 2014-02-03 | 2020-05-21 | Sangamo Biosciences Inc | Métodos e composições para tratamento de beta-talassemia |
JP6722176B2 (ja) | 2014-09-16 | 2020-07-15 | サンガモ セラピューティクス, インコーポレイテッド | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
MX2017014446A (es) | 2015-05-12 | 2018-06-13 | Sangamo Therapeutics Inc | Regulacion de expresion genica mediada por nucleasa. |
US9957501B2 (en) | 2015-06-18 | 2018-05-01 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
MX2018012729A (es) * | 2016-04-18 | 2019-07-04 | Crispr Therapeutics Ag | Materiales y metodos para el tratamiento de hemoglobinopatias. |
ES2900317T3 (es) | 2016-08-24 | 2022-03-16 | Sangamo Therapeutics Inc | Regulación de la expresión génica al usar nucleasas genomanipuladas |
KR102455249B1 (ko) | 2016-08-24 | 2022-10-17 | 상가모 테라퓨틱스, 인코포레이티드 | 가공된 표적 특이적 뉴클레아제 |
US11512287B2 (en) | 2017-06-16 | 2022-11-29 | Sangamo Therapeutics, Inc. | Targeted disruption of T cell and/or HLA receptors |
-
2020
- 2020-03-31 WO PCT/US2020/025919 patent/WO2020205838A1/en active Application Filing
- 2020-03-31 EP EP20722023.7A patent/EP3946384A1/en not_active Withdrawn
- 2020-03-31 MX MX2021012152A patent/MX2021012152A/es unknown
- 2020-03-31 CA CA3132167A patent/CA3132167A1/en active Pending
- 2020-03-31 SG SG11202110776TA patent/SG11202110776TA/en unknown
- 2020-03-31 JP JP2021559046A patent/JP2022519949A/ja active Pending
- 2020-03-31 BR BR112021019448A patent/BR112021019448A2/pt unknown
- 2020-03-31 CN CN202080040069.0A patent/CN113939586A/zh active Pending
- 2020-03-31 AU AU2020253362A patent/AU2020253362A1/en not_active Abandoned
- 2020-03-31 CN CN202210351840.7A patent/CN115141807A/zh active Pending
- 2020-03-31 US US16/835,776 patent/US20200316116A1/en not_active Abandoned
- 2020-03-31 KR KR1020217035085A patent/KR20210146986A/ko active Search and Examination
- 2020-03-31 MA MA055531A patent/MA55531A/fr unknown
- 2020-04-01 TW TW109111173A patent/TW202102665A/zh unknown
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2021
- 2021-09-30 IL IL286857A patent/IL286857A/en unknown
- 2021-11-02 CO CONC2021/0014747A patent/CO2021014747A2/es unknown
-
2023
- 2023-03-20 US US18/123,920 patent/US20230381225A1/en active Pending
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US20200316116A1 (en) | 2020-10-08 |
CN115141807A (zh) | 2022-10-04 |
SG11202110776TA (en) | 2021-10-28 |
EP3946384A1 (en) | 2022-02-09 |
TW202102665A (zh) | 2021-01-16 |
AU2020253362A1 (en) | 2021-11-04 |
KR20210146986A (ko) | 2021-12-06 |
JP2022519949A (ja) | 2022-03-25 |
MA55531A (fr) | 2022-02-09 |
IL286857A (en) | 2021-10-31 |
US20230381225A1 (en) | 2023-11-30 |
CA3132167A1 (en) | 2020-10-08 |
MX2021012152A (es) | 2021-11-03 |
BR112021019448A2 (pt) | 2021-11-30 |
WO2020205838A1 (en) | 2020-10-08 |
CN113939586A (zh) | 2022-01-14 |
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