ES2632444T5 - Compuestos que mejoran la transducción viral - Google Patents
Compuestos que mejoran la transducción viral Download PDFInfo
- Publication number
- ES2632444T5 ES2632444T5 ES12836507T ES12836507T ES2632444T5 ES 2632444 T5 ES2632444 T5 ES 2632444T5 ES 12836507 T ES12836507 T ES 12836507T ES 12836507 T ES12836507 T ES 12836507T ES 2632444 T5 ES2632444 T5 ES 2632444T5
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- Prior art keywords
- cells
- cell
- virus
- globin
- transduced
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- 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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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
- C12N15/63—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
-
- 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)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P5/00—Drugs for disorders of the endocrine system
- A61P5/38—Drugs for disorders of the endocrine system of the suprarenal hormones
-
- 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
- A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
-
- 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
-
- 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
- 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
-
- 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
- C12N15/867—Retroviral 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
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/02—Compounds of the arachidonic acid pathway, e.g. prostaglandins, leukotrienes
-
- 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
-
- 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
- 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
-
- 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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
- C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
- C12N2810/6072—Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses
- C12N2810/6081—Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses rhabdoviridae, e.g. VSV
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Hematology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Immunology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Molecular Biology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Cell Biology (AREA)
- Developmental Biology & Embryology (AREA)
- Endocrinology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161541736P | 2011-09-30 | 2011-09-30 | |
| US201161541736P | 2011-09-30 | ||
| PCT/US2012/057987 WO2013049615A1 (en) | 2011-09-30 | 2012-09-28 | Compounds for improved viral transduction |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| ES2632444T3 ES2632444T3 (es) | 2017-09-13 |
| ES2632444T5 true ES2632444T5 (es) | 2022-03-02 |
Family
ID=47996444
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES12836507T Active ES2632444T5 (es) | 2011-09-30 | 2012-09-28 | Compuestos que mejoran la transducción viral |
| ES19204363T Active ES2913633T3 (es) | 2011-09-30 | 2012-09-28 | Compuestos para transducción viral mejorada |
| ES17173567T Active ES2769270T3 (es) | 2011-09-30 | 2012-09-28 | Compuestos para transducción viral mejorada |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES19204363T Active ES2913633T3 (es) | 2011-09-30 | 2012-09-28 | Compuestos para transducción viral mejorada |
| ES17173567T Active ES2769270T3 (es) | 2011-09-30 | 2012-09-28 | Compuestos para transducción viral mejorada |
Country Status (19)
| Country | Link |
|---|---|
| US (5) | US9988644B2 (https=) |
| EP (4) | EP4095236A1 (https=) |
| JP (2) | JP6285863B2 (https=) |
| KR (1) | KR102011532B1 (https=) |
| CN (2) | CN103958667A (https=) |
| AU (1) | AU2012315699B2 (https=) |
| BR (1) | BR112014007782B1 (https=) |
| CA (1) | CA2850484C (https=) |
| CY (1) | CY1122654T1 (https=) |
| DK (2) | DK3269802T3 (https=) |
| EA (1) | EA032699B1 (https=) |
| ES (3) | ES2632444T5 (https=) |
| IL (2) | IL231812B (https=) |
| IN (1) | IN2014CN02518A (https=) |
| MX (2) | MX359398B (https=) |
| PT (2) | PT2760994T (https=) |
| SG (2) | SG11201401077PA (https=) |
| UA (1) | UA114796C2 (https=) |
| WO (1) | WO2013049615A1 (https=) |
Families Citing this family (55)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT3031907T (pt) | 2008-11-06 | 2021-01-28 | Univ Indiana Res & Tech Corp | Materiais e métodos para melhorar procedimentos de enxerto de células estaminais hematopoiéticas |
| PT2760994T (pt) | 2011-09-30 | 2017-08-24 | Bluebird Bio Inc | Compostos para melhor transdução viral |
| ES2869701T3 (es) | 2012-08-01 | 2021-10-25 | United Therapeutics Corp | Tratamiento de hipertensión arterial pulmonar con células progenitoras endoteliales tratadas con prostaciclina |
| CA2880808C (en) | 2012-08-01 | 2023-01-24 | United Therapeutics Corporation | Treatment of pulmonary arterial hypertension with mesenchymal stem cells |
| US20150216903A1 (en) * | 2012-08-10 | 2015-08-06 | Bluebird Bio, Inc. | Compounds for improved viral transduction |
| SG11201504038XA (en) | 2012-11-27 | 2015-06-29 | Childrens Medical Center | Targeting bcl11a distal regulatory elements for fetal hemoglobin reinduction |
| JP6431850B2 (ja) | 2013-01-09 | 2018-11-28 | ユナイテッド セラピューティクス コーポレイション | プロスタサイクリンおよび間葉系幹細胞による脈管障害の処置 |
| ES2747951T3 (es) * | 2013-10-24 | 2020-03-12 | Ospedale San Raffaele Srl | Método |
| US20170173083A1 (en) * | 2014-03-26 | 2017-06-22 | The Brigham And Women's Hospital, Inc. | Compositions and methods for ex vivo expansion of human hematopoietic stem/progenitor cells |
| MX375592B (es) | 2014-04-25 | 2025-03-06 | The Children´S Medical Center Corp | Composiciones y su uso para tratar hemoglobinopatias. |
| ES2835861T5 (en) | 2015-05-08 | 2025-02-18 | Childrens Medical Ct Corp | Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction |
| RU2630654C2 (ru) * | 2015-07-29 | 2017-09-11 | Федеральное государственное автономное образовательное учреждение высшего профессионального образования "Балтийский Федеральный Университет имени Иммануила Канта" (БФУ им. И. Канта) | Способ хемогенетической регистрации и коррекции нейрогенеза на основе генетических конструкций для трансфекции астроцитов и нейронов |
| CN105062979A (zh) * | 2015-08-21 | 2015-11-18 | 昆明理工大学 | 一种培养戊型肝炎病毒的方法 |
| WO2017139561A1 (en) * | 2016-02-12 | 2017-08-17 | Bluebird Bio, Inc. | Vcn enhancer compositions and methods of using the same |
| WO2017139576A1 (en) * | 2016-02-12 | 2017-08-17 | Bluebird Bio, Inc. | Vcn enhancer compositions and methods of using the same |
| CN109415687A (zh) * | 2016-04-07 | 2019-03-01 | 蓝鸟生物公司 | 嵌合抗原受体t细胞组合物 |
| US10898494B2 (en) | 2016-05-05 | 2021-01-26 | Liquidia Technologies, Inc. | Dry powder treprostinil for the treatment of pulmonary hypertension |
| ES2927406T3 (es) | 2016-10-24 | 2022-11-04 | United Therapeutics Corp | Potenciación de las propiedades inmunomoduladoras de las MSC mediante treprostinil |
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| SG11201908844YA (en) * | 2017-03-29 | 2019-10-30 | Bluebird Bio Inc | Vectors and compositions for treating hemoglobinopathies |
| US11261441B2 (en) | 2017-03-29 | 2022-03-01 | Bluebird Bio, Inc. | Vectors and compositions for treating hemoglobinopathies |
| US11788087B2 (en) | 2017-05-25 | 2023-10-17 | The Children's Medical Center Corporation | BCL11A guide delivery |
| WO2019055814A2 (en) * | 2017-09-15 | 2019-03-21 | The Medical College Of Wisconsin, Inc. | EPOXYEICOSATRIENOIC ACID (EET) ANALOGUES TARGETING KIDNEYS |
| FR3074189A1 (fr) * | 2017-11-30 | 2019-05-31 | Assistance Publique - Hopitaux De Paris | Methode de generation de greffons de cellules souches hematopoietiques |
| CN110799641B (zh) * | 2018-04-13 | 2021-02-19 | 诺未科技（北京）有限公司 | 丁酸钠的用途及含有丁酸钠的培养体系 |
| CN109207427B (zh) * | 2018-09-17 | 2020-12-08 | 诺未科技（银川）有限公司 | 一种将人造血祖细胞转变为造血干细胞的方法 |
| EP3784695B1 (en) * | 2018-04-27 | 2023-08-09 | The Medical College of Wisconsin, Inc. | Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders |
| US12522811B2 (en) | 2018-05-01 | 2026-01-13 | The Children's Medical Center Corporation | Enhanced BCL11A RNP / CRISPR delivery and editing using a 3XNLS-CAS9 |
| US12350284B2 (en) | 2018-05-02 | 2025-07-08 | The Children's Medical Center Corporation | BCL11A microRNAs for treating hemoglobinopathies |
| WO2020056400A1 (en) | 2018-09-14 | 2020-03-19 | The Children's Hospital Of Philadelphia | Compositions and methods for hemoglobin production |
| KR20210076048A (ko) * | 2018-10-12 | 2021-06-23 | 라이프 테크놀로지스 코포레이션 | 조혈 줄기 및 전구 세포 확장 시스템 |
| WO2020111229A1 (ja) * | 2018-11-30 | 2020-06-04 | 国立大学法人京都大学 | 溶液を用いた薬物送達系 |
| WO2020264488A1 (en) * | 2019-06-28 | 2020-12-30 | The Children's Hospital Of Philadelphia | Compositions and methods for treating anemia |
| WO2021055801A1 (en) | 2019-09-18 | 2021-03-25 | The Medical College Of Wisconsin, Inc. | Improved alpha-galactosidase protein for enzyme replacement therapy (ert) and methods of use |
| AU2020365133A1 (en) * | 2019-10-16 | 2022-04-28 | Orchard Therapeutics (Europe) Limited | Compositions and methods for modifying eukaryotic cells |
| CA3163572A1 (en) * | 2020-05-27 | 2021-12-02 | Oleksandr PASTUKHOV | Novel transduction enhancers and uses thereof |
| KR20230019156A (ko) * | 2020-06-02 | 2023-02-07 | 카탈렌트 파마 솔루션즈, 엘엘씨 | 유전자 삽입을 위한 다수의 도크들이 있는 세포주 |
| CN115125270A (zh) * | 2021-03-22 | 2022-09-30 | 华东师范大学 | 一种α-珠蛋白过表达载体及其应用 |
| CN113106098B (zh) * | 2021-04-21 | 2022-04-01 | 贵州医科大学 | 一种在红系细胞中特异表达人β珠蛋白的重组序列及其应用 |
| AU2021202658A1 (en) | 2021-04-28 | 2022-11-17 | Fondazione Telethon | Gene therapy |
| CN113336833A (zh) * | 2021-06-04 | 2021-09-03 | 广州伯尼兹生物科技有限公司 | 一种高效分泌表达猪流行性腹泻病毒s1蛋白的生产方法 |
| WO2023133595A2 (en) | 2022-01-10 | 2023-07-13 | Sana Biotechnology, Inc. | Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses |
| GB202206346D0 (en) | 2022-04-29 | 2022-06-15 | Ospedale San Raffaele Srl | Gene therapy |
| WO2024033802A2 (en) | 2022-08-11 | 2024-02-15 | Fondazione Telethon Ets | Gene therapy |
| EP4602153A1 (en) | 2022-10-11 | 2025-08-20 | Fondazione Telethon ETS | 3d cell culture methods |
| WO2024081820A1 (en) | 2022-10-13 | 2024-04-18 | Sana Biotechnology, Inc. | Viral particles targeting hematopoietic stem cells |
| WO2024119157A1 (en) | 2022-12-02 | 2024-06-06 | Sana Biotechnology, Inc. | Lipid particles with cofusogens and methods of producing and using the same |
| KR20260016952A (ko) | 2023-05-23 | 2026-02-04 | 사나 바이오테크놀로지, 인크. | 탠덤 퓨소젠 및 관련 지질 입자 |
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| IT202300022191A1 (it) | 2023-10-24 | 2025-04-24 | Fond Telethon Ets | Mezzi e metodi per la modifica genetica di uba1 |
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| CN118436665A (zh) * | 2024-03-22 | 2024-08-06 | 浙大城市学院 | 一类三萜结构及其类似物及在制备抗病毒药物中的应用 |
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