HUE068603T2 - Módosított IX-es faktor, valamint készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez - Google Patents
Módosított IX-es faktor, valamint készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhezInfo
- Publication number
- HUE068603T2 HUE068603T2 HUE16815331A HUE16815331A HUE068603T2 HU E068603 T2 HUE068603 T2 HU E068603T2 HU E16815331 A HUE16815331 A HU E16815331A HU E16815331 A HUE16815331 A HU E16815331A HU E068603 T2 HUE068603 T2 HU E068603T2
- Authority
- HU
- Hungary
- Prior art keywords
- organs
- tissues
- compositions
- cells
- methods
- Prior art date
Links
- 229960004222 factor ix Drugs 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 210000000056 organ Anatomy 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 title 1
Classifications
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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
- A61K38/16—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
- A61K38/36—Blood coagulation or fibrinolysis factors
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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
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
- A61K38/482—Serine endopeptidases (3.4.21)
- A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
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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
-
- 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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- 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
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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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
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/745—Blood coagulation or fibrinolysis factors
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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
- C12N15/52—Genes encoding for enzymes or proenzymes
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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
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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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
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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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
- C12N9/644—Coagulation factor IXa (3.4.21.22)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21022—Coagulation factor IXa (3.4.21.22)
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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
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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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
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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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Virology (AREA)
- Hematology (AREA)
- Toxicology (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562183599P | 2015-06-23 | 2015-06-23 | |
| US201662315453P | 2016-03-30 | 2016-03-30 | |
| US201662338315P | 2016-05-18 | 2016-05-18 | |
| US201662348781P | 2016-06-10 | 2016-06-10 | |
| US201662349572P | 2016-06-13 | 2016-06-13 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HUE068603T2 true HUE068603T2 (hu) | 2025-01-28 |
Family
ID=57586246
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE16815331A HUE068603T2 (hu) | 2015-06-23 | 2016-06-23 | Módosított IX-es faktor, valamint készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez |
| HUS2500003C HUS2500003I1 (hu) | 2015-06-23 | 2025-01-07 | Módosított IX-es faktor, valamint készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUS2500003C HUS2500003I1 (hu) | 2015-06-23 | 2025-01-07 | Módosított IX-es faktor, valamint készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez |
Country Status (25)
| Country | Link |
|---|---|
| US (6) | US10799566B2 (enExample) |
| EP (2) | EP4378487A3 (enExample) |
| JP (3) | JP6831779B2 (enExample) |
| KR (2) | KR102415896B1 (enExample) |
| CN (1) | CN108138159A (enExample) |
| AU (2) | AU2016282781A1 (enExample) |
| CA (1) | CA2990193A1 (enExample) |
| CO (1) | CO2018000134A2 (enExample) |
| DK (1) | DK3313991T3 (enExample) |
| ES (1) | ES2989774T3 (enExample) |
| FI (2) | FI3313991T3 (enExample) |
| FR (1) | FR25C1002I1 (enExample) |
| HR (1) | HRP20241180T1 (enExample) |
| HU (2) | HUE068603T2 (enExample) |
| IL (2) | IL309741A (enExample) |
| MX (2) | MX390848B (enExample) |
| NO (1) | NO2025003I1 (enExample) |
| PE (1) | PE20180675A1 (enExample) |
| PH (1) | PH12017502424A1 (enExample) |
| PL (1) | PL3313991T3 (enExample) |
| PT (1) | PT3313991T (enExample) |
| SA (1) | SA517390590B1 (enExample) |
| SG (1) | SG10202100131WA (enExample) |
| SI (1) | SI3313991T1 (enExample) |
| WO (1) | WO2016210170A1 (enExample) |
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| EP1866422B1 (en) | 2005-04-07 | 2016-04-06 | The Trustees of The University of Pennsylvania | Method of increasing the function of an aav vector |
| EP2337849B1 (en) | 2008-09-15 | 2018-06-13 | uniQure biopharma B.V. | Factor ix polypeptide mutant, its uses and a method for its production |
| RU2653444C2 (ru) * | 2012-02-17 | 2018-05-08 | Дзе Чилдрен'З Хоспитал Оф Филадельфия | Композиции вектора aav и способы переноса генов в клетки, органы и ткани |
| SI3024498T1 (sl) | 2013-07-22 | 2020-07-31 | The Children's Hospital Of Philadelphia | Različica AAV in sestavki, postopki in uporabe za prenos genov v celice, organe in tkiva |
| WO2016073693A2 (en) | 2014-11-05 | 2016-05-12 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| IL309741A (en) | 2015-06-23 | 2024-02-01 | Childrens Hospital Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
| US11219695B2 (en) | 2016-10-20 | 2022-01-11 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of Fabry disease |
| US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| ES2966692T3 (es) * | 2017-03-15 | 2024-04-23 | Univ North Carolina Chapel Hill | Vectores de virus adenoasociados poliploides y métodos de fabricación y uso de los mismos |
| TWI753168B (zh) * | 2017-05-22 | 2022-01-21 | 日商武田藥品工業股份有限公司 | 用於血友病ｂ基因療法之編碼具有增強表現的重組ｆｉｘ變異體之病毒載體 |
| CN108977452B (zh) * | 2017-05-31 | 2022-07-19 | 北卡罗来纳大学教堂山分校 | 优化的人凝血因子ix基因表达盒及其应用 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| PT3652326T (pt) * | 2017-07-10 | 2024-12-26 | Uniqure Ip Bv | Meios e métodos para terapia génica de aav em humanos |
| BR112020012336A2 (pt) * | 2017-12-19 | 2021-03-30 | Akouos, Inc. | Administração de anticorpos terapêuticos mediada por aav para o ouvido interno |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| CA3105953A1 (en) * | 2018-07-09 | 2020-01-16 | Flagship Pioneering Innovations V, Inc. | Fusosome compositions and uses thereof |
| WO2020020475A1 (en) * | 2018-07-27 | 2020-01-30 | Zentrum Für Forschungsförderung In Der Pädiatrie Gmbh | Improved concepts for the treatment of genetic disorders with high-capacity plal-generated gold nanoparticles |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| MX2021001901A (es) * | 2018-08-20 | 2021-05-27 | Ucl Business Ltd | Nucleotidos de codificacion del factor ix. |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB2576508A (en) * | 2018-08-20 | 2020-02-26 | Ucl Business Plc | Factor IX encoding nucleotides |
| CA3111047A1 (en) | 2018-08-24 | 2020-02-27 | Spark Therapeutics, Inc. | Optimized promoter sequences, intron-free expression constructs and methods of use |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| SG11202103734PA (en) | 2018-10-18 | 2021-05-28 | Intellia Therapeutics Inc | Compositions and methods for expressing factor ix |
| BR112021007102A2 (pt) * | 2018-11-09 | 2021-08-03 | Generation Bio Co. | dna com extremidade fechada modificada (cedna) que compreende repetições terminais invertidas modificadas simétricas |
| MX2021008131A (es) * | 2019-01-04 | 2021-10-13 | Sangamo Therapeutics Inc | Metodos y composiciones para el tratamiento de la enfermedad de fabry. |
| WO2020160303A1 (en) | 2019-02-01 | 2020-08-06 | Baxalta Incorporated | Gene therapy of hemophilia b using viral vectors encoding recombinant fix variants with increased expression |
| US11857641B2 (en) * | 2019-02-06 | 2024-01-02 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type I |
| CN110205297A (zh) * | 2019-05-15 | 2019-09-06 | 华北理工大学 | 一种重组脂肪干细胞及其重组方法 |
| EP4022097A4 (en) * | 2019-08-29 | 2022-12-14 | Siemens Healthcare Diagnostics Inc. | REAGENTS AND METHODS FOR DETECTING AAV EXCRETION |
| CN111647625A (zh) * | 2019-12-25 | 2020-09-11 | 深圳三智医学科技有限公司 | 一种提高人凝血因子ix表达水平的方法 |
| AU2021244555A1 (en) * | 2020-03-24 | 2022-11-24 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing factor ix therapeutics |
| JP2023521090A (ja) * | 2020-04-07 | 2023-05-23 | ザ・キュレーターズ・オブ・ザ・ユニバーシティ・オブ・ミズーリ | ＡＡＶ遺伝子治療のためのＣｐＧフリーＩＴＲ |
| CN113817759B (zh) * | 2020-07-10 | 2023-06-02 | 南京吉迈生物技术有限公司 | 修饰的因子ix、组合物、方法及其在基因治疗中的应用 |
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| WO2022208342A1 (en) | 2021-04-01 | 2022-10-06 | Pfizer Inc. | Pharmaceutical compositions containing adeno-associated viral vector |
| EP4079860A1 (en) * | 2021-04-23 | 2022-10-26 | uniQure biopharma B.V. | Methods and means for the prevention and/or treatment of joint damage in hemophilia |
| CA3216420A1 (en) | 2021-04-26 | 2022-11-03 | Seungil Han | Adeno-associated viral vector capsids with improved tissue tropism |
| US20240218397A1 (en) * | 2021-05-04 | 2024-07-04 | Regenxbio Inc. | Novel aav vectors and methods and uses thereof |
| CA3224755A1 (en) | 2021-06-22 | 2022-12-29 | Pfizer Inc. | Production of adeno-associated virus vector in insect cells |
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