AR117427A1 - Expresión estable de vectores de aav en sujetos jóvenes - Google Patents
Expresión estable de vectores de aav en sujetos jóvenesInfo
- Publication number
- AR117427A1 AR117427A1 ARP190101290A ARP190101290A AR117427A1 AR 117427 A1 AR117427 A1 AR 117427A1 AR P190101290 A ARP190101290 A AR P190101290A AR P190101290 A ARP190101290 A AR P190101290A AR 117427 A1 AR117427 A1 AR 117427A1
- Authority
- AR
- Argentina
- Prior art keywords
- aav
- vectors
- subject
- stable expression
- young subjects
- Prior art date
Links
- 239000013598 vector Substances 0.000 title abstract 2
- 230000010473 stable expression Effects 0.000 title 1
- 239000013607 AAV vector Substances 0.000 abstract 2
- 108700019146 Transgenes Proteins 0.000 abstract 2
- 210000004185 liver Anatomy 0.000 abstract 2
- 230000007774 longterm Effects 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 208000024891 symptom Diseases 0.000 abstract 1
Classifications
-
- 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
-
- 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
- A61K38/37—Factors VIII
-
- 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
- A61K38/44—Oxidoreductases (1)
-
- 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)
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
-
- 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
- C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
-
- 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
-
- 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
-
- 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
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
- C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
- C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
-
- 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)
-
- 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)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Hematology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Dermatology (AREA)
- Toxicology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862671271P | 2018-05-14 | 2018-05-14 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR117427A1 true AR117427A1 (es) | 2021-08-04 |
Family
ID=66770562
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP190101290A AR117427A1 (es) | 2018-05-14 | 2019-05-14 | Expresión estable de vectores de aav en sujetos jóvenes |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US20200069819A1 (https=) |
| EP (1) | EP3794112A1 (https=) |
| JP (2) | JP2021523198A (https=) |
| KR (1) | KR20210008491A (https=) |
| CN (1) | CN112424345A (https=) |
| AR (1) | AR117427A1 (https=) |
| AU (1) | AU2019270972A1 (https=) |
| BR (1) | BR112020023159A2 (https=) |
| CA (1) | CA3100000A1 (https=) |
| MX (1) | MX2020012167A (https=) |
| SG (1) | SG11202010832YA (https=) |
| TW (1) | TW202016298A (https=) |
| WO (1) | WO2019222132A1 (https=) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PE20190401A1 (es) | 2016-07-26 | 2019-03-13 | Biomarin Pharm Inc | Novedosas proteinas de la capside del virus adenoasociado |
| BR112020022722A8 (pt) | 2018-05-09 | 2022-01-18 | Biomarin Pharm Inc | Genoma e seu uso, vetor, partícula, molécula de ácido nucleico isolada, ácido nucleico, composição, tratamento, método |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| US20230407328A1 (en) | 2020-11-02 | 2023-12-21 | Biomarin Pharmaceutical Inc. | Process for enriching adeno-associated virus |
| US20240376496A1 (en) | 2021-09-03 | 2024-11-14 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| WO2023034990A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| US20240384242A1 (en) | 2021-09-03 | 2024-11-21 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| WO2023034980A1 (en) | 2021-09-03 | 2023-03-09 | Bomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| WO2023034994A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| EP4396203A1 (en) | 2021-09-03 | 2024-07-10 | BioMarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| KR20250108100A (ko) | 2022-09-22 | 2025-07-15 | 디나코 아게 | Aav 유전자 치료법 벡터를 이용한 심근병증의 치료 |
| UY40442A (es) | 2022-09-22 | 2024-02-15 | Biomarin Pharm Inc | Tratamiento de la miocardiopatía arritmogénica con vectores de genoterapia con aav |
| JP2025534900A (ja) * | 2022-10-08 | 2025-10-21 | リンイ バイオテック カンパニー リミテッド | 遺伝子発現を増強するためのコンストラクト |
| CN116350801A (zh) * | 2022-11-22 | 2023-06-30 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
| WO2025214477A1 (en) | 2024-04-12 | 2025-10-16 | Skyline Therapeutics (Shanghai) Co., Ltd. | Treatment of genetic cardiomyopathies with aav gene therapy vectors |
Family Cites Families (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US298A (en) | 1837-07-29 | Process for purifying salt-water preparatory to manufacturing salt | ||
| US6531A (en) | 1849-06-19 | Beed musical instrument | ||
| US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| IE58011B1 (en) | 1983-05-27 | 1993-06-16 | Texas A & M Univ Sys | Method for producing a recombinant baculovirus expression vector |
| ZA848495B (en) | 1984-01-31 | 1985-09-25 | Idaho Res Found | Production of polypeptides in insect cells |
| US4994371A (en) | 1987-08-28 | 1991-02-19 | Davie Earl W | DNA preparation of Christmas factor and use of DNA sequences |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| WO1999003496A1 (en) | 1997-07-21 | 1999-01-28 | The University Of North Carolina At Chapel Hill | Factor ix antihemophilic factor with increased clotting activity |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| US7943374B2 (en) * | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| US7943379B2 (en) | 2008-04-30 | 2011-05-17 | Nationwide Children's Hospital, Inc. | Production of rAAV in vero cells using particular adenovirus helpers |
| EP4219547A3 (en) | 2008-09-15 | 2023-10-18 | uniQure biopharma B.V. | Factor ix polypeptide mutant, its uses and a method for its production |
| GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| RS61039B1 (sr) | 2013-09-12 | 2020-12-31 | Biomarin Pharm Inc | Aav vektori koji sadrže gen koji kodira faktor viii |
| RU2742352C2 (ru) * | 2015-09-24 | 2021-02-05 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| CA3002524A1 (en) * | 2015-10-28 | 2017-05-04 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
| PE20231949A1 (es) | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| CN110770346B (zh) * | 2017-03-15 | 2024-01-12 | 北卡罗来纳-查佩尔山大学 | 多倍体腺相关病毒载体及其制备和使用方法 |
-
2019
- 2019-05-14 US US16/411,841 patent/US20200069819A1/en not_active Abandoned
- 2019-05-14 TW TW108116635A patent/TW202016298A/zh unknown
- 2019-05-14 BR BR112020023159-2A patent/BR112020023159A2/pt unknown
- 2019-05-14 CN CN201980047182.9A patent/CN112424345A/zh active Pending
- 2019-05-14 EP EP19728796.4A patent/EP3794112A1/en not_active Withdrawn
- 2019-05-14 KR KR1020207034760A patent/KR20210008491A/ko not_active Withdrawn
- 2019-05-14 AU AU2019270972A patent/AU2019270972A1/en not_active Abandoned
- 2019-05-14 AR ARP190101290A patent/AR117427A1/es not_active Application Discontinuation
- 2019-05-14 WO PCT/US2019/032092 patent/WO2019222132A1/en not_active Ceased
- 2019-05-14 CA CA3100000A patent/CA3100000A1/en active Pending
- 2019-05-14 MX MX2020012167A patent/MX2020012167A/es unknown
- 2019-05-14 JP JP2020564142A patent/JP2021523198A/ja active Pending
- 2019-05-14 SG SG11202010832YA patent/SG11202010832YA/en unknown
-
2023
- 2023-11-08 JP JP2023191118A patent/JP2024028696A/ja active Pending
-
2024
- 2024-05-13 US US18/662,491 patent/US20240285806A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP3794112A1 (en) | 2021-03-24 |
| TW202016298A (zh) | 2020-05-01 |
| CN112424345A (zh) | 2021-02-26 |
| US20240285806A1 (en) | 2024-08-29 |
| MX2020012167A (es) | 2022-07-13 |
| SG11202010832YA (en) | 2020-11-27 |
| JP2021523198A (ja) | 2021-09-02 |
| JP2024028696A (ja) | 2024-03-05 |
| CA3100000A1 (en) | 2019-11-21 |
| BR112020023159A2 (pt) | 2021-02-02 |
| WO2019222132A8 (en) | 2020-12-03 |
| US20200069819A1 (en) | 2020-03-05 |
| AU2019270972A1 (en) | 2020-12-03 |
| KR20210008491A (ko) | 2021-01-22 |
| WO2019222132A1 (en) | 2019-11-21 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA | Abandonment or withdrawal |