ES2859661T3 - Vectores de virus adenoasociados para el tratamiento de mucopolisacaridosis - Google Patents
Vectores de virus adenoasociados para el tratamiento de mucopolisacaridosisInfo
- Publication number
- ES2859661T3 ES2859661T3 ES17777541T ES17777541T ES2859661T3 ES 2859661 T3 ES2859661 T3 ES 2859661T3 ES 17777541 T ES17777541 T ES 17777541T ES 17777541 T ES17777541 T ES 17777541T ES 2859661 T3 ES2859661 T3 ES 2859661T3
- Authority
- ES
- Spain
- Prior art keywords
- vector
- aav
- cag
- gns
- aav9
- Prior art date
- 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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- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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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)
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
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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
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06014—N-Acetylglucosamine-6-sulfatase (3.1.6.14)
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
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- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/035—Animal model for multifactorial diseases
- A01K2267/0362—Animal model for lipid/glucose metabolism, e.g. obesity, type-2 diabetes
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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
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- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
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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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
- C12N2830/90—Vector systems having a special element relevant for transcription from vertebrates avian
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- Hematology (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
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| EP16382450 | 2016-09-30 | ||
| PCT/EP2017/074081 WO2018060097A1 (en) | 2016-09-30 | 2017-09-22 | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES2859661T3 true ES2859661T3 (es) | 2021-10-04 |
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| Application Number | Title | Priority Date | Filing Date |
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| ES17777541T Active ES2859661T3 (es) | 2016-09-30 | 2017-09-22 | Vectores de virus adenoasociados para el tratamiento de mucopolisacaridosis |
Country Status (10)
| Country | Link |
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| US (1) | US11338046B2 (OSRAM) |
| EP (1) | EP3519569B1 (OSRAM) |
| JP (1) | JP7059285B2 (OSRAM) |
| CN (1) | CN109863243B (OSRAM) |
| AR (1) | AR109752A1 (OSRAM) |
| AU (1) | AU2017333336B2 (OSRAM) |
| ES (1) | ES2859661T3 (OSRAM) |
| PT (1) | PT3519569T (OSRAM) |
| TW (1) | TW201814044A (OSRAM) |
| WO (1) | WO2018060097A1 (OSRAM) |
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| EP2605798A1 (en) | 2010-07-12 | 2013-06-26 | Universidad Autònoma de Barcelona | Gene therapy composition for use in diabetes treatment |
| EP2692868A1 (en) | 2012-08-02 | 2014-02-05 | Universitat Autònoma De Barcelona | Adeno-associated viral (AAV) vectors useful for transducing adipose tissue |
| EP3622821A1 (en) * | 2013-05-15 | 2020-03-18 | Regents of the University of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| EP3194430A1 (en) | 2014-09-16 | 2017-07-26 | Universitat Autònoma De Barcelona | Adeno-associated viral vectors for the gene therapy of metabolic diseases |
| WO2016087678A1 (en) | 2014-12-05 | 2016-06-09 | Universitat Autònoma De Barcelona | Viral vectors for the treatment of diabetes |
| EP3242945B1 (en) | 2015-01-07 | 2021-09-01 | Universitat Autònoma de Barcelona | Single-vector gene construct comprising insulin and glucokinase genes |
| RU2020134965A (ru) | 2018-04-27 | 2022-04-27 | Спейскрафт Севен, Ллк | Генная терапия при дегенерации цнс |
| AU2019275969B2 (en) * | 2018-05-30 | 2025-07-10 | Esteve Pharmaceuticals, S.A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type IV A |
| AU2019299970B2 (en) * | 2018-07-12 | 2025-12-11 | Spacecraft Seven, Llc | Gene therapy vectors for treatment of Danon disease |
| EP3914720B9 (en) | 2019-07-10 | 2023-09-27 | Masonic Medical Research Laboratory | Vgll4 with ucp-1 cis-regulatory element and method of use thereof |
| CN111718947B (zh) * | 2020-06-18 | 2022-08-23 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途 |
| IL300263A (en) | 2020-08-07 | 2023-03-01 | Spacecraft Seven Llc | Plakophilin-2 (PKP2) gene therapy using an AAV vector |
| CN114480454B (zh) * | 2020-10-27 | 2024-03-29 | 华东理工大学 | 用于治疗粘多糖贮积症ⅱ型的腺相关病毒载体、药物及其应用 |
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| US7232670B2 (en) * | 2001-09-28 | 2007-06-19 | St. Jude Children's Research Hospital | Targeting proteins to cells expressing mannose receptors via expression in insect cells |
| DK2325302T3 (en) * | 2003-02-11 | 2016-03-14 | Shire Human Genetic Therapies | Cells expressing a sulfatase and a C-formylglycine-generating enzyme and methods and uses thereof |
| EP2394667A1 (en) | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| AU2013216920B2 (en) | 2012-02-07 | 2016-09-22 | Global Bio Therapeutics, Inc. | Compartmentalized method of nucleic acid delivery and compositions and uses thereof |
| CN105916990B (zh) * | 2013-08-02 | 2020-02-04 | 巴塞罗那自治大学 | 用于转导脂肪组织的腺相关病毒载体 |
| CN105745326A (zh) * | 2013-10-24 | 2016-07-06 | 优尼科Ip有限公司 | 用于基因治疗神经疾病的aav-5假型载体 |
| GB201322798D0 (en) * | 2013-12-20 | 2014-02-05 | Oxford Biomedica Ltd | Production system |
| AR100419A1 (es) | 2014-05-14 | 2016-10-05 | Esteve Labor Dr | Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosómico |
| SG11201706445SA (en) | 2015-02-10 | 2017-09-28 | Genzyme Corp | Enhanced delivery of viral particles to the striatum and cortex |
| US9981021B1 (en) * | 2015-04-09 | 2018-05-29 | Kinetiq, Inc. | Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof |
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| AU2017333336B2 (en) | 2023-11-09 |
| US11338046B2 (en) | 2022-05-24 |
| EP3519569B1 (en) | 2020-12-02 |
| PT3519569T (pt) | 2021-03-04 |
| AR109752A1 (es) | 2019-01-23 |
| AU2017333336A1 (en) | 2019-04-11 |
| EP3519569A1 (en) | 2019-08-07 |
| JP2019528793A (ja) | 2019-10-17 |
| US20190231900A1 (en) | 2019-08-01 |
| CN109863243B (zh) | 2023-07-28 |
| JP7059285B2 (ja) | 2022-04-25 |
| TW201814044A (zh) | 2018-04-16 |
| CN109863243A (zh) | 2019-06-07 |
| WO2018060097A1 (en) | 2018-04-05 |
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