PT3519569T - Vetores de vírus adenoassociados para o tratamento de mucopolissacaridoses - Google Patents
Vetores de vírus adenoassociados para o tratamento de mucopolissacaridosesInfo
- Publication number
- PT3519569T PT3519569T PT177775418T PT17777541T PT3519569T PT 3519569 T PT3519569 T PT 3519569T PT 177775418 T PT177775418 T PT 177775418T PT 17777541 T PT17777541 T PT 17777541T PT 3519569 T PT3519569 T PT 3519569T
- Authority
- PT
- Portugal
- Prior art keywords
- mucopolysaccharidoses
- treatment
- virus vectors
- adenoassociated virus
- adenoassociated
- Prior art date
Links
- 241000702421 Dependoparvovirus Species 0.000 title 1
- 208000002678 Mucopolysaccharidoses Diseases 0.000 title 1
- 206010028093 mucopolysaccharidosis Diseases 0.000 title 1
- 239000013598 vector Substances 0.000 title 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
- 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
-
- 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)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- 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)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
-
- 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
-
- 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
- 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)
-
- 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
-
- 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
-
- 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
- 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
-
- 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
-
- 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
-
- 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
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)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Diabetes (AREA)
- Biophysics (AREA)
- Plant Pathology (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)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP16382450 | 2016-09-30 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PT3519569T true PT3519569T (pt) | 2021-03-04 |
Family
ID=57123944
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT177775418T PT3519569T (pt) | 2016-09-30 | 2017-09-22 | Vetores de vírus adenoassociados para o tratamento de mucopolissacaridoses |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US11338046B2 (enExample) |
| EP (1) | EP3519569B1 (enExample) |
| JP (1) | JP7059285B2 (enExample) |
| CN (1) | CN109863243B (enExample) |
| AR (1) | AR109752A1 (enExample) |
| AU (1) | AU2017333336B2 (enExample) |
| ES (1) | ES2859661T3 (enExample) |
| PT (1) | PT3519569T (enExample) |
| TW (1) | TW201814044A (enExample) |
| WO (1) | WO2018060097A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9309534B2 (en) | 2010-07-12 | 2016-04-12 | Universidad Autonoma 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 |
| RU2692251C2 (ru) | 2013-05-15 | 2019-06-24 | Риджентс Оф Зэ Юниверсити Оф Миннесота | Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему |
| US10973931B2 (en) | 2014-09-16 | 2021-04-13 | Universitat Autònoma De Barcelona | Adeno-associated viral vectors for the gene therapy of metabolic diseases |
| ES2824457T3 (es) | 2014-12-05 | 2021-05-12 | UNIV AUTòNOMA DE BARCELONA | Vectores virales para el tratamiento de la diabetes |
| ES2900973T3 (es) | 2015-01-07 | 2022-03-21 | UNIV AUTòNOMA DE BARCELONA | Constructo genético de vector individual que comprende genes de insulina y glucoquinasa |
| BR112020021903A2 (pt) | 2018-04-27 | 2021-03-02 | Rocket Pharmaceuticals, Ltd. | terapia genética para degeneração do snc |
| EP3802803A1 (en) * | 2018-05-30 | 2021-04-14 | Esteve Pharmaceuticals, S.A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type iv a |
| CA3106010A1 (en) * | 2018-07-12 | 2020-01-16 | Rocket Pharmaceuticals, Ltd. | Gene therapy vectors for treatment of danon disease |
| WO2021007473A1 (en) | 2019-07-10 | 2021-01-14 | Masonic Medical Research Institute | 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 |
| CN116194154A (zh) | 2020-08-07 | 2023-05-30 | 太空飞船七有限责任公司 | 使用aav载体的plakophilin-2(pkp2)基因疗法 |
| 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 |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US8227212B2 (en) * | 2003-02-11 | 2012-07-24 | Shire Human Genetic Therapies, Inc. | Cell that expresses a sulfatase and a formylglycine generating enzyme |
| EP2394667A1 (en) | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| CN107982548A (zh) | 2012-02-07 | 2018-05-04 | 全球生物疗法有限公司 | 核酸输送的区室化方法及其组合物和应用 |
| 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 |
| CA2927366A1 (en) * | 2013-10-24 | 2015-04-30 | Uniqure Ip B.V. | Aav-5 pseudotyped vector for gene therapy for neurological diseases |
| GB201322798D0 (en) * | 2013-12-20 | 2014-02-05 | Oxford Biomedica Ltd | Production system |
| MX383227B (es) | 2014-05-14 | 2025-03-13 | UNIV AUTòNOMA DE BARCELONA | Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosomico. |
| AU2016219398A1 (en) | 2015-02-10 | 2017-09-28 | Genzyme Corporation | 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 |
-
2017
- 2017-09-22 PT PT177775418T patent/PT3519569T/pt unknown
- 2017-09-22 US US16/337,953 patent/US11338046B2/en active Active
- 2017-09-22 ES ES17777541T patent/ES2859661T3/es active Active
- 2017-09-22 AU AU2017333336A patent/AU2017333336B2/en active Active
- 2017-09-22 WO PCT/EP2017/074081 patent/WO2018060097A1/en not_active Ceased
- 2017-09-22 EP EP17777541.8A patent/EP3519569B1/en active Active
- 2017-09-22 JP JP2019538714A patent/JP7059285B2/ja active Active
- 2017-09-22 CN CN201780060410.7A patent/CN109863243B/zh active Active
- 2017-09-29 AR ARP170102704A patent/AR109752A1/es unknown
- 2017-09-30 TW TW106133935A patent/TW201814044A/zh unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AU2017333336B2 (en) | 2023-11-09 |
| AU2017333336A1 (en) | 2019-04-11 |
| JP2019528793A (ja) | 2019-10-17 |
| CN109863243B (zh) | 2023-07-28 |
| US11338046B2 (en) | 2022-05-24 |
| TW201814044A (zh) | 2018-04-16 |
| EP3519569B1 (en) | 2020-12-02 |
| CN109863243A (zh) | 2019-06-07 |
| ES2859661T3 (es) | 2021-10-04 |
| US20190231900A1 (en) | 2019-08-01 |
| AR109752A1 (es) | 2019-01-23 |
| EP3519569A1 (en) | 2019-08-07 |
| WO2018060097A1 (en) | 2018-04-05 |
| JP7059285B2 (ja) | 2022-04-25 |
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