DK3268024T3 - Rekombinante glut1-adeno-associerede virusvektorkonstrukter og relaterede fremgangsmåder til genoprettelse af glut1-ekspression - Google Patents
Rekombinante glut1-adeno-associerede virusvektorkonstrukter og relaterede fremgangsmåder til genoprettelse af glut1-ekspression Download PDFInfo
- Publication number
- DK3268024T3 DK3268024T3 DK16762530.0T DK16762530T DK3268024T3 DK 3268024 T3 DK3268024 T3 DK 3268024T3 DK 16762530 T DK16762530 T DK 16762530T DK 3268024 T3 DK3268024 T3 DK 3268024T3
- Authority
- DK
- Denmark
- Prior art keywords
- glut1
- recovering
- associated virus
- recombinant
- related methods
- Prior art date
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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
- 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
- C12N5/069—Vascular Endothelial cells
-
- 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
- A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
-
- 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
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
-
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
-
- 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
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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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
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- 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)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Biophysics (AREA)
- Cell Biology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Toxicology (AREA)
- Psychology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Vascular Medicine (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562130899P | 2015-03-10 | 2015-03-10 | |
| PCT/US2016/021810 WO2016145217A1 (en) | 2015-03-10 | 2016-03-10 | Recombinant glut1 adeno-associated viral vector constructs and related methods for restoring glut1 expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK3268024T3 true DK3268024T3 (da) | 2020-12-07 |
Family
ID=56879055
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK16762530.0T DK3268024T3 (da) | 2015-03-10 | 2016-03-10 | Rekombinante glut1-adeno-associerede virusvektorkonstrukter og relaterede fremgangsmåder til genoprettelse af glut1-ekspression |
Country Status (19)
| Country | Link |
|---|---|
| US (2) | US20180042991A1 (da) |
| EP (1) | EP3268024B1 (da) |
| JP (3) | JP2018509164A (da) |
| KR (2) | KR20240119152A (da) |
| CN (1) | CN107635575A (da) |
| AU (1) | AU2016229000B2 (da) |
| CL (1) | CL2017002282A1 (da) |
| CO (1) | CO2017010149A2 (da) |
| DK (1) | DK3268024T3 (da) |
| EA (1) | EA036051B1 (da) |
| ES (1) | ES2836258T3 (da) |
| HU (1) | HUE052577T2 (da) |
| IL (2) | IL282053B2 (da) |
| MX (2) | MX2017011615A (da) |
| PL (1) | PL3268024T3 (da) |
| PT (1) | PT3268024T (da) |
| SG (1) | SG11201707116QA (da) |
| UA (1) | UA128249C2 (da) |
| WO (1) | WO2016145217A1 (da) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3215191B1 (en) | 2014-11-05 | 2024-08-14 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| JP6996728B2 (ja) * | 2016-01-07 | 2022-01-17 | 学校法人自治医科大学 | グルコーストランスポーター１発現用アデノ随伴ウイルスベクター |
| KR102673203B1 (ko) | 2016-04-20 | 2024-06-10 | 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. | Pklr의 유전자 발현 증강을 위한 조성물 및 방법 |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| MX2020003042A (es) * | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| US12441998B2 (en) | 2017-10-12 | 2025-10-14 | The Trustees Of Columbia University In The City Of New York | SLC2A1 lncRNA as a biologic and related treatments and methods |
| AU2018352583B2 (en) | 2017-10-16 | 2025-04-17 | Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. | Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency |
| US12539316B2 (en) | 2018-04-11 | 2026-02-03 | Spacecraft Seven, Llc | Compositions and methods for stem cell transplant |
| IL278213B2 (en) | 2018-04-27 | 2026-04-01 | Rocket Pharmaceuticals Ltd | A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd) |
| KR102877876B1 (ko) * | 2018-05-08 | 2025-10-28 | 루트거스, 더 스테이트 유니버시티 오브 뉴 저지 | Aav-상용성 라미닌-링커 중합 단백질 |
| CN112424359A (zh) | 2018-05-15 | 2021-02-26 | 沃雅戈治疗公司 | 用于治疗帕金森氏病的组合物和方法 |
| IL280465B2 (en) | 2018-07-30 | 2025-03-01 | Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P | Methods for gene modification of hematopoietic cells |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| US11162102B2 (en) | 2019-05-13 | 2021-11-02 | Dna Twopointo Inc. | Modifications of mammalian cells using artificial micro-RNA to alter their properties and the compositions of their products |
| CN114555814A (zh) * | 2019-09-13 | 2022-05-27 | 罗特格斯新泽西州立大学 | Aav相容的层粘连蛋白-连接子聚合蛋白 |
| EP4142775B1 (en) * | 2020-05-01 | 2026-03-25 | Ageronix SA | Treatment and/or prevention of a disease or a syndrome related to a virus infection |
| EP4178974A4 (en) * | 2020-07-09 | 2024-10-30 | The Board of Trustees of the Leland Stanford Junior University | IMMUNE CELLS WITH INCREASED GLYCOLYTIC FLOW |
| AU2021321412A1 (en) * | 2020-08-05 | 2023-04-06 | Spacecraft Seven, Llc | Adeno-associated viral vector for GLUT1 expression and uses thereof |
| WO2022032226A1 (en) | 2020-08-07 | 2022-02-10 | Spacecraft Seven, Llc | Plakophilin-2 (pkp2) gene therapy using aav vector |
| WO2022051627A2 (en) * | 2020-09-04 | 2022-03-10 | Dna Twopointo Inc. | Modifications of mammalian cells using artificial micro-rna to alter their properties and the compositions of their products |
| MX2024009685A (es) * | 2022-02-08 | 2024-08-19 | Spacecraft Seven Llc | Vector viral adenoasociado para la expresion de glut1 y usos del mismo. |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5399363A (en) | 1991-01-25 | 1995-03-21 | Eastman Kodak Company | Surface modified anticancer nanoparticles |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5543158A (en) | 1993-07-23 | 1996-08-06 | Massachusetts Institute Of Technology | Biodegradable injectable nanoparticles |
| IE80468B1 (en) | 1995-04-04 | 1998-07-29 | Elan Corp Plc | Controlled release biodegradable nanoparticles containing insulin |
| US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| CA2264482A1 (en) | 1996-09-06 | 1998-03-12 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| EP1648926B1 (en) * | 2003-05-02 | 2013-07-03 | Centre National De La Recherche Scientifique (Cnrs) | Glut-1 as a receptor for htlv envelopes and its uses |
| JP2006345703A (ja) * | 2003-09-09 | 2006-12-28 | Osaka Industrial Promotion Organization | 高発現型汎用哺乳動物宿主細胞 |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| DE23193606T1 (de) | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| US20100199362A1 (en) * | 2007-04-26 | 2010-08-05 | President And Fellows Of Harvard College | Wnt ligands involved in blood-brain barrier development and uses therefor |
| WO2010071832A1 (en) * | 2008-12-19 | 2010-06-24 | Nationwide Children's Hospital | Delivery of polynucleotides across the blood brain barrier using recombinant aav9 |
| US9610363B2 (en) * | 2009-04-16 | 2017-04-04 | President And Fellows Of Harvard College | Methods for inhibiting starvation of a cell |
| DK2561073T3 (da) * | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav-vektorer målrettet mod centralnervesystemet og fremgangsmåder til anvendelse deraf |
| EP2394667A1 (en) * | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
-
2016
- 2016-03-10 MX MX2017011615A patent/MX2017011615A/es unknown
- 2016-03-10 IL IL282053A patent/IL282053B2/en unknown
- 2016-03-10 PT PT167625300T patent/PT3268024T/pt unknown
- 2016-03-10 SG SG11201707116QA patent/SG11201707116QA/en unknown
- 2016-03-10 AU AU2016229000A patent/AU2016229000B2/en not_active Ceased
- 2016-03-10 JP JP2017548205A patent/JP2018509164A/ja active Pending
- 2016-03-10 WO PCT/US2016/021810 patent/WO2016145217A1/en not_active Ceased
- 2016-03-10 KR KR1020247024535A patent/KR20240119152A/ko not_active Ceased
- 2016-03-10 ES ES16762530T patent/ES2836258T3/es active Active
- 2016-03-10 EA EA201791936A patent/EA036051B1/ru unknown
- 2016-03-10 KR KR1020177028554A patent/KR20180016722A/ko not_active Ceased
- 2016-03-10 CN CN201680025266.9A patent/CN107635575A/zh active Pending
- 2016-03-10 PL PL16762530.0T patent/PL3268024T3/pl unknown
- 2016-03-10 HU HUE16762530A patent/HUE052577T2/hu unknown
- 2016-03-10 EP EP16762530.0A patent/EP3268024B1/en active Active
- 2016-03-10 DK DK16762530.0T patent/DK3268024T3/da active
- 2016-03-10 US US15/556,412 patent/US20180042991A1/en not_active Abandoned
- 2016-03-10 UA UAA201709758A patent/UA128249C2/uk unknown
-
2017
- 2017-09-03 IL IL254286A patent/IL254286B/en active IP Right Grant
- 2017-09-08 CL CL2017002282A patent/CL2017002282A1/es unknown
- 2017-09-08 MX MX2023001213A patent/MX2023001213A/es unknown
- 2017-10-05 CO CONC2017/0010149A patent/CO2017010149A2/es unknown
-
2020
- 2020-11-19 US US16/952,801 patent/US20210069292A1/en not_active Abandoned
-
2021
- 2021-03-01 JP JP2021032174A patent/JP2021097689A/ja active Pending
-
2023
- 2023-08-17 JP JP2023133147A patent/JP2023144087A/ja active Pending
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