EP3596203A4 - RATIONAL POLYPLOID ADENO-ASSOCIATED VIRUS VECTORS AND METHOD FOR MANUFACTURING AND USING THEREOF - Google Patents
RATIONAL POLYPLOID ADENO-ASSOCIATED VIRUS VECTORS AND METHOD FOR MANUFACTURING AND USING THEREOF Download PDFInfo
- Publication number
- EP3596203A4 EP3596203A4 EP18907477.6A EP18907477A EP3596203A4 EP 3596203 A4 EP3596203 A4 EP 3596203A4 EP 18907477 A EP18907477 A EP 18907477A EP 3596203 A4 EP3596203 A4 EP 3596203A4
- Authority
- EP
- European Patent Office
- Prior art keywords
- polyploid
- adeno
- rational
- manufacturing
- virus vectors
- 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.)
- Pending
Links
Classifications
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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
-
- 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
- A61K48/0025—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 wherein the non-active part clearly interacts with the delivered nucleic acid
-
- 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
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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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- 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
- A01K2267/0381—Animal model for diseases of the hematopoietic system
-
- 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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- 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
- C12N2750/14145—Special targeting system for 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
- 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
- C12N2750/14151—Methods of production or purification of viral material
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Virology (AREA)
- General Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Manufacture Or Reproduction Of Printing Formes (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862668056P | 2018-05-07 | 2018-05-07 | |
| US201862678675P | 2018-05-31 | 2018-05-31 | |
| PCT/US2018/044632 WO2019216932A1 (en) | 2018-05-07 | 2018-07-31 | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| EP3596203A1 EP3596203A1 (en) | 2020-01-22 |
| EP3596203A4 true EP3596203A4 (en) | 2021-12-01 |
Family
ID=68468205
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EP18907477.6A Pending EP3596203A4 (en) | 2018-05-07 | 2018-07-31 | RATIONAL POLYPLOID ADENO-ASSOCIATED VIRUS VECTORS AND METHOD FOR MANUFACTURING AND USING THEREOF |
Country Status (6)
| Country | Link |
|---|---|
| EP (1) | EP3596203A4 (https=) |
| JP (2) | JP2021522775A (https=) |
| CN (2) | CN110691846A (https=) |
| AU (1) | AU2018422759B2 (https=) |
| IL (1) | IL269184B2 (https=) |
| WO (1) | WO2019216932A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN110684800B (zh) * | 2018-11-02 | 2020-11-03 | 深圳益世康宁生物科技有限公司 | 一种携带肿瘤-睾丸抗原10基因的重组腺相关病毒载体及其应用价值 |
| WO2021142130A1 (en) * | 2020-01-07 | 2021-07-15 | The University Of North Carolina At Chapel Hill | Synthetic adeno-associated virus inverted terminal repeats and methods of their use as promoters |
| CN116096734A (zh) * | 2020-05-13 | 2023-05-09 | 沃雅戈治疗公司 | Aav衣壳的向性的重定向 |
| JP2024515626A (ja) * | 2021-04-16 | 2024-04-10 | アスクレピオス バイオファーマシューティカル, インコーポレイテッド | 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドaavビリオン |
| AU2022286647A1 (en) * | 2021-06-02 | 2023-11-30 | Uniqure Biopharma B.V. | Insect cell production of parvoviral vectors with modified capsid proteins |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20060088936A1 (en) * | 2002-05-01 | 2006-04-27 | Warrington Kenneth H | Raav expression systems for genetic modification of specific capsid proteins |
| US20130296409A1 (en) * | 2010-11-10 | 2013-11-07 | Fred Hutchinson Cancer Research Center | Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination |
| WO2018170310A1 (en) * | 2017-03-15 | 2018-09-20 | The University Of North Carolina At Chapel Hill | Polyploid adeno-associated virus vectors and methods of making and using the same |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1618201A1 (en) * | 2003-05-01 | 2006-01-25 | University of Florida Research Foundation, Inc. | Vp2 - modified raav vectors and uses thereof |
| EP1660123B1 (en) * | 2003-09-02 | 2013-03-13 | GlaxoSmithKline Biologicals s.a. | Rotavirus vaccine |
| EP2848253A1 (en) * | 2006-06-19 | 2015-03-18 | Asklepios Biopharmaceutical, Inc. | Modified factor VIII and factor IX genes and vectors for gene therapy |
| ES2685611T3 (es) * | 2011-02-14 | 2018-10-10 | The Children's Hospital Of Philadelphia | Vector VAA8 mejorado con una actividad funcional aumentada y métodos de utilización del mismo |
| GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| KR20240090694A (ko) * | 2013-07-22 | 2024-06-21 | 더 칠드런스 호스피탈 오브 필라델피아 | 변종 aav 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도 |
| WO2018109207A1 (en) * | 2016-12-16 | 2018-06-21 | Uniqure Ip B.V. | Immunoadsorption |
-
2018
- 2018-07-31 WO PCT/US2018/044632 patent/WO2019216932A1/en not_active Ceased
- 2018-07-31 AU AU2018422759A patent/AU2018422759B2/en active Active
- 2018-07-31 CN CN201880032343.2A patent/CN110691846A/zh active Pending
- 2018-07-31 CN CN202311045266.3A patent/CN117535247A/zh active Pending
- 2018-07-31 IL IL269184A patent/IL269184B2/en unknown
- 2018-07-31 EP EP18907477.6A patent/EP3596203A4/en active Pending
- 2018-07-31 JP JP2019565168A patent/JP2021522775A/ja active Pending
-
2023
- 2023-08-02 JP JP2023125955A patent/JP2023154428A/ja active Pending
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20060088936A1 (en) * | 2002-05-01 | 2006-04-27 | Warrington Kenneth H | Raav expression systems for genetic modification of specific capsid proteins |
| US20130296409A1 (en) * | 2010-11-10 | 2013-11-07 | Fred Hutchinson Cancer Research Center | Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination |
| WO2018170310A1 (en) * | 2017-03-15 | 2018-09-20 | The University Of North Carolina At Chapel Hill | Polyploid adeno-associated virus vectors and methods of making and using the same |
Non-Patent Citations (1)
| Title |
|---|
| ZHENG CHAI ET AL: "Application of polyploid adeno-associated virus vectors for transduction enhancement and neutralizing antibody evasion", JOURNAL OF CONTROLLED RELEASE, vol. 262, 1 September 2017 (2017-09-01), AMSTERDAM, NL, pages 348 - 356, XP055713109, ISSN: 0168-3659, DOI: 10.1016/j.jconrel.2017.08.005 * |
Also Published As
| Publication number | Publication date |
|---|---|
| IL269184A (en) | 2019-11-28 |
| AU2018422759B2 (en) | 2026-01-22 |
| EP3596203A1 (en) | 2020-01-22 |
| AU2018422759A1 (en) | 2020-01-02 |
| IL269184B2 (en) | 2025-08-01 |
| IL269184B1 (en) | 2025-04-01 |
| JP2023154428A (ja) | 2023-10-19 |
| WO2019216932A1 (en) | 2019-11-14 |
| NZ759802A (en) | 2025-08-29 |
| CN110691846A (zh) | 2020-01-14 |
| JP2021522775A (ja) | 2021-09-02 |
| CN117535247A (zh) | 2024-02-09 |
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| A4 | Supplementary search report drawn up and despatched |
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| RIC1 | Information provided on ipc code assigned before grant |
Ipc: C12N 15/86 20060101ALI20211026BHEP Ipc: A61K 48/00 20060101ALI20211026BHEP Ipc: C12N 5/10 20060101ALI20211026BHEP Ipc: C12N 15/864 20060101ALI20211026BHEP Ipc: C12N 15/35 20060101ALI20211026BHEP Ipc: C12N 7/00 20060101AFI20211026BHEP |
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| RAP3 | Party data changed (applicant data changed or rights of an application transferred) |
Owner name: THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL |
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| RAP3 | Party data changed (applicant data changed or rights of an application transferred) |
Owner name: THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL |