MX2020010191A - Composiciones de virus adenoasociado (aav), métodos de fabricación y métodos de uso. - Google Patents
Composiciones de virus adenoasociado (aav), métodos de fabricación y métodos de uso.Info
- Publication number
- MX2020010191A MX2020010191A MX2020010191A MX2020010191A MX2020010191A MX 2020010191 A MX2020010191 A MX 2020010191A MX 2020010191 A MX2020010191 A MX 2020010191A MX 2020010191 A MX2020010191 A MX 2020010191A MX 2020010191 A MX2020010191 A MX 2020010191A
- Authority
- MX
- Mexico
- Prior art keywords
- methods
- making
- aav compositions
- pharmaceutical compositions
- aav
- Prior art date
Links
Classifications
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- 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
- A61K38/162—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
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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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
- A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
- A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
-
- 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
- A61K38/45—Transferases (2)
-
- 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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
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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
- C12N5/0018—Culture media for cell or tissue culture
- C12N5/0031—Serum-free culture media
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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
- C12N5/0018—Culture media for cell or tissue culture
- C12N5/005—Protein-free medium
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y205/00—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5)
- C12Y205/01—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5) transferring alkyl or aryl groups, other than methyl groups (2.5.1)
- C12Y205/0106—Protein geranylgeranyltransferase type II (2.5.1.60)
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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
- 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
- C12N2750/14151—Methods of production or purification of viral material
-
- 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
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
-
- 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
- C12N9/10—Transferases (2.)
- C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Manufacturing & Machinery (AREA)
- Cell Biology (AREA)
- Ophthalmology & Optometry (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Abstract
Se describen métodos para la purificación de una partícula de AAV recombinante (rAAV) de un cultivo de células hospederas de mamífero. La descripción proporciona además una composición farmacéutica producida mediante los métodos de la descripción, las composiciones farmacéuticas y el método de uso de las composiciones farmacéuticas descritas aquí para el tratamiento de la enfermedad.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862653139P | 2018-04-05 | 2018-04-05 | |
US201862746980P | 2018-10-17 | 2018-10-17 | |
US201862773975P | 2018-11-30 | 2018-11-30 | |
PCT/US2019/026064 WO2019195729A1 (en) | 2018-04-05 | 2019-04-05 | Aav compositions, methods of making and methods of use |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2020010191A true MX2020010191A (es) | 2021-02-26 |
Family
ID=66223873
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2020010191A MX2020010191A (es) | 2018-04-05 | 2019-04-05 | Composiciones de virus adenoasociado (aav), métodos de fabricación y métodos de uso. |
Country Status (19)
Country | Link |
---|---|
US (1) | US20210145929A1 (es) |
EP (1) | EP3775234A1 (es) |
JP (1) | JP2021520233A (es) |
KR (1) | KR20210005045A (es) |
CN (1) | CN112384626A (es) |
AU (1) | AU2019247866A1 (es) |
BR (1) | BR112020020174A2 (es) |
CA (1) | CA3096051A1 (es) |
CL (1) | CL2020002566A1 (es) |
CO (1) | CO2020013699A2 (es) |
CR (1) | CR20200507A (es) |
IL (1) | IL277786A (es) |
JO (1) | JOP20200250A1 (es) |
MA (1) | MA52200A (es) |
MX (1) | MX2020010191A (es) |
PH (1) | PH12020551642A1 (es) |
SG (1) | SG11202009698PA (es) |
TW (1) | TW202000905A (es) |
WO (1) | WO2019195729A1 (es) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
IL296258A (en) * | 2020-03-16 | 2022-11-01 | Ultragenyx Pharmaceutical Inc | Methods for increasing the output of recombinant adeno-associated virus |
CN116806158A (zh) | 2020-09-02 | 2023-09-26 | 4D分子治疗有限公司 | 密码子优化的rep1基因及其用途 |
WO2022109247A1 (en) * | 2020-11-20 | 2022-05-27 | Biogen Ma Inc. | Viral vector potency assay |
WO2023165969A1 (en) | 2022-03-03 | 2023-09-07 | Evonik Operations Gmbh | Process to enhance viral vector production in cell-culture using glycyl-glutamine |
Family Cites Families (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
BR112012018899A2 (pt) * | 2010-01-28 | 2015-09-15 | Philadelphia Children Hospital | "método para purificar partículas de vetor de vírus adeno-associado." |
GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
EP3054007A1 (en) * | 2015-02-09 | 2016-08-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Recombinant adeno-associated virus particle purification comprising an immuno-affinity purification step |
-
2019
- 2019-04-05 US US17/045,093 patent/US20210145929A1/en not_active Abandoned
- 2019-04-05 CA CA3096051A patent/CA3096051A1/en not_active Withdrawn
- 2019-04-05 AU AU2019247866A patent/AU2019247866A1/en not_active Withdrawn
- 2019-04-05 EP EP19718558.0A patent/EP3775234A1/en not_active Withdrawn
- 2019-04-05 CN CN201980037670.1A patent/CN112384626A/zh not_active Withdrawn
- 2019-04-05 MX MX2020010191A patent/MX2020010191A/es unknown
- 2019-04-05 MA MA052200A patent/MA52200A/fr unknown
- 2019-04-05 BR BR112020020174-0A patent/BR112020020174A2/pt not_active Application Discontinuation
- 2019-04-05 KR KR1020207031797A patent/KR20210005045A/ko unknown
- 2019-04-05 WO PCT/US2019/026064 patent/WO2019195729A1/en active Application Filing
- 2019-04-05 CR CR20200507A patent/CR20200507A/es unknown
- 2019-04-05 JP JP2021504132A patent/JP2021520233A/ja not_active Withdrawn
- 2019-04-05 JO JOP/2020/0250A patent/JOP20200250A1/ar unknown
- 2019-04-05 SG SG11202009698PA patent/SG11202009698PA/en unknown
- 2019-04-08 TW TW108112198A patent/TW202000905A/zh unknown
-
2020
- 2020-10-04 IL IL277786A patent/IL277786A/en unknown
- 2020-10-05 PH PH12020551642A patent/PH12020551642A1/en unknown
- 2020-10-05 CL CL2020002566A patent/CL2020002566A1/es unknown
- 2020-10-30 CO CONC2020/0013699A patent/CO2020013699A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
KR20210005045A (ko) | 2021-01-13 |
JOP20200250A1 (ar) | 2020-10-04 |
WO2019195729A1 (en) | 2019-10-10 |
BR112020020174A2 (pt) | 2021-01-19 |
SG11202009698PA (en) | 2020-10-29 |
CL2020002566A1 (es) | 2021-04-16 |
CN112384626A (zh) | 2021-02-19 |
CO2020013699A2 (es) | 2021-04-08 |
IL277786A (en) | 2020-11-30 |
PH12020551642A1 (en) | 2021-07-26 |
US20210145929A1 (en) | 2021-05-20 |
EP3775234A1 (en) | 2021-02-17 |
AU2019247866A1 (en) | 2020-10-22 |
TW202000905A (zh) | 2020-01-01 |
CR20200507A (es) | 2021-03-02 |
CA3096051A1 (en) | 2019-10-10 |
MA52200A (fr) | 2021-02-17 |
JP2021520233A (ja) | 2021-08-19 |
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