CA2713338C - Recombinant virus production using mammalian cells in suspension - Google Patents
Recombinant virus production using mammalian cells in suspension Download PDFInfo
- Publication number
- CA2713338C CA2713338C CA2713338A CA2713338A CA2713338C CA 2713338 C CA2713338 C CA 2713338C CA 2713338 A CA2713338 A CA 2713338A CA 2713338 A CA2713338 A CA 2713338A CA 2713338 C CA2713338 C CA 2713338C
- Authority
- CA
- Canada
- Prior art keywords
- suspension
- mammalian cells
- recombinant virus
- virus production
- methods
- 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.)
- Active
Links
- 239000000725 suspension Substances 0.000 title abstract 2
- 241000700605 Viruses Species 0.000 title 1
- 210000004962 mammalian cell Anatomy 0.000 title 1
- 238000000034 method Methods 0.000 abstract 3
- 239000002245 particle Substances 0.000 abstract 3
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 238000001415 gene therapy Methods 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
- 230000003612 virological effect Effects 0.000 abstract 1
Classifications
-
- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- 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
-
- 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
-
- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
- C12N2710/16641—Use of virus, viral particle or viral elements as a vector
- C12N2710/16643—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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
- C12N2710/16661—Methods of inactivation or attenuation
- C12N2710/16662—Methods of inactivation or attenuation by genetic engineering
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/50—Vectors for producing vectors
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
The invention generally provides methods for producing recombinant AAV viral particles using cells grown in suspension. The invention provides recombinant AAV particles for use in methods for delivering genes encoding therapeutic proteins, and methods for using the recombinant AAV particles in gene therapy.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US6281908P | 2008-01-29 | 2008-01-29 | |
| US61/062,819 | 2008-01-29 | ||
| PCT/US2009/000577 WO2009097129A1 (en) | 2008-01-29 | 2009-01-29 | Recombinant virus production using mammalian cells in suspension |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CA2713338A1 CA2713338A1 (en) | 2009-08-06 |
| CA2713338C true CA2713338C (en) | 2021-10-26 |
Family
ID=40913147
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2713338A Active CA2713338C (en) | 2008-01-29 | 2009-01-29 | Recombinant virus production using mammalian cells in suspension |
Country Status (7)
| Country | Link |
|---|---|
| US (3) | US20110229971A1 (en) |
| EP (1) | EP2242840B1 (en) |
| AU (1) | AU2009209408B2 (en) |
| CA (1) | CA2713338C (en) |
| DK (1) | DK2242840T3 (en) |
| ES (1) | ES2751999T3 (en) |
| WO (1) | WO2009097129A1 (en) |
Families Citing this family (92)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3591055A1 (en) * | 2011-01-07 | 2020-01-08 | Applied Genetic Technologies Corporation | Promoters derived from the 5' nontranslated region (5'-ntr) of the human cyclic nucleotide gated beta 3 (cngb3) gene, expression cassettes and vectors comprising said promoters, for use treating achromatopsia and other diseases |
| GB201103062D0 (en) | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| US10196636B2 (en) | 2011-04-21 | 2019-02-05 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of myotilin |
| WO2012145597A2 (en) * | 2011-04-21 | 2012-10-26 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of myotilin |
| SG11201509419QA (en) | 2013-05-15 | 2015-12-30 | Univ Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| US9644216B2 (en) | 2013-11-26 | 2017-05-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Adeno-associated virus vectors for treatment of glycogen storage disease |
| JP6635942B2 (en) * | 2014-04-15 | 2020-01-29 | アプライド ジェネティック テクノロジーズ コーポレイション | Codon-optimized nucleic acid encoding retinitis pigmentosa GTPase regulator (RPGR) |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| GB201417042D0 (en) * | 2014-09-29 | 2014-11-12 | Fkd Therapies Oy | Method |
| SG11201703148TA (en) | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| US11827680B2 (en) * | 2014-11-06 | 2023-11-28 | Case Western Reserve University | Compounds and methods of treating usher syndrome III |
| CN107207556B (en) | 2014-11-14 | 2020-12-08 | 沃雅戈治疗公司 | regulatory polynucleotide |
| CN114717264A (en) | 2014-11-14 | 2022-07-08 | 沃雅戈治疗公司 | Compositions and methods for treating amyotrophic lateral sclerosis (ALS) |
| WO2016086227A2 (en) * | 2014-11-26 | 2016-06-02 | The Regents Of The University Of California | Therapeutic compositions comprising transcription factors and methods of making and using the same |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| US10415044B2 (en) | 2014-12-23 | 2019-09-17 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Adeno-associated virus vectors encoding modified G6PC and uses thereof |
| IL315273A (en) | 2015-01-16 | 2024-10-01 | Univ Washington | New microdystrophins and associated method of use |
| EP3719134B1 (en) | 2015-03-11 | 2023-08-23 | The United States of America, as represented by The Secretary, Department of Health and Human Services | Rp2 vectors for treating x-linked retinitis pigmentosa |
| EP3626274B1 (en) | 2015-04-16 | 2025-01-22 | Emory University | Recombinant promoters and vectors for protein expression in liver and use thereof |
| KR20180002706A (en) | 2015-04-23 | 2018-01-08 | 워싱턴 스테이트 유니버시티 | Smad7 gene delivery as a therapeutic |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| AU2016341983B8 (en) | 2015-10-21 | 2022-10-06 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Codon-optimized reduced-size ATP7A cDNA and uses for treatment of copper transport disorders |
| EP3368054A4 (en) | 2015-10-28 | 2019-07-03 | Voyager Therapeutics, Inc. | REGULATORY EXPRESSION USING AN ADENO-ASSOCIATED VIRUS (AAV) |
| SI3411484T1 (en) | 2016-02-05 | 2023-12-29 | Emory University | Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid |
| EP3448987A4 (en) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR THE TREATMENT OF DISEASES |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| IL297576B2 (en) | 2016-05-18 | 2024-02-01 | Voyager Therapeutics Inc | Preparations and methods for treating Huntington's disease |
| KR102652994B1 (en) | 2016-05-18 | 2024-04-01 | 보이저 테라퓨틱스, 인크. | Modulatory polynucleotides |
| EP3506817A4 (en) | 2016-08-30 | 2020-07-22 | The Regents of The University of California | METHOD FOR BIOMEDICAL TARGETING AND RELEASE, AND DEVICES AND SYSTEMS FOR IMPLEMENTING THEM |
| US20190367944A1 (en) | 2017-01-30 | 2019-12-05 | The U.S.A., As Represented By The Secretary, Department Of Health And Human Services | Recombinant virus vectors for the treatment of glycogen storage disease |
| CN111108198A (en) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | Compositions and methods for treating Huntington's disease |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | COMPOSITIONS AND TREATMENT METHODS FOR AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| CN111108196A (en) | 2017-05-09 | 2020-05-05 | 爱莫里大学 | Coagulation factor variants and uses thereof |
| JOP20190269A1 (en) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| AU2018302016A1 (en) | 2017-07-17 | 2020-02-06 | The Regents Of The University Of California | Trajectory array guide system |
| KR102883778B1 (en) | 2017-09-22 | 2025-11-12 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Gene therapy for the treatment of type II mucopolysaccharidosis |
| WO2019067840A1 (en) | 2017-09-29 | 2019-04-04 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
| CA3077426A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| CA3091810A1 (en) | 2018-03-02 | 2019-09-06 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Use of il-34 to treat retinal inflammation and neurodegeneration |
| EP3787771A1 (en) | 2018-04-29 | 2021-03-10 | REGENXBIO Inc. | Scalable clarification process for recombinant aav production |
| WO2019212922A1 (en) | 2018-04-29 | 2019-11-07 | Regenxbio Inc. | Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles |
| CA3099306A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| IL279193B2 (en) | 2018-06-14 | 2025-07-01 | Regenxbio Inc | Anion exchange chromatography for recombinant aav production |
| EP3818161A1 (en) | 2018-07-02 | 2021-05-12 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
| CN110699367B (en) * | 2018-07-09 | 2021-06-11 | 武汉纽福斯生物科技有限公司 | Nucleic acid for coding human NADH dehydrogenase subunit 4 protein and application thereof |
| MX2021000810A (en) | 2018-07-24 | 2021-04-28 | Voyager Therapeutics Inc | Systems and methods for producing gene therapy formulations. |
| US20210163991A1 (en) | 2018-08-10 | 2021-06-03 | Regenxbio Inc. | Scalable method for recombinant aav production |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| TW202035689A (en) | 2018-10-04 | 2020-10-01 | 美商航海家醫療公司 | Methods for measuring the titer and potency of viral vector particles |
| US20210324483A1 (en) | 2018-10-15 | 2021-10-21 | Regenxbio Inc. | Method for measuring the infectivity of replication defective viral vectors and viruses |
| AU2019405758B2 (en) | 2018-12-18 | 2026-02-05 | Ultragenyx Pharmaceutical Inc. | Methods and compositions for treating glycogen storage diseases |
| PL3906066T3 (en) | 2019-01-04 | 2024-09-02 | Ultragenyx Pharmaceutical Inc. | Gene therapy constructs for treating wilson disease |
| TW202039858A (en) | 2019-01-18 | 2020-11-01 | 美商航海家醫療公司 | Methods and systems for producing aav particles |
| MX2021008832A (en) | 2019-02-08 | 2021-09-08 | Krystal Biotech Inc | COMPOSITIONS AND METHODS FOR ADMINISTRATION OF CFTR POLYPEPTIDES. |
| JP2022522166A (en) | 2019-02-28 | 2022-04-14 | ベニテック アイピー ホールディングス インコーポレーテッド | Compositions and Methods for Treating Oculopharyngeal Muscular Dystrophy (OPMD) |
| DK3953483T3 (en) | 2019-04-11 | 2023-12-18 | Regenxbio Inc | SIZE CHROMATOGRAPHY METHODS FOR CHARACTERIZING COMPOSITIONS OF RECOMBINANT ADENO-ASSOCIATED VIRUS |
| US20220143115A1 (en) | 2019-04-19 | 2022-05-12 | Regenxbio Inc. | Adeno-Associated Virus Vector Formulations and Methods |
| WO2020223274A1 (en) | 2019-04-29 | 2020-11-05 | Voyager Therapeutics, Inc. | SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS |
| EP4010465A1 (en) | 2019-08-09 | 2022-06-15 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| EP4022070A1 (en) | 2019-08-26 | 2022-07-06 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| WO2021062012A1 (en) | 2019-09-25 | 2021-04-01 | Emory University | Use of klk10 and engineered derivatizations thereof |
| EP4077688A1 (en) * | 2019-12-20 | 2022-10-26 | Krystal Biotech, Inc. | Compositions and methods for gene delivery to the airways and/or lungs |
| JP7703543B2 (en) | 2020-01-17 | 2025-07-07 | ザ ユナイテッド ステイツ オブ アメリカ アズ リプリゼンテッド バイ ザ セクレタリー、デパートメント オブ ヘルス アンド ヒューマン サービシーズ | Gene therapy for the treatment of CRX autosomal dominant retinopathy - Patents.com |
| KR20220140537A (en) | 2020-02-14 | 2022-10-18 | 울트라제닉스 파마수티컬 인코포레이티드 | Gene therapy to treat CDKL5 deficiency disorder |
| EP4192514A1 (en) | 2020-08-06 | 2023-06-14 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| CN112107696A (en) * | 2020-09-14 | 2020-12-22 | 台州和和生物科技有限公司 | Innovative gene therapy medicine for special route administration and medicine box thereof |
| US20230383278A1 (en) | 2020-09-18 | 2023-11-30 | The United States Of America,As Represented By The Secretary,Department Of Health And Human Services | Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency |
| KR20230120128A (en) | 2020-12-16 | 2023-08-16 | 리젠엑스바이오 인크. | Methods for producing recombinant viral particles |
| WO2022159662A1 (en) | 2021-01-21 | 2022-07-28 | Regenxbio Inc. | Improved production of recombinant polypeptides and viruses |
| WO2022187548A1 (en) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| US20240141377A1 (en) | 2021-03-03 | 2024-05-02 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| EP4322975A4 (en) * | 2021-04-14 | 2025-03-26 | Capsida, Inc. | Plasmids and methods of production of adeno-associated viruses |
| WO2023018854A2 (en) | 2021-08-11 | 2023-02-16 | Solid Biosciences Inc. | Treatment of muscular dystrophy |
| CN115873987A (en) * | 2021-09-30 | 2023-03-31 | 北京五加和基因科技有限公司 | Method and kit for detecting infection titer of adeno-associated virus vector |
| CN118202060A (en) | 2021-10-05 | 2024-06-14 | 再生生物股份有限公司 | Compositions and methods for recombinant AAV production |
| WO2023060113A1 (en) | 2021-10-05 | 2023-04-13 | Regenxbio Inc. | Compositions and methods for recombinant aav production |
| WO2023114816A1 (en) | 2021-12-14 | 2023-06-22 | Neurogene, Inc. | Recombinant optimized galc constructs and methods for treating galc-associated disorders |
| GB202201242D0 (en) | 2022-01-31 | 2022-03-16 | Univ Edinburgh | Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders |
| US20250213727A1 (en) | 2022-03-25 | 2025-07-03 | Regenxbio Inc. | Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy |
| WO2023196898A1 (en) | 2022-04-07 | 2023-10-12 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Beta globin mimetic peptides and their use |
| GB202206336D0 (en) | 2022-04-29 | 2022-06-15 | Univ Edinburgh | Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders |
| WO2023239627A2 (en) | 2022-06-08 | 2023-12-14 | Regenxbio Inc. | Methods for recombinant aav production |
| WO2024054983A1 (en) | 2022-09-08 | 2024-03-14 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| WO2024168358A1 (en) | 2023-02-10 | 2024-08-15 | Expression Therapeutics, Llc | Lentiviral system |
| EP4669654A1 (en) | 2023-02-22 | 2025-12-31 | The United States of America, as represented by The Secretary, Department of Health and Human Services | Recombinant adeno-associated viral vectors without immunodominant T-cell epitope and use thereof |
| AU2024240009A1 (en) | 2023-03-17 | 2025-09-18 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Methods for treatment of age-related macular degeneration |
| EP4689137A1 (en) | 2023-04-07 | 2026-02-11 | REGENXBIO Inc. | Compositions and methods for recombinant aav production |
| WO2024233529A2 (en) | 2023-05-07 | 2024-11-14 | Regenxbio Inc. | Compositions and methods for recombinant aav production |
| WO2025090962A1 (en) | 2023-10-25 | 2025-05-01 | Regenxbio Inc. | Compositions and methods for recombinant aav production |
| WO2025106374A1 (en) | 2023-11-13 | 2025-05-22 | Juno Therapeutics, Inc. | Aav production method |
| WO2026017965A1 (en) | 2024-07-19 | 2026-01-22 | The University Court Of The University Of Edinburgh | Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6686200B1 (en) * | 1993-08-31 | 2004-02-03 | Uab Research Foundation | Methods and compositions for the large scale production of recombinant adeno-associated virus |
| DE4438833A1 (en) * | 1994-10-31 | 1996-05-02 | Bayer Ag | Method for the analytical separation of viruses |
| DE19830141A1 (en) * | 1998-07-06 | 2000-01-13 | Regine Heilbronn | Recombinant herpes viruses for the generation of recombinant adeno-associated viruses |
| US6793926B1 (en) * | 1999-05-27 | 2004-09-21 | Genovo, Inc. | Methods for production of a recombinant adeno-associated virus |
| GB2375479B (en) * | 2002-02-23 | 2003-03-26 | Beldore Ltd | Waste water outlet unit |
| US7091029B2 (en) | 2002-09-23 | 2006-08-15 | Applied Genetics Technologies Corporation | High titer recombinant AAV production |
| US20070202587A1 (en) | 2002-09-23 | 2007-08-30 | Applied Genetic Technologies Corporation | Recombinant AAV production in mammalian cells |
| WO2007059473A2 (en) | 2005-11-12 | 2007-05-24 | Introgen Therapeutics, Inc. | Methods for the production and purification of adenoviral vectors |
| US20110014232A1 (en) * | 2009-07-16 | 2011-01-20 | Agricultural Research Council | Chimeric foot and mouth disease viruses |
-
2009
- 2009-01-29 DK DK09706778.9T patent/DK2242840T3/en active
- 2009-01-29 ES ES09706778T patent/ES2751999T3/en active Active
- 2009-01-29 WO PCT/US2009/000577 patent/WO2009097129A1/en not_active Ceased
- 2009-01-29 CA CA2713338A patent/CA2713338C/en active Active
- 2009-01-29 AU AU2009209408A patent/AU2009209408B2/en not_active Ceased
- 2009-01-29 EP EP09706778.9A patent/EP2242840B1/en active Active
-
2010
- 2010-07-13 US US12/812,671 patent/US20110229971A1/en not_active Abandoned
-
2012
- 2012-11-21 US US13/683,577 patent/US9783826B2/en active Active
-
2017
- 2017-09-07 US US15/697,655 patent/US20180312872A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| AU2009209408A1 (en) | 2009-08-06 |
| US20180312872A1 (en) | 2018-11-01 |
| EP2242840A4 (en) | 2012-12-19 |
| US20110229971A1 (en) | 2011-09-22 |
| DK2242840T3 (en) | 2019-10-21 |
| CA2713338A1 (en) | 2009-08-06 |
| EP2242840B1 (en) | 2019-07-24 |
| EP2242840A1 (en) | 2010-10-27 |
| US9783826B2 (en) | 2017-10-10 |
| ES2751999T3 (en) | 2020-04-02 |
| WO2009097129A1 (en) | 2009-08-06 |
| US20130244331A1 (en) | 2013-09-19 |
| AU2009209408B2 (en) | 2015-06-11 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CA2713338C (en) | Recombinant virus production using mammalian cells in suspension | |
| CY1124905T1 (en) | Variant CAPSID-ASSOCIATED VIRUS VIRUS PARTICLES AND METHODS OF USING THEREOF | |
| WO2010138263A3 (en) | Novel aav 's and uses thereof | |
| US20180327706A1 (en) | Crispr-cas9 delivery to hard-to-transfect cells via membrane deformation | |
| PH12020551039A1 (en) | Non-viral dna vectors and uses thereof for antibody and fusion protein production | |
| MX2021000211A (en) | Methods and products for transfecting cells. | |
| EP4372008A3 (en) | Tropism-modified recombinant viral vectors and uses thereof for the targeted introduction of genetic material into human cells | |
| MX340102B (en) | A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy. | |
| WO2008156702A3 (en) | Bacteria mediated gene silencing | |
| WO2009120762A3 (en) | Human cardiovascular progenitor cells | |
| MX2014003744A (en) | Fibronectin type iii repeat based protein scaffolds with alternative binding surfaces. | |
| EP2330200A3 (en) | Gene expression technique | |
| WO2011139379A3 (en) | A method of treating patients undergoing protein replacement therapy, gene replacement therapy, or other therapeutic modalities | |
| AU2009201827B2 (en) | Nerve regeneration | |
| WO2014186160A8 (en) | Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases | |
| WO2006042158A3 (en) | Methods and compositions for improving recombinant protein production | |
| WO2008122811A3 (en) | Adenoviral vectors encoding a pathogen or tumour antigen | |
| WO2005086667A3 (en) | High efficiency peptide production in plant cells | |
| WO2013119371A3 (en) | Mini-intronic plasmid vectors | |
| WO2011039646A3 (en) | Papilloma virus -like particles for targeted gene delivery | |
| WO2012094193A3 (en) | Methods for enhancing the delivery of gene-transduced cells | |
| WO2019089034A1 (en) | Crispr-cas9 delivery to hard-to-transfect cells via membrane deformation | |
| WO2010098561A3 (en) | Novel beta-galactosidase having a high transglycosylation activity, and a use therefor | |
| WO2013046216A8 (en) | Two plasmid mammalian expression system | |
| EP1149917A3 (en) | Hepatitis B virus vectors for gene therapy |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20140107 |