IL221158B - Methods for vector purification of the aav virus - Google Patents
Methods for vector purification of the aav virusInfo
- Publication number
- IL221158B IL221158B IL221158A IL22115812A IL221158B IL 221158 B IL221158 B IL 221158B IL 221158 A IL221158 A IL 221158A IL 22115812 A IL22115812 A IL 22115812A IL 221158 B IL221158 B IL 221158B
- Authority
- IL
- Israel
- Prior art keywords
- methods
- viral vector
- aav viral
- vector purification
- purification
- Prior art date
Links
- 238000000034 method Methods 0.000 title 1
- 238000000746 purification Methods 0.000 title 1
- 239000013603 viral 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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/12—Viral antigens
- A61K39/23—Parvoviridae, e.g. feline panleukopenia virus
-
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2/00—Peptides of undefined number of amino acids; Derivatives thereof
-
- 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
- C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
-
- 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
- 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
-
- 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
- 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
- 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
-
- 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
- C12N2750/14051—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
- 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
-
- 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
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/01—Carboxylic ester hydrolases (3.1.1)
- C12Y301/01064—Retinoid isomerohydrolase (3.1.1.64)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Cell Biology (AREA)
- Manufacturing & Machinery (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Mycology (AREA)
- Diabetes (AREA)
- Hematology (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)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US29918410P | 2010-01-28 | 2010-01-28 | |
| PCT/US2011/022371 WO2011094198A1 (en) | 2010-01-28 | 2011-01-25 | A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL221158B true IL221158B (en) | 2018-07-31 |
Family
ID=44319714
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL221158A IL221158B (en) | 2010-01-28 | 2012-07-26 | Methods for vector purification of the aav virus |
Country Status (13)
| Country | Link |
|---|---|
| US (4) | US9408904B2 (https=) |
| EP (1) | EP2529020B1 (https=) |
| JP (4) | JP2013517798A (https=) |
| CN (2) | CN102947453A (https=) |
| AU (1) | AU2011209743B2 (https=) |
| CA (1) | CA2787827C (https=) |
| DK (1) | DK2529020T3 (https=) |
| ES (1) | ES2680915T3 (https=) |
| IL (1) | IL221158B (https=) |
| IN (1) | IN2012DN06629A (https=) |
| MX (1) | MX340102B (https=) |
| PT (1) | PT2529020T (https=) |
| WO (1) | WO2011094198A1 (https=) |
Families Citing this family (124)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| DK2529020T3 (en) * | 2010-01-28 | 2018-08-06 | Childrens Hospital Philadelphia | SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY |
| WO2011133874A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Multicistronic expression constructs |
| EP2826860B1 (en) | 2010-04-23 | 2018-08-22 | University of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
| PT2744895E (pt) * | 2011-09-08 | 2016-02-08 | Uniqure Ip Bv | Remoção de vírus contaminantes de preparações de aav |
| EP2934532B1 (en) | 2012-12-20 | 2019-10-23 | Purdue Research Foundation | Chimeric antigen receptor-expressing t cells as anti-cancer therapeutics |
| FR3002237B1 (fr) * | 2013-02-15 | 2017-12-15 | Genethon | Methodes pour la production de particules virales aav double brin |
| WO2015081101A1 (en) | 2013-11-26 | 2015-06-04 | 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 |
| GB201401707D0 (en) | 2014-01-31 | 2014-03-19 | Sec Dep For Health The | Adeno-associated viral vectors |
| US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
| EP4410805A3 (en) | 2014-03-18 | 2024-11-27 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| FI3628334T3 (fi) | 2014-03-21 | 2023-09-15 | Genzyme Corp | Geenihoito verkkokalvon pigmenttirappeuma |
| ES2876409T3 (es) | 2014-04-25 | 2021-11-12 | Univ Pennsylvania | Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol |
| EP3134522B1 (en) * | 2014-04-25 | 2021-10-06 | University of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| US10711270B2 (en) | 2014-10-03 | 2020-07-14 | University Of Massachusetts | High efficiency library-identified AAV vectors |
| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| AU2015335923B2 (en) | 2014-10-21 | 2021-04-29 | University Of Massachusetts | Recombinant AAV variants and uses thereof |
| FI3224376T4 (fi) * | 2014-11-28 | 2023-08-31 | Dna-epäpuhtaudet koostumuksessa, joka käsittää parvovirusvirionin | |
| JP6824169B2 (ja) | 2014-12-23 | 2021-02-03 | ザ ユナイテッド ステイツ オブ アメリカ, アズ リプレゼンテッド 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 | 改変g6pcをコードするアデノ随伴ウイルスベクターおよびその使用 |
| SG11201704919SA (en) | 2015-01-13 | 2017-07-28 | Alfa Wassermann Inc | Methods of purifying adeno-associated virus (aav) and/or recombinant adeno-associated virus (raav) |
| EP3054006A1 (en) | 2015-02-09 | 2016-08-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Recombinant adeno-associated virus particle purification with multiple-step anion exchange chromatography |
| 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 |
| WO2016131009A1 (en) | 2015-02-13 | 2016-08-18 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
| EP3262162A4 (en) * | 2015-02-23 | 2018-08-08 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
| CN115074366A (zh) | 2015-04-16 | 2022-09-20 | 埃默里大学 | 用于肝脏中蛋白质表达的重组启动子和载体及其用途 |
| CA2983808C (en) | 2015-04-23 | 2022-03-22 | Washington State University | Smad7 gene delivery as a therapeutic |
| US11046955B2 (en) | 2015-04-24 | 2021-06-29 | University Of Massachusetts | Modified AAV constructs and uses thereof |
| WO2017070525A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance in neurodegenerative disease |
| CA3002980A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| CN105420275A (zh) * | 2015-11-27 | 2016-03-23 | 中国科学院苏州生物医学工程技术研究所 | 制备外源功能基因定点整合的人神经干细胞的方法 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
| IL303850A (en) * | 2015-12-11 | 2023-08-01 | Univ Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| US11028372B2 (en) * | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
| IL297018A (en) | 2015-12-18 | 2022-12-01 | Sangamo Therapeutics Inc | Directed cleavage of cell mhc receptor |
| AU2016369490C1 (en) | 2015-12-18 | 2021-12-23 | Sangamo Therapeutics, Inc. | Targeted disruption of the T cell receptor |
| WO2017136536A1 (en) | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
| EP3411056A4 (en) | 2016-02-02 | 2019-10-02 | Sangamo Therapeutics, Inc. | COMPOUNDS FOR NETWORKING DNA BINDING DOMAINS AND SPLITTING DOMAINS |
| PL3411484T3 (pl) | 2016-02-05 | 2024-02-19 | Emory University | Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego |
| US11060088B2 (en) | 2016-02-12 | 2021-07-13 | University Of Massachusetts | Anti-angiogenic miRNA therapeutics for inhibiting corneal neovascularization |
| BR112018070256A2 (pt) | 2016-03-31 | 2019-01-29 | Spark Therapeutics, Inc. | processo de fabricação de raav totalmente escalável à base de colunas |
| US11207426B2 (en) | 2016-04-05 | 2021-12-28 | University Of Massachusetts | Compositions and methods for selective inhibition of grainyhead-like protein expression |
| JP7282521B2 (ja) | 2016-04-08 | 2023-05-29 | パーデュー・リサーチ・ファウンデイション | Car t細胞療法のための方法および組成物 |
| US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
| US11882815B2 (en) | 2016-06-15 | 2024-01-30 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
| GB201612248D0 (en) | 2016-07-14 | 2016-08-31 | Puridify Ltd | New process |
| KR102695908B1 (ko) * | 2016-07-21 | 2024-08-14 | 스파크 테라퓨틱스, 인코포레이티드 | 재조합 아데노-관련 바이러스(rAAV) 벡터를 고수율로 생산하기 위한 확장 가능한 고회수 방법 및 이에 의해 생산된 재조합 아데노-관련 바이러스(rAAV) 벡터 |
| WO2018022511A1 (en) | 2016-07-25 | 2018-02-01 | The Trustees Of The University Of Pennsylvania | Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof |
| JP2019530737A (ja) | 2016-08-23 | 2019-10-24 | アコーオス インコーポレイテッド | ヒト対象において非加齢性聴力障害を治療するための組成物および方法 |
| SG10201913948PA (en) | 2016-08-24 | 2020-03-30 | Sangamo Therapeutics Inc | Engineered target specific nucleases |
| LT3504229T (lt) | 2016-08-24 | 2021-12-10 | Sangamo Therapeutics, Inc. | Genų raiškos reguliavimas, panaudojant rekombinantines nukleazes |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| EP3526333A4 (en) | 2016-10-13 | 2020-07-29 | University of Massachusetts | AAV CAPSIDE DESIGNS |
| KR102482744B1 (ko) | 2016-11-04 | 2022-12-30 | 다케다 야쿠힌 고교 가부시키가이샤 | 아데노-연관 바이러스 정제 방법 |
| EP3321357A1 (en) | 2016-11-09 | 2018-05-16 | Deutsches Krebsforschungszentrum | Scalable process for oncolytic rat parvovirus h-1 production and purification based on isoelectric point-based elimination of empty particles |
| AU2018212002A1 (en) | 2017-01-30 | 2019-08-29 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Recombinant virus vectors for the treatment of glycogen storage disease |
| CN110612119B (zh) | 2017-02-07 | 2024-10-29 | 西雅图儿童医院(Dba西雅图儿童研究所) | 磷脂醚(ple)car t细胞肿瘤靶向(ctct)剂 |
| US11554147B2 (en) | 2017-02-20 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| JP7178355B2 (ja) | 2017-02-28 | 2022-11-25 | エンドサイト・インコーポレイテッド | Car t細胞療法のための組成物および方法 |
| EP3622065A1 (en) | 2017-05-09 | 2020-03-18 | Emory University | Clotting factor variants and their use |
| WO2018208972A1 (en) | 2017-05-09 | 2018-11-15 | University Of Massachusetts | Methods of treating amyotrophic lateral sclerosis (als) |
| AU2018281306B2 (en) | 2017-06-07 | 2025-04-17 | Spark Therapeutics, Inc. | Enhancing agents for improved cell transfection and/or rAAV vector production |
| MY207534A (en) * | 2017-06-30 | 2025-03-03 | Spark Therapeutics Inc | Aav vector column purification methods |
| CA3071683A1 (en) | 2017-08-08 | 2019-02-14 | Sangamo Therapeutics, Inc. | Chimeric antigen receptor mediated cell targeting |
| CA3075643A1 (en) | 2017-09-22 | 2019-03-28 | University Of Massachusetts | Sod1 dual expression vectors and uses thereof |
| AU2018351528B2 (en) | 2017-10-20 | 2025-02-27 | Research Institute At Nationwide Children's Hospital | Methods and materials for NT-3 gene therapy |
| MX2020004830A (es) * | 2017-11-08 | 2020-11-11 | Avexis Inc | Medios y metodo para preparar vectores virales y usos de los mismos. |
| JP7549303B2 (ja) | 2018-01-22 | 2024-09-11 | エンドサイト・インコーポレイテッド | Car t細胞の使用方法 |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| CN111902164A (zh) | 2018-02-01 | 2020-11-06 | 同源药物公司 | 用于恢复pah基因功能的腺相关病毒组合物及其使用方法 |
| SG11202007426XA (en) | 2018-02-06 | 2020-09-29 | Seattle Childrens Hospital Dba Seattle Childrens Res Inst | Fluorescein-specific cars exhibiting optimal t cell function against fl-ple labelled tumors |
| CN112041437A (zh) | 2018-02-19 | 2020-12-04 | 同源药物公司 | 用于恢复f8基因功能的腺相关病毒组合物和其使用方法 |
| CN112105382A (zh) | 2018-02-23 | 2020-12-18 | 恩多塞特公司 | 用于car t细胞疗法的顺序方法 |
| WO2019169291A1 (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 |
| KR20210005045A (ko) * | 2018-04-05 | 2021-01-13 | 나이트스타엑스 리미티드 | Aav 조성물, 제조 방법 및 사용 방법 |
| US12163129B2 (en) | 2018-06-08 | 2024-12-10 | University Of Massachusetts | Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells |
| CN112601557B (zh) | 2018-06-08 | 2025-06-06 | 诺华股份有限公司 | 用于测量药物产品效力的基于细胞的测定 |
| KR20240014102A (ko) | 2018-06-29 | 2024-01-31 | 우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니 | 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법 |
| CA3109432A1 (en) | 2018-08-20 | 2020-02-27 | Wuhan Neurophth Biotechnology Limited Company | Compositions and methods for treating leber's hereditary optic neuropathy |
| IL281615B2 (en) | 2018-09-21 | 2026-01-01 | Acuitas Therapeutics Inc | Systems and methods for producing lipid nanoparticles and liposomes |
| JP7543259B2 (ja) | 2018-10-18 | 2024-09-02 | アクイタス セラピューティクス インコーポレイテッド | 活性剤の脂質ナノ粒子送達のための脂質 |
| WO2020113034A1 (en) * | 2018-11-30 | 2020-06-04 | Avexis, Inc. | Aav viral vectors and uses thereof |
| ES2969222T3 (es) | 2018-12-18 | 2024-05-17 | Ultragenyx Pharmaceutical Inc | Métodos y composiciones para el tratamiento de enfermedades del almacenamiento de glucógeno |
| KR20210112339A (ko) | 2019-01-04 | 2021-09-14 | 울트라제닉스 파마수티컬 인코포레이티드 | 윌슨병을 치료하기 위한 유전자 요법 구축물 |
| MY202837A (en) | 2019-01-11 | 2024-05-24 | Acuitas Therapeutics Inc | Lipids for lipid nanoparticle delivery of active agents |
| JP2022522995A (ja) * | 2019-01-18 | 2022-04-21 | ボイジャー セラピューティクス インコーポレイテッド | Aav粒子を生成するための方法及びシステム |
| EP3990636A1 (en) | 2019-06-28 | 2022-05-04 | CRISPR Therapeutics AG | Materials and methods for controlling gene editing |
| WO2021014428A1 (en) | 2019-07-25 | 2021-01-28 | Novartis Ag | Regulatable expression systems |
| WO2021062012A1 (en) | 2019-09-25 | 2021-04-01 | Emory University | Use of klk10 and engineered derivatizations thereof |
| CN113025633B (zh) | 2019-12-09 | 2024-08-27 | 武汉纽福斯生物科技有限公司 | 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用 |
| WO2021124152A1 (en) | 2019-12-19 | 2021-06-24 | Pfizer Inc. | Compositions and methods for nucleic acid transfection using cationic polymers and stabilizers |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| JP7703543B2 (ja) | 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 | Crx常染色体優性網膜症の処置のための遺伝子治療 |
| AU2021217003A1 (en) | 2020-02-04 | 2022-09-15 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Anti-dinitrophenol chimeric antigen receptors |
| MX2022009982A (es) | 2020-02-14 | 2022-09-12 | Ultragenyx Pharmaceutical Inc | Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5. |
| JP7807380B2 (ja) | 2020-02-21 | 2026-01-27 | アコーオス インコーポレイテッド | ヒト対象において非加齢関連聴力障害を処置するための組成物および方法 |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復pah基因功能的腺相關病毒組成物及其使用方法 |
| WO2022016070A1 (en) | 2020-07-16 | 2022-01-20 | Acuitas Therapeutics, Inc. | Cationic lipids for use in lipid nanoparticles |
| JP7526360B2 (ja) * | 2020-08-18 | 2024-07-31 | ザルトリウス ビーアイエー セパレーションズ ディー.オー.オー. | マルチモーダル金属アフィニティー処理aavカプシド |
| WO2022061002A1 (en) | 2020-09-18 | 2022-03-24 | 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 |
| EP4229205B1 (en) | 2020-10-15 | 2025-05-14 | F. Hoffmann-La Roche AG | Nucleic acid constructs for va rna transcription |
| WO2022079082A1 (en) | 2020-10-15 | 2022-04-21 | F. Hoffmann-La Roche Ag | Nucleic acid constructs for simultaneous gene activation |
| WO2023042104A1 (en) | 2021-09-16 | 2023-03-23 | Novartis Ag | Novel transcription factors |
| CA3242402A1 (en) | 2021-12-16 | 2023-06-22 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
| 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 |
| CN118974274A (zh) | 2022-04-13 | 2024-11-15 | 豪夫迈·罗氏有限公司 | 用于确定aav基因组的方法 |
| US20250353883A1 (en) | 2022-05-06 | 2025-11-20 | Novartis Ag | Novel recombinant aav vp2 fusion polypeptides |
| CN119256220A (zh) | 2022-05-23 | 2025-01-03 | 豪夫迈·罗氏有限公司 | 用于区分aav颗粒血清型和aav颗粒荷载状态的基于拉曼的方法 |
| EP4532737A1 (en) | 2022-06-03 | 2025-04-09 | F. Hoffmann-La Roche AG | Method for producing recombinant aav particles |
| CA3261054A1 (en) | 2022-07-14 | 2024-01-18 | F. Hoffmann-La Roche Ag | PROCESS FOR THE PRODUCTION OF RECOMBINANT ADENO-ASSOCIATED VIRUS PARTICLES |
| WO2024056561A1 (en) | 2022-09-12 | 2024-03-21 | F. Hoffmann-La Roche Ag | Method for separating full and empty aav particles |
| AU2024216670A1 (en) | 2023-02-07 | 2025-07-31 | F. Hoffmann-La Roche Ag | Method for the detection of anti-aav particle antibodies |
| WO2024168358A1 (en) | 2023-02-10 | 2024-08-15 | Expression Therapeutics, Llc | Lentiviral system |
| JP2026508221A (ja) | 2023-02-22 | 2026-03-10 | ザ ユナイテッド ステイツ オブ アメリカ, アズ リプレゼンテッド 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 | 免疫優性t細胞エピトープを欠く組換えアデノ随伴ウイルスベクターおよびその使用 |
| 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 |
| CN120835797A (zh) | 2023-03-21 | 2025-10-24 | 豪夫迈·罗氏有限公司 | 用于产生重组aav颗粒制备物的方法 |
| WO2025090942A1 (en) * | 2023-10-26 | 2025-05-01 | University Of Notre Dame Du Lac | Gene therapy for treatment of neurometabolic disease |
| WO2025168663A1 (en) | 2024-02-09 | 2025-08-14 | F. Hoffmann-La Roche Ag | Method for producing recombinant adeno-associated viral particles |
| WO2025252480A1 (en) | 2024-06-07 | 2025-12-11 | F. Hoffmann-La Roche Ag | Method for purifying plasmid dna |
| US20260022401A1 (en) | 2024-07-22 | 2026-01-22 | Hoffmann-La Roche Inc. | Novel AAV rep ORFs and Rep polypeptides |
| WO2026037757A1 (en) | 2024-08-13 | 2026-02-19 | F. Hoffmann-La Roche Ag | Modified aav particles |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| US6004797A (en) | 1995-11-09 | 1999-12-21 | Avigen, Inc. | Adenovirus helper-free recombinant AAV Virion production |
| DE69841982D1 (de) * | 1997-03-14 | 2010-12-16 | Philadelphia Children Hospital | Zusammensetzungen zum einsatz in einer gentherapie zur behandlung von hämophilie |
| US6995006B2 (en) * | 1997-09-05 | 2006-02-07 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| ES2340230T3 (es) * | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| CA2369848C (en) | 1999-06-30 | 2011-09-20 | Robert F. Garry | Human endogenous retrovirus in breast cancer |
| WO2001070763A1 (en) * | 2000-03-22 | 2001-09-27 | The Children's Hospital Of Philadelphia | Modified blood clotting factors and methods of use |
| US6593123B1 (en) | 2000-08-07 | 2003-07-15 | Avigen, Inc. | Large-scale recombinant adeno-associated virus (rAAV) production and purification |
| AU2003253595A1 (en) * | 2002-04-05 | 2003-11-03 | The Children's Hospital Of Philadelphia | Methods for the production of chimeric adeno-associated virus (aav) vectors, compositions of chimeric aav vectors, and methods of use thereof |
| DE602004030327D1 (de) * | 2003-05-21 | 2011-01-13 | Genzyme Corp | Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind |
| EP1751275B1 (en) * | 2004-06-01 | 2017-08-16 | Avigen, Inc. | Compositions and methods to prevent aav vector aggregation |
| CN1873012A (zh) * | 2005-06-01 | 2006-12-06 | 上海二医新生基因科技有限公司 | 重组腺相关病毒(rAAV)载体大规模生产新工艺 |
| EP2018421B1 (en) * | 2006-04-28 | 2012-12-19 | The Trustees of the University of Pennsylvania | Scalable production method for aav |
| WO2008128251A1 (en) * | 2007-04-17 | 2008-10-23 | The Children's Hospital Of Philadelphia | Humanized viral vectors and methods of use thereof |
| PL2307551T3 (pl) * | 2008-06-18 | 2017-07-31 | Oxford Biomedica (Uk) Limited | Oczyszczanie wektorów retrowirusowych |
| DK2529020T3 (en) * | 2010-01-28 | 2018-08-06 | Childrens Hospital Philadelphia | SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY |
-
2011
- 2011-01-25 DK DK11737508.9T patent/DK2529020T3/en active
- 2011-01-25 MX MX2012008757A patent/MX340102B/es active IP Right Grant
- 2011-01-25 CN CN2011800166162A patent/CN102947453A/zh active Pending
- 2011-01-25 WO PCT/US2011/022371 patent/WO2011094198A1/en not_active Ceased
- 2011-01-25 JP JP2012551228A patent/JP2013517798A/ja active Pending
- 2011-01-25 PT PT117375089T patent/PT2529020T/pt unknown
- 2011-01-25 CN CN201610109900.9A patent/CN105838737A/zh active Pending
- 2011-01-25 CA CA2787827A patent/CA2787827C/en not_active Expired - Fee Related
- 2011-01-25 EP EP11737508.9A patent/EP2529020B1/en active Active
- 2011-01-25 IN IN6629DEN2012 patent/IN2012DN06629A/en unknown
- 2011-01-25 ES ES11737508.9T patent/ES2680915T3/es active Active
- 2011-01-25 AU AU2011209743A patent/AU2011209743B2/en active Active
-
2012
- 2012-07-26 IL IL221158A patent/IL221158B/en active IP Right Grant
- 2012-07-30 US US13/561,753 patent/US9408904B2/en active Active
-
2016
- 2016-03-25 US US15/081,001 patent/US10328145B2/en active Active
- 2016-05-30 JP JP2016107298A patent/JP2016185153A/ja active Pending
-
2017
- 2017-09-27 JP JP2017186157A patent/JP2018046820A/ja active Pending
-
2018
- 2018-04-04 JP JP2018072783A patent/JP6991095B2/ja active Active
-
2019
- 2019-05-23 US US16/421,397 patent/US20190321463A1/en not_active Abandoned
-
2020
- 2020-09-10 US US17/016,993 patent/US11878056B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| US9408904B2 (en) | 2016-08-09 |
| JP2013517798A (ja) | 2013-05-20 |
| CN102947453A (zh) | 2013-02-27 |
| EP2529020A4 (en) | 2013-10-16 |
| AU2011209743B2 (en) | 2016-03-10 |
| JP6991095B2 (ja) | 2022-01-12 |
| BR112012018899A2 (pt) | 2015-09-15 |
| CA2787827C (en) | 2020-11-10 |
| US20190321463A1 (en) | 2019-10-24 |
| EP2529020B1 (en) | 2018-04-25 |
| IN2012DN06629A (https=) | 2015-10-23 |
| MX2012008757A (es) | 2012-11-06 |
| US20130072548A1 (en) | 2013-03-21 |
| US20160206706A1 (en) | 2016-07-21 |
| US20200405845A1 (en) | 2020-12-31 |
| CA2787827A1 (en) | 2011-08-04 |
| PT2529020T (pt) | 2018-07-30 |
| EP2529020A1 (en) | 2012-12-05 |
| US10328145B2 (en) | 2019-06-25 |
| JP2018046820A (ja) | 2018-03-29 |
| CN105838737A (zh) | 2016-08-10 |
| AU2011209743A1 (en) | 2012-08-23 |
| JP2016185153A (ja) | 2016-10-27 |
| MX340102B (es) | 2016-06-24 |
| JP2018121653A (ja) | 2018-08-09 |
| ES2680915T3 (es) | 2018-09-11 |
| DK2529020T3 (en) | 2018-08-06 |
| WO2011094198A1 (en) | 2011-08-04 |
| US11878056B2 (en) | 2024-01-23 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IL221158B (en) | Methods for vector purification of the aav virus | |
| HRP20181743T1 (hr) | Poboljšani postupci za pročišćavanje rekombinantnih aav vektora | |
| IL225650A0 (en) | Protein purification processes | |
| SG10201507003VA (en) | Reduced graphene oxide-based-composites for the purification of water | |
| ZA201403108B (en) | Purification of anti-c-met antibodies | |
| IL228438A0 (en) | New methods for protein purification | |
| SG10201802316VA (en) | Protein purification | |
| PL2534150T3 (pl) | Sposoby leczenia infekcji wirusowej | |
| SG11201406800UA (en) | Purification of air | |
| SI2552941T1 (sl) | Čiščenje vmesnih spojin kaspofungina | |
| EP2723759A4 (en) | PURIFICATION OF CHIMERIC PROTEIN | |
| GB2502195B (en) | Purification of nucleic acids | |
| IL231876A0 (en) | A system for the purification of viral vectors | |
| GB201004143D0 (en) | Promoter sequence for DNA and viral vectors | |
| EP2714713A4 (en) | PURIFICATION OF ANTIBODIES | |
| ZA201306342B (en) | The treatment of viral infections | |
| EP2558412A4 (en) | PREPARED CLEANING OF SILICIUM | |
| GB201012168D0 (en) | Treatment of viral infections | |
| PL2585230T3 (pl) | Czyszczenie urządzeń do alkilowania katalizowanego HF | |
| GB201001821D0 (en) | Treatment of viral infections | |
| AU2011900691A0 (en) | Viral vector | |
| GB201010255D0 (en) | Improvement of nucleic acid purification yields |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FF | Patent granted | ||
| KB | Patent renewed | ||
| KB | Patent renewed |