[go: up one dir, main page]

MX2023007315A - Método para producir una partícula de virus adenoasociado recombinante. - Google Patents

Método para producir una partícula de virus adenoasociado recombinante.

Info

Publication number
MX2023007315A
MX2023007315A MX2023007315A MX2023007315A MX2023007315A MX 2023007315 A MX2023007315 A MX 2023007315A MX 2023007315 A MX2023007315 A MX 2023007315A MX 2023007315 A MX2023007315 A MX 2023007315A MX 2023007315 A MX2023007315 A MX 2023007315A
Authority
MX
Mexico
Prior art keywords
producing
transferring
volume
virus particle
transfection reagent
Prior art date
Application number
MX2023007315A
Other languages
English (en)
Inventor
Robert Stadelman
Donald Startt
Original Assignee
Regenxbio Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Regenxbio Inc filed Critical Regenxbio Inc
Publication of MX2023007315A publication Critical patent/MX2023007315A/es

Links

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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0603—Embryonic cells ; Embryoid bodies
    • 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
    • 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
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Developmental Biology & Embryology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Reproductive Health (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

En la presente memoria se proporcionan métodos mejorados para producir partículas de virus recombinante. En algunas modalidades, un método para producir partículas de virus recombinante descrito en la presente memoria comprende proporcionar un cultivo de células en suspensión que comprende una población de células capaces de producir la partícula viral, transferir un primer volumen de complejos de polinucleótido:reacti vo de transfección al cultivo en suspensión para transfectar las células, y transferir un segundo volumen de complejos de polinucleótido:reactivo de transfección al cultivo en suspensión, donde la transferencia del primer y segundo volumen de complejos de polinucleótido:reactivo de transfección se realiza simultánea o consecutivamente en cualquier orden. En algunas modalidades, las partículas de virus recombinante son partículas de AAV recombinante (rAAV).
MX2023007315A 2020-12-16 2021-12-16 Método para producir una partícula de virus adenoasociado recombinante. MX2023007315A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063126405P 2020-12-16 2020-12-16
PCT/US2021/063739 WO2022133051A1 (en) 2020-12-16 2021-12-16 Method of producing a recombinant adeno-associated virus particle

Publications (1)

Publication Number Publication Date
MX2023007315A true MX2023007315A (es) 2023-08-22

Family

ID=80050899

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023007315A MX2023007315A (es) 2020-12-16 2021-12-16 Método para producir una partícula de virus adenoasociado recombinante.

Country Status (10)

Country Link
US (1) US20240167054A1 (es)
EP (1) EP4263841A1 (es)
JP (1) JP2023554389A (es)
KR (1) KR20230120128A (es)
CN (1) CN116761892A (es)
AU (1) AU2021403076A1 (es)
CA (1) CA3201743A1 (es)
IL (1) IL303614A (es)
MX (1) MX2023007315A (es)
WO (1) WO2022133051A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116983434B (zh) * 2023-09-28 2024-03-15 康霖生物科技（杭州）有限公司 用于基因治疗的核酸构建体及其用途

Family Cites Families (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
AU781958C (en) 1999-08-09 2006-03-30 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
SG10201506627PA (en) 2001-11-13 2015-10-29 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
CN102212558B (zh) 2003-09-30 2016-08-03 宾夕法尼亚大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
US7183969B2 (en) 2004-12-22 2007-02-27 Raytheon Company System and technique for calibrating radar arrays
CN101203613B (zh) 2005-04-07 2012-12-12 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 振幅誤差補償装置及び直交度誤差補償装置
DK2242840T3 (da) 2008-01-29 2019-10-21 Applied Genetic Tech Corporation Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension
EP3257937B1 (en) 2008-02-19 2022-08-03 uniQure IP B.V. Optimisation of expression of parvoviral rep and cap proteins in insect cells
US20110212526A1 (en) * 2008-03-18 2011-09-01 National Tsing Hua University Method for producing recombinant adeno-associated virus
CN102439157B (zh) 2009-04-30 2015-09-16 宾夕法尼亚大学托管会 包含腺伴随病毒构建体的靶向传导气道细胞组合物
NZ596595A (en) 2009-05-12 2013-05-31 Transgene Sa Method for orthopoxvirus production and purification
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2011097447A2 (en) * 2010-02-04 2011-08-11 Neurologix, Inc. Production of recombinant virus
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US10738326B2 (en) 2010-10-27 2020-08-11 Jichi Medical University Adeno-associated virus vector for gene transfer to nervous system cells
DK2673289T3 (da) 2011-02-10 2023-07-24 Univ North Carolina Chapel Hill Virusvektorer med modificerede transduktionsprofiler og fremgangsmåder til fremstilling og anvendelse deraf
EP2699270B1 (en) 2011-04-22 2017-06-21 The Regents of The University of California Adeno-associated virus virions with variant capsid and methods of use thereof
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
US9677088B2 (en) 2012-05-09 2017-06-13 Oregon Health & Science University Adeno associated virus plasmids and vectors
EP2970946A4 (en) 2013-03-13 2016-09-07 Philadelphia Children Hospital ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF
KR102413498B1 (ko) 2013-04-20 2022-06-24 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 엑손 2-표적 U7snRNA 폴리뉴클레오티드 작제물의 재조합형 아데노 부속 바이러스 전달
PH12016500162B1 (en) 2013-07-22 2024-02-21 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene trnsfer to cells, organs, and tissues
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
PT3459965T (pt) 2013-10-11 2021-02-25 Massachusetts Eye & Ear Infirmary Métodos de previsão de sequências de vírus ancestrais e suas utilizações
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
PL3198018T3 (pl) 2014-09-24 2021-07-19 City Of Hope Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposoby
JP6665466B2 (ja) 2015-09-26 2020-03-13 日亜化学工業株式会社 半導体発光素子及びその製造方法
WO2017070491A1 (en) 2015-10-23 2017-04-27 Applied Genetic Technologies Corporation Ophthalmic formulations
US12487152B2 (en) * 2017-05-09 2025-12-02 Ultragenyx Pharmaceutical Inc. Scalable method for producing transfection reagents
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
EP3787771A1 (en) 2018-04-29 2021-03-10 REGENXBIO Inc. Scalable clarification process for recombinant aav production
IL279193B2 (en) 2018-06-14 2025-07-01 Regenxbio Inc Anion exchange chromatography for recombinant AAV production
US20210163991A1 (en) 2018-08-10 2021-06-03 Regenxbio Inc. Scalable method for recombinant aav production

Also Published As

Publication number Publication date
CN116761892A (zh) 2023-09-15
KR20230120128A (ko) 2023-08-16
EP4263841A1 (en) 2023-10-25
AU2021403076A1 (en) 2023-06-29
WO2022133051A1 (en) 2022-06-23
JP2023554389A (ja) 2023-12-27
IL303614A (en) 2023-08-01
US20240167054A1 (en) 2024-05-23
CA3201743A1 (en) 2022-06-23

Similar Documents

Publication Publication Date Title
Lehnertz et al. HLF expression defines the human hematopoietic stem cell state
Dai et al. One-step generation of modular CAR-T cells with AAV–Cpf1
Bak et al. CRISPR-mediated integration of large gene cassettes using AAV donor vectors
Costariol et al. Demonstrating the manufacture of human CAR‐T cells in an automated stirred‐tank bioreactor
EP3983542A2 (en) Methods and compositions for improved homology directed repair
Cappabianca et al. Metabolic priming of GD2 TRAC-CAR T cells during manufacturing promotes memory phenotypes while enhancing persistence
AU2024204373A1 (en) Improved methods of genetically modifying animal cells
CN102321587B (zh) 肺癌药物筛选细胞株的建立
MX2023007315A (es) Método para producir una partícula de virus adenoasociado recombinante.
CN118660958A (zh) 适应无血清悬浮培养的hek293细胞株及其应用
Yang et al. Optimized two‐step electroporation process to achieve efficient nonviral‐mediated gene insertion into primary T cells
Vargas et al. Spatial distribution of bacterial communities on volumetric and planar anodes in single‐chamber air‐cathode microbial fuel cells
Porreca et al. An aptamer-mediated base editing platform for simultaneous knockin and multiple gene knockout for allogeneic CAR-T cells generation
Merten et al. Viral vectors for gene therapy
Audouze-Chaud et al. Efficient and stable CRISPR/Cas9-mediated genome-editing of human type 2 innate lymphoid cells
CN111996598A (zh) 一种单细胞染色质可及性的建库方法
CN113881671A (zh) 增强nk细胞杀伤力的基因靶标及应用
Ribeiro et al. A quantitative method to evaluate mesenchymal stem cell lipofection using real‐time PCR
US20220249625A1 (en) Compositions comprising an endonuclease and methods for purifying an endonuclease
CN116162659B (zh) 一种原代mdsc的体外基因表达干预方法
CN111808880A (zh) 一种电转染缓冲液及其应用
Mamia et al. T cell correction pipeline for Inborn Errors of Immunity
WO2024086595A3 (en) Systems and methods for gene therapy
US20240336937A1 (en) Method for cell transduction and microfluidic chip for cell transduction
CN118910070A (zh) 一种基因修饰的内源基因及其应用