MX2020004830A - Medios y metodo para preparar vectores virales y usos de los mismos. - Google Patents

Medios y metodo para preparar vectores virales y usos de los mismos.

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Publication number
MX2020004830A
MX2020004830A MX2020004830A MX2020004830A MX2020004830A MX 2020004830 A MX2020004830 A MX 2020004830A MX 2020004830 A MX2020004830 A MX 2020004830A MX 2020004830 A MX2020004830 A MX 2020004830A MX 2020004830 A MX2020004830 A MX 2020004830A
Authority
MX
Mexico
Prior art keywords
same
viral vectors
preparing viral
preparing
compositions
Prior art date
Application number
MX2020004830A
Other languages
English (en)
Inventor
Robert Emil Hodge
Brian K Kaspar
James Michael Hatfield
Joseph Balleydier
Allan Arman Kaspar
Original Assignee
Avexis 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 Avexis Inc filed Critical Avexis Inc
Publication of MX2020004830A publication Critical patent/MX2020004830A/es

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    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the administration regime
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • 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
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    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)
    • 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
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/525—Virus
    • A61K2039/5256—Virus expressing foreign proteins
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    • 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
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material

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  • Virology (AREA)
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Abstract

Se proporcionan métodos para preparar y purificar partículas virales, y composiciones y usos que comprenden las mismas.
MX2020004830A 2017-11-08 2018-11-01 Medios y metodo para preparar vectores virales y usos de los mismos. MX2020004830A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762583035P 2017-11-08 2017-11-08
PCT/US2018/058744 WO2019094253A1 (en) 2017-11-08 2018-11-01 Means and method for preparing viral vectors and uses of same

Publications (1)

Publication Number Publication Date
MX2020004830A true MX2020004830A (es) 2020-11-11

Family

ID=64362706

Family Applications (3)

Application Number Title Priority Date Filing Date
MX2020004830A MX2020004830A (es) 2017-11-08 2018-11-01 Medios y metodo para preparar vectores virales y usos de los mismos.
MX2025001503A MX2025001503A (es) 2017-11-08 2020-07-13 Medios y metodo para preparar vectores virales y usos de los mismos
MX2025009360A MX2025009360A (es) 2017-11-08 2020-07-13 Medios y metodo para preparar vectores virales y usos de los mismos

Family Applications After (2)

Application Number Title Priority Date Filing Date
MX2025001503A MX2025001503A (es) 2017-11-08 2020-07-13 Medios y metodo para preparar vectores virales y usos de los mismos
MX2025009360A MX2025009360A (es) 2017-11-08 2020-07-13 Medios y metodo para preparar vectores virales y usos de los mismos

Country Status (17)

Country Link
US (2) US12168777B2 (es)
EP (2) EP4585229A3 (es)
JP (2) JP7587422B2 (es)
KR (2) KR20260052185A (es)
CN (4) CN119033965A (es)
AU (3) AU2018365677C1 (es)
BR (1) BR112019013268A2 (es)
CA (2) CA3309109A1 (es)
CL (3) CL2020001193A1 (es)
CO (1) CO2020006900A2 (es)
IL (2) IL274430B2 (es)
MX (3) MX2020004830A (es)
MY (1) MY208779A (es)
SA (1) SA520411930B1 (es)
SG (1) SG11202004406VA (es)
TW (1) TWI827560B (es)
WO (1) WO2019094253A1 (es)

Families Citing this family (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11219696B2 (en) * 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
US20200164008A1 (en) * 2017-07-17 2020-05-28 Spark Therapeutics, Inc. Apheresis methods and uses
EP4585229A3 (en) 2017-11-08 2026-01-14 Novartis AG Means and method for preparing viral vectors and uses of same
JP7478675B2 (ja) * 2018-06-08 2024-05-07 ノバルティス アーゲー 薬物製品の効力を測定するための細胞ベースアッセイ
JP2021534154A (ja) * 2018-08-15 2021-12-09 バイオジェン・エムエイ・インコーポレイテッドBiogen MA Inc. 脊髄性筋萎縮症のための併用療法
EP3886919A1 (en) * 2018-11-30 2021-10-06 Novartis AG Aav viral vectors and uses thereof
HRP20251506T1 (hr) * 2019-04-10 2026-01-02 Prevail Therapeutics, Inc. Genska terapija za lizosomske poremećaje
US10801042B1 (en) * 2019-07-15 2020-10-13 Vigene Biosciences, Inc. Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
CA3147875A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
CN114174514A (zh) 2019-07-25 2022-03-11 诺华股份有限公司 可调节的表达系统
WO2021026461A2 (en) * 2019-08-08 2021-02-11 Biogen Ma Inc. Potency assays for viral vector production
WO2021087282A1 (en) * 2019-10-30 2021-05-06 Amicus Therapeutics, Inc. Recombinant cdkl5 proteins, gene therapy and production methods
EP4087949A1 (en) * 2020-01-28 2022-11-16 Lonza Walkersville, Inc. Methods of determining viral titer
EP4136231A1 (en) 2020-04-17 2023-02-22 Friedrich Miescher Institute for Biomedical Research Use of an orphan motif to increase expression of a heterologous transgene
RU2742837C1 (ru) * 2020-06-02 2021-02-11 Общество С Ограниченной Ответственностью "Анабион" Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение
EP4175969A4 (en) * 2020-07-01 2024-07-24 Medimmune, LLC DETERGENT AND METHOD FOR PURIFYING A BIOTHERAPY AGENT
CN112121179A (zh) * 2020-09-15 2020-12-25 山东兴瑞生物科技有限公司 一种组合物及其在治疗脊髓性肌萎缩症中的应用
JP2023545830A (ja) * 2020-10-15 2023-10-31 プリベイル セラピューティクス，インコーポレーテッド 組換えアデノ随伴ウイルス組成物及びその生産方法
CN112251440A (zh) * 2020-10-29 2021-01-22 大连理工大学 一种CRISPR/Cas9基因载体、其制备方法及应用
CN116529381A (zh) 2020-12-03 2023-08-01 弗里德里克·米谢尔生物医学研究所 利用孤儿基序和CpG密度的组合来控制异源转基因的表达
EP4324907A4 (en) * 2021-04-15 2025-06-18 The University of Tokyo METHOD FOR PRODUCING VIRUS PARTICLES
US20240254483A1 (en) 2021-07-30 2024-08-01 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
AU2022318664A1 (en) 2021-07-30 2024-02-29 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
JP2024532700A (ja) * 2021-08-04 2024-09-10 武田薬品工業株式会社 カチオン交換体によるアデノ随伴ウイルス分離
JP2024534410A (ja) 2021-09-16 2024-09-20 ノバルティス アーゲー 新規転写因子
CN118119710A (zh) * 2021-10-15 2024-05-31 上海天泽云泰生物医药有限公司 用于治疗脊髓性肌萎缩的重组腺相关病毒载体
JP2024539123A (ja) 2021-10-25 2024-10-28 ノバルティス アーゲー アデノ随伴ウイルス（ａａｖ）送達を改善する方法
WO2023085382A1 (ja) * 2021-11-12 2023-05-19 Ａｇｃ株式会社 ウイルスの精製方法
US20250001013A1 (en) 2021-11-24 2025-01-02 Novartis Ag Modulators of adeno-associated virus transduction and uses thereof
CA3255627A1 (en) 2022-05-06 2023-11-09 Novartis Ag NEW VP2 FUSION POLYPEPTIDES OF RECOMBINANT AAV
EP4554967A2 (en) 2022-07-12 2025-05-21 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation
CN114990162A (zh) * 2022-07-18 2022-09-02 苏州吉纳星辰生物技术有限公司 一种降低aav病毒载体生产中质粒残留的方法
WO2024027632A1 (en) * 2022-08-01 2024-02-08 Skyline Therapeutics (Shanghai) Co., Ltd. Novel plasmid backbone to reduce dna impurities in raav preparation
CN115354071A (zh) * 2022-08-10 2022-11-18 上海药明生基医药科技有限公司 一种检测重组腺相关病毒宿主细胞dna残留的方法
CN116350801A (zh) 2022-11-22 2023-06-30 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024194280A1 (en) 2023-03-21 2024-09-26 F. Hoffmann-La Roche Ag Method for the production of recombinant aav particle preparations
WO2024252182A1 (en) * 2023-06-08 2024-12-12 Meiragtx Uk Ii Limited Cation exchange chromatography for aav capture
WO2025157137A1 (zh) * 2024-01-25 2025-07-31 鼐济医药科技(杭州)有限公司 Aav载体、载体组合物、嵌合载体和治疗或预防sma的方法
AU2025218748A1 (en) 2024-02-09 2026-08-06 F. Hoffmann-La Roche Ag Method for producing recombinant adeno-associated viral particles
WO2025188316A1 (en) * 2024-03-08 2025-09-12 Jaguar Gene Therapy, Llc Aav pharmaceutical formulations and methods thereof
WO2025190867A1 (en) * 2024-03-11 2025-09-18 Sartorius Stedim Biotech Gmbh Two-step intensified cell lysis to release a viral vector
WO2025217224A1 (en) * 2024-04-08 2025-10-16 Advanced Medicine Partners, LLC Method of manufacture of aav particles
KR102915956B1 (ko) * 2024-04-17 2026-01-20 한국로봇융합연구원 3차원 오비탈 용접로봇
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
WO2026028089A1 (en) 2024-07-29 2026-02-05 Novartis Ag Compositions and methods for the treatment of lysosomal acid lipase deficiency (lal-d)
WO2026037757A1 (en) 2024-08-13 2026-02-19 F. Hoffmann-La Roche Ag Modified aav particles
WO2026094009A1 (en) 2024-11-04 2026-05-07 Insmed Incorporated Compositions and methods for treating duchenne muscular dystrophy
WO2026132012A1 (en) 2024-12-20 2026-06-25 F. Hoffmann-La Roche Ag Aav particle processing using affinity chromatography with gradient elution
WO2026132139A1 (en) 2024-12-20 2026-06-25 F. Hoffmann-La Roche Ag Purification process for viral vectors
WO2026150302A1 (en) 2025-01-09 2026-07-16 Novartis Ag Novel aav rep/cap encoding constructs

Family Cites Families (59)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173418A (en) 1985-05-10 1992-12-22 Benzon Pharma, A/S Production in Escherichia coli of extracellular Serratia spp. hydrolases
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
DE69433922T2 (de) 1993-11-09 2005-07-28 Medical College Of Ohio, Toledo Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
EP0733103B1 (en) 1993-11-09 2004-03-03 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
WO1996017947A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびａａｖの精製
JPH11514853A (ja) 1995-09-08 1999-12-21 ジエンザイム コーポレイション 遺伝子治療のための改良されたａａｖベクター
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
EP1696036B1 (en) 1996-09-06 2010-04-21 The Trustees of The University of Pennsylvania Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells
US6756207B1 (en) 1997-02-27 2004-06-29 Cellomics, Inc. System for cell-based screening
CA2995542A1 (en) 1997-09-05 1999-03-11 Genzyme Corporation Methods for generating high titer helper-free preparations of recombinant aav vectors
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP2003518380A (ja) * 1999-12-29 2003-06-10 ジェンザイム・コーポレイション 核酸および他の細胞性汚染混入物質からウイルスベクターを分離するための濾過助剤の使用方法
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
EP1463805B1 (en) 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
JP4559429B2 (ja) 2003-05-21 2010-10-06 ジェンザイム・コーポレーション 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP1682075A4 (en) 2003-11-07 2009-12-09 Univ Florida BREEDING AND DIFFERENTIATION OF NEURAL CREATOR CELLS
ES2932278T3 (es) 2004-06-01 2023-01-17 Genzyme Corp Composiciones y métodos para prevenir la agregación del vector AAV
PT2548560E (pt) 2005-06-23 2015-09-22 Isis Pharmaceuticals Inc Composições e métodos de modulação da excisão-união de smn2
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
CA2729121C (en) 2008-06-30 2019-04-09 Centocor Ortho Biotech Inc. Differentiation of pluripotent stem cells
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
WO2010071832A1 (en) 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
US11219696B2 (en) * 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
SI3988660T1 (sl) 2009-05-02 2025-09-30 Genzyme Corporation Genska terapija za nevrodegenerativne motnje
KR20220084437A (ko) 2009-06-17 2022-06-21 바이오젠 엠에이 인코포레이티드 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법
RU2664864C1 (ru) 2009-12-23 2018-08-23 Янссен Байотек, Инк. Способы увеличения экспрессии ngn3 и nkx6.1 в эндокринных клетках поджелудочной железы
EP2529020B1 (en) * 2010-01-28 2018-04-25 The Children's Hospital of Philadelphia A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
RU2015100900A (ru) 2012-06-14 2016-08-10 Янссен Байотек, Инк. Обработка плюрипотентных клеток
CN104704123A (zh) * 2012-06-21 2015-06-10 肌肉学研究协会 基因治疗载体的广泛的基因递送
CA2880653C (en) 2012-08-01 2022-05-17 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
BR112015027336A2 (pt) 2013-05-01 2017-09-26 Genzyme Corp composições e processos para tratamento de atrofia muscular espinhal
KR102069561B1 (ko) 2013-07-11 2020-01-23 다카라 바이오 가부시키가이샤 외피 비보유 바이러스의 제조 방법
RU2664471C2 (ru) 2013-07-26 2018-08-17 Юниверсити Оф Айова Рисерч Фаундейшн Способы и композиции для лечения болезней мозга
RS63942B1 (sr) 2013-08-27 2023-02-28 Res Inst Nationwide Childrens Hospital Proizvodi i postupci za lečenje amiotrofične lateralne skleroze
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
WO2015141521A1 (ja) 2014-03-21 2015-09-24 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及び記録媒体
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
US10526583B2 (en) * 2014-07-02 2020-01-07 University Of Florida Research Foundation, Incorporated Compositions and methods for purifying recombinant adeno-associated virus
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
JP6754361B2 (ja) 2014-12-16 2020-09-09 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
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
EP3387138B2 (en) * 2015-12-11 2026-05-27 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
PH12018502104B1 (en) * 2016-03-31 2024-03-27 Spark Therapeutics Inc Column-based fully scalable raav manufacturing process
US11253612B2 (en) * 2016-04-15 2022-02-22 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type II
EP3299456A1 (en) 2016-09-26 2018-03-28 Université de Strasbourg Recombinant dgkk gene for fragile x syndrome gene therapy
IL322146A (en) 2016-11-17 2025-09-01 Nationwide Childrens Hospital Inc Intrathecal administration of adenovirus encoding methyl-CpG-binding protein 2
WO2019011817A1 (en) 2017-07-08 2019-01-17 Genethon TREATMENT OF SPINAL AMYOTROPHY
EP4585229A3 (en) 2017-11-08 2026-01-14 Novartis AG Means and method for preparing viral vectors and uses of same
JP7478675B2 (ja) 2018-06-08 2024-05-07 ノバルティス アーゲー 薬物製品の効力を測定するための細胞ベースアッセイ

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