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CL2025001440A1 - Medios y método para preparar vectores virales y usos de los mismos - Google Patents

Medios y método para preparar vectores virales y usos de los mismos

Info

Publication number
CL2025001440A1
CL2025001440A1 CL2025001440A CL2025001440A CL2025001440A1 CL 2025001440 A1 CL2025001440 A1 CL 2025001440A1 CL 2025001440 A CL2025001440 A CL 2025001440A CL 2025001440 A CL2025001440 A CL 2025001440A CL 2025001440 A1 CL2025001440 A1 CL 2025001440A1
Authority
CL
Chile
Prior art keywords
viral vectors
preparing viral
preparing
compositions
methods
Prior art date
Application number
CL2025001440A
Other languages
English (en)
Inventor
k kaspar Brian
Michael Hatfield James
Balleydier Joseph
Arman Kaspar Allan
Emil Hodge Robert
Original Assignee
Novartis Gene Therapies 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 Novartis Gene Therapies Inc filed Critical Novartis Gene Therapies Inc
Publication of CL2025001440A1 publication Critical patent/CL2025001440A1/es

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    • 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
    • A61K48/0091—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
    • 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
    • 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
    • C12N15/11—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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • 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
    • 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
    • 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)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Manufacturing & Machinery (AREA)

Abstract

Se proporcionan métodos para preparar y purificar partículas virales, y composiciones y usos que comprenden las mismas.
CL2025001440A 2017-11-08 2025-05-15 Medios y método para preparar vectores virales y usos de los mismos CL2025001440A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762583035P 2017-11-08 2017-11-08

Publications (1)

Publication Number Publication Date
CL2025001440A1 true CL2025001440A1 (es) 2025-09-05

Family

ID=64362706

Family Applications (3)

Application Number Title Priority Date Filing Date
CL2020001193A CL2020001193A1 (es) 2017-11-08 2020-05-06 Medios y método para preparar vectores virales y usos de los mismos.
CL2024004045A CL2024004045A1 (es) 2017-11-08 2024-12-26 Medios y método para preparar vectores virales y usos de los mismos
CL2025001440A CL2025001440A1 (es) 2017-11-08 2025-05-15 Medios y método para preparar vectores virales y usos de los mismos

Family Applications Before (2)

Application Number Title Priority Date Filing Date
CL2020001193A CL2020001193A1 (es) 2017-11-08 2020-05-06 Medios y método para preparar vectores virales y usos de los mismos.
CL2024004045A CL2024004045A1 (es) 2017-11-08 2024-12-26 Medios y método para preparar vectores virales y usos de los mismos

Country Status (17)

Country Link
US (2) US12168777B2 (es)
EP (2) EP3707264A1 (es)
JP (2) JP7587422B2 (es)
KR (1) KR20200086292A (es)
CN (4) CN111566220A (es)
AU (2) AU2018365677C1 (es)
BR (1) BR112019013268A2 (es)
CA (1) CA3082136A1 (es)
CL (3) CL2020001193A1 (es)
CO (1) CO2020006900A2 (es)
IL (2) IL314692A (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 (47)

* 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
WO2019018439A1 (en) * 2017-07-17 2019-01-24 Spark Therapeutics, Inc. APHERESE METHODS AND ASSOCIATED USES
CN111566220A (zh) 2017-11-08 2020-08-21 阿维克斯公司 制备病毒载体的手段和方法及其用途
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
JP7677887B2 (ja) * 2018-11-30 2025-05-15 ノバルティス アーゲー Ａａｖウイルスベクター及びその使用
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
WO2021016075A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
CA3147574A1 (en) 2019-07-25 2021-01-28 Novartis Ag Regulatable expression systems
JP2022543656A (ja) * 2019-08-08 2022-10-13 バイオジェン・エムエイ・インコーポレイテッド ウイルスベクター産生のための効力アッセイ
TW202136514A (zh) * 2019-10-30 2021-10-01 美商阿米庫斯醫療股份有限公司 重組ｃｄｋｌ５蛋白、基因療法及生產方法
KR20220133908A (ko) * 2020-01-28 2022-10-05 론자 워커스빌 아이엔씨. 바이러스 역가를 결정하는 방법
US20230220402A1 (en) 2020-04-17 2023-07-13 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
EP4229206A2 (en) * 2020-10-15 2023-08-23 Prevail Therapeutics, Inc. Recombinant adeno-associated virus compositions and methods for producing same
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)
US20240252684A1 (en) 2021-07-30 2024-08-01 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
CA3226803A1 (en) * 2021-08-04 2023-02-09 Christian Fiedler Adeno-associated virus separation on a cation exchanger
JP2024534410A (ja) 2021-09-16 2024-09-20 ノバルティス アーゲー 新規転写因子
WO2023061499A1 (en) * 2021-10-15 2023-04-20 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating spinal muscular atrophy
WO2023073526A1 (en) 2021-10-25 2023-05-04 Novartis Ag Methods for improving adeno-associated virus (aav) delivery
CN118632860A (zh) 2021-11-24 2024-09-10 诺华股份有限公司 腺相关病毒转导调节剂及其用途
CA3255627A1 (en) 2022-05-06 2023-11-09 Novartis Ag NEW VP2 FUSION POLYPEPTIDES OF RECOMBINANT AAV
CA3261865A1 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS
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
CN115554418B (zh) * 2022-11-22 2023-04-14 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
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)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
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
WO2025168663A1 (en) 2024-02-09 2025-08-14 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
WO2026022064A1 (en) 2024-07-22 2026-01-29 F. Hoffmann-La Roche Ag 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)

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
JPH09509564A (ja) 1993-11-09 1997-09-30 ターゲテッド ジェネティックス コーポレイション 高力価組換えａａｖベクターの生成
WO1995013392A1 (en) 1993-11-09 1995-05-18 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
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
CA2230758A1 (en) 1995-09-08 1997-03-13 Genzyme Corporation Improved aav vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
ATE465267T1 (de) 1996-09-06 2010-05-15 Univ Pennsylvania Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen
US6756207B1 (en) 1997-02-27 2004-06-29 Cellomics, Inc. System for cell-based screening
ES2399232T3 (es) 1997-09-05 2013-03-26 Genzyme Corporation Procedimientos para la generación de preparaciones de vectores de AAV recombinantes de títulos altos exentas de auxiliares
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
US20010043916A1 (en) * 1999-12-29 2001-11-22 Mcneilly David S. Method using filtration aids for the separation of virus vectors from nucleic acids and other cellular contaminants
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
ES2526341T3 (es) 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
PL1625210T3 (pl) * 2003-05-21 2011-05-31 Genzyme Corp Sposoby wytwarzania preparatów rekombinowanych wirionów AAV zasadniczo niezawierających pustych kapsydów
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
JP2007522796A (ja) 2003-11-07 2007-08-16 ユニヴァーシティ オブ フロリダ リサーチ ファウンデーション，インク． 神経前駆細胞の培養と分化
DK1751275T3 (da) 2004-06-01 2017-11-27 Avigen Inc Sammensætninger og fremgangsmåder til forebyggelse af aggregering af aav-vektorer
PL2548560T3 (pl) 2005-06-23 2015-11-30 Biogen Ma Inc Kompozycje i sposoby modulowania splicingu SMN2
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
AU2009267137A1 (en) 2008-06-30 2010-01-07 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
SG10202109219SA (en) 2009-05-02 2021-10-28 Genzyme Corp Gene therapy for neurodegenerative disorders
KR101965868B1 (ko) 2009-06-17 2019-04-05 바이오젠 엠에이 인코포레이티드 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법
CN102712902B (zh) 2009-12-23 2019-01-08 詹森生物科技公司 人胚胎干细胞的分化
JP2013517798A (ja) * 2010-01-28 2013-05-20 ザ チルドレンズ ホスピタル オブ フィラデルフィア ウイルスベクター精製における拡張可能な製造プラットフォームおよび遺伝子治療における使用のための高純度ウイルスベクター
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
CA2876671A1 (en) 2012-06-14 2013-12-27 Janssen Biotech, Inc. Treatment of pluripotent cells
EP2864488A1 (en) * 2012-06-21 2015-04-29 Association Institut de Myologie Widespread gene delivery of gene therapy vectors
BR112015002168A2 (pt) 2012-08-01 2017-11-07 Nationwide Childrens Hospital liberação intratecal de vírus 9 adeno-associado recombinante
AU2013388083B2 (en) 2013-05-01 2019-08-22 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
US10072250B2 (en) 2013-07-11 2018-09-11 Takara Bio Inc. Method for manufacturing non-enveloped virus
CN105764532B (zh) 2013-07-26 2021-08-31 衣阿华大学研究基金会 治疗脑疾病的方法和组合物
PL3702466T3 (pl) 2013-08-27 2023-04-24 Research Institute At Nationwide Children's Hospital Produkty i sposoby do leczenia stwardnienia zanikowego bocznego
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
JP6202701B2 (ja) 2014-03-21 2017-09-27 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プログラム
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
WO2016004319A1 (en) * 2014-07-02 2016-01-07 University Of Florida Research Foundation, Inc. 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
CA2967468A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
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
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
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
RU2754467C2 (ru) 2016-03-31 2021-09-02 Спарк Терапьютикс, Инк. ПОЛНОСТЬЮ МАСШТАБИРУЕМЫЙ СПОСОБ ПРОИЗВОДСТВА rAAV НА ОСНОВЕ КОЛОНОК
BR112018071156A2 (pt) 2016-04-15 2019-03-12 The Trustees Of The University Of Pennsylvania terapia de gene para tratar mucopolissacaridose tipo ii
EP3299456A1 (en) 2016-09-26 2018-03-28 Université de Strasbourg Recombinant dgkk gene for fragile x syndrome gene therapy
MX2019005874A (es) 2016-11-17 2020-02-07 Nationwide Childrens Hospital Inc Administracion intratecal de virus recombinante adeno-asociado que codifica la proteina 2 que une metilo-cpg.
EP3652324A1 (en) 2017-07-08 2020-05-20 Genethon Treatment of spinal muscular atrophy
CN111566220A (zh) 2017-11-08 2020-08-21 阿维克斯公司 制备病毒载体的手段和方法及其用途
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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