SG11202004406VA - Means and method for preparing viral vectors and uses of same - Google Patents

Means and method for preparing viral vectors and uses of same

Info

Publication number
SG11202004406VA
SG11202004406VA SG11202004406VA SG11202004406VA SG11202004406VA SG 11202004406V A SG11202004406V A SG 11202004406VA SG 11202004406V A SG11202004406V A SG 11202004406VA SG 11202004406V A SG11202004406V A SG 11202004406VA SG 11202004406V A SG11202004406V A SG 11202004406VA
Authority
SG
Singapore
Prior art keywords
same
viral vectors
preparing viral
preparing
vectors
Prior art date
Application number
SG11202004406VA
Other languages
English (en)
Inventor
Brian K Kaspar
James Michael Hatfield
Joseph Balleydier
Allan Arman Kaspar
Robert Emil Hodge
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 SG11202004406VA publication Critical patent/SG11202004406VA/en

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
    • 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
    • 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
    • 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)
    • 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

Landscapes

  • 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)
SG11202004406VA 2017-11-08 2018-11-01 Means and method for preparing viral vectors and uses of same SG11202004406VA (en)

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
SG11202004406VA true SG11202004406VA (en) 2020-06-29

Family

ID=64362706

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202004406VA SG11202004406VA (en) 2017-11-08 2018-11-01 Means and method for preparing viral vectors and uses of same

Country Status (17)

Country Link
US (2) US12168777B2 (https=)
EP (2) EP4585229A3 (https=)
JP (2) JP7587422B2 (https=)
KR (1) KR20200086292A (https=)
CN (4) CN111566220A (https=)
AU (2) AU2018365677C1 (https=)
BR (1) BR112019013268A2 (https=)
CA (1) CA3082136A1 (https=)
CL (3) CL2020001193A1 (https=)
CO (1) CO2020006900A2 (https=)
IL (2) IL314692A (https=)
MX (3) MX2020004830A (https=)
MY (1) MY208779A (https=)
SA (1) SA520411930B1 (https=)
SG (1) SG11202004406VA (https=)
TW (1) TWI827560B (https=)
WO (1) WO2019094253A1 (https=)

Families Citing this family (49)

* 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
MX2020000713A (es) * 2017-07-17 2020-08-31 Spark Therapeutics Inc Metodos de aferesis y sus usos.
MX2020004830A (es) 2017-11-08 2020-11-11 Avexis Inc Medios y metodo para preparar vectores virales y usos de los mismos.
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
EP3837374A4 (en) * 2018-08-15 2022-06-08 Biogen MA Inc. COMBINATION THERAPY FOR SPINAL MUSCLE ATROPHY
WO2020113034A1 (en) * 2018-11-30 2020-06-04 Avexis, Inc. Aav viral vectors and uses thereof
PL3953377T3 (pl) * 2019-04-10 2026-02-16 Prevail Therapeutics, Inc. Terapie genowe zaburzeń lizosomalnych
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
WO2021014428A1 (en) 2019-07-25 2021-01-28 Novartis Ag Regulatable expression systems
CA3149825A1 (en) * 2019-08-08 2021-02-11 Biogen Ma Inc. Potency assays for viral vector production
CA3156506A1 (en) * 2019-10-30 2021-05-06 Amicus Therapeutics, Inc. Recombinant cdkl5 proteins, gene therapy and production methods
KR20220133908A (ko) * 2020-01-28 2022-10-05 론자 워커스빌 아이엔씨. 바이러스 역가를 결정하는 방법
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
CN115996940A (zh) * 2020-07-01 2023-04-21 免疫医疗有限责任公司 用于纯化生物治疗剂的洗涤剂和方法
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
KR20230088393A (ko) * 2020-10-15 2023-06-19 프리베일 테라퓨틱스, 인크. 재조합 아데노 연관 바이러스 조성물 및 이의 제조 방법
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
WO2022118237A1 (en) 2020-12-03 2022-06-09 Friedrich Miescher Institute For Biomedical Research Use of a combination of an orphan motif and cpg density to control expression of a heterologous transgene
EP4324907A4 (en) 2021-04-15 2025-06-18 The University of Tokyo METHOD FOR PRODUCING VIRUS PARTICLES
EP4377459A2 (en) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
WO2023010135A1 (en) 2021-07-30 2023-02-02 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
WO2023042104A1 (en) 2021-09-16 2023-03-23 Novartis Ag Novel transcription factors
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
US20250235554A1 (en) 2021-10-25 2025-07-24 Novartis Ag Methods for improving adeno-associated virus (aav) delivery
WO2023085382A1 (ja) * 2021-11-12 2023-05-19 Ａｇｃ株式会社 ウイルスの精製方法
EP4436982A1 (en) 2021-11-24 2024-10-02 Novartis AG Modulators of adeno-associated virus transduction and uses thereof
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
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
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)
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
WO2024252182A1 (en) * 2023-06-08 2024-12-12 Meiragtx Uk Ii Limited Cation exchange chromatography for aav capture
CN120677245A (zh) * 2024-01-25 2025-09-19 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
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

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
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
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
AU707866B2 (en) 1994-12-06 1999-07-22 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
AU722196B2 (en) 1995-08-30 2000-07-27 Genzyme Corporation Chromatographic purification of adenovirus and AAV
EP1983057A3 (en) 1995-09-08 2009-01-07 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
EP0932418B1 (en) 1996-09-06 2007-12-05 The Trustees Of The University Of Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
US6756207B1 (en) 1997-02-27 2004-06-29 Cellomics, Inc. System for cell-based screening
JP2001514845A (ja) 1997-09-05 2001-09-18 ターゲティッド ジェネティクス コーポレイション 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法
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
WO2001048155A2 (en) * 1999-12-29 2001-07-05 Genzyme Corporation 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
AU2002359284A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
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
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
US20050153446A1 (en) 2003-11-07 2005-07-14 Steindler Dennis A. Culturing and differentiating neural precursor cells
EP1751275B1 (en) * 2004-06-01 2017-08-16 Avigen, Inc. Compositions and methods to prevent aav vector aggregation
SI2548560T1 (sl) 2005-06-23 2015-12-31 Isis Pharmaceuticals, Inc. Sestavki in postopki za moduliranje izrezovanja smn2
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
PL2310492T3 (pl) 2008-06-30 2015-12-31 Janssen Biotech Inc Różnocowanie pluripotencjalnych komórek macierzystych
US11219696B2 (en) * 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides 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
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
PT3305302T (pt) 2009-06-17 2018-12-14 Biogen Ma Inc Composições e métodos de modulação de excisões de smn2 em um sujeito
CA2784425A1 (en) 2009-12-23 2011-06-30 Centocor Ortho Biotech Inc. Differentiation of human embryonic stem cells
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
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
EP2861723A4 (en) 2012-06-14 2016-01-20 Janssen Biotech Inc DIFFERENTIATION OF HUMAN EMBRYONAL STEM CELLS IN ENDOCRINE PANCREAS CELLS
WO2013190059A1 (en) 2012-06-21 2013-12-27 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
AU2013296425B2 (en) 2012-08-01 2018-06-07 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
MX380973B (es) 2013-05-01 2025-03-12 Genzyme Corp Composiciones y metodos para tratar la atrofia muscular espinal.
EP3020808B1 (en) 2013-07-11 2019-09-11 Takara Bio Inc. Method for manufacturing adeno-associated virus
EP3024497B1 (en) 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
LT3702466T (lt) 2013-08-27 2023-02-27 Research Institute At Nationwide Children's Hospital Produktai ir būdai, skirti šoninės amiotrofinės sklerozės gydymui
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
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
CN107249646B (zh) 2014-12-16 2021-06-29 内布拉斯加大学董事会 用于青少年巴滕病的基因疗法
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
BR112018070256A2 (pt) * 2016-03-31 2019-01-29 Spark Therapeutics, Inc. processo de fabricação de raav totalmente escalável à base de colunas
IL305449B2 (en) * 2016-04-15 2026-01-01 Univ Pennsylvania Gene therapy for the treatment of mucocutaneous type ii diabetes
EP3299456A1 (en) 2016-09-26 2018-03-28 Université de Strasbourg Recombinant dgkk gene for fragile x syndrome gene therapy
BR112019009834B1 (pt) 2016-11-17 2022-05-17 Ohio State Innovation Foundation Vetor viral adeno-associado recombinante (raav), vírus, composição farmacêutica e uso de um vetor raav
US20200370069A1 (en) 2017-07-08 2020-11-26 Genethon Treatment of spinal muscular atrophy
MX2020004830A (es) 2017-11-08 2020-11-11 Avexis Inc Medios y metodo para preparar vectores virales y usos de los mismos.
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

Also Published As

Publication number Publication date
AU2025203480B2 (en) 2026-04-09
US20210317474A1 (en) 2021-10-14
MX2025001503A (es) 2025-03-07
CN111566220A (zh) 2020-08-21
MX2025009360A (es) 2025-09-02
IL274430B1 (en) 2024-09-01
US12168777B2 (en) 2024-12-17
CL2020001193A1 (es) 2020-11-13
EP3707264A1 (en) 2020-09-16
BR112019013268A2 (pt) 2019-12-17
TW201930590A (zh) 2019-08-01
CA3082136A1 (en) 2019-05-16
AU2018365677B2 (en) 2025-04-03
CL2025001440A1 (es) 2025-09-05
MY208779A (en) 2025-05-28
TWI827560B (zh) 2024-01-01
AU2018365677A1 (en) 2020-05-14
CO2020006900A2 (es) 2020-06-19
CL2024004045A1 (es) 2025-08-01
RU2020118557A3 (https=) 2022-04-15
EP4585229A2 (en) 2025-07-16
AU2018365677C1 (en) 2025-09-11
EP4585229A3 (en) 2026-01-14
IL274430A (en) 2020-06-30
CN120505370A (zh) 2025-08-19
CN120501895A (zh) 2025-08-19
IL314692A (en) 2024-10-01
JP7587422B2 (ja) 2024-11-20
AU2025203480A1 (en) 2025-06-05
CN119033965A (zh) 2024-11-29
JP2021502123A (ja) 2021-01-28
SA520411930B1 (ar) 2025-04-07
MX2020004830A (es) 2020-11-11
JP2025017361A (ja) 2025-02-05
IL274430B2 (en) 2025-01-01
US20250066813A1 (en) 2025-02-27
WO2019094253A1 (en) 2019-05-16
KR20200086292A (ko) 2020-07-16
RU2020118557A (ru) 2021-12-08

Similar Documents

Publication Publication Date Title
IL274430A (en) Means and method for preparing viral vectors and their uses
ZA201900347B (en) Bispecific proteins and methods for preparing same
IL257628A (en) Methods and compositions for stealth virus antibody vectors
GB2545810B (en) Bagging station support frame and method of forming the same
IL245366A0 (en) A multi-input and multi-output radar system (mimo) and a method for using it
SI3433285T1 (sl) Izboljšan postopek za pripravo sugamadeksa
SI3380530T1 (sl) Izboljšan postopek za pripravo sugamadeksa in njegovih intermediatov
GB201710126D0 (en) Vectors & Methods
GB201519128D0 (en) Solid forms and methods of preparing the same
HUE073342T2 (hu) PRRSV minor fehérjét tartalmazó rekombináns vírusvektorok és eljárások elõállításukra és alkalmazásukra
ZA202003590B (en) Process for the preparation of opicapone and intermediates thereof
EP3238027A4 (en) Method and apparatus for variably expanding between mask and vector registers
GB201706945D0 (en) Self-inactivating viral vector
GB201807691D0 (en) Systems and methods for updating sequence of services
IL256067B (en) System of multiple bags and method for preparing blood components
SG10201600388WA (en) Method of processing single-crystal member
IL259096A (en) Peptides and methods of treating endometriosis using the same
EP3451161B8 (en) Apparatus and method for executing operations of maximum value and minimum value of vectors
IL272559A (en) A method for the preparation of 4-(heptafluoro-2-propyl) aniline
IL293192A (en) Methods for the preparation of indolinobenzodiazepine derivatives
PT3440208T (pt) Vetores para clonagem e expressão de proteínas, métodos e suas aplicações
GB201803172D0 (en) System and method for configuration of self-storage
IL267491B (en) A process for preparing temozolomide and a binder
ZA201805711B (en) Multigenome retroviral vector preparations and methods and systems for producing and using same
PL3608324T3 (pl) Sposób wytwarzania 2-arylomalonoamidu i zastosowania tego sposobu