AU2021297965A1 - Methods for the removal of free factor VIII from preparations of lentiviral vectors modified to express said protein - Google Patents

Methods for the removal of free factor VIII from preparations of lentiviral vectors modified to express said protein Download PDF

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Publication number
AU2021297965A1
AU2021297965A1 AU2021297965A AU2021297965A AU2021297965A1 AU 2021297965 A1 AU2021297965 A1 AU 2021297965A1 AU 2021297965 A AU2021297965 A AU 2021297965A AU 2021297965 A AU2021297965 A AU 2021297965A AU 2021297965 A1 AU2021297965 A1 AU 2021297965A1
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Prior art keywords
viral vector
transgene protein
transgene
chromatography
composition
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Pending
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AU2021297965A
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English (en)
Inventor
Ayman Ismail
Tongyao Liu
Mukesh MAYANI
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Fondazione Telethon
Ospedale San Raffaele SRL
Bioverativ Therapeutics Inc
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Fondazione Telethon
Ospedale San Raffaele SRL
Bioverativ Therapeutics Inc
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Publication of AU2021297965A1 publication Critical patent/AU2021297965A1/en
Assigned to OSPEDALE SAN RAFFAELE S.R.L., BIOVERATIV THERAPEUTICS INC., FONDAZIONE TELETHON ETS reassignment OSPEDALE SAN RAFFAELE S.R.L. Amend patent request/document other than specification (104) Assignors: BIOVERATIV THERAPEUTICS INC., FONDAZIONE TELETHON, OSPEDALE SAN RAFFAELE S.R.L.
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    • 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
    • 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
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/10—Selective adsorption, e.g. chromatography characterised by constructional or operational features
    • B01D15/18—Selective adsorption, e.g. chromatography characterised by constructional or operational features relating to flow patterns
    • B01D15/1864—Selective adsorption, e.g. chromatography characterised by constructional or operational features relating to flow patterns using two or more columns
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/26—Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/36—Selective adsorption, e.g. chromatography characterised by the separation mechanism involving ionic interaction, e.g. ion-exchange, ion-pair, ion-suppression or ion-exclusion
    • B01D15/361—Ion-exchange
    • B01D15/363—Anion-exchange
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/26—Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/38—Selective adsorption, e.g. chromatography characterised by the separation mechanism involving specific interaction not covered by one or more of groups B01D15/265 and B01D15/30 - B01D15/36, e.g. affinity, ligand exchange or chiral chromatography
    • B01D15/3804—Affinity chromatography
    • 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
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15051—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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16051—Methods of production or purification of viral material

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Analytical Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Manufacturing & Machinery (AREA)
  • Animal Behavior & Ethology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2021297965A 2020-06-24 2021-06-24 Methods for the removal of free factor VIII from preparations of lentiviral vectors modified to express said protein Pending AU2021297965A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063043697P 2020-06-24 2020-06-24
US63/043,697 2020-06-24
US202063062120P 2020-08-06 2020-08-06
US63/062,120 2020-08-06
PCT/US2021/038871 WO2021262963A1 (en) 2020-06-24 2021-06-24 Methods for the removal of free factor viii from preparations of lentiviral vectors modified to express said protein

Publications (1)

Publication Number Publication Date
AU2021297965A1 true AU2021297965A1 (en) 2023-02-23

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AU2021297965A Pending AU2021297965A1 (en) 2020-06-24 2021-06-24 Methods for the removal of free factor VIII from preparations of lentiviral vectors modified to express said protein

Country Status (12)

Country Link
US (2) US12173307B2 (https=)
EP (1) EP4171657A1 (https=)
JP (1) JP2023537565A (https=)
KR (1) KR20230074703A (https=)
CN (1) CN116033927A (https=)
AU (1) AU2021297965A1 (https=)
BR (1) BR112022026127A2 (https=)
CA (1) CA3183557A1 (https=)
IL (1) IL299333A (https=)
MX (1) MX2023000156A (https=)
TW (1) TW202208619A (https=)
WO (1) WO2021262963A1 (https=)

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US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
CA3012695A1 (en) 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Optimized factor viii genes
KR20220097891A (ko) 2019-09-30 2022-07-08 바이오버라티브 테라퓨틱스 인크. 렌티바이러스 벡터 제형
MX2023000156A (es) 2020-06-24 2023-02-16 Bioverativ Therapeutics Inc Metodos para la eliminacion de factor viii libre de preparaciones de vectores lentivirales modificados para expresar dicha proteina.
GB202117844D0 (en) * 2021-12-09 2022-01-26 Oxford Biomedica Ltd Purification method
CN119256087A (zh) * 2022-03-14 2025-01-03 布拉默生物有限责任公司 慢病毒制造平台
US20230331773A1 (en) * 2022-04-15 2023-10-19 Pall Corporation Chromatography device and method of use
CN115505578A (zh) * 2022-09-20 2022-12-23 苏州博腾生物制药有限公司 可放大的用于纯化重组病毒的方法
WO2025106701A1 (en) * 2023-11-15 2025-05-22 Tectaria Bio Llc Compositions and methods for increased protein production

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