AU2020336230B2 - Methods and compositions for the modification and delivery of lymphocytes - Google Patents

Methods and compositions for the modification and delivery of lymphocytes

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Publication number
AU2020336230B2
AU2020336230B2 AU2020336230A AU2020336230A AU2020336230B2 AU 2020336230 B2 AU2020336230 B2 AU 2020336230B2 AU 2020336230 A AU2020336230 A AU 2020336230A AU 2020336230 A AU2020336230 A AU 2020336230A AU 2020336230 B2 AU2020336230 B2 AU 2020336230B2
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Australia
Prior art keywords
cells
cell
lymphocytes
modified
cell formulation
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AU2020336230A
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AU2020336230A1 (en
Inventor
Gregory Ian Frost
Farzad Haerizadeh
Anirban Kundu
James Joseph Onuffer Jr.
Frederic Vigant
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Exuma Biotech Corp
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Exuma Biotech Corp
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Priority claimed from PCT/US2019/049259 external-priority patent/WO2020047527A2/en
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Publication of AU2020336230A1 publication Critical patent/AU2020336230A1/en
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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
    • C12N15/79—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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/39—Medicinal preparations containing antigens or antibodies characterised by the immunostimulating additives, e.g. chemical adjuvants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
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    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K40/15—Natural-killer [NK] cells; Natural-killer T [NKT] cells
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    • A61K40/41—Vertebrate antigens
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    • A—HUMAN NECESSITIES
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    • A61K40/00—Cellular immunotherapy
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0636—T lymphocytes
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
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    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0646—Natural killers cells [NK], NKT cells
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/80—Vaccine for a specifically defined cancer
    • A61K2039/804—Blood cells [leukemia, lymphoma]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2121/00—Preparations for use in therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/31—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterized by the route of administration
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/38—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the dose, timing or administration schedule
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
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    • C12N2510/00—Genetically modified cells
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    • C12N2740/10011—Retroviridae
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    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01035—Hyaluronoglucosaminidase (3.2.1.35), i.e. hyaluronidase

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AU2020336230A 2019-09-01 2020-08-31 Methods and compositions for the modification and delivery of lymphocytes Active AU2020336230B2 (en)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US201962894849P 2019-09-01 2019-09-01
US201962894853P 2019-09-01 2019-09-01
US201962894852P 2019-09-01 2019-09-01
US62/894,852 2019-09-01
US62/894,849 2019-09-01
US62/894,853 2019-09-01
US201962894926P 2019-09-02 2019-09-02
PCT/US2019/049259 WO2020047527A2 (en) 2018-09-02 2019-09-02 Methods and compositions for genetically modifying lymphocytes in blood or in enriched pbmcs
US62/894,926 2019-09-02
AUPCT/US2019/049259 2019-09-02
US201962943207P 2019-12-03 2019-12-03
US62/943,207 2019-12-03
US202062985741P 2020-03-05 2020-03-05
US62/985,741 2020-03-05
PCT/US2020/048843 WO2021042072A1 (en) 2019-09-01 2020-08-31 Methods and compositions for the modification and delivery of lymphocytes

Publications (2)

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AU2020336230A1 AU2020336230A1 (en) 2022-03-31
AU2020336230B2 true AU2020336230B2 (en) 2026-04-16

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US (1) US20220340927A1 (https=)
EP (1) EP4022035A4 (https=)
JP (2) JP2022546101A (https=)
KR (1) KR20220070449A (https=)
CN (1) CN116057181A (https=)
AU (1) AU2020336230B2 (https=)
BR (1) BR112022003617A2 (https=)
CA (1) CA3152780A1 (https=)
MX (1) MX2022002521A (https=)
WO (1) WO2021042072A1 (https=)

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US11111505B2 (en) 2016-03-19 2021-09-07 Exuma Biotech, Corp. Methods and compositions for transducing lymphocytes and regulating the activity thereof
US11325948B2 (en) 2016-03-19 2022-05-10 Exuma Biotech Corp. Methods and compositions for genetically modifying lymphocytes to express polypeptides comprising the intracellular domain of MPL
US12590321B2 (en) 2016-03-19 2026-03-31 Exuma Biotech Corp. Methods and compositions for genetically modifying and expanding lymphocytes and regulating the activity thereof
CN113840911A (zh) * 2018-09-02 2021-12-24 埃克苏马生物技术公司 用于在血液中或在富集pbmc中基因修饰淋巴细胞的方法和组合物
WO2021231569A1 (en) * 2020-05-14 2021-11-18 Seattle Children's Hospital (dba Seattle Children's Research Institute) Dual promoter systems
WO2023015310A2 (en) * 2021-08-06 2023-02-09 Seattle Children's Hospital D/B/A Seattle Children's Research Institute T-cell manufacturing methods
EP4463135A2 (en) 2022-01-10 2024-11-20 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
EP4469586A1 (en) * 2022-03-01 2024-12-04 Exuma Biotech Corp. Viral particles with membrane-bound hyaluronidase
WO2024026377A1 (en) 2022-07-27 2024-02-01 Sana Biotechnology, Inc. Methods of transduction using a viral vector and inhibitors of antiviral restriction factors
IL320156A (en) * 2022-10-10 2025-06-01 Kite Pharma Inc New large scale car-t immune cell manufacturing method utilizing lentiviral vector transfection
IL326194A (en) * 2023-07-31 2026-03-01 Bio4T2 Llc Methods for the expansion and in vivo uptake of CAR cells mediated by blood cells
EP4589007A1 (en) * 2024-01-17 2025-07-23 Revvity Gene Delivery GmbH Production of viral vectors
WO2025213071A1 (en) * 2024-04-05 2025-10-09 Baylor College Of Medicine Synthetic gene expression regulatory switches

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