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WO2024220447A3 - Engineered cell preparations for treatment of niemann pick b disease - Google Patents

Engineered cell preparations for treatment of niemann pick b disease Download PDF

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Publication number
WO2024220447A3
WO2024220447A3 PCT/US2024/024830 US2024024830W WO2024220447A3 WO 2024220447 A3 WO2024220447 A3 WO 2024220447A3 US 2024024830 W US2024024830 W US 2024024830W WO 2024220447 A3 WO2024220447 A3 WO 2024220447A3
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WIPO (PCT)
Prior art keywords
disease
treatment
engineered cell
cell preparations
niemann pick
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
PCT/US2024/024830
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French (fr)
Other versions
WO2024220447A2 (en
Inventor
Timothy James MULLEN
Ishara Datta
Hanlan Liu
Huy Anh TU
Anja Fides Hohmann
Claudia FIORINI
Sean Philip Leary ARLAUCKAS
Richard A. Morgan
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.)
Be Biopharma Inc
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Be Biopharma Inc
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Publication date
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Publication of WO2024220447A2 publication Critical patent/WO2024220447A2/en
Publication of WO2024220447A3 publication Critical patent/WO2024220447A3/en
Anticipated expiration legal-status Critical
Pending legal-status Critical Current

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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
    • C12N5/0635—B lymphocytes
    • 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
    • A61K40/13—B-cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/20—Cytokines; Chemokines
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    • C12N2501/20—Cytokines; Chemokines
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    • C12N2501/20—Cytokines; Chemokines
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
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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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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Zoology (AREA)
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  • Genetics & Genomics (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Cell Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

The present disclosure provides methods and compositions for culturing, engineering, and administering engineered B lineage cell populations that produce Sphingomyelin Phosphodiesterase 1 (SMPD1).
PCT/US2024/024830 2023-04-17 2024-04-16 Engineered cell preparations for treatment of niemann pick b disease Pending WO2024220447A2 (en)

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US202363459983P 2023-04-17 2023-04-17
US63/459,983 2023-04-17
US202363502636P 2023-05-16 2023-05-16
US63/502,636 2023-05-16
US202363592497P 2023-10-23 2023-10-23
US63/592,497 2023-10-23
US202463549943P 2024-02-05 2024-02-05
US63/549,943 2024-02-05
US202463553522P 2024-02-14 2024-02-14
US63/553,522 2024-02-14

Publications (2)

Publication Number Publication Date
WO2024220447A2 WO2024220447A2 (en) 2024-10-24
WO2024220447A3 true WO2024220447A3 (en) 2025-02-13

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ID=93153463

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/024830 Pending WO2024220447A2 (en) 2023-04-17 2024-04-16 Engineered cell preparations for treatment of niemann pick b disease

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Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190352614A1 (en) * 2017-01-26 2019-11-21 Sangamo Therapeutics, Inc. B-cell engineering
US20220193129A1 (en) * 2017-04-27 2022-06-23 Immusoft Corporation B cells for in vivo delivery of therapeutic agents and dosages thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190352614A1 (en) * 2017-01-26 2019-11-21 Sangamo Therapeutics, Inc. B-cell engineering
US20220193129A1 (en) * 2017-04-27 2022-06-23 Immusoft Corporation B cells for in vivo delivery of therapeutic agents and dosages thereof

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
ANONYMOUS: "Human Peripheral Blood CD19+ B Cells, Frozen ", STEMCELL TECHNOLOGIES, 5 October 2022 (2022-10-05), pages 1 - 5, XP093281151, Retrieved from the Internet <URL:https://www.stemcell.com/products/human-peripheral-blood-cd19-b-cells-frozen.html> *

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WO2024220447A2 (en) 2024-10-24

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