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WO2019113375A3 - Dosing regimens for the mobilization of hematopoietic stem and progenitor cells - Google Patents

Dosing regimens for the mobilization of hematopoietic stem and progenitor cells Download PDF

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Publication number
WO2019113375A3
WO2019113375A3 PCT/US2018/064335 US2018064335W WO2019113375A3 WO 2019113375 A3 WO2019113375 A3 WO 2019113375A3 US 2018064335 W US2018064335 W US 2018064335W WO 2019113375 A3 WO2019113375 A3 WO 2019113375A3
Authority
WO
WIPO (PCT)
Prior art keywords
progenitor cells
hematopoietic stem
mobilization
cells
compositions
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.)
Ceased
Application number
PCT/US2018/064335
Other languages
French (fr)
Other versions
WO2019113375A2 (en
Inventor
Dwight Morrow
Patrick C. Falahee
Anthony Boitano
Michael P. Cooke
Kevin A. GONCALVES
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.)
Dianthus Therapeutics Inc
Original Assignee
Magenta Therapeutics 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
Priority claimed from US15/834,017 external-priority patent/US10058573B1/en
Priority to IL315735A priority Critical patent/IL315735A/en
Priority to BR112020011186-4A priority patent/BR112020011186A2/en
Priority to AU2018378804A priority patent/AU2018378804B2/en
Priority to CA3083783A priority patent/CA3083783A1/en
Priority to MX2020005878A priority patent/MX2020005878A/en
Priority to EA202091092A priority patent/EA202091092A1/en
Priority to CN201880088815.6A priority patent/CN111712262A/en
Priority to IL275077A priority patent/IL275077B2/en
Application filed by Magenta Therapeutics Inc filed Critical Magenta Therapeutics Inc
Priority to EP18829615.6A priority patent/EP3720494A2/en
Priority to KR1020207019222A priority patent/KR20200096942A/en
Priority to SG11202004913TA priority patent/SG11202004913TA/en
Priority to JP2020531468A priority patent/JP2021505172A/en
Priority to US16/352,578 priority patent/US11260079B2/en
Publication of WO2019113375A2 publication Critical patent/WO2019113375A2/en
Publication of WO2019113375A3 publication Critical patent/WO2019113375A3/en
Anticipated expiration legal-status Critical
Priority to CONC2020/0007275A priority patent/CO2020007275A2/en
Priority to JP2023188499A priority patent/JP2024023226A/en
Ceased legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19Cytokines; Lymphokines; Interferons
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • A61P35/02Antineoplastic agents specific for leukemia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0647Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/569Immunoassay; Biospecific binding assay; Materials therefor for microorganisms, e.g. protozoa, bacteria, viruses
    • G01N33/56966Animal cells
    • G01N33/56972White blood cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/50Cell markers; Cell surface determinants
    • C12N2501/599Cell markers; Cell surface determinants with CD designations not provided for elsewhere
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/435Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
    • G01N2333/52Assays involving cytokines
    • G01N2333/521Chemokines
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/435Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
    • G01N2333/705Assays involving receptors, cell surface antigens or cell surface determinants
    • G01N2333/70596Molecules with a "CD"-designation not provided for elsewhere in G01N2333/705
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Hematology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biomedical Technology (AREA)
  • Cell Biology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
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  • Diabetes (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Urology & Nephrology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Analytical Chemistry (AREA)
  • Transplantation (AREA)
  • General Physics & Mathematics (AREA)
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Abstract

The invention provides compositions and methods useful for mobilizing populations of hematopoietic stem and progenitor cells within a donor, as well as for determining whether samples of mobilized cells are suitable for release for ex vivo expansion and/or therapeutic use. In accordance with the compositions and methods described herein, mobilized hematopoietic stem and progenitor cells can be withdrawn from a donor and administered to a patient for the treatment of various stem cell disorders, including hematopoietic diseases, metabolic disorders, cancers, and autoimmune diseases, among others. In certain embodiments, the compositions and methods described herein lead to the mobilization of a population of CD34dim cells that have immunosuppressive effects and that can reduce the incidence of graft vs. host disease.
PCT/US2018/064335 2017-12-06 2018-12-06 Dosing regimens for the mobilization of hematopoietic stem and progenitor cells Ceased WO2019113375A2 (en)

Priority Applications (15)

Application Number Priority Date Filing Date Title
KR1020207019222A KR20200096942A (en) 2017-12-06 2018-12-06 Dosing regimen for mobilization of hematopoietic stem and progeny cells
BR112020011186-4A BR112020011186A2 (en) 2017-12-06 2018-12-06 dosing regimens for stem cell mobilization and hematopoietic progenitors
AU2018378804A AU2018378804B2 (en) 2017-12-06 2018-12-06 Dosing regimens for the mobilization of hematopoietic stem and progenitor cells
CA3083783A CA3083783A1 (en) 2017-12-06 2018-12-06 Dosing regimens for the mobilization of hematopoietic stem and progenitor cells
MX2020005878A MX2020005878A (en) 2017-12-06 2018-12-06 DOSAGE GUIDELINES FOR THE MOBILIZATION OF HEMATOPOIETIC STEM AND PROGENITOR CELLS.
EA202091092A EA202091092A1 (en) 2018-11-30 2018-12-06 DOSED ADMINISTRATION SCHEME FOR MOBILIZATION OF HEMOPOETIC STEM CELLS AND PRECURSOR CELLS
CN201880088815.6A CN111712262A (en) 2017-12-06 2018-12-06 Dosing regimen for mobilization of hematopoietic stem and progenitor cells
IL275077A IL275077B2 (en) 2017-12-06 2018-12-06 Dosing Regimens for the Mobilization of Hematopoietic Stem and Progenitor Cells
EP18829615.6A EP3720494A2 (en) 2017-12-06 2018-12-06 Dosing regimens for the mobilization of hematopoietic stem and progenitor cells
IL315735A IL315735A (en) 2017-12-06 2018-12-06 Dosing regimens for the mobilization of stem cells and hematopoietic progenitor cells
JP2020531468A JP2021505172A (en) 2017-12-06 2018-12-06 Dosing regimen to mobilize hematopoietic stem cells and progenitor cells
SG11202004913TA SG11202004913TA (en) 2017-12-06 2018-12-06 Dosing regimens for the mobilization of hematopoietic stem and progenitor cells
US16/352,578 US11260079B2 (en) 2017-12-06 2019-03-13 Dosing regimens for the mobilization of hematopoietic stem and progenitor cells
CONC2020/0007275A CO2020007275A2 (en) 2017-12-06 2020-06-16 Dosage guidelines for mobilization of hematopoietic stem and progenitor cells
JP2023188499A JP2024023226A (en) 2017-12-06 2023-11-02 Dosing regimen to mobilize hematopoietic stem and progenitor cells

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US15/834,017 2017-12-06
US15/834,017 US10058573B1 (en) 2017-12-06 2017-12-06 Dosing regimens for the mobilization of hematopoietic stem cells
US201762596056P 2017-12-07 2017-12-07
US62/596,056 2017-12-07
US16/101,676 2018-08-13
US16/101,676 US20190167726A1 (en) 2017-12-06 2018-08-13 Dosing Regimens for the Mobilization of Hematopoietic Stem Cells
US201862753656P 2018-10-31 2018-10-31
US62/753,656 2018-10-31
US201862773954P 2018-11-30 2018-11-30
US62/773,954 2018-11-30

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
US16/101,676 Continuation-In-Part US20190167726A1 (en) 2017-12-06 2018-08-13 Dosing Regimens for the Mobilization of Hematopoietic Stem Cells

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US16/352,578 Continuation US11260079B2 (en) 2017-12-06 2019-03-13 Dosing regimens for the mobilization of hematopoietic stem and progenitor cells

Publications (2)

Publication Number Publication Date
WO2019113375A2 WO2019113375A2 (en) 2019-06-13
WO2019113375A3 true WO2019113375A3 (en) 2019-07-18

Family

ID=66749948

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2018/064335 Ceased WO2019113375A2 (en) 2017-12-06 2018-12-06 Dosing regimens for the mobilization of hematopoietic stem and progenitor cells

Country Status (12)

Country Link
EP (1) EP3720494A2 (en)
JP (2) JP2021505172A (en)
KR (1) KR20200096942A (en)
CN (1) CN111712262A (en)
AU (1) AU2018378804B2 (en)
BR (1) BR112020011186A2 (en)
CA (1) CA3083783A1 (en)
CO (1) CO2020007275A2 (en)
IL (2) IL315735A (en)
MX (2) MX2020005878A (en)
SG (1) SG11202004913TA (en)
WO (1) WO2019113375A2 (en)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11260079B2 (en) 2017-12-06 2022-03-01 Magenta Therapeutics, Inc. Dosing regimens for the mobilization of hematopoietic stem and progenitor cells
EP4051298A1 (en) * 2019-11-01 2022-09-07 Magenta Therapeutics, Inc. Dosing regimens for the mobilization of hematopoietic stem and progentor cells
IL297690A (en) * 2020-04-27 2022-12-01 Magenta Therapeutics Inc Methods and compositions for transduction of hematopoietic stem cells and progenitor cells in a living body.
EP4308694A1 (en) * 2021-03-16 2024-01-24 Magenta Therapeutics, Inc. Dosing regimens for hematopoietic stem cell mobilization for stem cell transplants in multiple myeloma patients
JP2025519561A (en) * 2022-06-10 2025-06-26 ジーピーシーアール セラピューティクス,インク. GPCR inhibitors and uses thereof

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