JP2021503008A - 造血幹細胞および前駆細胞移植療法のための組成物および方法 - Google Patents

造血幹細胞および前駆細胞移植療法のための組成物および方法 Download PDF

Info

Publication number
JP2021503008A
JP2021503008A JP2020544558A JP2020544558A JP2021503008A JP 2021503008 A JP2021503008 A JP 2021503008A JP 2020544558 A JP2020544558 A JP 2020544558A JP 2020544558 A JP2020544558 A JP 2020544558A JP 2021503008 A JP2021503008 A JP 2021503008A
Authority
JP
Japan
Prior art keywords
optionally substituted
cells
hematopoietic stem
group
patient
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
JP2020544558A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021503008A5 (https=
Inventor
ボイタノ アンソニー
ボイタノ アンソニー
マイケル クック
クック マイケル
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
Application filed by Magenta Therapeutics Inc filed Critical Magenta Therapeutics Inc
Publication of JP2021503008A publication Critical patent/JP2021503008A/ja
Publication of JP2021503008A5 publication Critical patent/JP2021503008A5/ja
Pending legal-status Critical Current

Links

Classifications

    • 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
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • 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
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/4427Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems
    • A61K31/444Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring heteroatom, e.g. amrinone
    • 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
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/4985Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
    • 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
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A61K31/52Purines, e.g. adenine
    • 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
    • A61K35/48Reproductive organs
    • A61K35/51Umbilical cord; Umbilical cord blood; Umbilical stem cells
    • 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
    • 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
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • 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
    • 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
    • C12N2501/10Growth factors
    • C12N2501/125Stem cell factor [SCF], c-kit ligand [KL]
    • 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
    • C12N2501/10Growth factors
    • C12N2501/145Thrombopoietin [TPO]
    • 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
    • C12N2501/20Cytokines; Chemokines
    • C12N2501/23Interleukins [IL]
    • C12N2501/2306Interleukin-6 (IL-6)
    • 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
    • C12N2501/20Cytokines; Chemokines
    • C12N2501/26Flt-3 ligand (CD135L, flk-2 ligand)
    • 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
    • C12N2501/999Small molecules not provided for elsewhere
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Hematology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Cell Biology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Diabetes (AREA)
  • Reproductive Health (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2020544558A 2017-10-31 2018-10-31 造血幹細胞および前駆細胞移植療法のための組成物および方法 Pending JP2021503008A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762579776P 2017-10-31 2017-10-31
US62/579,776 2017-10-31
US201762596661P 2017-12-08 2017-12-08
US62/596,661 2017-12-08
PCT/US2018/058562 WO2019089833A1 (en) 2017-10-31 2018-10-31 Compositions and methods for hematopoietic stem and progenitor cell transplant therapy

Publications (2)

Publication Number Publication Date
JP2021503008A true JP2021503008A (ja) 2021-02-04
JP2021503008A5 JP2021503008A5 (https=) 2021-12-09

Family

ID=64650470

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020544558A Pending JP2021503008A (ja) 2017-10-31 2018-10-31 造血幹細胞および前駆細胞移植療法のための組成物および方法

Country Status (7)

Country Link
US (1) US20190343885A1 (https=)
EP (1) EP3703715A1 (https=)
JP (1) JP2021503008A (https=)
CN (1) CN111683669A (https=)
AU (1) AU2018358054A1 (https=)
CA (1) CA3079404A1 (https=)
WO (1) WO2019089833A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX392743B (es) 2017-04-12 2025-03-12 Magenta Therapeutics Inc Antagonistas del receptor de hidrocarburo de arilo y sus usos.
EP3735412B1 (en) * 2018-01-03 2024-02-21 EdiGene Biotechnology, Inc. Compositions and methods for the expansion of hematopoietic stem and progenitor cells and treatment of inherited metabolic disorders

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016164502A1 (en) * 2015-04-06 2016-10-13 President And Fellows Of Harvard College Compositions and methods for non-myeloablative conditioning

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5233044A (en) 1989-06-08 1993-08-03 Millipore Corporation Active esters for solid phase peptide synthesis
US5021409A (en) 1989-12-21 1991-06-04 Johnson Matthey Plc Antiviral cyclic polyamines
US5512421A (en) 1991-02-19 1996-04-30 The Regents Of The University Of California Generation, concentration and efficient transfer of VSV-G pseudotyped retroviral vectors
GB9126677D0 (en) 1991-12-16 1992-02-12 Johnson Matthey Plc Improvements in chemical compounds
MD1367C2 (ro) 1992-11-13 2000-11-30 Idec Pharmaceuticals Corporation Metode de tratament al limfomului celulelor B, anticorpi anti-CD20, hibridom.
US6447766B1 (en) 1993-06-08 2002-09-10 Smithkline Beecham Corporation Method of mobilizing hematopoietic stem cells
DE69433301T2 (de) 1993-06-08 2004-09-09 Smithkline Beecham Corp. Methoden zur erhöhung der biologischen aktivität von chemokinen
GB9400411D0 (en) 1994-01-11 1994-03-09 Johnson Matthey Plc Improvements in chemical compounds
US5547892A (en) 1995-04-27 1996-08-20 Taiwan Semiconductor Manufacturing Company Process for forming stacked contacts and metal contacts on static random access memory having thin film transistors
US6506770B1 (en) 1996-06-06 2003-01-14 Anormed, Inc. Antiviral compounds
US5801030A (en) 1995-09-01 1998-09-01 Genvec, Inc. Methods and vectors for site-specific recombination
HU226994B1 (en) 1997-02-11 2010-04-28 Mallinckrodt Reactor and method for solid phase peptide synthesis
EP1068357B2 (en) 1998-03-30 2014-12-10 NorthWest Biotherapeutics, Inc. Therapeutic and diagnostic applications based on the role of the cxcr-4 gene in tumorigenesis
BR0010655A (pt) 1999-03-24 2002-02-13 Anormed Inc Compostos heterocìclicos para ligação ao receptor de quimiocina
DE60042030D1 (de) 1999-12-17 2009-05-28 Genzyme Corp Chemokinrezeptor-bindende heterocyclische verbindungen
CA2407651C (en) 2000-04-27 2013-07-02 Max-Delbruck-Centrum Fur Molekulare Medizin Sleeping beauty, a transposon vector with a broad host range for the genetic transformation in vertebrates
DE60103052T2 (de) 2000-05-09 2005-03-03 The University Of British Columbia, Vancouver Verwendung von cxcr4 antagonisten zur behandlung von krebs und autoimmunkrankheiten
CA2412436C (en) 2000-06-05 2013-05-21 The Trustees Of Columbia University In The City Of New York Identification and use of human bone marrow-derived endothelial progenitor cells to improve myocardial function after ischemic injury
CA2456977C (en) 2001-08-16 2011-06-07 The Trustees Of The University Of Pennsylvania Synthesis and use of reagents for improved dna lipofection and/or slow release prodrug and drug therapies
US20030199464A1 (en) 2002-04-23 2003-10-23 Silviu Itescu Regeneration of endogenous myocardial tissue by induction of neovascularization
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
JP2008509928A (ja) 2004-08-13 2008-04-03 アノーメド インコーポレイテッド 前駆/幹細胞を動員するためのケモカインの組み合わせ
CA2891996A1 (en) 2005-10-18 2007-04-26 Duke University Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity
US8383095B2 (en) * 2006-02-14 2013-02-26 Cellerant Therapeutics, Inc. Methods and compositions for enhancing engraftment of hematopoietic stem cells
KR20090021217A (ko) 2006-06-14 2009-02-27 추가이 세이야쿠 가부시키가이샤 조혈 줄기세포 증가 촉진제
EP1995316A1 (de) 2007-05-25 2008-11-26 Qiagen GmbH Verfahren zur schonenden Zellaufreinigung, Zellgewinnung und Transfektion von Zellen
EP2009095A1 (en) 2007-06-28 2008-12-31 Innovalor AG Method of generating glucose-responsive cells
EP2215223B1 (en) 2007-10-31 2013-05-01 Precision Biosciences, Inc. Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
PE20100362A1 (es) 2008-10-30 2010-05-27 Irm Llc Derivados de purina que expanden las celulas madre hematopoyeticas
WO2010085699A2 (en) 2009-01-23 2010-07-29 The Johns Hopkins University Mammalian piggybac transposon and methods of use
EP2270025A1 (en) 2009-06-29 2011-01-05 Centre National pour la Recherche Scientifique (CNRS) Solid phase peptide synthesis of peptide alcohols
US9388212B2 (en) 2013-02-21 2016-07-12 Chemical & Biopharmaceutical Laboratories Of Patras S.A. Solid phase peptide synthesis via side chain attachment
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
JP2016516672A (ja) * 2013-02-28 2016-06-09 プレジデント アンド フェローズ オブ ハーバード カレッジ 幹細胞を動員するための方法および組成物
US9169287B2 (en) 2013-03-15 2015-10-27 Massachusetts Institute Of Technology Solid phase peptide synthesis processes and associated systems

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016164502A1 (en) * 2015-04-06 2016-10-13 President And Fellows Of Harvard College Compositions and methods for non-myeloablative conditioning

Also Published As

Publication number Publication date
AU2018358054A1 (en) 2020-05-07
EP3703715A1 (en) 2020-09-09
CA3079404A1 (en) 2019-05-09
WO2019089833A1 (en) 2019-05-09
US20190343885A1 (en) 2019-11-14
CN111683669A (zh) 2020-09-18

Similar Documents

Publication Publication Date Title
JP7412341B2 (ja) 造血幹細胞および前駆細胞の増幅のための組成物および方法
US20230130646A1 (en) Dosing regimens for the mobilization of hematopoietic stem cells
JP2024023226A (ja) 造血幹細胞及び前駆細胞を動員させるための投薬レジメン
US20250032549A1 (en) Dosing regimens for the mobilization of hematopoietic stem and progenitor cells
US11260079B2 (en) Dosing regimens for the mobilization of hematopoietic stem and progenitor cells
JP2021503008A (ja) 造血幹細胞および前駆細胞移植療法のための組成物および方法
EP3735412B1 (en) Compositions and methods for the expansion of hematopoietic stem and progenitor cells and treatment of inherited metabolic disorders
JP2022505615A (ja) 造血幹・前駆細胞移植療法の方法
US20220401481A1 (en) Dosing regimens for the mobilization of hematopoietic stem and progenitor cells
JPWO2020092694A5 (https=)

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211029

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20211029

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20221025

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20230523