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WO2002031110A3 - Hematopoietic stem cell gene therapy - Google Patents

Hematopoietic stem cell gene therapy Download PDF

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Publication number
WO2002031110A3
WO2002031110A3 PCT/IB2001/002739 IB0102739W WO0231110A3 WO 2002031110 A3 WO2002031110 A3 WO 2002031110A3 IB 0102739 W IB0102739 W IB 0102739W WO 0231110 A3 WO0231110 A3 WO 0231110A3
Authority
WO
WIPO (PCT)
Prior art keywords
cells
thymus
patient
hematopoietic stem
lhrh
Prior art date
Application number
PCT/IB2001/002739
Other languages
French (fr)
Other versions
WO2002031110A2 (en
Inventor
Richard Boyd
Original Assignee
Richard Boyd
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 AUPR0745A external-priority patent/AUPR074500A0/en
Priority claimed from US09/966,576 external-priority patent/US20020071833A1/en
Priority to JP2002534480A priority Critical patent/JP2004511495A/en
Priority to BR0114639-4A priority patent/BR0114639A/en
Priority to AU2002216320A priority patent/AU2002216320C1/en
Priority to APAP/P/2003/002800A priority patent/AP2003002800A0/en
Application filed by Richard Boyd filed Critical Richard Boyd
Priority to CA002462073A priority patent/CA2462073A1/en
Priority to KR10-2003-7005255A priority patent/KR20030082542A/en
Priority to IL15541301A priority patent/IL155413A0/en
Priority to EP01986714A priority patent/EP1357978A4/en
Priority to AU1632002A priority patent/AU1632002A/en
Publication of WO2002031110A2 publication Critical patent/WO2002031110A2/en
Publication of WO2002031110A3 publication Critical patent/WO2002031110A3/en

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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
    • 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
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/36—Skin; Hair; Nails; Sebaceous glands; Cerumen; Epidermis; Epithelial cells; Keratinocytes; Langerhans cells; Ectodermal cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/08—Peptides having 5 to 11 amino acids
    • A61K38/09—Luteinising hormone-releasing hormone [LHRH], i.e. Gonadotropin-releasing hormone [GnRH]; Related peptides
    • A—HUMAN NECESSITIES
    • 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
    • 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
    • A61K40/46—Viral antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N2510/00—Genetically modified cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Immunology (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (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)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Zoology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Genetics & Genomics (AREA)
  • Hematology (AREA)
  • Molecular Biology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Dermatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Mycology (AREA)
  • Reproductive Health (AREA)
  • Endocrinology (AREA)
  • AIDS & HIV (AREA)
  • Gastroenterology & Hepatology (AREA)

Abstract

The present disclosure provides methods for gene therapy utilizing hematopoietic stem cells, lymphoid progenitor cells, and/or myeloid progenitor cells. The cells are genetically modified to provide a gene that is expressed in these cells and their progeny after differentiation. In a preferred embodiment the cells contain a gene or gene fragment that confers to the cells resistance to HIV infection and/or replication. The cells are administered to a patient in conjunction with treatment to reactivate the patient's thymus. The cells may be autologous, syngeneic, allogeneic or xenogeneic, as tolerance to foreign cells is created in the patient during reactivation of the thymus. In a preferred embodiment the hematopoietic stem cells are CD34+. The patient's thymus is reactivated by disruption of sex steroid mediated signaling to the thymus. In a preferred embodiment, this disruption is created by administration of LHRH agonists, LHRH antagonists, anti-LHRH receptor antibodies, anti-LHRH vaccines or combinations thereof.
PCT/IB2001/002739 2000-10-13 2001-10-12 Hematopoietic stem cell gene therapy WO2002031110A2 (en)

Priority Applications (9)

Application Number Priority Date Filing Date Title
AU1632002A AU1632002A (en) 2000-10-13 2001-10-12 Hematopoietic stem cell gene therapy
EP01986714A EP1357978A4 (en) 2000-10-13 2001-10-12 Gene therapy with haematopoietic stem cells
BR0114639-4A BR0114639A (en) 2000-10-13 2001-10-12 Hematopoietic stem cell gene therapy
AU2002216320A AU2002216320C1 (en) 2000-10-13 2001-10-12 Hematopoietic stem cell gene therapy
APAP/P/2003/002800A AP2003002800A0 (en) 2000-10-13 2001-10-12 Hematopoietic stem cell gene therapy
JP2002534480A JP2004511495A (en) 2000-10-13 2001-10-12 Hematopoietic stem cell gene therapy
CA002462073A CA2462073A1 (en) 2000-10-13 2001-10-12 Hematopoietic stem cell gene therapy
KR10-2003-7005255A KR20030082542A (en) 2000-10-13 2001-10-12 Hematopoietic Stem Cell Gene Therapy
IL15541301A IL155413A0 (en) 2000-10-13 2001-10-12 Hematopoietic stem cell gene therapy

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US79528600A 2000-10-13 2000-10-13
US79530200A 2000-10-13 2000-10-13
US09/795,286 2000-10-13
AUPR0745 2000-10-13
AUPR0745A AUPR074500A0 (en) 2000-10-13 2000-10-13 Treatment of t cell disorders
US09/795,302 2000-10-13
US75891001A 2001-01-10 2001-01-10
US09/758,910 2001-01-10
US09/966,576 US20020071833A1 (en) 1999-04-15 2001-09-26 Hematopoietic stem cell gene therapy
US09/966,576 2001-09-26
US96951001A 2001-10-01 2001-10-01
US09/969,510 2001-10-01

Publications (2)

Publication Number Publication Date
WO2002031110A2 WO2002031110A2 (en) 2002-04-18
WO2002031110A3 true WO2002031110A3 (en) 2002-06-20

Family

ID=27542982

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2001/002739 WO2002031110A2 (en) 2000-10-13 2001-10-12 Hematopoietic stem cell gene therapy

Country Status (10)

Country Link
EP (1) EP1357978A4 (en)
JP (1) JP2004511495A (en)
KR (1) KR20030082542A (en)
CN (1) CN1582178A (en)
AP (1) AP2003002800A0 (en)
AU (1) AU1632002A (en)
BR (1) BR0114639A (en)
CA (1) CA2462073A1 (en)
IL (1) IL155413A0 (en)
WO (1) WO2002031110A2 (en)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040258672A1 (en) * 1999-04-15 2004-12-23 Monash University Graft acceptance through manipulation of thymic regeneration
JP2007518699A (en) * 2003-04-18 2007-07-12 ノーウッド・イミュノロジー・リミテッド Disease prevention and vaccination before thymus regeneration
CN101952454B (en) 2008-02-08 2014-06-04 米迪缪尼有限公司 Anti-IFNAR1 antibodies with reduced Fc ligand affinity
PT2635607T (en) 2010-11-05 2019-12-11 Zymeworks Inc Stable heterodimeric antibody design with mutations in the fc domain

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996014853A1 (en) * 1994-11-10 1996-05-23 University Of Washington Intrathymic stem cell implantation
WO2000062657A2 (en) * 1999-04-15 2000-10-26 Monash University Improvement of t cell mediated immunity

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996014853A1 (en) * 1994-11-10 1996-05-23 University Of Washington Intrathymic stem cell implantation
WO2000062657A2 (en) * 1999-04-15 2000-10-26 Monash University Improvement of t cell mediated immunity

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
BONYHADI M. ET AL: "RevM10-expressing T cells derived in vivo from transduced human hematopoietic stem-progenitor cells inhibit human immunodeficiency virus replication", J. VIROL., vol. 71, no. 6, 1997, pages 4707 - 4716, XP002969707 *
FABRIS N. ET AL: "Plasticity of neuro-endocrine-thymus interaction during aging", CELLULAR AND MOLEC. BIOL., vol. 43, no. 4, 1997, pages 529 - 541, XP002969786 *
GREENSTEIN B. ET AL: "Regeneration of the thymus in old male rats treated with a stable analogue of LHRH", J. ENDOCR., vol. 112, 1987, pages 345 - 350, XP002969788 *
NIKOLIC B. ET AL: "A novel application of cyclosporin A in nonmyeloablative pretransplant host conditioning for allogeneic BMT", BLOOD, vol. 96, no. 3, August 2000 (2000-08-01), pages 1166 - 1172, XP002969706 *
PENIT C. ET AL: "Cell proliferation and thymocyte subset reconstitution in sublethally irradiated mice: Compared kinetics of endogenous and intrathymically transferred progenitors", PROC. NATL. ACAD. SCI. USA, vol. 86, 1989, pages 5547 - 5551, XP002969787 *

Also Published As

Publication number Publication date
CN1582178A (en) 2005-02-16
KR20030082542A (en) 2003-10-22
AU1632002A (en) 2002-04-22
WO2002031110A2 (en) 2002-04-18
IL155413A0 (en) 2003-11-23
EP1357978A2 (en) 2003-11-05
JP2004511495A (en) 2004-04-15
BR0114639A (en) 2004-01-20
CA2462073A1 (en) 2002-04-18
EP1357978A4 (en) 2004-05-19
AP2003002800A0 (en) 2003-06-30

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