WO2001025398A3 - Process for inducing functional tolerance to gene transfer products - Google Patents

Process for inducing functional tolerance to gene transfer products Download PDF

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Publication number
WO2001025398A3
WO2001025398A3 PCT/US2000/026946 US0026946W WO0125398A3 WO 2001025398 A3 WO2001025398 A3 WO 2001025398A3 US 0026946 W US0026946 W US 0026946W WO 0125398 A3 WO0125398 A3 WO 0125398A3
Authority
WO
WIPO (PCT)
Prior art keywords
gene transfer
inducing functional
tolerance
methods
transfer products
Prior art date
Application number
PCT/US2000/026946
Other languages
French (fr)
Other versions
WO2001025398A2 (en
Inventor
Goran K Andersson
Original Assignee
Biotransplant 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 Biotransplant Inc filed Critical Biotransplant Inc
Priority to CA002385750A priority Critical patent/CA2385750A1/en
Priority to EP00967159A priority patent/EP1578897A2/en
Priority to AU77406/00A priority patent/AU7740600A/en
Priority to JP2001528553A priority patent/JP2003531816A/en
Publication of WO2001025398A2 publication Critical patent/WO2001025398A2/en
Publication of WO2001025398A3 publication Critical patent/WO2001025398A3/en

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    • 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
    • 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
    • A61K39/0005—Vertebrate antigens
    • A61K39/001—Preparations to induce tolerance to non-self, e.g. prior to transplantation
    • 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
    • A61K39/46—Cellular immunotherapy
    • A61K39/461—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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/462—Cellular immunotherapy characterized by the effect or the function of the cells
    • A61K39/4621—Cellular immunotherapy characterized by the effect or the function of the cells immunosuppressive or immunotolerising
    • 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
    • A61K39/46—Cellular immunotherapy
    • A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643—Vertebrate antigens
    • A61K39/46433—Antigens related to auto-immune diseases; Preparations to induce self-tolerance
    • 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
    • A61K39/46—Cellular immunotherapy
    • A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643—Vertebrate antigens
    • A61K39/46434—Antigens related to induction of tolerance to non-self
    • 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
    • A61K39/46—Cellular immunotherapy
    • A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643—Vertebrate antigens
    • A61K39/4644—Cancer antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • A61K2035/124—Materials 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
    • 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 A61K39/46
    • A61K2239/31—Indexing codes associated with cellular immunotherapy of group A61K39/46 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 A61K39/46
    • A61K2239/46—Indexing codes associated with cellular immunotherapy of group A61K39/46 characterised by the cancer treated
    • A61K2239/48—Blood cells, e.g. leukemia or lymphoma
    • 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 A61K39/46
    • A61K2239/46—Indexing codes associated with cellular immunotherapy of group A61K39/46 characterised by the cancer treated
    • A61K2239/59—Reproductive system, e.g. uterus, ovaries, cervix or testes

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Mycology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Transplantation (AREA)
  • Oncology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pulmonology (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Methods of inducing functional tolerance for the expression products of transgenes in somatic cells are disclosed, which methods comprise the introduction into the recipient of stem cells, such as hematopoietic stem cells, transgenically modified so as to express one or more neoantigens, such procedure optionally preceded by a myeloreductive procedure. The purpose of the disclosed methods is to induce tolerance to these same antigens when later expressed by cells or vectors to be introduced as part of a gene therapy treatment.
PCT/US2000/026946 1999-10-01 2000-09-29 Process for inducing functional tolerance to gene transfer products WO2001025398A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
CA002385750A CA2385750A1 (en) 1999-10-01 2000-09-29 Process for inducing functional tolerance to gene transfer products
EP00967159A EP1578897A2 (en) 1999-10-01 2000-09-29 Process for inducing functional tolerance to gene transfer products
AU77406/00A AU7740600A (en) 1999-10-01 2000-09-29 Process for inducing functional tolerance to gene transfer products
JP2001528553A JP2003531816A (en) 1999-10-01 2000-09-29 Methods for inducing functional tolerance in transgenic products

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US15723399P 1999-10-01 1999-10-01
US60/157,233 1999-10-01

Publications (2)

Publication Number Publication Date
WO2001025398A2 WO2001025398A2 (en) 2001-04-12
WO2001025398A3 true WO2001025398A3 (en) 2008-03-20

Family

ID=22562877

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2000/026946 WO2001025398A2 (en) 1999-10-01 2000-09-29 Process for inducing functional tolerance to gene transfer products

Country Status (5)

Country Link
EP (1) EP1578897A2 (en)
JP (1) JP2003531816A (en)
AU (1) AU7740600A (en)
CA (1) CA2385750A1 (en)
WO (1) WO2001025398A2 (en)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003245752A1 (en) * 2002-06-28 2004-01-19 Bio Transplant, Inc. Process for promoting graft acceptance by depletion of hematopoietic stem cells
AU2002952834A0 (en) * 2002-09-16 2002-12-05 The Walter And Eliza Hall Institute Of Medical Research A method of treating an autoimmune disease
SG163615A1 (en) 2005-07-11 2010-08-30 Macrogenics Inc Methods for the treatment of autoimmune disorders using immunosuppressive monoclonal antibodies with reduced toxicity
JP2009544761A (en) 2006-06-14 2009-12-17 マクロジェニクス，インコーポレーテッド Method of treating autoimmune disease using low toxicity immunosuppressive monoclonal antibody
US20100313284A1 (en) * 2007-11-01 2010-12-09 Deltacell B.V. Means and methods for eliciting an immune response
CA2924668A1 (en) 2013-09-23 2015-03-26 Wilson Wolf Manufacturing Corporation Improved methods of genetically modifying animal cells

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5876708A (en) * 1992-02-19 1999-03-02 The General Hospital Corporation Allogeneic and xenogeneic transplantation
US5928638A (en) * 1996-06-17 1999-07-27 Systemix, Inc. Methods for gene transfer
US6096537A (en) * 1994-03-31 2000-08-01 Diacrin, Inc. Cells with multiple altered epitopes on a surface antigen for use in transplantation

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5876708A (en) * 1992-02-19 1999-03-02 The General Hospital Corporation Allogeneic and xenogeneic transplantation
US6096537A (en) * 1994-03-31 2000-08-01 Diacrin, Inc. Cells with multiple altered epitopes on a surface antigen for use in transplantation
US5928638A (en) * 1996-06-17 1999-07-27 Systemix, Inc. Methods for gene transfer

Also Published As

Publication number Publication date
AU7740600A (en) 2001-05-10
JP2003531816A (en) 2003-10-28
WO2001025398A2 (en) 2001-04-12
EP1578897A2 (en) 2005-09-28
CA2385750A1 (en) 2001-04-12

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