WO1995027512A3 - Compositions and methods for gene therapy to treat disease - Google Patents

Compositions and methods for gene therapy to treat disease Download PDF

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Publication number
WO1995027512A3
WO1995027512A3 PCT/US1995/004397 US9504397W WO9527512A3 WO 1995027512 A3 WO1995027512 A3 WO 1995027512A3 US 9504397 W US9504397 W US 9504397W WO 9527512 A3 WO9527512 A3 WO 9527512A3
Authority
WO
WIPO (PCT)
Prior art keywords
disease
gene therapy
disorders
compositions
methods
Prior art date
Application number
PCT/US1995/004397
Other languages
French (fr)
Other versions
WO1995027512A2 (en
Inventor
Savio L C Woo
Louis C Smith
Lawrence C B Chan
Allain Rolland
Mark A Kay
Randy C Eisensmith
Larry Moss
Tadeusz Kolodka
Tina M Hahn
Original Assignee
Baylor College Medicine
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 Baylor College Medicine filed Critical Baylor College Medicine
Priority to EP95916281A priority Critical patent/EP0755268A1/en
Priority to AU22834/95A priority patent/AU695618B2/en
Publication of WO1995027512A2 publication Critical patent/WO1995027512A2/en
Publication of WO1995027512A3 publication Critical patent/WO1995027512A3/en

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/2242—Atrial natriuretic factor complex: Atriopeptins, atrial natriuretic protein [ANP]; Cardionatrin, Cardiodilatin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1816—Erythropoietin [EPO]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/27—Growth hormone [GH] (Somatotropin)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/33—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans derived from pro-opiomelanocortin, pro-enkephalin or pro-dynorphin
    • A61K38/34—Melanocyte stimulating hormone [MSH], e.g. alpha- or beta-melanotropin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/38—Albumins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/62—Insulins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

Abstract

Method of gene therapy for the treatment of a variety of genetic defects or diseases comprising the steps of either introducing transformed cells into an organism such as a human or the in vivo transformation of cells. The transformed cells contain a nucleic acid vector which will produce the protein necessary to correct the deficiency or disease. Examples of diseases which can be treated this way include diabetes mellitus, Parkinson's disease, cardiovascular disease, hypercholesterolemia, hypertension, anemia, thrombosis, growth disorders, metabolic disorders such as PKU and serum protein disorders, such as hemophilia A.
PCT/US1995/004397 1994-04-11 1995-04-10 Compositions and methods for gene therapy to treat disease WO1995027512A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
EP95916281A EP0755268A1 (en) 1994-04-11 1995-04-10 Compositions and methods for gene therapy to treat disease
AU22834/95A AU695618B2 (en) 1994-04-11 1995-04-10 Compositions and methods for gene therapy to treat disease

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US22584094A 1994-04-11 1994-04-11
US08/225,840 1994-04-11

Publications (2)

Publication Number Publication Date
WO1995027512A2 WO1995027512A2 (en) 1995-10-19
WO1995027512A3 true WO1995027512A3 (en) 1995-12-21

Family

ID=22846464

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1995/004397 WO1995027512A2 (en) 1994-04-11 1995-04-10 Compositions and methods for gene therapy to treat disease

Country Status (4)

Country Link
EP (1) EP0755268A1 (en)
AU (1) AU695618B2 (en)
CA (1) CA2188675A1 (en)
WO (1) WO1995027512A2 (en)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5658729A (en) * 1994-10-11 1997-08-19 The University Of British Columbia Method, reagent and kit for evaluating susceptibility to premature atherosclerosis
US6814962B1 (en) 1994-06-02 2004-11-09 Aventis Pharma S.A. Recombinant viruses and their use for treatment of atherosclerosis and other forms of coronary artery disease and method, reagent, and kit for evaluating susceptibility to same
CA2197427A1 (en) * 1994-08-26 1996-03-07 Christopher Wrighton Gene therapy for transplantation and inflammatory or thrombotic conditions
US6818439B1 (en) 1994-12-30 2004-11-16 Chiron Corporation Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders
ZA969435B (en) * 1995-11-16 1997-06-02 Antonomous University Of Barce Treatment of diabetes with a glucokinase gene
US6087129A (en) 1996-01-19 2000-07-11 Betagene, Inc. Recombinant expression of proteins from secretory cell lines
WO1997030167A1 (en) * 1996-02-13 1997-08-21 The Trustees Of The University Of Pennsylvania Method of treating liver disorders
US6027721A (en) * 1996-05-20 2000-02-22 Cytotherapeutics, Inc. Device and method for encapsulated gene therapy
US5952226A (en) * 1996-11-05 1999-09-14 Modex Therapeutiques Hypoxia responsive EPO producing cells
US6518412B1 (en) * 1997-09-29 2003-02-11 Jean-Louis Dasseux Gene therapy approaches to supply apolipoprotein A-I agonists and their use to treat dyslipidemic disorders
JP2002533124A (en) 1998-12-31 2002-10-08 カイロン コーポレイション Improved expression of HIV polypeptide and generation of virus-like particles
ES2311464T3 (en) * 1999-06-24 2009-02-16 The University Of British Columbia THERAPY WITH LIPOPROTEIN LIPASA VARIANTE (LPL).
US7220718B2 (en) 2001-08-03 2007-05-22 United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Oral treatment of hemophilia
JP4319908B2 (en) 2001-08-24 2009-08-26 ユニバーシティ オブ ビクトリア イノベーション アンド ディベロップメント コーポレーション Proaerolysin containing a protease activating sequence and methods of use for the treatment of prostate cancer
AU2003228751A1 (en) 2002-05-01 2003-11-17 Cell Genesys, Inc. Lentiviral vector particles resistant to complement inactivation
US10842822B2 (en) 2015-10-05 2020-11-24 Orig3N, Inc. Diagnosis and treatment of parkinson's disease based on identification and amelioration of liver dysfunction
AU2016369612B2 (en) 2015-12-17 2023-06-01 Modernatx, Inc. Polynucleotides encoding methylmalonyl-CoA mutase
MA50801A (en) 2017-11-22 2020-09-30 Modernatx Inc POLYNUCLEOTIDES CODING FOR PHENYLALANINE HYDROXYLASE FOR THE TREATMENT OF PHENYLKETONURIS

Citations (7)

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Publication number Priority date Publication date Assignee Title
WO1988008306A1 (en) * 1987-05-01 1988-11-03 The General Hospital Corporation Transkaryotic implantation
WO1989007136A2 (en) * 1988-02-05 1989-08-10 Whitehead Institute For Biomedical Research Modified hepatocytes and uses therefor
WO1992015680A1 (en) * 1991-03-06 1992-09-17 Board Of Regents, The University Of Texas System Methods and compositions for the selective inhibition of gene expression
WO1992021756A1 (en) * 1991-06-03 1992-12-10 Board Of Regents, The University Of Texas System Methods and compositions relating to genetically engineered cells that produce insulin in response to glucose
WO1993011247A1 (en) * 1991-12-06 1993-06-10 Genentech, Inc. Prohormone convertase transformed cells
WO1993013807A1 (en) * 1992-01-10 1993-07-22 Georgetown University A method of administering genetically engineered endothelial cells to sites of angiogenesis for effecting genetic therapy
WO1994020624A1 (en) * 1993-03-01 1994-09-15 Genentech, Inc. Prohormone convertase transformed cells and polypeptide synthesis

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1988008306A1 (en) * 1987-05-01 1988-11-03 The General Hospital Corporation Transkaryotic implantation
WO1989007136A2 (en) * 1988-02-05 1989-08-10 Whitehead Institute For Biomedical Research Modified hepatocytes and uses therefor
WO1992015680A1 (en) * 1991-03-06 1992-09-17 Board Of Regents, The University Of Texas System Methods and compositions for the selective inhibition of gene expression
WO1992021756A1 (en) * 1991-06-03 1992-12-10 Board Of Regents, The University Of Texas System Methods and compositions relating to genetically engineered cells that produce insulin in response to glucose
WO1993011247A1 (en) * 1991-12-06 1993-06-10 Genentech, Inc. Prohormone convertase transformed cells
WO1993013807A1 (en) * 1992-01-10 1993-07-22 Georgetown University A method of administering genetically engineered endothelial cells to sites of angiogenesis for effecting genetic therapy
WO1994020624A1 (en) * 1993-03-01 1994-09-15 Genentech, Inc. Prohormone convertase transformed cells and polypeptide synthesis

Non-Patent Citations (5)

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Title
BOWLES ET AL: "GENE THERAPY FOR INSULIN-DEPENDENT DIABETES MELLITUS BY THE HEPATIC EXPRESSION OF INSULIN", JOURNAL OF CELLULAR BIOCHEMISTRY,SUPPLEMENT 21A, 14 March 1995 (1995-03-14), pages 375 *
GROSKREUTZ ET AL: "GENETICALLY ENGINEERED PROINSULIN CONSTITUTIVELY PROCESSED AND SECRETED AS MATURE,ACTIVE INSULIN", THE JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 269, no. 8, 25 February 1994 (1994-02-25), pages 6241 - 6245 *
VALERA ET AL: "APPROACHES TO DIABETES GENE THERAPY USING INSULIN GENE DRIVEN BY P-ENOLPYRUVATE CARBOXIKINASE PROMOTER", JOURNAL OF CELLULAR BIOCHEMISTRY,SUPPLEMENT 17E, pages 252 *
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Also Published As

Publication number Publication date
AU2283495A (en) 1995-10-30
EP0755268A1 (en) 1997-01-29
AU695618B2 (en) 1998-08-20
CA2188675A1 (en) 1995-10-19
WO1995027512A2 (en) 1995-10-19

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