CA2188675A1 - Compositions et procedes de therapie genique pour traiter des maladies - Google Patents

Compositions et procedes de therapie genique pour traiter des maladies

Info

Publication number
CA2188675A1
CA2188675A1 CA 2188675 CA2188675A CA2188675A1 CA 2188675 A1 CA2188675 A1 CA 2188675A1 CA 2188675 CA2188675 CA 2188675 CA 2188675 A CA2188675 A CA 2188675A CA 2188675 A1 CA2188675 A1 CA 2188675A1
Authority
CA
Canada
Prior art keywords
nucleic acid
transformed cell
vector
cell
functional portion
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.)
Abandoned
Application number
CA 2188675
Other languages
English (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
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.)
Baylor College of Medicine
Genemedicine Inc
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA2188675A1 publication Critical patent/CA2188675A1/fr
Abandoned legal-status Critical Current

Links

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], i.e. 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Public Health (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Zoology (AREA)
  • Endocrinology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Cell Biology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Cardiology (AREA)
  • Hematology (AREA)
  • Biotechnology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Procédé de thérapie génique destiné au traitement de toute une série d'anomalies ou maladies génétiques, qui consiste soit à introduire des cellules transformées dans un organisme tel qu'un être humain, soit à transformer les cellules in vivo. Les cellules transformées contiennent un vecteur d'acide nucléique qui produira la protéine nécessaire pour corriger l'anomalie ou la maladie. Parmi les maladies qui peuvent être traitées de cette manière, on trouve le diabète sucré, la maladie de Parkinson, les maladies cardio-vasculaires, l'hypercholestérolémie, l'hypertension, l'anémie, la thrombose, les troubles de la croissance, les troubles métaboliques tels que la polycétonurie et les troubles des protéines sériques, tels que l'hémophilie A.
CA 2188675 1994-04-11 1995-04-10 Compositions et procedes de therapie genique pour traiter des maladies Abandoned CA2188675A1 (fr)

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 (1)

Publication Number Publication Date
CA2188675A1 true CA2188675A1 (fr) 1995-10-19

Family

ID=22846464

Family Applications (1)

Application Number Title Priority Date Filing Date
CA 2188675 Abandoned CA2188675A1 (fr) 1994-04-11 1995-04-10 Compositions et procedes de therapie genique pour traiter des maladies

Country Status (4)

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

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US5658729A (en) * 1994-10-11 1997-08-19 The University Of British Columbia Method, reagent and kit for evaluating susceptibility to premature atherosclerosis
AU3518695A (en) * 1994-08-26 1996-03-22 Beth Israel Deaconess Medical Center Gene therapy for transplantation and inflammatory or thrmbotic 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
AU2251897A (en) * 1996-02-13 1997-09-02 Trustees Of The University Of Pennsylvania, The 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
AU2221600A (en) 1998-12-31 2000-07-31 Chiron Corporation Improved expression of hiv polypeptides and production of virus-like particles
ATE404217T1 (de) * 1999-06-24 2008-08-15 Univ British Columbia Therapie mit lipoproteinlipase (lpl) variant
EP1418810A4 (fr) 2001-08-03 2006-08-02 Us Gov Health & Human Serv Traitement de l'hemophilie par voie orale
PT1567641E (pt) 2001-08-24 2012-08-21 Uvic Industry Partnerships Inc Proaerolisina contendo sequência de activação de proteases e métodos de uso para o tratamento do cancro da próstata
WO2003093417A2 (fr) 2002-05-01 2003-11-13 Cell Genesys, Inc. Particules de vecteur lentiviral resistantes a l'inactivation d'un complement
JP2018537118A (ja) 2015-10-05 2018-12-20 オリジェン， インコーポレイテッド 肝機能不全の同定および緩和に基づくパーキンソン病の診断および処置
JP7065036B2 (ja) 2015-12-17 2022-05-11 モダーナティエックス・インコーポレイテッド メチルマロニルＣｏＡムターゼをコードするポリヌクレオチド
EP3714047A2 (fr) 2017-11-22 2020-09-30 ModernaTX, Inc. Polynucléotides codant pour la phénylalanine hydroxylase pour le traitement de la phénylcétonurie

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0289034B1 (fr) * 1987-05-01 1996-11-27 The General Hospital Corporation Implantation transcaryotique
ATE152169T1 (de) * 1988-02-05 1997-05-15 Whitehead Biomedical Inst Modifizierte hepatozyten und deren anwendung
US5427940A (en) * 1991-06-03 1995-06-27 Board Of Regents, The University Of Texas System Engineered cells producing insulin in response to glucose
CA2108144A1 (fr) * 1991-03-06 1992-09-07 Jack A. Roth Methodes et compositions pour l'inhibition selective de l'expression genique
PT616645E (pt) * 1991-12-06 2002-02-28 Genentech Inc Celulas transformadas para prohormona convertase
US6348327B1 (en) * 1991-12-06 2002-02-19 Genentech, Inc. Non-endocrine animal host cells capable of expressing variant proinsulin and processing the same to form active, mature insulin and methods of culturing such cells
WO1993013807A1 (fr) * 1992-01-10 1993-07-22 Georgetown University Procede permettant d'administrer des cellules endotheliales produites par genie genetique au niveau de sites d'angiogenese afin d'effectuer une therapie genetique

Also Published As

Publication number Publication date
WO1995027512A3 (fr) 1995-12-21
WO1995027512A2 (fr) 1995-10-19
EP0755268A1 (fr) 1997-01-29
AU2283495A (en) 1995-10-30
AU695618B2 (en) 1998-08-20

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