title: [Quality control of gene therapy products: approach of the French Agency for the Safety of Health Products].
abstract: Gene therapy is a new therapeutic strategy which can constitute in some diseases a true alternative or a complement to the "classical treatments". Regarding the innovative features, the complexity and the extreme diversity of the gene therapy products (naked DNA, synthetic vectors, viral vectors, genetically modified cells), these new products presently in clinical trials have to be precisely evaluated and controlled for their medicine quality as well as their biological origin and/or their specific characteristics of genetically modified organisms. The French Agency for the Safety of Health Products engaged an in-depth scientific review concerning the control of this very heterogeneous class of potential therapeutics through the creation of a working group. The objectives of this group were to determine the testings to be performed by a national authority for each type of gene therapy products and to select the appropriate techniques or methods to be developed. Controls considered as essential are listed and include the verification of the identity, the purity, the transfer and expression efficiency as well as the microbiological and viral safety of the products. This implies the development of diverse techniques of molecular biology, cellular biology, physico-chemistry, animal testing, histology and microbiology. Finally, in order to define the basis of testings of these emerging products, the marketing of which should be effective for some of them in the next years, it appears extremely important to harmonize the quality, efficiency and safety criteria, to develop specific references and standards and to create specific guidelines for the control of gene therapy products.
authors.affiliation: Unité biologie cellulaire et moléculaire, Direction des Laboratoires et des Contrôles, 635, rue de la Garenne, 34740 Vendargues, France.
authors.forename: Xavier
authors.initials: X
authors.lastname: Chenivesse
authors.forename: Valérie
authors.initials: V
authors.lastname: Ridoux
authors.forename: Marie-Hélène
authors.initials: MH
authors.lastname: Tissier
mesh_terms: D005602:France; D015316:Genetic Therapy / Q000592:standards*; D017408:Guidelines as Topic; D006801:Humans; D009313:National Health Programs; D011786:Quality Control; D017751:Safety Management / Q000592:standards