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This Conference and Practical Course, held at the ‘Université d’ Evry – Val d’Essonne’, was the 3rd of a series of training initiatives on gene therapy (GT) on behalf of a training project called Vecteurotrain. This project was initiated in 2002, with the 1st Eurolabcourse on vectorology and GT, which took place at Généthon-Genocentre in Evry, and continued in 2004 with a 2nd European Conference and Course on the basic techniques of in vivo gene transfer into animals, held at the ‘Universitat Autònoma de Barcelona’ in Bellaterra in 1–14 February 2004. The 1st Eurolabcourse in 2002 emphasized the general aspects of gene transfer and therapy, and delivered practical training on vector production, purification and characterization (see The Journal of Gene Medicine 2004; 6(S1): 1–228). The 2nd European Conference and Course in 2004 delivered theoretical and practical training on preclinical gene transfer assessment for clinical GT (see Gene Therapy 2004; 11 (S1): 1–172), which is indispensable in the GT development pathway. The topics developed in the 3rd event in Evry in June 2004 focused on the biotechnological issues of GT products (vectors, plasmids, cell lines, etc), when they have to be prepared at industrial scale in a pharmaceutical production environment, which is a mandatory step in the development process of GT (see Figure 2 in Gonin et al in this supplement). During the conference, 114 scientific communications (36 full lectures, 23 short communications and 55 posters) were presented and highlighted the biology, use and industrial development of the main gene vectors, with general and expert information on (i) retroviral vectors (RVV), lentiviral vectors (LVV), adenoviral vector (AdV) and parvovirus-based, nonviral and other viral (alphavirus and herpes-based) vector systems; (ii) some applications in gene transfer into the liver, muscle, central nervous system and hematopoietic stem cells; (iii) biotechnological issues of scaling up, that is, theory and praxis of purification, cell cultures techniques and devices for industrial production of RVV, AdV and adeno-associated vector (AAV) vectors, and quality control issues; (iv) regulatory and ethical issues of gene transfer and GT. In a special session/round table, (v) some vector production facilities and services that are currently available for the scientific community in Europe and USA were presented. During the practical course, 32 selected researchers acquired hands-on experience on advanced methods for: (i) the production of AAV (ie transient transfection of 293 cells or infection of insect cells with recombinant baculoviruses) and purification with simple and fast methods using new ion-exchange chromatographic devices; (ii) preparations of large batches of AdV purified with chromatographic procedures; (iii) production of LVV vectors at large scale with improved packaging cell lines; (iv) new strategies to purify RVV and LVV pseudotyped with vesicular stomatitis virus protein G protein; and (v) characterization of viral vectors for basic biosafety parameters (ie physical and functional titration and assessment of adventitious contaminants and replication-competent particle).
Therefore, researchers and other GT professionals attending this course were trained in the scientific, technological and regulatory issues concerning the evolutionary process of GT products towards new biopharmaceuticals. This could be achieved by integrating the competence of different professions: namely, the researchers, who sign the gene transfer prototypes and protocols, and perform animal experimentation to validate proofs of principle; the regulators, who define the legal frame governing the clinical use of GT products; the industrialists, who allow technology transfers from the research benches to the biotech production plants, in order to transform the prototypes into exploitable products; and the physicians, involved in the last steps that lead to the completion of a clinical trial. Indeed, many GT operators from industries and other professions participated in all past events (see Table 1, bottom line), indicating that these workshops correspond to a real need of these nonacademic sectors. In the 3rd Conference and Course in Evry, 25 companies and SMEs participated as exhibitors, sponsors, lecturers, and attendees of the conference and/or attendees of the practical course (see list of industrial participants). The participation of professionals from industry, regulatory/ethical organizations and patients’ associations in the three events indicates that the courses, by providing the GT community with specific workshops and round-table discussions, greatly contributed to implement the dialogue between the different operators, which is necessary to allow the flux of information, technologies and materials. These aspects of the developmental process of GT are discussed in the article by Gonin et al in this supplement. Therefore, our training programme, which schedules a series of eight future events up to 2010 (see www.vecteurotrain.org), will provide a permanent forum for the major GT stakeholders to work together in identifying and addressing the issues related to the GT development. This will greatly help the process of standardization of GT products and the harmonization of the regulations in Europe.
Mezzina, M. The 3rd European conference and practical course on advanced gene vector technologies: training on pharmaceutical development of gene therapy products.
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(Suppl 1), S1–S4 (2005). https://doi.org/10.1038/sj.gt.3302652