The field of gene therapy has matured and the prospects are exciting and hopeful since some treatments have now been shown to be effective in the clinic. However, precise quality and safety standards for clinical gene transfer have yet to be defined. Defining optimal methods for the production of standard vector systems would pave the way for accelerated development and improved safety. This would be of enormous value to industry, individual investigators and regulators. The NoE goal is the creation of a European Network for the Advancement of Clinical Gene Transfer and Therapy (CLINIGENE) integrating multidisciplinary research & development in gene therapy and mobilising all major stakeholders involved in the development of gene therapy medicinal products: academia, industry, regulatory bodies, clinics and patients. CLINIGENE will generate platform databases for particular vectors with respect to their safety and efficacy to ensure product manufacturing according to well-defined quality and safety standards in order to accelerate clinical trials. This will be achieved by compiling all available information and then ranking test and control methods by comparison and through validation by expert partners. The JPA comprises 1. Integration activites: sharing facilities, exchange and high-level training of personnel, e-communication, collaboration with the ESGT 2. Research activities: 6 horizontal activities serving integration towards the generation of reference/standard profile data-bases - AAV, gamma-retrovirus, lentivirus, adenovirus, genetically-modified cells & non-viral vectors - & 4 vertical activities defining a path to optimised clinical protocols - quality and efficacy (manufacture), safety (pharm-tox and virus safety); pre-clinical models and novel assessment tools, clinical trials 3. Dissemination activities: training, high-level education, communication (including a web-site with scientific & medical data-bases), management of shared information, IPR.
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