Since 1984, the European Commission has undertaken seven framework programs (FPs), dedicated to research and innovation, that ran for 5–7-year periods. During the fifth, sixth, and seventh FPs, more than €351 million has been invested in 110 projects in the gene transfer and gene therapy field (Table 1). While in FP5 and FP6, support was allocated mostly to small projects addressing specific problems such as refining targeted gene delivery or transgene expression in specific cells, the large-scale collaborative projects of FP6 and FP7 brought this research field from bench to bedside by gathering the critical mass of scientific expertise.
Presently, 15 FP7 gene therapy projects include clinical trials mostly for patients suffering from a rare disease or cancer, but also from more frequent disorders such as severe fetal growth restriction or age-related macular degeneration. Some of those projects (as well as their participants) derive directly from research and technologies developed in previous FPs. For example, the projects Cell-PID, performing clinical trials for primary immunodeficiencies (PIDs), and SUPERSIST, developing scale-up production of targeted genetic correction of hematopoietic stem cells and T cells for PIDs and leukemia, both derive from former FP7 PERSIST (refinement of gene therapy vectors for persisting transgenesis) and FP6 CONSERT (safety and efficiency evaluation of retroviral transgenesis) collaborative projects (ec.europa.eu/research/health/biotechnology/new-therapies/projects-fp7-gt_en.html). The specificity of these collaborative grants lies in gathering international expertise in various research disciplines, from scale-up and good manufacturing practice, development of new preclinical models, and toxicology and monitoring tests to regulatory and ethical issues of clinical trials.
The eighth research and innovation program, Horizon 2020 (2014–2020), is conceived as one of the central means to drive economic growth and job creation by addressing the entire innovation cycle, from basic research to implementation. In structure, Horizon 2020 has three main pillars: excellence in science, industrial leadership, and societal challenges. The challenge addressing primarily health research under the new program will have a slightly larger budget, €7.4 bn, compared with previous programs (€2.65 bn in FP6 and €6.2 bn in FP7). The first calls for societal challenges of “health, demographic change, and wellbeing” are published. Applicants are expected to focus on problem solving, and therefore gene and cell therapy projects could find funding opportunities in topics advertising chronic or rare disease research as well as regenerative medicine or advanced therapies.
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