Table 2.

Selection of Horizon 2020 Funding Opportunities for Gene and Cell Therapy Projects

Topics from societal challenges	Specific challenge	Scope summary
2014/2015—Clinical research on regenerative medicine	Specific research is needed for proving safety, efficacy, and repeatability of new treatments in regenerative medicine clinical trials.	Proposals should focus on regenerative medicine therapies ready for clinical (in-patient) research and may address any disease or condition.
		Proposers should have or be close to have the necessary ethical and regulatory authorizations to carry out the work.
2014—New therapies for chronic noncommunicable diseases	Development of new therapies is stagnating because of lack of clinical validation.	Clinical trial(s) supporting proof of concept of novel therapeutic concept(s) and/or optimization of available therapies.
		Application may build on preexisting preclinical research and results from databases.
		Feasibility assessment and effectiveness should be provided if relevant.
2014—ERA NET: Rare disease research implementing IRDiRC objectives	In the area of rare diseases, promote transnational cooperation and pooling of resources while developing common standards and research priorities.	Proposals should coordinate national and regional programs for research on rare diseases to implement IRDiRC objectives.
		Further the understanding of disease mechanisms and natural history of rare diseases with objective to develop new diagnostic tools and treatments.
		Proposals should be complementary with other funding programs and activities on European and international level.
2015—New therapies for rare diseases	Specific problems posed in rare disease therapy development include the following:  small and dispersed patient populations  nature of the therapies proposed  regulatory hurdles  limited market providing low commercial return	Proposals may address one or more of the following:  development of new or improved therapeutic approaches, from small molecule to gene or cell therapy  repurposing of existing therapies  preclinical research, animal model development, and GMP production
		Clinical trials only supported in cases where “orphan designation” has been given and where its design takes into account recommendations given by the European Medicines Agency, including a clear patient recruitment strategy.
		Considerations of effectiveness/potential clinical benefit should be integrated in the application if relevant.
		Selected proposals should contribute to the objectives of and follow the guidelines and policies of IRDiRC.
2015—Tools and technologies for advanced therapies	New therapies, such as gene or cell therapies, tissue engineering, or regenerative medicine often require technological innovation to bring these innovative treatments to the patient and achieving regulatory compliance.	Proposals should focus on refining a particular technological step or component needed by the therapeutic approach.
		Establishing proof of concept for the new technology and carrying out preclinical research may be included if needed.
		Regulatory aspects of the new technology should be addressed as appropriate.
2015—Support for the European Reference Networks: Efficient network modeling and validation	Centres of Expertise will be able to collaborate, coordinate, and share their knowledge across borders. The demonstration and validation project of ERNs as an innovative model of cooperation and work between highly specialized health-care providers is required.	Proposals should provide coordinated support to the activities of the ERNs under the framework of a directive concerning validation of a model for the optimal organization, governance, maintenance, and continuous monitoring and evaluation of the ERNs and their centers.

ERNs, European reference networks; IRDiRC, International Rare Diseases Research Consortium.