EIC Transition Challenge: RNA-based therapies and diagnostics for complex or rare genetic diseases

The starting point in the project should be a preliminary technology or protocol of an RNA-based therapy for complex or rare genetic diseases with unmet medical needs that demonstrates, in a lab or preclinical context, the essential features that underpin the disruptive nature of the innovation (TRL 3-4). The endpoint in the project should be a completely functional version of the technology suitable for clinical validation (TRL5-6), supported by a sound and implementable commercialisation/exploitation strategy.

Proposals are expected to contribute to at least one of the following outcomes:

• novel technological solutions leading to more effective and safer RNA delivery methods applicable to a wide range of non-infectious diseases;
• utilisation of RNAs to molecularly classify sub-types of different solid tumours that would allow for stratification of patients leading to more effective and precise treatments in complex diseases with high-unmet medical needs;
• novel and sound ideas for the development and validation of RNA-based therapeutic platforms and drugs;
• all the projects should lead to a sufficiently mature and sound data for being ready to be up taken to the (pre-) clinical trials.

EIC Transition aims at maturing both your technology and business idea thus increasing its technology and market readiness. The expected outcomes of an EIC Transition project are a) a technology that is demonstrated to be effective for its intended application and b) a business model, its initial validation and a business plan for its development to market. It is also expected that the intellectual property generated by the EIC Transition project is formally protected in an adequate way.

For more details, see the WP 2022.

Proposals submitted to this EIC Transition Challenge call should focus on one or more of the following specific objectives:

• advance, beyond the state-of-the-art, RNA delivery methods, including robust mRNA formulations, that would enable effective and safe delivery of mRNA into the cells;
• design, develop and preclinical validate of novel miRNAs (miRNA lncRNA, tRNA or siRNA-based) therapies for complex or rare genetic diseases;
• develop and validate novel RNA-based diagnostics and RNA-based predictive biomarkers that would allow for early and more accurate diagnosis and for favourable or non- post-treatment prognosis, respectively.

EIC Transition funds innovation activities that go beyond the experimental proof of principle in laboratory. It supports both the maturation and validation of a novel technology from the lab to the relevant application environments (by making use of prototyping, formulation, models, user testing or other validation tests) as well as explorations and development of a sustainable business case and business model towards commercialisation.