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

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.

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