REFINE proposes a Regulatory Science Framework for the risk-benefit assessment of medical products and medical devices that are based on nanomedicines and biomaterials. The heart of our framework is the development of a product-specific Decision Support System that identifies the most efficient way to deliver the data required by regulation by the best-fitting methods. The decision tree will explicate the product’s specific regulatory challenges and the priorities of both missing data and missing methods to match these challenges. It will thus allow planning a cost-and time efficient strategy both for necessary measurements and for the advancement of methods. Our approach is aligned with the industrial R&D practice of stage gating. We will demonstrate the relevance of the framework for the most pressing regulatory challenges, which are: borderline products, nanosimilars, and products combining several functionalities. In order to do so, we will identify the regulatory challenges with Regulation Authorities from Europe and abroad, and design methods for tiered decision tree, guided by the latest scientific knowledge. We will study/predict physiological distribution of nanomedicines and biomaterials, as well as develop and validate new analytical or experimental methods and assays requested by the regulators. These latter development will be performed in a quality management system, ensuring the possible standardisation of our assays. REFINE will gather a wide community of stakeholders in regulation, industry, science, technology development, patients, and end-users, into a Consortium for the Advancement of Regulatory Science in Biomaterials and Nanomedicine.

This project will develop an innovative therapeutic approach for the treatment of Cystic Fibrosis (CF). This condition originates from the defective function of the CFTR protein, a chloride and bicarbonate permeable transmembrane channel. This project will evaluate small molecules capable of facilitating the transmembrane transport of anions such as chloride and bicarbonate and will thus enable CF treatment by replacing the missing CFTR anion permeation activity. This represents an unexplored path in the treatment of CF and a paradigm shift with respect to current strategies searching for a cure for CF. Instead of focusing on the development of mutation-specific treatments, we plan to develop a therapy applicable to CF patients, regardless of the type of mutation they harbor. Thus, this therapeutic approach overcomes the limitation of current mutation-specific treatments and is applicable to CF patients in general. To achieve this goal we have set up a comprehensive program to validate a research concept and complete the preclinical development of a new lead compound, making it ready for early clinical development. A rmultidisciplinary team of qualified researchers have been assembled to bring to conclusion a truly translational project from the synthesis of new compounds to validation on animal models. Cystic Fibrosis affects more people than any other rare disease. Therefore, it could be said, at least in quantitative terms, that CF qualifies as the main target of the topic. This project aims to complete the preclinical development of novel, innovative drugs based on a radically new concept in Cystic Fibrosis therapies. This result fully addresses the expected impact set out in the work programme of advancing the development of new therapeutic options for patients living with rare diseases as well as contributing to reach the IRDiRC objective to deliver 200 new therapies for rare diseases by 2020.

The novel corona virus causing COVID-19 overwhelmed Europe and the world in just a few weeks’ time. Small and Medium-sized Entrerprises (SMEs are put at very high risk by the slowdown or shutdown of economic life in their countries – exactly those companies who ensure the development of innovative solutions urgently required for counteracting the impacts of COVID-19 and for helping to control possible future outbreaks alike. In view of this situation, accelerated uptake of innovative technologies to tackle COVID-19 is most needed. In order to give an efficient and fast answer to this challenging situation, we propose building an Innovation Hub for the addressed technologies: an innovative open access platform to offer companies and reference laboratories the capabilities, expertise, networks and services required for the assessment, development, prototyping, testing, scaling-up, pilot production and market exploitation of innovative technologies to tackle COVID-19 pandemic. This approach will be based in the Open Innovation Test Beds (OITBs) concept, to incorporate lessons learned from those Innovation Actions, reducing time for putting in place such HUB and ensuring its successful implementation for a swift impact to the current situation. Our approach will include leading applied research and innovation centers in Europe, together with entities specialized in building OITBs, and reference clinical sites. INNO4COV19 will provide funding to third parties to help them achieve the desired objectives. Presence of leading applied research EU institutions in the Consortium will help to obtain a huge impact and to find and attract innovators across Europe to offer new solutions for COVID-19 pandemic and other potential future outbreaks. Europe needs to reinforce its technological leadership and rebuild an industrial sector capable to protect our citizen’s safety and well-being, and INNO4COV-19 will help in catalyzing the change.

280M of people worldwide suffers from major depressive disorders (MDD). Although a well-populated therapeutic landscape of anti-depre280M of people worldwide suffers from major depressive disorders (MDD). Although a well-populated therapeutic landscape of anti-depressants, the number of patients in remission is particularly low with not more than 6% of the patients who benefit from the current therapeutic journey. OPADE objective is to identify key biomarkers that support the decision-making process of the healthcare providers. The project focuses on the microbiota – brain -axis which plays a major role in mental health and in particular MDD. Through clinical investigations, the consortium partners will study the combination between genetics, epigenetics, microbiome and inflammatory networks to: - Establish patient profiles to predict and optimise the efficacy of the antidepressants prescribed with an increase in the remission rate and reduction of impairment of real-life functioning, - Establish the possible correlation between neuroinflammatory indices, target indicators of the microbiome, metabolomics, immune-profile linked, epigenomic, enzymatic algorithms, - Evaluate molecular and non-molecular biomarkers that may represent predictive indices of recurrence - Discover new molecular targets for a personalised approach, - Improve the diagnostic accuracy for primary prevention, - Evaluate retrospectively, using accurate anamnesis, the onset of depressive symptoms in adolescence. - Establish how much and to what extent do blood biomarkers correlate with other specific biomarkers 350 patients between 14 and 50 years will be recruited in 6 EU and international countries for 24 months. Real-time EEG and patient cognitive assessment will be collected with blood, stool and saliva samples. Results and analysis will be used to train the AI / ML predictive tool, the main outcome of the project. A patient empowerment tool will be deployed over the project duration.