RNA molecules play key roles in biological processes and diseases. An emerging avenue to develop innovative medicines is therefore to design small molecule drugs that bind to RNA and affect downstream biological functions. However, to advance this field of research, a new generation of scientists is needed that can further develop and ultimately master the challenging process of drug design applied to RNA. Therefore, the TargetRNA network aims to educate and nurture the next generation of scientists with cutting-edge interdisciplinary skills in the discovery and biological characterization of RNA modulators and thus generate fundamental knowledge on what drives affinity and specificity of small molecules binding to RNA. The objective of TargetRNA is to develop compounds with antimicrobial activity through their interaction with RNA targets, and to use these to selectively reshape the human gut microbiome, an area with vast implications for medicine. Training through research activities, spanning from computational and medicinal chemistry over structural and molecular biology to microbiology and in vivo models, will be supplemented by an innovative training programme in specific and transferable skills for interdisciplinary drug discovery strategies to target RNA, including FAIR data principles, open science practices as well as responsible development and sustainable implementation of new innovations. To achieve the ambitious research and training goals, the TargetRNA network unites leading scientists from 13 academic partners (8 beneficiaries, 5 associated) and 4 industry partners (1 beneficiary, 3 associated) based in 9 countries.

EU-PEARL has the ambition of transforming the current approach of conducting single-compound clinical trials into the use of cross-company Integrated Research Platforms (IRPs), taking into consideration both patients’ interests and the opportunities from novel molecules for addressing medical needs. Patient-centric data and knowledge sharing have the potential to accelerate the development of new treatments and reduce the operational costs of clinical trials. EU-PEARL will improve clinical effectiveness, patients’ satisfaction and societal access to timely and affordable medicines and it will shape the clinical trials of the future. This will change the industry paradigm from competition to cooperation in four disease areas and provide the framework for designing IRPs in other disease areas. The main objectives of EU-PEARL are: (1) To create a reusable, accessible and sustainable modular IRP for the design and execution of patient-centric, cross- company IRP in any disease area with unmet needs; (2) To set up the open, dynamic, patient inclusive IRP governance structure that will manage the appropriate regulatory, ethical, legal, statistical and data utilisation requirements of the IRP; (3) To disseminate and exploit the EU-PEARL paradigm through the provision of the necessary common tools, procedures, expertise and operational skills working to the highest scientific, regulatory and ethical standards and best practices, developed jointly by public and industry partners in a consensus-based approach; and (4) To create trial-ready IRP networks in the four disease areas of Major Depressive Disorder (MDD), Tuberculosis (TB), Non-Alcoholic Steatohepatitis (NASH) and Neurofibromatosis (NF).

Europe is generating huge amounts of patient-level information contained in Electronic Health Record (EHR) systems and other types of health databases. These include structured data in the form of diagnoses, medications, laboratory test results, etc., and unstructured data in clinical narratives. The Electronic Health Data in a European Network (EHDEN) Consortium leverages these vast volumes of data to improve future clinical practice and individual patient outcomes by increasing our understanding of disease and treatment pathways. EHDEN will galvanize transparent and reproducible analytics that will generate valid real-world evidence to improve patient care, and enable medical outcomes-based research at an unprecedented scale. The EHDEN Consortium provides the infrastructure and eco-system supporting disease-specific projects in the IMI Big Data for Better Outcomes (BD4BO) programme. The core of EHDEN is the use of a common data model (OMOP-CDM), standardised outcome assessment (ICHOM), and transparent open-source analytics (OHDSI). The objective of the EHDEN consortium is to provide all the necessary services that enable a distributed European data network to perform fast, scalable and highly reproducible research, while respecting privacy regulations, local data provenance and governance. This will include services and tools to perform data standardization, analytical pipelines, tools to share study results, and tools for stakeholder engagement and training. The EHDEN Consortium combines active participation of stakeholder representatives with proven experience in: a) integrating different data types, methods and technologies to utilize diverse clinical datasets; b) platform development to make methods and datasets Findable, Accessible, Interoperable and Reusable (FAIR); and c) engaging a wide variety of stakeholders, including health technology assessment agencies, regulators and patients.