Counteracting high attrition rates in oncology drug development and providing optimal therapeutic management of cancer patients require preclinical models that properly recapitulate the complexity and diversity of human tumours. Patient-derived tumour xenografts (PDXs), established by transplanting tumour fragments into immunodeficient mice, are being widely embraced by the scientific community as preclinical tools for target and biomarker discovery. The overall goal of EDIReX is to establish a cutting-edge European infrastructure offering Trans-national Access (TA) of PDX resources to academic and industrial cancer researchers, including the distribution of cryopreserved samples to third parties, the structured biobanking of user-developed models, and the performance of efficacy studies. To ensure interoperability in services, TA initiatives will be backed by Networking Activities (NAs); these will be mainly centred around the establishment of standard procedures for PDX quality control, long-term storage and therapeutic mouse trials. NAs will also entail the adoption of shared ethics parameters for animal experimentation, the wide dissemination of services and project results, and the design of plans to ensure sustainability of the infrastructure. User outreach will be maximised by Joint Research Activities through a three-pronged approach: i) the implementation of a public Data Portal for efficient and user-friendly query and visualisation of clinical, molecular and pharmacological annotation of the models; ii) a cross-validation mouse trial to harmonise inter-laboratory procedures, thus improving the quality and reliability of service provision; and iii) the development of exploratory, more advanced PDX-based preclinical platforms, such as orthotopic and humanised models and in vitro PDX-derived cells. Capitalising on all these assets, EDIReX will contribute to structuring the European Research Area and global cooperation of research infrastructures.

BRECISE is a Public-Private Partnership Project, co-funded by the Innovative Health Initiative (IHI) of the European commission aiming at creating a collaborative ecosystem to accelerate the clinical validation of Next-Generation Sequencing (NGS)-based, multi-modality Artificial Intelligence (AI) oncology biomarkers and related technologies. By addressing technical and regulatory challenges, the consortium unites diverse expertise and resources to validate novel prostate and bladder cancer biomarkers, ensuring their efficacy and clinical utility. This initiative aligns with the goal of advancing precision medicine in oncology, ultimately improving patient outcomes and streamlining personalized cancer care. BRECISE's primary objective is to enhance biomarker-driven approaches for patient risk stratification, disease progression prediction, and treatment response assessment, within the framework of precision medicine. The project addresses the current challenge of limited access to clinically validated prognostic and predictive biomarkers in oncology. By providing healthcare professionals with NGS-based, multi-modality AI oncology biomarkers, BRECISE aims to enable precise disease risk assessment and informed treatment selection, contributing to more personalized and effective patient care. The impact of BRECISE is transformative, aiming to revolutionize risk assessment and treatment selection in oncology, facilitating the implementation of precision medicine. This will significantly reduce unnecessary and ineffective treatments, optimizing patient care and minimizing side effects. The validated biomarker technologies empower researchers to develop safer and more effective personalized treatments, advancing precision medicine. These advancements will enhance the competitiveness of European health industries, positioning them at the forefront of innovation and healthcare excellence. Overall, BRECISE is expected to significantly improve patient outcomes, enhance healthcare efficiency, and bolster the global standing of European health industries.

TransQST will develop a Quantitative Systems Toxicology (QST) approach, employing pre-existing data where possible, in order to yield new mechanistic insight into drug-induced toxicity. A central tenet of our programme will be to ensure the human physiological and pharmacological relevance of any test system that has been (or will be) used for generating the input data for modelling. By adopting this approach, we will be able to accurately interpret what happens when test systems are perturbed by drug exposure, and ensure translatability of modelling tools. Mechanistic translational biomarkers are a core aspect of our approach and will be applied in parallel with evidence for understanding how to develop, model and apply such biomarkers in a QST setting. The project is structured in 8 work packages to provide the following outcomes: curate the best available experimental data suitable for modelling adverse drug reactions; provide fit-for-purpose QST models that will address key toxicity measures for liver, kidney, heart and GI-tract; provide quantitative risk assessment for off-target toxicity in man based on in vitro and in vivo models; provide a quantitative mechanistic read-across from species (in vivo and in vitro) currently used for the toxicological evaluation of a new drug; provide definition and applicability of the human physiological relevance of preclinical test systems; provide a battery of translational biomarkers that can be used for quantitative read-across from in vitro systems to man and which relate to intracellular pathways (and systems) relevant to drug toxicity. Led by the University of Liverpool, TransQST brings together 14 partners, characterized by their scientific rigour and proven track record. Collectively they will enable achievement of the goals of the call, thanks to their complementarity, proven ability to work together (and with EFPIA partners), and their understanding of how to ensure the relevance of QST to human biology.

The EU outermost regions (ORs) face unique challenges and opportunities in the biomedical sector due to their geographical remoteness, insularity, and specific socio-economic conditions. Despite significant R&I initiatives in the past years, which hold great potential to address the health challenges faced by these ORs, the creation of new businesses and jobs remains low. In addition, the lack of specialized biomedical researchers and innovators hampers the ability of ORs to conduct cutting-edge research. FOCUS-4R aims to enhance the R&I capacities of the ORs, helping them to gain autonomy, competitiveness, and attractiveness in the biomedical field. The FOCUS-4R project brings together 3 emblematic ORs (Reunion Island, Canary Islands and Madeira) to leverage the expertise they have developed over recent years to create growth opportunities and increase the excellence of their institutions. Bringing together an intersectoral and interdisciplinary network of 6 academic institutions and 5 non-academic partners from 4 Widening regions and 4 Advanced countries, the project will offer cross-sectoral training and talent mobility through secondments and workshops. FOCUS-4R aims to develop a relevant path to assess therapeutic effects of molecules rapidly and at a low cost without ignoring the ethical issues by focusing on identified techniques that meet the principles of 4R (Replacement, Reduction, Refinement and Responsibility). Through training and mentoring from advanced partners, FOCUS-4R will develop transferable skills essential to meet the industrial and market needs and enhance entrepreneurship in the ORs. By providing training, lifelong learning opportunities and by consolidating knowledge transfer, FOCUS-4R will contribute to bridge the gap between ORs and advanced regions, developing long-lasting collaborations, creating new employment and career opportunities and leading to the creation of a sustainable R&I ecosystem fostering Excellence and Innovation.

Drug repurposing (or repositioning) is the application of previously identified drugs or compounds to treat new indications. The International Rare Diseases Research Consortium (IRDiRC) set as main goal for 2027 that 1000 new therapies for rare diseases will be approved. One of the levers to achieve this goal includes the repurposing of already existing and marketed drugs. In this context, DRUGtrain offers a timely paradigm for multidisciplinary research ideal for improving drug repurposing and development strategies of compounds, with Autosomal Dominant Polycystic Kidney Disease (ADPKD) as example. Identifying compounds for repurposing requires a broad variety of approaches. Therefore, the aim of DRUGtrain is to offer a multidisciplinary research training program to young researchers preparing them to become leading scientists able to integrate molecular and pharmacological data, and translate fundamental research questions to the (pre)clinic and vice versa. The research objectives of DRUGtrain are: 1) To identify potential drug targets and drug candidates for repurposing using innovative bio-informatics and cheminformatics approaches; 2) To perform drug testing/drug screenings in advanced in vitro and in vivo models, ultimately leading to testing of new treatment strategies; 3) To clarify drug mechanisms, and identify and reduce adverse events using state-of-the-art technologies from in silico and wet lab methodologies. Complementary training through courses will cover additional topics in drug repurposing and drug discovery as well as scientific skills, personal development, commerce and entrepreneurship. In DRUGtrain, 6 academic and 4 private sector beneficiaries/partner organisations and 1 NGO are collaborating in joining forces to intensify network activities between academia, industry, and a patient organization. The training offered should support the ESRs in becoming the next generation of true multidisciplinary researchers in (bio)pharmaceutical sciences.