GeneH proposes to create an excellence hub in gene therapy through strategic partnerships within and beyond Widening countries. Despite gene therapy's recognized priority in genomic medicine and its potential for high-curative economic impact, challenges persist in its development process, from conceptualization to commercialization. GeneH intends to address these challenges by serving as a catalyst for overcoming barriers and expediting research translation. To achieve this, GeneH will establish a robust and sustainable hub that builds upon already established ecosystems in the field and recent H2020 WIDENING projects to implement centers of excellence in gene therapy (GeneT in PT and CTGCT in SL). The academic partners at its core, namely, the University of Coimbra (UC) in Portugal and the National Institute of Chemistry (KI/NIC) in Slovenia , have reached substantial achievements by partnering with industry (Bluepharma, Biocant Park, Biosistemika, Jafral), hospitals (CHUC EPE), policy makers (CCDRC, RRA LUR), ecosystem/innovation supporters (SIH EEIG, IPN), and patient associations (APAHE, ZOPS). Partners in the two ecosystems share a common goal: treating life-threatening or chronically debilitating diseases considered incurable. By harnessing the combined expertise and resources of its partners, GeneH aims to drive cross-sectoral collaboration through its 4-helix network, in a multidimensional approach that will gather further investment, address ethical and regulatory aspects, implement de-risking approaches, educational and training programes, joint R&I programs, and prepare the path for clinical trials of new gene therapy products, while actively engaging society. By positioning itself as a leading hub in gene therapy, in line with regional, national, and European strategies, GeneH seeks to contribute to European competitive leadership globally, advancing healthcare, fostering economic growth, and making a positive societal impact.

Europe still sees a quarter of the world's cancer cases each year, making cancer the second leading cause of death and illness in the region after cardiovascular diseases. Unless we take decisive action, lives lost to cancer in the EU are set to increase by more than 24% by 2035, making it the leading cause of death in the EU. Cross-border collaboration can address this challenge by combining data from various modalities and sources, extracting meaningful insights to deepen our understanding of cancer. However, ethical, legal, and national regulations, along with data access processes, including differing interpretations of the EU GDPR create significant hurdles. Technical interoperability issues across European cancer RIs, and patients' and citizens' rights to control who uses their personal information and for what purposes further complicate data sharing. The project will provide European researchers, SMEs, and innovators with a decentralized collaborative network, “UNCAN-CONNECT,” for cancer research. It consists of both technical components, a governance, compliance, and operational framework based on the UNCAN blueprint, with the goal of operationalizing it. The objective is to facilitate access to cancer data, promote open science, and revolutionize cancer research and treatment by co-creating an open-source federation of federations platform. It will be developed using specific use cases focused on six major cancer types: Paediatric, Lymphoid malignancies, Pancreatic cancer, Ovarian, Lung, and Prostate cancers and active collaboration with a diverse range of stakeholders, including researchers, SMEs, industrial end users, and citizens. It will build on existing European RIs such as BBMRI as well as initiatives like EOSC4CANCER, CanSERV, EUCAIM, to enable seamless storage, access, sharing, and processing of data across Member States and associated countries. This approach will foster interoperability and collaboration, accelerating progress in cancer research. This action is part of the Cancer Mission clusters of projects 'Understanding' established in 2022.

Brain conditions such as neurodegenerative diseases, stroke and traumatic injuries are a mayor burden in modern society. Because neural stem cells (NSCs) can differentiate into new neural cells including neurons, the regulation of their proliferation, differentiation and migration represent a promising regenerative/therapeutic strategy. The hypothesis of NanoStem project is that novel nanoparticles (NPs) combined with recent identified/developed pharmaceuticals can be an efficient approach to control the biological activity of NSCs. The scientific project is organized in three work packages (WP), each focusing on specific objectives and containing well-defined deliverables. WP1 will focus on the synthesis of the nanomaterials and the uploading of the therapeutics. WP2 will take the best nanoparticles and focus more on the evaluation of the permeation and efficacy of the formulations against NSCs. WP3 will focus on in vivo studies to evaluate the formulations developed to tackle the brain and to assess their toxicity. The project will train 14 ESRs in areas including organic and polymer chemistry, cell biology, pharmacology, neuroscience, nanobiotechnology and clinical sciences. The project will also provide a substantial training program in complementary skills, targeting both professional and personal development, and translation of research and entrepreneurship. The project brings together six academic partners, one hospital and two industrial teams as full partners, complemented by four associate partners, one of which is an industrial team, distributed over a total of seven European Union member states. The consortium brings together groups with a very interdisciplinary expertise ranging from nanoparticle synthesis and characterization, computational modeling, in vitro BBB models and BBB transport, neural stem cells, animal testing and clinical expertise.

GLORIA aims to prove that the addition of chronic low dose glucocorticoids (GC) to current antirheumatic therapy is highly cost-effective and safe in elderly patients with rheumatoid arthritis (RA). RA is a frequent (affecting > 2% of the elderly population), painful and disabling chronic disease with high societal costs. RA is associated with multiple comorbidities, polypharmacy and adverse events; these problems, together with challenges in compliance (adherence) are dramatically increased in the elderly population. About 50% of patients are chronically treated with low-dose glucocorticoids (GC) in combination with other antirheumatic drugs, but without good evidence on the balance of benefit and harm. Thus, existing guidelines and information on safety and efficacy of GC are inadequate. GLORIA will address these problems by conducting a large pragmatic trial: 800 elderly (>65y) RA patients receiving standard of care will be randomized to additionally receive 5 mg prednisolone daily or placebo for 2 years. Very liberal eligibility criteria will ensure representativeness to the target population, and most data will be collected from routine clinical practice, minimizing patient and physician load, and operating costs. A novel tool will monitor compliance; it can send personalized reminders to a patient’s smart device. The efficacy of this technology will be tested in a nested trial. Compliance and other characteristics will be entered into a model that will allow personalized risk and benefit assessment in the future. Qualitative research in patients and physicians of member states will explore expectations and challenges in guideline implementation. This information and the study results will enable an update to existing guidelines and patient information, in collaboration with guideline committees and regulatory agencies. Networking conferences will improve health technology assessment in the elderly in general.

Cerebrovascular diseases (CVD) represent a major public health burden in Europe, afflicting, in particular, Easter Widening Regions. By bringing together a multidisciplinary and cross-sector agenda, CHAngeing will transform and interconnect two quadruple helix-based excellence ecosystems located in southern Europe (Portugal and Greece). This will be accomplished by science-based interventions: 1) promoting healthy lifestyles supported by Mediterranean diet culture to prevent CVD; 2) improving health technology-based rehabilitation of stroke patients. The objectives of CHAngeing are aligned with call outcomes, are supported by the Smart Specialization Strategy (RIS3) of both regions, and will contribute to deliver impact in both hubs and between hubs, in a synergistic manner: 1) reinforcing the excellence of both hubs as poles of attraction of new talents; 2) developing a common R&I strategy on ageing research, supported by action and investment plans; 3) promoting a culture of cross-sector fertilization in R&I and student’s training in both regions and across regions; 4) building strong links between research and business, filling innovation gaps and foster knowledge transfer and job creation; 5) implementing pilots and demonstrators towards healthy lifestyles and rehabilitation of stroke patients; 6) efficiently communicate, disseminate and exploit results of CHAngeing, ensuring high societal impact and long-term sustainability. The proposal encloses seven work packages: WP1- Coordination and management; WP2- Exploitation and sustainability (action and investment plans); WP3- R&I strategy; WP4- Advanced training and twinning; WP5- Connecting academia & business; WP6- Pilots & demonstrators; WP7- Communication, dissemination and societal impact. CHAngeing will be a catalyser of excellence synergistically supported by the success of established flagship projects in both regions, including Teaming for Excellence, ERA Chairs, Twinning, ERC’s, REGPOT, AAL and EIT Health