ReachGLIO aims to improve glioblastoma therapy by combining blood–brain barrier opening strategies with targeted, drug-loaded nanoparticles. The project evaluates SNGR‑TNF (a stable derivative of NGR‑TNF) alongside standard chemotherapy and selects nanomedicines designed to penetrate the tumour and reach cancer stem cells. Candidate formulations are tested in advanced BBB/BTB models and 3D glioblastoma models, and then validated in mouse models and in spontaneous gliomas in dogs. Raman spectroscopy-based imaging makes it possible to track drug distribution and release, generating preclinical evidence to accelerate clinical translation.
Enfermedad Archives
Machine Learning and Artificial Intelligence for Nanomedicine Optimization
MELINA drives an AI platform in nanomedicine that combines curated data, standardized experimental workflows, and advanced machine learning models to optimize therapeutic nanoparticles. The project integrates information on nanoparticle synthesis, physicochemical characterization, and biological evaluation in cellular models to identify which variables most influence efficacy and safety. It also incorporates high-throughput methods, image analysis, and Quality by Design strategies to guide the development of new formulations in a more systematic and reproducible manner. By reducing trial-and-error and sharing data and tools openly, MELINA aims to accelerate innovation in nanomedicine and strengthen translational research.
Lipid replacement therapy for patients with congenital ichthyosis using nanostructured topical delivery systems of acylceramides.
Hereditary ichthyoses are rare genetic diseases (13.3 cases per million in Europe) that appear at birth or in early childhood, causing scaling, skin thickening, dryness, itching, and pain. Approximately half of the patients, especially those with Autosomal Recessive Congenital Ichthyosis (ARCI), present a defect in the synthesis of CerEOS, a ceramide essential for the epidermal barrier. Current treatments are only symptomatic and not very effective. The LIPARCI II project develops a lipid replacement therapy based on CerEOS encapsulated in biomimetic nanoparticles, capable of restoring the skin barrier in preclinical ARCI models. Additionally, LIPARCI II includes a phase 0 clinical trial to evaluate bioavailability, will advance large-scale production, and will develop new models and formulations to expand the number of beneficiary patients.
Hybrid extracellular vesicles for advanced treatment of lysosomal storage diseases
HEAL aims to transform the treatment of lysosomal storage disorders, focusing on Fabry disease, through third-generation nanoparticles that combine extracellular vesicles and liposomes (EVs) to deliver highly active enzymes to target organs. The project integrates artificial intelligence to optimise design, targeting and scalable manufacturing, and evaluates efficacy in advanced preclinical models that better replicate human disease. The goal is to improve stability, biodistribution and potential access to the brain, overcoming the limitations of conventional ERT.
Interdisciplinary doctoral training in oligonucleotide-based therapies for Myotonic Dystrophy
Rare diseases pose significant diagnostic and therapeutic challenges, and Myotonic Dystrophy (DM), the most common hereditary muscular dystrophy in adults, remains without effective treatments due to its genetic complexity and the limited efficacy of antisense oligonucleotides evaluated. ENTRY-DM brings together an interdisciplinary and intersectoral consortium with 9 academic and 2 non-academic partners to train 14 high-potential doctoral candidates in ASO design and delivery, disease mechanism research, advanced experimental models, and clinical trial preparation, creating a framework transferable to other RNA-mediated diseases and aligned with European and international priorities in rare diseases. By integrating cutting-edge science, international mobility, and interdisciplinary training to overcome barriers, from genotype-phenotype correlations to ASO biodistribution, the program strengthens European leadership in ASO-based therapies and lays the foundation for future clinical trials.