University of Milan (P.I. Paolo Ciana) boasts internationally recognized technical and scientific expertise in preclinical and clinical pharmacology, particularly focusing on biotech drugs. The research groups excel in developing innovative experimental models for in vitro and in vivo studies (zebrafish and mice), with a well-recognized expertise in toxicology and immunology. The affiliate researchers organize and participate across a large interdisciplinary national and international network dedicated to drug research and development, molecular biology, and genetics. Furthermore, they maintain strong collaborations with both international and national biotech and pharmaceutical companies. The University of Milan has an experienced Technology Transfer Office (TTO) that provides vital support for the translation of biomedical research. Finally, the pharmacological expertise includes clinical pharmacology that enjoys close interactions with regulatory bodies within the European Union and throughout Italy.

National Research Council of Italy – CNR (coordinator Daniele Catalucci).
CNR participants possess expertise in studying the molecular mechanisms underlying the synapse dysfunction and the influence of immune molecules on this process to identify new targets suitable for innovative treatments. CNR studies also include exploring the molecular mechanisms underlying the contractility/relaxation of cardiomyocytes and the formulation of biomimetic nanoparticles for the targeted delivery of specific drugs to the heart.

Humanitas University – HUNIMED (coordinators Michela Matteoli and Simona Lodato).
HUNIMED participants investigate neurodevelopment, neurodegenerative, and psychiatric diseases. The affiliate possesses established expertise in iPS-derived brain cells, 3D brain organoids, and RNA drugs, which potentially target complex CNS diseases. Additionally, HUNIMED has extensive experience in RNA drug delivery within the Central Nervous System (CNS) in animal models using various methodologies, as well as in modelling the blood-brain barrier through microfluidics and lab-on-chip systems.

University of Naples Federico II – UNINA (Coordinator: Angelo Izzo).
UNINA participants possess extensive expertise in developing 2D/3D models and organoids (including EMT, methylomics, and transcriptomics) as well as transgenic models for studying pharmacodynamics, efficacy, and organ/systemic toxicity. Animal models are available for various conditions including cardiovascular and metabolic diseases, inflammation, cancer, dermatology, skeletal muscle diseases, neuropharmacology, respiratory, and gastrointestinal pharmacology/toxicology. Additionally, a UNINA research group specializes in creating pharmacoeconomic models and frameworks for building regulatory reimbursement models using Real-World Evidence (RWE).

University of Padua – UNIPD (Coordinator: Marisa Brini).
The UNIPD group enjoys international recognition for having developed SPLITS reporters for microscopy applications to map organelle contact sites both in vitro and in vivo. This expertise is essential for leveraging split-Green Fluorescent Protein (GFP) technology in testing the efficiency of nucleic acid-based therapies. Additionally, the group possesses well-established skills in isolating, characterizing, and applying extracellular vesicles in drug therapy, along with expertise in conducting clinical assays to assess the toxicity of gene therapy with a particular expertise for liver toxicity.

University of Pavia – UNIPV (Coordinator: Maria Cristina Bonferroni).
UNIPV participants possess extensive expertise in the utilization of natural polymers in drug delivery, particularly for treating local infections and promoting tissue repair, while also assessing safety profiles. Additionally, the affiliate offers several models for testing RNA drugs and their carriers, with specific expertise in neuroinflammation.

Sapienza University of Rome – UNIROMA1 (Coordinator: Arca Marcello).
UNIROME1 participants have consolidated experience in the treatment of rare disorders of lipid metabolism with antisense RNA technology. They have developed innovative approaches by using extracellular vesicles (EVs) for delivery targeted RNA therapy and organoid models for several diseases including familial chylomicronemia syndrome (FCS), homozygous familial hypercholesterolemia (HoFH), BRAF mutated thyroid cancer, and Amyotrophic Lateral Sclerosis (ALS).

Antares Vision contributes to the design of quality control methodologies and offers smart data and tools for the digitalization of the process.

Chiesi Group contributes through its collaboration with Moderna Inc., on identifying RNA drugs most suitable for development and defining technologies for studying RNA drug distribution, with particular emphasis on translating dosage studies conducted in laboratory animals into doses for human use.

Dompé Pharmaceuticals focuses on RNA vaccine immunotoxicology to develop new methods in vitro and in vivo models for immuno-toxicological evaluations, as well as Good Laboratory Practice (GLP) toxicology studies.