Leading a New Era in Rare Disease Treatment - Italy's First Phase 1 Clinical Trial Center

Modena 28 February 2025 – Committed to advancing innovative treatments for rare diseases, establishing Italy’s first Phase 1 Clinical Trial Center (CTC) is a significant milestone of the National Center’s mission.

Foreseen to be operational by the end of 2025 at the University Hospital of Modena, the CTC facility represents a crucial step in accelerating the bench-to-bedside, thus supporting the initial human trials of RNA-based drugs developed by National Center researchers.

Designed to bridge the gap between preclinical studies and human trials, the state-of-the-art CTC facilities will ensure the safety, efficacy, and feasibility of cutting-edge therapies for rare genetic diseases, including those affecting the skin, blood, eyes, kidneys, neuromuscular system, and metabolism.

A key feature of the CTC is its integrated GLP (Good Laboratory Practice) laboratory, which enables the collection of pharmacokinetic and biomarker data—critical elements for advancing clinical trials and refining treatment strategies. Additionally, its proximity to the Spoke 1 Flagship Laboratory, home to a next-generation DNA sequencing platform, allows researchers to conduct high-precision molecular and genomic analyses.