The San Raffaele Scientific Institute is pleased to announce that Prof. Danilo De Gregorio, researcher and project leader of the Division of Neuroscience, is the winner of a European ERDERA (European Rare Diseases Research Alliance) grant, co-funded by the Fondazione Regionale per la Ricerca Biomedica (Regional Foundation for Biomedical Research, FRRB).

Prof. De Gregorio’s project has received total funding of 360,000 euros and is part of a research consortium involving six national and international partners, coordinated by McGill University (Montreal, Canada). The project is expected to last up to a maximum of three years.

What the research project involves

Prof. De Gregorio’s lab will participate in an international research project aiming to test the therapeutic potential of an already patented molecule for two rare diseases associated with autism, specifically Fragile X syndrome (FXS) — a rare genetic condition that can affect cognitive development, behavior, and communication skills — and Phelan–McDermid syndrome (PMS), which is characterized by delays in motor and intellectual development.

Currently, there are no specific treatments available for these syndromes. The overall project, which includes Prof. De Gregorio’s research group among its participants, aims to evaluate the therapeutic potential of COS01, a new drug that targets the melatonin MT2 receptor, that has already demonstrated efficacy and safety in modulating sleep and certain brain functions. Using preclinical models of the two syndromes, the study will assess the effects of this molecule on sleep, social behavior, and other symptoms typical of autism spectrum disorders, with the goal of making it ready for clinical trials in humans.

Who is Prof. Danilo De Gregorio

An internationally trained pharmacologist, Prof. De Gregorio earned his degree in Pharmacy from the University of Naples “Federico II” and his PhD in Pharmacological Sciences and Experimental Medicine from the University of Campania “Vanvitelli.” After a postdoctoral research period in the Department of Psychiatry at McGill University in Montreal, Canada, he returned to Italy in 2021 as Associate Professor of Pharmacology at Vita-Salute San Raffaele University and project leader of the Neuropsychopharmacology group.

His research combines advanced behavioral and electrophysiological techniques in animal models to study the psychopharmacology of psychoactive compounds—including hallucinogens and cannabinoids—and their role in mental disorders. His work, published in leading journals such as Nature, PNAS, and Nature Communications, has earned him numerous awards and research grants, including funding from the Canadian Institutes of Health Research and the Fonds pour la recherche en santé du Québec.

Published on 19/12/2025