Genespire
Biotechnology ResearchLombardy, Italy11-50 Employees
Genespire is a Milan-based biotechnology company focused on developing durable and transformative gene therapies for genetic diseases, with a particular emphasis on primary immunodeficiencies and inherited metabolic disorders. It was founded in March 2020 as a spin-off of SR-Tiget, a cell and gene therapy research institute, by Prof. Luigi Naldini, Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. The company operates within the biotechnology research landscape aiming to bring therapies to pediatric patients affected by these conditions. In 2025, Genespire reported positive preclinical data on a liver-directed in vivo gene therapy for methylmalonic acidemia, presented at the ASGCT annual meeting in Baltimore. The firm is pursuing a proprietary approach to gene delivery, including the development of a vector platform with lead candidate GENE202 that is moving toward clinical development.