New Gene Therapy Lab at the Bambino Gesù Children’s Hospital – IRCCS

28 October 2025, Rome — The National Center proudly announces the inauguration of the new Gene Therapy Research Laboratory at the Bambino Gesù Pediatric Hospital (IRCCS) in Rome. This milestone marks a significant advancement in Italy’s national strategy to strengthen gene therapy research and clinical innovation, reaffirming the country’s leadership in medicine and translational science.

The event coincided with the 40th anniversary of Bambino Gesù’s designation as a Scientific Institute for Research, Hospitalization and Healthcare (IRCCS), a recognition first granted in 1985. The inauguration therefore celebrated not only a state-of-the-art scientific achievement but also four decades of excellence and innovation in pediatric care and biomedical research. The establishment of the new Gene Therapy Laboratory was made possible thanks to the decisive contribution of the National Center, which funded the project through PNRR funds for a total amount of approximately €19.5 million.

Held in the Auditorium of the San Paolo Fuori le Mura Campus, the ceremony was attended by key representatives of Italy’s scientific and institutional community, including:

  • Prof. Tiziano Onesti, Presidente dell’Ospedale Pediatrico Bambino Gesù
  •  Sua Eminenza Reverendissima Cardinale Pietro Parolin, Segretario di Stato
  • Prof. Roberto Gualtieri, Sindaco di Roma
  • Avv. Francesco Rocca, Presidente della Regione Lazio
  •  Prof. Orazio Schillaci, Ministro della Salute
  • Prof. Andrea Onetti Muda, Direttore Scientifico dell’Ospedale Pediatrico Bambino Gesù

Their presence underscored the unity of purpose between national institutions, regional authorities, and the healthcare sector in supporting scientific progress and ensuring access to cutting-edge therapies.

During the ceremony, Prof Franco Locatelli, Head of the Department of Pediatric Hematology and Oncology and Cell and Gene Therapy at Bambino Gesù, offered an overview of the National Center’s mission and achievements. Presenting the new facility and its state of the art equipment, Prof Locatelli emphasized how coordinated national investments are accelerating the development of gene therapy approaches from preclinical research to clinical application. He also highlighted the close collaboration between research institutions, government agencies, and healthcare organizations, a partnership that has been fundamental in achieving this milestone and that continues to drive Italy’s leadership in the field of precision medicine.

The new Gene Therapy Laboratory occupies approximately 700 square meters and is dedicated to the research and development of genetically modified cell drugs, which will be produced in the hospital’s adjacent pharmaceutical manufacturing facility. With an infrastructure investment of around €3.5 million and new technologies worth €6.5 million, the laboratory represents a significant scientific and strategic investment. It also includes substantial resources devoted to researchers and clinicians, whose expertise will enable the development of innovative clinical trials targeting oncological, genetic, and metabolic diseases.

Within the framework of the National Center, Bambino Gesù coordinates the activities of Spoke 10, which focuses on preclinical development, good manufacturing practices, and clinical testing of gene therapy medicinal products. Through this collaboration, the hospital works alongside numerous Italian public and private centers of excellence, forming a national network that integrates expertise, facilities, and scientific knowledge. This coordinated approach strengthens Italy’s ability to translate research discoveries into therapeutic solutions, offering pediatric patients access to advanced, personalized treatments.

The new Gene Therapy Laboratory stands as a national and international reference point for innovation in pediatric gene therapy. It symbolizes the convergence of scientific ambition, public investment, and collaborative research, providing the infrastructure and talent necessary to bring cutting-edge therapies from the laboratory to the clinic.

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