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Prof. Locatelli gave a fantastic presentation at the recent ESGCT conference in ROME, on gene editing, titled “Haemoglobinopathies gene Therapy”


Prof. Locatelli, one of the most important Researchers in ATMP arena, is the Head of the Department of Paediatric Haematology and Oncology, IRCCS Bambino Gesù Children’s Hospital, Rome and Full Professor of Pediatrics at the Sapienza, University of Rome, Italy. He leads the largest programme of childhood allogeneic haematopoietic stem cell transplantation (HSCT) in Italy and in 2019 was appointed President of the Italian Higher Council of Health, the technical scientific advisory body to the Ministry of Health. He is also serving as coordinator of the Technical-Scientific Committee for the COVID-19 pandemic.

Professor Locatelli is an expert in haematological and oncological malignancies of childhood. He has been the President of the Italian Association for Pediatric Hematology-Oncology AIEOP from

Prof. Locatelli gave a fantastic presentation in the Plenary Session, of the recent ESGCT conference in Rome in the splendid setting of the Rome Convention Center called “Nuvola”.

The Rome Convention Center, the masterpiece of the architect Massimiliano Fuksas is a work of extraordinary artistic value, characterized by innovative solutions, an eco-compatible approach and technologically advanced materials, built within a context that is dominated by the architectural rationalism of the 'Thirties and 'Forties.

The Plenary session was full of people, extremely interested in the presentation with many questions at the end and great appreciation for the speaker.

Prof. Locatelli with his presentation matched the ESGCT objective, showing his recent results in clinical gene therapy, facilitating education, the exchange of information and technology to stakeholder communities and regulatory bodies in Europe.

Some of his final results were outstanding underlying how gene editing:

  • Represent apromising potentially curative approach in patients with Hbpaties
  • Offer the advantages of being applicable in any patient with a preserved hematopietic reservoir and a limited iron overload related organ damage
  • Most patients treated so far had early and sustained increases in total hemoglobin with pancellular distribution

Prof. Locatelli concluded that the long-term safety profile of these innovative therapies needs to be carefully monitored.

If you would like more information, you could visit the ESGCT Conference website https://www.esgct.eu

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