Thalassaemia: gene therapy makes its debut at the Brotzu Hospital in Cagliari

He has not needed any transfusions for a month. This is the patient, who suffers from thalassaemia major, who was treated with gene therapy at the ARNAS Brotzu Hospital in Cagliari. The Brotzu Hospital in Cagliari is the only accredited centre in Sardinia for this procedure and is among the first in Italia. This is an initiative that the regional government considers strategic and which aims to guarantee Sardinian patients access to the most innovative treatments.

“This is an extraordinary achievement for our healthcare system and, above all, for people living with thalassaemia,” said Alessandra Todde, President of the Region and Acting Councillor for Health. “ “This achievement demonstrates that, even in Sardinia, it is possible to guarantee access to the most advanced treatments and to transform scientific innovation into new treatment options.”

Todde then highlights another aspect, specifically linked to the various forms of Mediterranean anaemia and the hardships and challenges faced by patients and their families. ‘Sardinia knows the burden of thalassaemia better than any other region. That is why we have fought to ensure that our region could, for the first time, gain access to the National Fund for Innovative Medicines, and we will defend the allocation earmarked for Sardinia at all costs,’ he adds. ‘We have also chosen to invest directly in innovation, combining regional resources with national ones, because access to treatment must be determined by people’s clinical conditions,’ he adds.

This strategy also includes the new apheresis service at Arnas Brotzu, commissioned by the Regional Government and inaugurated in February 2026, which represents a further step towards strengthening the range of care and treatment services on offer. “Behind this achievement lie years of work and, above all, the expertise of the professionals at Brotzu, including the three centres at Businco, Microcitemico and San Michele, whom I would like to thank for their tireless efforts and for the quality of care they provide every day,” concludes Todde. “It is vital to support this initiative. We now have our first tangible result. The public health service must have this ambition: to bring the most advanced therapies to Sardinia and transform scientific research into new treatment options for people’.