If so far over 400 people in the world, with an unfortunate diagnosis and little hope of life, are in good health conditions, it is due to the discoveries of the Italian scientist Luigi Naldini, pioneer in the field of gene therapy and “father” of lentiviral vectors, tools today used in clinics and derived from one of the most feared human viruses, HIV. After a life spent in research, starting from the mid-nineties, and numerous awards first in the United States and then in Italy, for Naldini – since 2008 director of the San Raffaele-Telethon Institute for gene therapy (Sr-Tiget) of Milan and full professor of Histology and Gene and Cellular Therapy at the Vita-Salute San Raffaele University – now comes the Phacilitate Lifetime Achievement Award 2024 for innovations in the field of advanced therapies. «Without his dedication and perseverance – said Sven Kili, president of the jury during the ceremony on January 18 in Miami, Florida – gene therapy would not be where it is today, capable of changing patients’ lives thanks to drugs currently approved for genetic diseases such as Ada-Scid and metachromatic leukodystrophy, not to mention the numerous others still in the clinical development phase”. At the San Raffaele Hospital alone, using blood stem cells corrected via lentiviral vectors, almost a hundred patients with serious genetic diseases such as metachromatic leukodystrophy, Wiskott-Aldrich syndrome, beta thalassemia and type 1 mucopolysaccharidosis have been treated. Meanwhile, the commitment of the director of Sr-Tiget continues: «I will continue to work with renewed energy and enthusiasm – commented Naldini – in the search for solutions based on gene and cell therapy, for the many patients who need innovative therapies to deal with diseases hitherto considered fatal.”
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