Orchard Therapeutics announced that the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) has published encouraging results from a clinical trial of OTL-300, an autologous ex vivo lentiviral gene therapy program being studied in individuals with transfusion-dependent beta-thalassemia. The paper, published in Nature Medicine, provides an interim analysis of long-term efficacy outcomes in seven of the nine treated patients with more than one year of follow up from this ongoing trial. The clinical trial conducted at SR-Tiget in Milan is led by Giuliana Ferrari, professor at The Vita-Salute San Raffaele University and made possible by the strategic alliance between San Raffaele Hospital, Telethon Foundation and Orchard Therapeutics, following the transfer of GSK’s gene therapy portfolio to Orchard in April 2018. The publication of the trial results was the result of the collaboration between basic researchers and clinicians, and in partnership with the Pediatric Immunohematology Unit headed by Alessandro Aiuti, the Hematology and Hematopoietic Stem Cell Transplantation Unit headed by Fabio Ciceri, and the Rare Diseases Centre at the Policlinico Hospital headed by Maria Domenica Cappellini. The trial, with the clinical coordination of doctor Sarah Marktel of SR-Tiget, has involved other Italian centers specializing in thalassemia and the cooperation of patient organizations.