A First-in-human Clinical Trial Using a Gene Therapy With Patient's Own Stem Cells to Treat Early Type 1 Diabetes

The intervention is intended for newly diagnosed T1D patients with residual beta-cell function, who can receive the Drug Product (DP) within 180 days from the first insulin administration. Mobilized autologous HSPCs, collected by leukapheresis, are ex vivo transduced with a LVV encoding for hPD-L1 and formulated in a cryopreservation medium containing DMSO (DP). After release, the DP is thawed and administered to the patient at a dose ranging between 5 and 20 million of cells per kilogram of body weight. The intervention plans for a single DP injection.