XJ-Bio has recently secured orphan drug status from the US Food and Drug Administration (FDA) for its groundbreaking cellular therapy, XJ-PLT-001, designed for platelet injection. This therapy is specifically tailored to address Bernard-Soulier syndrome. Derived from hematopoietic stem cells, XJ-PLT-001 leverages a directed differentiation system to produce high-quality platelets, distinguished by its absence of gene editing and residual contaminating cells.