05/11/2025 Leukogene Therapeutics' LTI-214 received the FDA orphan drug designation for AML (Ref) The US FDA granted the orphan drug designation to Leukogene Therapeutics' LTI-214 (M2T-CD33; myeloid-targeted immunotherapy) for the treatment of Acute Myeloid Leukemia (AML). Sandeep Gupta, CEO, Leukogene: “We are honored that the FDA has recognized the therapeutic promise of LTI-214 by granting Orphan Drug Designation. AML remains one of the most challenging hematologic cancers