Onco-Summaries: Daily Oncology Updates at a Glance

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, and outcomes for relapsed or refractory patients remain poor. The LTI-214 program embodies our commitment to advancing new immunotherapy approaches that are both potent and safer for patients. This designation represents an important step toward our goal of transforming the treatment paradigm for AML.”

  
  

• Nathan Dolloff, Founder and CSO, Leukogene: “This is an important step forward for Leukogene and the company’s Major Histocompatibility Complex Class II (MHCII) engager technology. The M2T™ platform is a completely new approach to cancer immunotherapy and the endorsement from FDA is a testament to its high impact potential.”