Ingenium Therapeutics, a company in next-generation NK cell therapies, has announced the receipt of Orphan Drug Designation from the US Food and Drug Administration (FDA) for its investigational therapy IGNK001 (Gengluecel) in the treatment of acute myeloid leukemia (AML).

The company's innovative approach utilizes proprietary technology to differentiate and proliferate natural killer (NK) cells, resulting in superior anti-cancer efficacy. By specifically targeting CD3-negative immune cells, Ingenium can generate a large quantity of highly pure NK cells within a short timeframe. This allogeneic NK cell therapy, derived from healthy donors, exhibits potent anti-cancer effects characterized by high purity and activity.

Ko Jin-ok, CEO of Ingenium Therapeutics, expressed excitement about the Orphan Drug Designation, stating, "We are delighted to have received Orphan Drug Designation for IGNK001 from the FDA, recognizing the potential of our innovative NK cell therapy technology." 

He further elaborated on the progress of Gengluecel, highlighting its approval for phase 2 clinical trials by the Korean Ministry of Food and Drug Safety. Additionally, preparations are underway to initiate clinical trials in the US, targeting 80 subjects with the objective of completing studies by December 2027.

The Orphan Drug Designation comes with various development incentives for Ingenium, including tax credits for qualified clinical trials, exemption from FDA application fees, and the potential for a seven-year period of market exclusivity upon regulatory approval.