In a potential development for clinical trial translations to come, the U.S. Food and Drug Administration (FDA) authorized orphan drug designation (ODD) to Nkarta’s engineered natural killer (NK) cell therapy NKX101 for the treatment of acute myeloid leukemia (AML). AML is a blood cancer that disrupts the production of normal blood cells in the bone marrow and the five-year survival rate is 26% for patients diagnosed with the condition. NKX101 is an investigational, off-the-shelf cancer immunotherapy that uses NK cells derived from the blood of healthy donors and is engineered to induce a cell-killing immune response through the detection of stress ligands that are widely expressed on cancer cells. Additional common treatments for AML include chemotherapy, targeted drug treatments, or bone marrow transplants. Nkarta is a clinical-stage biotechnology company that develops off-the-shelf NK cell therapies for cancer patients using proprietary cell engineering technologies and CRISPR-based genome engineering capabilities.

Read more here.