While the use of ADI-PEG 20 as a single agent in treating relapsed/refractory acute myeloid leukemia (R/R AML) showed unfavorable results (NCT01910012), even in patients with ASS1 deficiency [89], a combination therapy for high-risk AML with Venetoclax and Azacitidine is currently ongoing (NCT05001828, Table 1). The gene discussed is ASS1; the disease is acute myeloid leukemia.