ORY-2001 (Vafidemstat), a dual LSD1/MAO-B inhibitor developed by Oryzon Genomics, has recently been approved to enter IIa clinical trial to evaluate the safety, tolerability, and preliminary efficacy of ORY-2001 in patients with mild to moderate Alzheimer’s disease (ClinicalTrials.gov Identifier: NCT03867253). The gene discussed is KDM1A; the disease is Alzheimer disease.