Thus, according to publicly available information, Calporta Therapeutics, acquired by Merck in 2019, has developed preclinical stage TRPML1 agonists for potential treatment of Niemann-Pick C disease (NPC) and other lysosomal storage diseases, as well as amyotrophic lateral sclerosis, Alzheimer’s disease, and PD. The gene discussed is MCOLN1; the disease is lysosomal storage disease.