 Laterzinomab is a new drug being developed to treat frontotemporal dementia, FTD, caused by heterozygous loss of function mutations in the prognulin, PGIN, gene. It works by reducing the amount of sotilin, a protein that binds to PGIN and transports it to the lysosome for degradation. This reduces the amount of PGIN available for degradation, leading to higher levels of PGIN in the body. Laterzinomab has been shown to reduce sotilin levels in white blood cells, increase plasma and cerebrospinal fluid, CSF, PGIN levels, and restore PGIN levels in asymptomatic individuals who carry the genetic mutation associated with FTD. This article was authored by Michael Kernelis, Ononia Mitra, Tina Schwab, and others.