TEE375

TEE375 is a small molecule drug candidate being developed by the French biotechnology company Apteeus for the treatment of Acid Ceramidase Deficiency, also known as Farber disease. Farber disease is a rare lysosomal storage disorder caused by mutations in the ASAH1 gene, which leads to the accumulation of ceramides in various tissues. TEE375 was identified using Apteeus's proprietary TEEpS (Therapeutic Efficiency Evaluation on Patients' Samples) drug repositioning platform, which screens existing approved drugs to find new therapeutic applications for rare diseases. By repurposing an established pharmaceutical compound, Apteeus aims to accelerate the development of a treatment that can modulate the ceramidase pathway and address the underlying metabolic deficiency. As of 2021, TEE375 is in Phase 1 clinical development.