The mitochondrion is a double-membrane-bound discrete organelle found in most eukaryotic organisms, generating most of the cell's supply of adenosine triphosphate (ATP) and controlling the cellular basal metabolic rate, called as the cell's powerhouses. In addition to supplying cellular energy, mitochondria are the major source of ROS (reactive oxygen species) that reflect the level of cellular oxidative stress and play an important role in mitochondria ROS signaling such as apoptosis, proliferation, and aging, etc. In addition, the fine modulation of mitochondrial calcium (Ca2+) homeostasis plays a fundamental role in many of the processes involving this organelle. Mitochondrial Ca2+ accumulation is a tightly controlled process, in turn regulating functions as diverse as aerobic metabolism and induction of cell death. Mitochondrial DNA mutations may lead to many mitochondrial metabolic disorders, and are thought to contribute to aging by promoting apoptosis. Mitochondria therefore represent an attractive drug target for metabolic diseases, neurodegeneration, or hyperproliferative diseases (cancer). A number of pre-clinical and clinical data have shown that mitochondria as drug targets have great potential. Small molecule drugs or biologics can act on mitochondria through various pathways including ETC inhibition, OXPHOS uncoupling, mitochondrial Ca2+ modulation, and control of oxidative stress via decrease or increase of mitochondrial ROS accumulation.

Mitochondria-Targeted Compound Library from TargetMol, a unique collection of 812 compounds targeting mitochondria, can be used for research in mitochondrial medicine and related target study.