Scientific Founder, Stealth Biotherapeutics
Discovery and Clinical Development of Elamipretide
Mitochondria play a central role in energy generation in the cell, providing ATP to carry out essential biological functions. As energy output declines, the most energetic tissues are preferentially affected, including the skeletal muscles, heart and retina. Age-related decline in mitochondrial function is associated with numerous chronic degenerative diseases. This talk will describe the discovery of a family of mitochondria-targeted tetrapeptides (SS peptides) that preserve mitochondrial structure and restore cellular bioenergetics by targeting cardiolipin, a unique phospholipid on the inner mitochondrial membrane. Preclinical and clinical development of the first drug candidate (Elamipretide) for rare genetic mitochondrial diseases (myopathy, Leber’s hereditary optic neuropathy, Barth Syndrome) and common age-related mitochondrial diseases (heart failure, macular degeneration, muscle weakness) will be presented.