Novel Approach to Treat Rare Disease May Lead to Drugs for Common Ones

Mitochondria, the powerhouses of the cell, play a critical role in a range of rare and common diseases. Retrotope, a development-stage company, thinks it’s found a way to repair the damage done by various disease processes to mitochondira. The company is just concluding an early-stage trial of its experimental therapy to treat the rare disease Freidreich’s ataxia. We spoke to Bob Molinari, CEO of Retrotope, and Harry Saal, chairman of the company, about mitochondrial diseases, the approach the company is taking, and why its work may have broad implications for a wide range of degenerative diseases.