It is well known that mitochondria, the so-called “powerhouses of the cell,” are not only important in cellular energy and cell death, but have been implicated in a large number of diseases, from cancer and obesity to heart disease and Alzheimer’s. Given the structure and location of these organelles they are very difficult to target with conventional therapeutics. Despite this, mitochondria are one of the most studied parts of the cell and present a significant untapped opportunity to modulate disease, if accessible to modulating compounds.
Our platform has been engineered to overcome many of the hurdles involved in targeting the mitochondria by taking advantage of the physiology of the cell. We also take advantage of similar physiological characteristics to provide for a more drug-like profile for our therapeutics including aspects such as drug-target specificity and appropriate pharmacokinetics and biodistribution.
Our platform has been validated in a variety of pre-clinical models across a number of diseases with data published in a number of peer-reviewed journals. Our lead development candidates target key components of cellular metabolism known to be up-regulated in many forms of cancer. We expect to complete pre-clinical testing of our lead and begin clinical trials by the second half of 2016.