An opportunity to make a difference
Our goal is to transform the lives of patients with rare genetic disease. By intervening early, we believe we will be able to restore the function of aberrant genes before the patients experience irreversible declines in function.
Our technology platform is modular in nature; our initial product candidates will leverage our existing knowledge to address diseases by targeting the liver, including a category of diseases known as inborn errors of metabolism. We expect that our future product candidates will also draw from programs that address genetic diseases by targeting other tissues including the central nervous system (CNS) and muscle.
Our focus on metabolic diseases has led us to intensive study of organic acidemias, which are caused by an enzymatic defect in the metabolism of amino acids, leading to a dangerously high level of organic acid in the blood and body tissues. We are pursuing methylmalonic acidemia (MMA) as an initial indication. Learn more about our therapeutic focus:
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