A foundation in protein stabilization
Our approach builds on the pioneering work of our cofounders, Dr. Jeffery W. Kelly and Dr. Richard Labaudinière, who discovered tafamidis—the first-in-class pharmacological chaperone approved for treating transthyretin amyloidosis. Tafamidis stabilizes transthyretin and is approved in the United States for adults with transthyretin amyloid cardiomyopathy. Read the tafamidis prescribing information.
We draw on decades of expertise in protein biophysics, medicinal chemistry, and clinical development to investigate protein stabilization at other targets. Each molecule and disease setting requires its own evidence of safety and efficacy.
Our lead program, PROT-001, is investigational. Its safety and efficacy in patients with AL amyloidosis have not been established.
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