Scientific Foundation

Building on the proven success of tafamidis, the first-in-class pharmacological chaperone discovered by our cofounders.

Building on Proven Success

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. Their breakthrough validated the concept that small molecules can selectively stabilize disease-causing proteins and deliver meaningful clinical benefit.

We draw on decades of expertise in protein biophysics, medicinal chemistry, and clinical development to extend this proven mechanism to new therapeutic targets—bringing hope to patients with untreatable protein misfolding diseases.

Protein Structure - CPK Model