Pharmacological Chaperones

Pharmacological Chaperones

Pharmacological chaperones are small molecules that bind target proteins and can help stabilize their folded state. The effects depend on the target protein and the molecule.

Tafamidis provides a clinical precedent for protein stabilization in transthyretin amyloidosis. It stabilizes transthyretin and is approved in the United States for adults with transthyretin amyloid cardiomyopathy. Read the tafamidis prescribing information.

Protego is applying protein-stabilization research to other targets, including lambda light chains in AL amyloidosis. Evidence for tafamidis in its own disease setting does not establish the safety or efficacy of PROT-001.

PROT-001 is investigational. Its safety and efficacy in patients with AL amyloidosis have not been established.

Explore PROT-001 →