We develop small molecules designed to stabilize target proteins and reduce the misfolding processes that can contribute to disease. Our lead program focuses on lambda light chains in AL amyloidosis.
Dive deeper into our scientific approach, from the fundamental challenge of protein misfolding to our lead therapeutic program.
Misfolded proteins can lose function, disrupt cellular proteostasis, and drive pathogenic self-association. Disease-causing variants, age-related stress, and destabilizing cellular environments push proteins away from their native fold—leading to loss of activity, accelerated clearance, or toxic aggregation.
Read MoreWe design small molecule pharmacological chaperones intended to bind target proteins and stabilize their folded state. Our approach combines protein biophysics, computation and medicinal chemistry.
Read MorePROT-001 is an investigational oral small molecule designed to stabilize lambda light chains. It is in clinical development for AL amyloidosis.
Its safety and efficacy in patients with AL amyloidosis have not been established.
Explore PROT-001Learn how the clinical precedent of tafamidis in transthyretin amyloidosis informs research into protein stabilization at other targets.
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