Harnessing the power of pharmacological chaperones to restore protein function and transform patient outcomes.
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 that bind and stabilize target proteins in their native, functional state, improving folding efficiency and preserving activity. Our approach combines protein biophysics, structure-informed chemistry, computation, and translational pharmacology, backed by deep expertise in mechanism-based drug discovery for conformational diseases.
Read MoreOur most advanced program targeting AL amyloidosis, now advancing into pivotal clinical studies.
Read MoreBuilt on the pioneering discovery of tafamidis by our cofounders, extending proven pharmacological chaperone science to new targets.
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