Pharmacological Chaperones
Our therapeutic approach centers on pharmacological chaperones—small molecules that bind selectively to a target protein and enhance its conformational stability. The mechanism is clinically validated by tafamidis, a breakthrough treatment for transthyretin amyloidosis discovered by our cofounders.
Pharmacological chaperones work by:
- Binding selectively to the target protein
- Stabilizing its native, functional conformation
- Preventing misfolding and aggregation
- Preserving cellular proteostasis
