Transthyretin (TTR) is a relatively abundant protein in the blood, named after its role in transporting thyroxine and retinol. Due to a genetic mutation or environmental factors, the protein can become unstable and misfold, causing it to accumulate as toxic amyloid aggregates in the heart or peripheral nerves.
Eidos aims to halt TTR amyloidosis by treating the root cause of the disease, destabilized and misfolded TTR. Inspired by a natural TTR mutation that protects carriers from the disease, Eidos is developing a small molecule (named AG10) that binds and stabilizes TTR in the blood. Pre-clinical studies have shown promising data, paving the way for clinical testing.