Rare disorders have traditionally often been neglected by pharmaceutical companies. RTT remains dedicated to the identification and development of therapeutic options even if the number of patients suffering from the condition is very low.
Our lead product candidate, Emcitate® , is an oral small molecule that has been shown to restore dysfunctional thyroid hormone signalling in several situations. Studies have shown that Emcitate can enter into cells regardless of a functioning MCT8 transporter.
Thyroid hormone is a key metabolic regulator in the human body with effects on almost all cell types and plays an important role in the development and proper function of multiple organs.
MCT8 is an important transporter protein for the correct trafficking of thyroid hormone. Thyroid hormone was long believed to be able to enter into cells without the need for specific transporters, and it was only in 2000 that MCT8 was described as the first thyroid hormone specific transporter.