In the pharmaceutical context, TTR typically refers to Transthyretin, a protein primarily produced in the liver and choroid plexus.
Transthyretin's Role
Transthyretin (TTR), also known as prealbumin, is a transport protein in the blood and cerebrospinal fluid. Its primary functions include:
- Transporting Thyroxine (T4): TTR carries the thyroid hormone thyroxine, playing a crucial role in thyroid hormone distribution throughout the body.
- Transporting Retinol (Vitamin A): TTR binds to retinol-binding protein (RBP), which then carries retinol (vitamin A). This is essential for vitamin A transport and utilization.
Relevance in Pharma
In the pharmaceutical industry, TTR is particularly relevant due to its involvement in transthyretin amyloidosis (ATTR), a disease caused by misfolded TTR proteins that aggregate and deposit in various tissues and organs.
- Drug Development: Several pharmaceutical companies are developing drugs to treat ATTR amyloidosis. These therapies aim to:
- Stabilize TTR: Prevent the misfolding and aggregation of TTR protein. Tafamidis (Vyndaqel, Vyndamax) is an example of a TTR stabilizer.
- Reduce TTR Production: Decrease the amount of TTR protein produced in the liver. Examples include Inotersen (Tegsedi) and Patisiran (Onpattro), which are RNA interference (RNAi) therapies.
- Diagnostic Testing: TTR levels and mutations are important biomarkers in diagnosing and monitoring ATTR amyloidosis. Diagnostic assays are used to identify individuals at risk or with the disease.
- Clinical Trials: TTR is a key endpoint and biomarker in clinical trials evaluating new therapies for ATTR amyloidosis.
ATTR Amyloidosis Types
ATTR amyloidosis can be categorized into two main types:
- Hereditary ATTR (hATTR) Amyloidosis: Caused by a genetic mutation in the TTR gene, leading to the production of unstable TTR protein.
- Wild-Type ATTR (wtATTR) Amyloidosis: Occurs when the normal TTR protein misfolds and aggregates, typically affecting older individuals.
Therefore, when discussing "TTR" in a pharmaceutical context, it most often relates to the Transthyretin protein and its role in ATTR amyloidosis, a disease being actively targeted by drug development efforts.
