Protein / target
Transthyretin
Protein at a glance
Biological role
Protein-containing complex binding
Primary system
Endocrine & metabolic
Strongest disease association
amyloidosis, hereditary systemic 1
Therapeutic maturity
Clinically validated target
Druggability
Small molecule
Clinical development
12 approved · 1 in clinical development
Derived from structured UniProt, Open Targets and literature data on this page.
Protein profile
Canonical identity and biological annotation from UniProt.
Function
Thyroid hormone-binding protein. Probably transports thyroxine from the bloodstream to the brain
Subcellular location
Domains and Gene Ontology detail (12)Hide
Gene Ontology
- Cazurophil granule lumen
- Cextracellular exosome
- Cextracellular region
- Cextracellular space
- Cprotein-containing complex
- Fhormone activity
- Fhormone binding
- Fidentical protein binding
- Fmolecular sequestering activity
- Fprotein-containing complex binding
- Ppurine nucleobase metabolic process
- Pretinoid metabolic process
Biological roles
What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.
View supporting evidenceHide supporting evidence
Metabolic enzyme activity
- ·purine nucleobase metabolic process
- ·retinoid metabolic process
View underlying pathways (6)Hide underlying pathways
Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.
Interaction neighbourhood
Proteins with the strongest functional or physical association. Node size and line weight reflect STRING confidence; hover or select a partner to inspect one association.
Functional and physical associations from STRING v12.0. Only associations with this protein are drawn — partner-to-partner links are not part of this evidence.
Drugs targeting this protein
Whether each drug engages this protein directly or through a complex, and how many other targets are recorded for it. Direct binders with few recorded targets are listed first.
Transthyretin stabiliser
Appears in clinical studies involving amyloidosis, cardiomyopathy, AL amyloidosis, Familial transthyretin-related amyloidosis
ChEMBL mechanism, action type and target identity. Open Targets clinical status and indications.
Translational evidence
Why this target matters therapeutically — disease associations, drugs in development, tractability and safety, from Open Targets.
Strongest genetic associations
Human genetic evidence — the most direct causal link between this target and a disease.
Highest-confidence therapeutic associations
Diseases where a drug acting on this target has already reached clinical development.
Show all associationsHide all associations
Open Targets ranks 1,611 associations for this target and we store the top 10 by aggregated score. The long tail is largely literature co-mention and RNA-expression evidence, not evidence that this target drives those diseases.
Known drugs · 13 total
cardiac amyloidosis · cardiomyopathy · male infertility
amyloidosis · cardiomyopathy · cardiac amyloidosis
familial amyloid neuropathy · familial amyloid neuropathy · amyloidosis
amyloidosis · cardiomyopathy · AL amyloidosis
AL amyloidosis · amyloidosis · cardiomyopathy
hereditary amyloidosis · familial amyloid neuropathy · familial amyloid neuropathy
polyneuropathy · amyloidosis · hereditary amyloidosis
hereditary amyloidosis · familial amyloid neuropathy · polyneuropathy
cardiovascular disorder · familial amyloid neuropathy · amyloidosis
amyloidosis · hereditary amyloidosis · cardiac amyloidosis
Tractability
Safety liabilities
Clinical trials
Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein.
Show remaining trials (14)Hide
ClinicalTrials.gov via the drug-target graph.
Related literature
Papers indexed under “Prealbumin” — a subject heading that covers this protein without being specific to it. Shown as context; not counted as this protein's own research activity.
Europe PMC literature, reached through a MeSH descriptor linked to this protein.
Forefront confidence
Our own weighting of the evidence behind each disease association. Human genetics and clinical evidence count for most; literature co-mention counts for little, because two entities sharing an abstract is not evidence that one drives the other.
The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the underlying per-type scores are shown above so the calculation can be checked.
What's happening now
Recent activity around this target, drawn from one canonical event stream. Every item is reached through a drug that targets this protein, so each event is news about that drug rather than about the protein directly.
- CHMP positive opinion
CHMP positive opinion: Tafamidis Accord (EMA)
- New publicationEffect of Tafamidis on Cardiac Function in Patients With Transthyretin Amyloid Cardiomyopathy: A Post Hoc Analysis of the ATTR-ACT Randomized Clinical Trial.
- Regulatory approval
Approval: Vyndaqel (EMA)
Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.