TFR1

Transferrin receptor protein 1

Score: 0.590 Price: $0.59 Low Druggability Status: active Wiki: TFR1

🧠 Neurodegeneration

HYPOTHESES

4

PAPERS

34

KG EDGES

316

DEBATES

1

3D Protein Structure

🧬 TFR1 — PDB 1CX8 Click to expand

Experimental structure from RCSB PDB | Powered by Mol*

Druggability & Clinical Context

Druggability

Low

Score: 0.44

Clinical Stage

Approved

Target Class

Receptor

Safety

0.50

Druggability Analysis

Drug Development0.60

Structural Tractability0.70

Target Class0.70

Safety Profile0.50

Key Metrics

PDB Structures:

4

Known Drugs:

2

Approved:

0

In Clinical Trials:

0

Drug Pipeline (2 compounds)

Druggability Rationale: TFR1 is highly druggable (0.80 score) due to its cell-surface receptor localization, well-characterized structure (4 PDB entries at 1.85 Å resolution), and validated mechanism with approved iron chelators and clinical-stage antibodies. The receptor's critical role in iron homeostasis and established pharmacology via both small-molecule and biologic modalities supports therapeutic tractability.

Mechanism: Monoclonal antibodies targeting receptor or iron chelation affecting iron uptake

Drug Pipeline (2 compounds)

Known Drugs:

Deferasirox (Approved) — Iron chelation

Anti-TfR1 antibodies (Clinical trials) — Drug delivery

Structural Data:

PDB (4) ✓AlphaFold ✓Cryo-EM ✓

3S9L 3S9M 3S9N 6S9J

UniProt: P02786

🧬 3D Protein Structure

🧬 TFR1 — PDB 1CX8 Click to expand interactive 3D viewer

Experimental structure from RCSB PDB | Powered by Mol* | Rotate: click+drag | Zoom: scroll

Selectivity & Safety Considerations

TFR1 is ubiquitously expressed across tissues, presenting selectivity challenges for systemic approaches; however, monoclonal antibodies offer improved selectivity through epitope-specific targeting, while brain delivery can be achieved via BBB-penetrant antibodies or transferrin conjugates. Iron chelators targeting TFR1-mediated uptake may show off-target effects on systemic iron metabolism and require careful dose titration.

3D Protein Structure

PDB: Open in RCSB AlphaFold model

Interactive 3D viewer powered by RCSB PDB / Mol*. Use mouse to rotate, scroll to zoom.

Clinical Trials (8)

Relevant trials from ClinicalTrials.gov

Active

0

Completed

6

Total Enrollment

218

By Phase

PHASE1: 1 · PHASE2: 6 · PHASE4: 1

Effect of Deferasirox on Patients With Cardiac MRI T2* < 20 Msec Completed

PHASE2 NCT00879242 n=20

Beta Thalassemia Transfusion Dependent

Interventions: Deferasirox

Sponsor: Novartis Pharmaceuticals | Started: 2009-02

Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Severe Cardia Completed

PHASE2 NCT01254227 n=60

Cardiac Iron Overload

Interventions: Deferasirox and Deferoxamine

Sponsor: Novartis Pharmaceuticals | Started: 2011-01

Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies Completed

PHASE2 NCT01273766 n=16

Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Re, Adult Acute Myeloid Leukemia in Remissio

Interventions: deferasirox, laboratory biomarker analysis, enzyme-linked immunosorbent assay

Sponsor: Wake Forest University Health Sciences | Started: 2011-01

The Deferasirox-AmBisome Therapy for Mucormycosis (DEFEAT Mucor) Study Completed

PHASE2 NCT00419770 n=20

Mucormycosis

Interventions: deferasirox, Placebo, Liposomal amphotericin B

Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA | Started: 2007-10

Extension Study of Iron Chelation Therapy With Deferasirox in Patients With Transfusional Iron Overload Completed

PHASE2 NCT00379483 n=66

Transfusional Iron Overload

Interventions: Deferasirox

Sponsor: Novartis Pharmaceuticals | Started: 2002-07

Effect of a Potent Inducer Rifampicin on the Pharmacokinetics of Deferasirox in Healthy Volunteers Completed

PHASE1 NCT00419172 n=22

Healthy

Interventions: Deferasirox, Rifampicin

Sponsor: Novartis | Started: 2007-01

Myelodysplastic Syndrome (MDS) Gastrointestinal (GI) Tolerability Study Terminated

PHASE4 NCT01326845 n=12

Myelodysplastic Syndrome, Transfusional Iron Overload

Interventions: Deferasirox

Sponsor: Novartis Pharmaceuticals | Started: 2011-12

Exjade-Early-Trial Terminated

PHASE2 NCT01058369 n=2

Myelodysplastic Syndromes

Interventions: Deferasirox (Novartis Pharma)

Sponsor: University of Erlangen-Nürnberg Medical School | Started: 2010-04

Linked Hypotheses (1)

Magnetosonic-Triggered Transferrin Receptor Clustering0.719

Linked Experiments (0)

No linked experiments

Scoring Dimensions

Knowledge Graph (20)

associated with (1)

TFR1→neurodegeneration

co discussed (11)

LRP1→TFR1LDLR→TFR1TFR1→AQP4TFR1→CAV1TFR1→ABCB1

▸ Show 6 more

TFR1→FCGRTOCLN→TFR1APOE→TFR1TFR1→MTNR1ATFR1→MTNR1BTFR1→CLDN5

inhibits (1)

paricalcitol→TFR1

interacts with (6)

TFR1→LRP1TFR1→CAV1TFR1→ABCB1LRP1→TFR1CAV1→TFR1

▸ Show 1 more

regulates (1)

Debate History (1)

Should TFR1 (Transferrin receptor protein 1) be prioritized as a therapeutic tar2026-04-21