Druggability & Clinical Context
Druggability
High
Score: 0.75
Druggability Analysis
Structural Tractability0.85
Key Metrics
PDB Structures:
21
Known Drugs:
1
Approved:
0
In Clinical Trials:
0
Drug Pipeline (1 compounds)
Druggability Rationale: LDLR is highly druggable (0.75 score) due to its established receptor class, extensive structural characterization (21 PDB structures at 1.26 Å resolution), and validated clinical precedent with approved PCSK9 inhibitors. The combination of well-defined extracellular ligand-binding domains and proven pharmacological modulation through both antibody and small molecule approaches supports continued drug development potential.
Mechanism: Upregulation through PCSK9 inhibition or direct receptor modulation
Drug Pipeline (1 compounds)
Known Drugs:PCSK9 inhibitors (Approved) — Hypercholesterolemia
Structural Data:PDB (21) ✓AlphaFold ✓Cryo-EM ✓
Selectivity & Safety Considerations
LDLR selectivity is generally favorable given its restricted expression pattern on hepatocytes and peripheral tissues; however, off-target engagement with related LDL receptor family members (LRP1, LDLRL1) must be monitored. Small molecule approaches targeting the ligand-binding domain may have advantages over PCSK9 modulation for tissue-specific CNS penetration relevant to neurodegeneration indications.
Clinical Trials (8)
Relevant trials from ClinicalTrials.gov
By Phase
NA: 5 · PHASE1: 1 · PHASE2: 1 · Unknown: 1
NA
NCT00486551
n=26
Tourette Syndrome, Chronic Tic Disorder, Oppositional Defiant Disorder
Interventions: Anger control training
Sponsor: Yale University | Started: 2001-08
NA
NCT06909045
n=130
Deep Brain Stimulation, Parkinson Disease
Interventions: Adaptive DBS, Continue DBS
Sponsor: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC | Started: 2026-01-27
PHASE1
NCT00843739
n=90
Parkinson's Disease
Interventions: EMST - Active Treatment, sham EMST
Sponsor: University of Florida | Started: 2004-01
Unknown
NCT03292575
n=441
Stroke
Interventions: Anticoagulants
Sponsor: Centre Hospitalier Universitaire Dijon | Started: 2016-01
NA
NCT01924312
n=80
Cerebrovascular Disease, Mild Cognitive Impairment
Interventions: Heart Health Intervention
Sponsor: Gregory Jicha, 323-5550 | Started: 2013-05
NA
NCT06306365
n=35
Executive Functions
Interventions: Modern board game-based learning
Sponsor: European University Miguel de Cervantes | Started: 2024-02-07
NA
NCT02260167
n=25
Alzheimer's Disease, Dementia
Interventions: A mix of natural treatments and medicati
Sponsor: Practitioners Alliance Network | Started: 2014-09
PHASE2
NCT03987295
n=33
Frontotemporal Dementia
Interventions: AL001
Sponsor: Alector Inc. | Started: 2019-09-27