Biomarker & Intervention Discovery
Primary Sclerosing Cholangitis (PSC)
Pharmacologically actionable gene targets, therapeutic agents ranked by evidence tier, and active clinical trials.
Spontaneous remissionProgressive cholestatic disease; median time to liver failure or transplant 12-15 years; spontaneous remission…
Best-intervention remissionLiver transplantation: 5-year survival 80-85% (curative but recurrence in 20% post-transplant); ursodeoxycholic acid…
Gap sizeVery large — no approved disease-modifying therapy exists; liver transplantation is the only definitive treatment
Primary barrierbiological
Full remission profile →
Remission & chronicity
Spontaneous remission
Progressive cholestatic disease; median time to liver failure or transplant 12-15 years; spontaneous remission essentially zero; biliary strictures progress in majority; cholangiocarcinoma risk 10-15% lifetime
Best-intervention remission
Liver transplantation: 5-year survival 80-85% (curative but recurrence in 20% post-transplant); ursodeoxycholic acid (UDCA): improvement in LFTs but no transplant-free survival benefit (Phase 3 — no benefit, possibly harm at high dose); no pharmacotherapy proven to alter the natural history
Gap size
Very large — no approved disease-modifying therapy exists; liver transplantation is the only definitive treatment
Primary barrier
biological
PSC has resisted all pharmacotherapy attempts; UDCA widely prescribed despite no survival benefit (at high doses — possibly harmful); the field has 20+ failed Phase 2/3 trials; norUDCA, nor-UDCA, and several FXR agonists are in Phase 3; gut-liver axis and gut microbiome increasingly implicated (80% of PSC patients have concurrent IBD)
Biomarker Targets & Therapeutic Agents
Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC.
Agents ranked: ● Clinical >
● Mechanistic >
● Correlative.
Pipeline results not yet available. Run: python -m biomarker_pipeline.run_for_diseases --skip-llm
Clinical Trials
Recruiting and recently completed trials from ClinicalTrials.gov.
Data retrieved 2026-07-06.
No recent clinical trials found on ClinicalTrials.gov for this condition.
Agents Found By Disease-Level Search
Searched directly from “Primary Sclerosing Cholangitis (PSC)” via ClinicalTrials.gov interventions and Open Targets'
disease→drug data — independent of the 0-gene target panel above.
This surfaces agents whose mechanism doesn't route through a curated gene (combination
therapies, standard-of-care drugs, targets outside the panel).
Not yet run. python -m biomarker_pipeline.run_disease_agent_discovery --disease "..."