Gene → Disease → Drug, in one search box
Three questions come up constantly at the bench: which drugs hit my gene? what are the top druggable genes for my disease? and which drugs target this whole pathway? All three answers live inside the Open Targets Platform — but the full platform is a lot to drive. BioTransfer's Target ↔ Disease Explorer is a thin, honest layer that turns each of those questions into a single search.
The problem: a goldmine you have to mine
Open Targets is one of the best resources in biomedicine — it fuses genetics, somatic mutations, known drugs, pathways, expression, and literature into evidence-based target–disease association scores, and it tracks the clinical pipeline for thousands of drugs. But the platform is built for deep, interactive exploration. If you just want a quick, linkable answer — "is MYCN druggable?", "what's in trials for neuroblastoma?" — you end up clicking through several views to assemble it. This tool assembles it for you.
Three ways to explore
1 · By gene → its diseases & druggability
Type a gene and you get its evidence-ranked associated diseases plus a druggability (tractability) readout. Search MYCN, the classic neuroblastoma oncogene, and the honest answer jumps out: it's a transcription factor with almost no small-molecule tractability — a famously hard target. That's a useful thing to learn in one glance.
2 · By disease → ranked target genes, each with its drugs
Search a disease and you get its top target genes ranked by association score, and — the important part — each gene paired with the pipeline drugs that actually act on it. Search neuroblastoma and ALK tops the list (score ≈ 0.84), sitting next to lorlatinib, ceritinib and crizotinib — the real ALK inhibitors used in ALK-driven neuroblastoma. No guessing which drug hits which gene; the link is explicit.
3 · By pathway → its druggable nodes & their drugs
Pick a signalling pathway and the tool shows its key druggable nodes, each with the drugs targeting it, approved ones first. Open RTK → RAS–RAF–MEK–ERK and you see EGFR paired with afatinib and cetuximab, KRAS with sotorasib and adagrasib, BRAF with its inhibitors — the pathway's whole druggable surface on one screen.
Why the pathway view matters
Cancers rarely depend on a single gene — they depend on pathways. When one node is blocked, tumours often escape by re-wiring through another node in the same cascade, which is exactly why combination and pathway-level thinking drives modern targeted therapy. Seeing every druggable node of a pathway alongside the drugs that hit it — and how far each has progressed clinically — is a fast way to reason about targets, resistance, and repurposing. The tool covers the most-studied signalling pathways (RTK/MAPK, PI3K–AKT–mTOR, cell cycle/CDK, DNA-damage/PARP, JAK–STAT, apoptosis/BCL-2, WNT, Hedgehog, NOTCH, VEGF, epigenetic, immune checkpoints, nuclear hormone receptors, NF-κB, TGF-β).
How it works
Everything comes live from the free, public Open Targets GraphQL API — no API key, no login, no local data. A gene or disease lookup is two lightweight calls; a whole pathway is just two batched calls (resolve the node symbols, then pull each node's drugs). Every gene, disease, and drug in the results links straight back to its Open Targets page, so the tool is a fast index into the source, never a copy of it.
Honest about the limits
- Scores are computational. Association scores aggregate evidence (genetics, somatic mutations, known drugs, pathways, expression, literature) — they're for target prioritisation and discovery, not clinical or prescribing advice.
- Pathways use curated nodes. Each pathway is a hand-picked set of its key druggable nodes (the most-cited ones), not the full Reactome membership — kept focused so a pathway resolves in two fast calls.
- Drugs are "any indication." In pathway view, a node's drugs are all drugs acting on that gene, not filtered to one disease — use disease mode when you need the disease-specific pipeline.
No login. Search by gene, disease, or pathway — ranked results, druggability, and the drug pipeline, every row linked back to Open Targets.
Open the tool →Pair it with the GEO Dataset Finder for expression data, or the Neuroblastoma Surface Targets atlas for immunotherapy target discovery.