OnCo
targetsTarget

WRN helicase (MSI-high cancers)

A DNA-unwinding enzyme that mismatch-repair-deficient cancers cannot live without; the first WRN inhibitors are in trials as a chemotherapy-free option for MSI-high tumours that fail immunotherapy.

CRISPR screens (2019) identified WRN as a synthetic-lethal dependency in microsatellite-unstable (MSI-H/dMMR) cancers because expanded TA-dinucleotide repeats form secondary structures that only WRN helicase can resolve. First-in-class covalent WRN inhibitors (HRO761, RO7589831/VVD-133214, GSK4418959) entered phase 1 in 2023-24 with early responses in MSI-H colorectal cancer, including after checkpoint inhibitors. Relevant to ~15% of colorectal, ~30% of endometrial and ~20% of gastric cancers, and Lynch syndrome.

WRN helicase (MSI-high cancers): what it is and how drugs act on it · animated generic schematic, not to scale
  • Target · the protein and the cell it sits on
  • Drug · antibody, small molecule, cell or radioligand
  • Effect · signal, damage or kill

In plain words · A DNA-unwinding enzyme that mismatch-repair-deficient cancers cannot live without; the first WRN inhibitors are in trials as a chemotherapy-free option for MSI-high tumours that fail immunotherapy.

  1. 1 · What it is

    A DNA-unwinding enzyme that mismatch-repair-deficient cancers cannot live without; the first WRN inhibitors are in trials as a chemotherapy-free option for MSI-high tumours that fail immunotherapy.

  2. 2 · What goes wrong in cancer

    RecQ family 3'→5' DNA helicase and exonuclease; germline loss causes Werner progeroid syndrome; in MSI cancers its helicase (not exonuclease) activity prevents replication fork collapse at expanded (TA)n repeats.

  3. 3 · How drugs use it

    No product in this corpus aims at WRN helicase (MSI-high cancers) yet. Inhibitors are shaped to fit the enzyme's active site so the reaction the cancer relies on stops.

Biology

RecQ family 3'→5' DNA helicase and exonuclease; germline loss causes Werner progeroid syndrome; in MSI cancers its helicase (not exonuclease) activity prevents replication fork collapse at expanded (TA)n repeats.

Where it is found
  • MSI-H colorectal cancer (~15% of CRC; dependency)
  • MSI-H endometrial cancer (~30%)
  • MSI-H gastric cancer (~20%)
  • Lynch syndrome tumours
Class
enzyme · WRN

How common it is, by cancer

CancerPrevalenceSource
Endometrial cancer
30%
Gastric & gastro-oesophageal junction cancer
20%
Colorectal cancer
15%

Approximate, population-level figures; the measure column says what was counted. Ranges show the midpoint as a bar.

Key papers

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Latest papers

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Latest papers · live from Europe PMC
Open in Europe PMC

Query for this target: (TITLE:"WRN helicase" OR ABSTRACT:"WRN helicase" OR TITLE:"MSI-high cancers" OR ABSTRACT:"MSI-high cancers" OR TITLE:"WRN" OR ABSTRACT:"WRN") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about WRN helicase (MSI-high cancers), not a curated reading list.

Connected

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