A first-in-class investigational drug with the potential to extend and enhance lives
Imetelstat is a first-in-class telomerase inhibitor that could change the course of blood cancers—changing lives for the better.
The development of imetelstat is grounded on Nobel Prize-winning scientific discoveries by early Geron collaborators that expanded the understanding of telomeres and telomerase and their role in the proliferation of cancer cells. Using this knowledge, Geron scientists pioneered a new approach in which oligonucleotides (short single strands of synthetic DNA or RNA) inhibit the activity of telomerase, an enzyme that helps maintain telomeres and enables the continued proliferation of malignant cells.
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How imetelstat works
Imetelstat binds to telomerase and inhibits its activity, selectively killing the malignant stem and progenitor cells in the bone marrow that are the source of disease in blood cancers, such as myelodysplastic syndromes (MDS) and myelofibrosis (MF).
By inhibiting the proliferation of these malignant stem and progenitor cells, which enables the recovery of non-malignant bone marrow and blood cell production, imetelstat has exhibited disease-modifying activity and clinical benefits for patients in Phase 2 clinical trials of lower risk MDS and MF. This mechanism of action differentiates imetelstat from other approved and investigational treatments in these blood cancers.