Imetelstat is a telomerase inhibitor. Telomerase is an enzyme that helps cancer cells keep their telomeres (protective caps on the ends of chromosomes) long, which allows these cells to keep dividing and growing. By blocking telomerase, imetelstat causes the telomeres in cancer cells to shorten, which can slow down or stop the growth of these cells and may lead to their death.

In blood cancers like myelodysplastic syndromes (MDS), and potentially myelofibrosis, increased telomerase activity is often seen in malignant stem and progenitor cells. Imetelstat binds to the RNA component of telomerase, inhibiting its activity, reducing the proliferation of these abnormal cells, and promoting their death. This can help reduce the need for blood transfusions and improve blood counts in some patients.

imetelstat

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