Marina Kremyanskaya, MD, PhD, Icahn School of Medicine at Mount Sinai, discusses the development of gene silencer divesiran for treatment of polycythemia vera (PV).

 


 

PV is a myeloproliferative disorder characterized by an elevated absolute red blood cell mass caused by uncontrolled red blood cell production. PV is also frequently associated with uncontrolled white blood cell and platelet production. Current standard of care includes phlebotomies and medications to reduce red blood cell count.

Divesiran is a TMPRSS6 gene silencer therapy, leading to increased hepcidin-a peptide hormone and iron balance. These increases in turn lower red blood cell production. In previous studies, divesiran was shown eliminating the need for phlebotomy in well-controlled patients with infrequent dosing. Additionally, tolerability and safety has been favorable.

The ongoing SANRECO phase 1/2 clinical trial is further evaluating divesiran in patients with PV.

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To learn more about PV and other rare cancers, visit https://checkrare.com/diseases/cancers/