Frequently Asked Questions

PV is a rare blood cancer characterized by an excess production of red blood cells². PV affects approximately 3.5 million people worldwide³.

The current standard of care includes a procedure called a phlebotomy that reduces the percentage of red blood cells in the blood, and/or medications to reduce red blood cell counts. There is currently no cure for PV and people living with the disease are at an increased risk of cardiovascular events and potential progression to myelofibrosis or leukemia^2,4,5.

Divesiran works by temporarily silencing the production of the TMPRSS6 protein found in liver cells, which in turn increases the body’s production of another protein called hepcidin. By increasing hepcidin levels, divesiran aims to redirect iron delivery away from the bone marrow, lowering red blood cell production and potentially reducing the high red blood cell count in people living with PV. This may reduce the need for phlebotomies and lower the risk of blood clots and strokes. In addition, PV related symptoms such as fatigue, brain fog, and itchy skin may be reduced.

siRNA is a short interfering RNA. siRNA therapeutics work by leveraging the body’s natural process of RNA interference and aim to treat disease by “silencing” the genes that cause or contribute to them. Unlike gene therapy or editing, it does not produce permanent changes.

A clinical trial is a research study where experts study potential treatments. The treatments might be new drugs or devices. Clinical trials, also known as clinical studies, must meet specific standards and regulations. Should you decide to join a clinical study, know that your well-being is the clinical study team’s top priority. And you can leave a study at any time.

Clinical studies are essential for understanding whether new treatments are safe and effective. They help advance medical research and can lead to better care in the future. Participation is voluntary and personal—many people join to help others with the same condition or to find a better treatment or to access a potential new therapy.

There are typically four main phases of clinical studies (phase 1 to phase 4). Each phase has a specific purpose and study design. A phase 1 study focuses on evaluating the safety, dose range and side effects of a medication. A phase 2 study assesses the effectiveness of a medication and further evaluates the safety profile. A phase 3 study is used to confirm the effectiveness of the medication, monitor side effects, and compare the medication to standard treatments. Finally, a phase 4 study is typically conducted after a medication has received regulatory approval and is used to monitor the long-term effectiveness and safety of the new medication.