The initial gene therapy to cure Hemophilia B in grown-ups has been authorized by the FDA.

Introducing Hemgenix: The First Gene Therapy Treatment for Hemophilia B

The Prevalence and Symptoms of Hemophilia

Hemophilia is a serious blood disorder that affects the body’s ability to clot, leading to excessive bleeding even after minor injuries. It is a rare disorder that affects an estimated one in 10,000 newborn babies worldwide, with over 400,000 people currently living with the condition. Hemophilia A and B are the two primary types of hemophilia, with hemophilia A being the most common form. Symptoms can range from mild to life-threatening and include bleeding into joints, easy bruising, bleeding of the mouth and gums, frequent nosebleeds, and prolonged bleeding from minor cuts and scrapes.

The Inheritance and Treatment of Hemophilia

Hemophilia is often inherited from parents via their genetics, with the faulty gene located on the X chromosome. Females carrying the faulty gene are considered “carriers” of the condition and may experience mild to moderate symptoms. Treatment options for hemophilia are intended for symptom management and include concentrated blood clotting products, aminocaproic acid, and non-factor replacement therapies. However, these treatments are not without complications and drawbacks, such as the development of “inhibitor” antibodies over time.

The Promise of Hemgenix

Hemgenix is a gene therapy medication that has been FDA-approved for treating certain adults with hemophilia B. It uses AAV5, a modified virus, to deliver a functional copy of the gene for factor IX into the body’s cells. After a single dose, cells within the body can produce clotting factor IX at a rate 5-8 times greater than before. Clinical trials have shown promising results, with 94% of participants able to reduce or stop their concentrated FIX dosages and a 54% decrease in annual bleeds. However, the medication comes with a hefty price tag of $3.5 million per infusion.

The Future of Hemophilia Treatment

While Hemgenix may seem incredibly expensive, it is the first step into gene therapy for treating hemophilia and may be the future of managing the disease. Gene therapy offers many benefits to people with hemophilia, including increased peace of mind, less time spent receiving care, and proven long-term efficacy and safety. This breakthrough could also open the door for effective gene therapy for other genetic conditions. More clinical research and studies are needed for researchers to learn more about other potential gene therapy options and hopefully more affordable treatment options for people with hemophilia.

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