The Barbara Ann Karmanos Cancer Institute in Detroit has become the first and only independent cancer center in the U.S. to provide a new hemophilia gene therapy treatment.
Etranacogene dezaparvovec-drlb, also known by its brand name Hemgenix, is a one-time gene therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with hemophilia B.
Steve W. of Clinton Township was the first patient to receive the therapy at Karmanos Cancer Center in Detroit on Oct. 28, 2025. In the U.S., Steve is the 52nd patient treated after clinical studies.
“I’m just very blessed to be in the right spot at the right time with the right people,” Steve says. “And it’s working. I didn’t have any problems the first few days … I did have some days where I was just tired, but about a week after the infusion, I was good.”
Hemophilia B is a rare blood-clotting disorder caused by a deficiency of factor IX, a clotting protein. As a result, hemophilia B patients may have excessive bleeding after injury or surgery. Severe deficiency can result in spontaneous bleeding, particularly to the joints. Hemophilia B can be a life-threatening disease in the case of severe injury or surgery without proper therapy.
“Although most hemophilia is hereditary, sometimes patients can be diagnosed with this disease without a family history,” says Dr. Indryas Woldie, head of the Hemophilia and Other Bleeding Disorders Clinic at Karmanos. “Official diagnosis of this condition usually happens when a newborn is circumcised, or later in life when a patient experiences excessive bleeding, potentially after a procedure, and in severe cases, spontaneous bleeding.
“Males inherit the X chromosome from their mothers, and if it is hemophilic, they will have hemophilia. Although female carriers may not have severe disease manifestations, they can still have bleeding, particularly during menstrual cycles. In addition, they will need to be properly treated during surgery and in the event of injuries. Depending on the factor IX level, females can also have mild and occasionally moderate hemophilia.”
Hemophilia B requires consistent medical care. The current standard of care is lifelong, routine infusions of factor IX replacement therapy (prophylaxis) to help reduce bleeding risk. Before the gene therapy, Steve would have to inject himself once a week with prophylaxis to increase his factor IX count in his blood. Now, after receiving the treatment, he doesn’t have to treat himself routinely, unless he needs a procedure.
According to Woldie, the gene coding for factor IX is placed into a vector containing an inactive viral capsid. It is then infused into the patient, where the vector preferentially travels to the liver and helps it make the deficient protein, factor IX.
Patients are monitored during and after the infusion for at least three hours to ensure there are no infusion-related reactions. Potential symptoms during this time may include:
- Elevated levels of a specific blood enzyme.
- Fatigue.
- Feeling unwell.
- Flu-like symptoms.
- Headache.
- Infusion-related reactions.
- Liver enzyme elevations.
- Nausea.
“Patients will start making their own factor IX from the first few weeks of infusion up to six months and longer,” Woldie says. “There have been good long-term results of patients having a factor level that precludes the use of prophylaxis.”
According to CSL, the company that makes Hemgenix, 94 percent of patients in the international clinical trial discontinued their routine factor IX prophylaxis. At the five-year mark after treatment, 94 percent of those patients maintained mild to normal factor IX levels.
For more information about Hemgenix, visit here.
“My team is excited to be able to offer this gene therapy for patients,” says Surender Kanaparthi, chief pharmacy officer at Karmanos Cancer Hospital. “Our mission has always been to expand access to the most advanced treatments available, and gene therapy is the future of hematology and oncology care.”
Hemophilia B patients who are eligible to receive this one-time therapy must currently use factor IX prophylaxis therapy, have current or historical life-threatening bleeding, or have repeated, severe spontaneous bleeding episodes.
