Karmanos Cancer Institute Pioneers Hemgenix Gene Therapy for Hemophilia B

The Barbara Ann Karmanos Cancer Institute in Detroit, Michigan, has grow the first and only independent cancer center in the United States to offer Hemgenix® (etranacogene dezaparvovec-drlb), a groundbreaking one-time gene therapy for adults with hemophilia B. This advancement provides a potential long-term treatment option for individuals living with this rare genetic bleeding disorder.

Understanding Hemophilia B

Hemophilia B is a rare, inherited bleeding disorder caused by a deficiency in factor IX, a crucial protein needed for blood clotting FDA. Without sufficient factor IX, individuals with hemophilia B experience prolonged bleeding after injuries or surgery, and in severe cases, spontaneous bleeding, particularly into joints. While most cases are hereditary, a diagnosis can sometimes occur without a family history, often during infancy (such as after circumcision) or later in life following excessive bleeding after a procedure Karmanos.

Hemophilia B predominantly affects males, who inherit the affected X chromosome from their mothers. Females can be carriers of the gene and may experience milder bleeding symptoms, particularly during menstruation or surgery Karmanos.

How Hemgenix Works: A Gene Therapy Approach

Hemgenix represents a paradigm shift in hemophilia B treatment. Prior to gene therapy, the standard of care involved lifelong, routine infusions of factor IX replacement therapy (prophylaxis) to prevent bleeding episodes. Hemgenix offers a potential alternative by addressing the underlying genetic cause of the disease.

“The gene coding for factor IX is placed into a vector containing an inactive viral capsid. We then infuse it into the patient intravenously, where the vector preferentially travels to the liver and helps it produce the deficient protein, factor IX,” explained Indryas Woldie, M.D., a hematologist and medical oncologist at Karmanos Karmanos.

The therapy aims to enable patients to produce their own factor IX, potentially eliminating or significantly reducing the necessitate for regular infusions. Clinical trial data indicates that 94% of patients in an international trial were able to discontinue routine factor IX prophylaxis, with 94% maintaining mild to normal factor IX levels at the five-year mark Karmanos.

Patient Experience and Eligibility

Steve W. Of Clinton Township, Michigan, was the first patient to receive Hemgenix at Karmanos on October 28, 2025, and the 52nd patient treated in the U.S. After clinical studies Karmanos. He previously required weekly factor IX injections. “I’m just particularly blessed to be in the right spot at the right time with the right people,” Steve expressed. “And it’s working… I did have some days where I was just tired, but about a week after the infusion, I was good.”

Hemgenix is indicated for adults with hemophilia B who currently apply factor IX prophylaxis, have a history of life-threatening bleeding, or experience repeated, severe spontaneous bleeding episodes FDA.

Karmanos’ Commitment to Advanced Therapies

“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,” said Surender Kanaparthi, R.Ph., MBA, DPLA, FACHE, chief pharmacy officer at Karmanos Cancer Hospital Karmanos. The implementation of Hemgenix required extensive training for healthcare teams at Karmanos to ensure safe and effective administration.

For more information about Hemgenix and eligibility criteria, visit karmanos.org/hemgenix or call 1-800-KARMANOS.