Canadian first: One-time gene therapy given to London, Ont., hemophilia B patient

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A London, Ont., patient has become the first in Canada to receive a publicly funded gene therapy for hemophilia B, in what specialists say could mark a major step forward in treatment.

London Health Sciences Centre says it has administered HEMGENIX, a one-time gene therapy for adults with hemophilia B, a rare genetic bleeding disorder caused by a deficiency of clotting Factor IX.

According to Harvard Health, the disease prevents blood from clotting properly, often resulting in a lifelong dependence on medication to manage bleeding. People living with hemophilia B may experience spontaneous or prolonged bleeding episodes that can lead to joint damage, chronic pain, reduced mobility, and other long-term complications that affect quality of life.

Dr. Chai Phua, who oversaw the treatment, says the milestone signals a shift in how the condition may be managed in Canada.

The patient, identified as Basel A., said the therapy represents a significant change after years of living with the physical and emotional burden of the disease.

Hemophilia B typically requires ongoing treatment, including regular transfusions and careful monitoring to prevent bleeding episodes. Basel says the condition meant constant vigilance in daily life.

The gene therapy is designed as a single infusion, replacing the need for frequent treatments.

Experts say the development reflects how gene therapies are beginning to move from experimental promise to real-world care in Canada.

“This is a meaningful moment for the bleeding disorders community in Canada,” said Emil Wijnker, president of the Canadian Hemophilia Society.“For many, hemophilia B is a constant consideration in everyday life. Advances like gene therapy signal real progress, with the potential to ease that burden and meaningfully change how people manage their condition over time.”

Hemophilia affects an estimated 3,800 Canadians. The condition can lead to repeated bleeding episodes, long-term joint damage and chronic pain, while lifelong treatment can carry significant physical, emotional and financial costs for patients and the health-care system.

HEMGENIX is currently eligible for public reimbursement in Ontario and British Columbia for patients who meet specific clinical criteria.

Health officials say the London case marks a milestone in access to advanced therapies for eligible patients and their families.