July 31,2024
Cure 4 The Kids Foundation (C4K) announced it has completed Nevada’s first successful infusion of Hemgenix®, a gene therapy option for adult patients with hemophilia B — a genetic bleeding disorder in which people do not produce a protein needed to create blood clots.
Depending on severity, a hemophilia B patient can require multiple infusions of factor IX on a weekly basis to prevent a bleed. However, after the one-time Hemgenix® infusion, the gene therapy is expected to enable the individual’s body to produce their own Factor IX, eliminating weekly infusions.
Under the medical supervision of Dr. Aimee Foord, director of C4K’s bleeding and clotting clinic along with Dr. Joseph Lasky, medical director of C4K and the entire C4K medical team, the infusion was completed in C4K’s infusion suite.
The patient, a 39-year-old male from Arizona, will be monitored on a regular basis to ensure the treatment’s success.
“This treatment is ground-breaking for a number of reasons,” said Annette Logan-Parker, chief innovation and advocacy officer at C4K. “Most importantly, Hemgenix® gives the patient a freedom from daily or weekly factor infusions they have not had since their original hemophilia diagnosis. From a larger perspective, being approved to provide this life-changing treatment is another example of how Cure 4 The Kids Foundation is bringing innovation and cutting-edge therapies to the patients of Nevada and the western United States.”
Hemgenix® (etranacogene dezaparvovec-drlb) is the first gene therapy for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening bleeding, or have repeated, serious spontaneous bleeding episodes. Although Hemgenix® is not a cure for hemophilia B patients, the medical advancement may reduce, if not, eliminate weekly preventative factor replacement and bleeds.
Hemgenix® relies upon a gene transfer that is administered through intravenous (IV) infusion. The approach introduces a “working” or functional gene into liver cells to “instruct them” to produce factor IX protein, which is deficient in patients with hemophilia B, and which helps form blood clots to prevent prolonged, excessive or life-threatening bleeding.
Hemgenix® has the potential to fundamentally transform the treatment paradigm through a single, one-time infusion in people living with hemophilia B by addressing the genetic cause of the condition.
Because the genes that cause hemophilia A and B are located on the sex-determining X chromosome, the disorder disproportionately affects males. According to the U.S. Centers for Disease Control and Prevention (CDC), as many as 33,000 males in the United States are living with hemophilia. Hemophilia B is a rarer form of the disorder, found in just 3.7 cases per 100,000 males.
