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LLU Health Introduces Transformative Gene Therapy for Hemophilia

Breakthrough has the potential to transform the lives of countless children and adults.

Linda Ha, Loma Linda University Health News
LLU Health Introduces Transformative Gene Therapy for Hemophilia
Physician Akshat Jain and his hemophilia patient Edgar Larios. [Photo: Loma Linda University Health News]

Loma Linda University Health’s Center of Excellence Hemophilia Program now offers HEMGENIX, the first gene therapy for hemophilia B approved by the US Food and Drug Administration (FDA), alongside “dirloctocogene samoparvovec,” a second-generation advanced gene therapy for hemophilia A.

This breakthrough has the potential to transform the lives of countless children and adult patients suffering from these debilitating conditions, expert said.

Hemophilia is a genetic disorder that impairs the blood’s ability to clot and has long been life-altering for those affected. Hemophilia can turn minor injuries into serious health risks due to uncontrolled bleeding. It is often referred to as the “royal disease” due to its prevalence in the descendants of Queen Victoria of the United Kingdom, according to the National Library of Medicine. Queen Victoria was a carrier of hemophilia, a genetic disorder that affects the blood’s ability to clot, leading to excessive bleeding from even minor injuries. The disorder is typically passed down through the X chromosome, which is why it primarily affects males, while females are usually carriers.

Traditionally, treating hemophilia has required patients to undergo frequent infusions of clotting factors. This often means multiple self-injections each week to prevent dangerous bleeding episodes, a regimen that can be both painful and restrictive.

HEMGENIX allows patients’ bodies to produce their own missing clotting factors. This one-time treatment involves a three-hour infusion that delivers a modified gene to the liver, enabling the natural production of clotting factors.

“HEMGENIX has proven to be transformative for patients,” said Akshat Jain, physician and director of Inherited Bleeding Disorders at Loma Linda University Children’s Health. “It raises clotting factor levels from less than 1 percent to around 30 to 40 percent, which is a dramatic improvement. While the normal range is 55 percent and up, this increase significantly reduces the frequency and severity of bleeding episodes, allowing patients to lead near-normal lives.”

Patients can walk, play sports, and work without the constant fear of life-threatening bleeding.

Edgar Larios, 18, a hemophilia patient, is being considered for gene therapy treatment. “My life would change dramatically as I would not have to keep getting injections hundreds of times and would be able to be more active without fear of bleeding out,” Larios said.

LLUH has structured its program to ensure that financial barriers do not prevent access to this life-changing therapy, with many patients relying on Medicare or Medicaid.

“Our center’s wholistic approach includes extensive infrastructure and safety protocols to handle gene therapy,” Jain said. “We are one of the few centers in the United States and the only one in Inland Empire [a region in Southern California] activated to provide this advanced treatment.”

The original version of this story was posted on Loma Linda University Health news site.

Linda Ha, Loma Linda University Health News