Hackensack Meridian Children’s Health Begins Curative Gene Therapy Treatment of Sickle Cell Disease

First patient initiated process for federally approved, one-time Lyfgenia gene therapy treatment in summer 2024

Hackensack Meridian Children’s Health is the first hospital in New Jersey, and in the New York metropolitan area, to administer the Bluebird-manufactured Lyfgenia™ treatment as a curative gene therapy treatment for sickle cell disease (SAD).

A 19-year-old patient has begun the long process of undergoing the gene-modifying treatment at Joseph M. Sanzari Children’s Hospital at Hackensack Meridian Hackensack University Medical Center. The hospital is one of a few pediatric hospitals in the country offering this treatment as one of Bluebird’s designated Qualified Treatment Centers for the gene therapy treatment.

“As the oldest and largest treatment center for sickle cell disease in New Jersey, we are pleased to be the first center in the state to provide this next-generation gene therapy treatment to patients,” said Alfred Gillio, M.D., director of Bone Marrow and Cellular Transplantation. “The Lyfgenia treatment protocol is a very big step in the fight against sickle cell.”

Lyfgenia treatment is available to patients cared for at Sanzari and at K. Hovnanian Children’s Hospital at Hackensack Meridian Jersey Shore University Medical Center, the network’s other children’s hospital. The one-time gene therapy was approved last year by the U.S. Food and Drug Administration for patients ages 12 and older who experience vaso-occlusive events (VOEs).

Lyfgenia treatment involves collecting patients’ stem cells and treating them with Lyfgenia before infusing them back into the patient via a stem cell transplant. Before the transplant, patients must undergo a round of chemotherapy to eliminate faulty blood stem cells. After the transplant, red blood cells generated from the engineered stem cells should produce the anti-sickling hemoglobin, limiting red blood cell sickling and future VOEs.

In addition to gene therapy treatment, which prior to the FDA approval of Lyfgenia was only available when enrolled in a clinical trial, Joseph M. Sanzari has provided curative treatment via bone marrow stem cell transplants as well. Hackensack Meridian Children’s Health provides the only pediatric stem cell transplant and cellular program in New Jersey.

This is a joint program in partnership with the Adult Stem Cell Transplantation and Cellular Therapy available at the John Theurer Cancer Center which is also located on the Hackensack University Medical Center campus. The joint program, which serves children and adults, is accredited by Foundation for the Accreditation for Cellular Therapy (FACT).

Lyfgenia, previously known as LentiGlobin, received U.S. Food and Drug Administration (FDA) approval as a gene therapy treatment for sickle cell disease for adults and children aged 12 and older in December 2023. In 2019, Joseph M. Sanzari Children’s Hospital became one of only a few U.S. hospitals, and the only in New Jersey to offer a LentiGlobin clinical trial.

Lyfgenia treatment is available to patients cared for at Sanzari and at K. Hovnanian Children’s Hospital at Hackensack Meridian Jersey Shore University Medical Center, the network’s other children’s hospital.

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