First Patient Completes Treatment with Curative Gene Therapy for Sickle Cell Disease

An 18-year-old patient has been discharged after receiving curative treatment for sickle cell disease with the FDA-approved gene-modifying therapy Lyfgenia™ at Hackensack Meridian Children’s Health Joseph M. Sanzari Children’s Hospital.

As the first pediatric health network in New Jersey to administer Lyfgenia, the Joseph M. Sanzari patient was the first to complete treatment outside of a clinical trial.

The Children’s Hospital, along with Hackensack University Medical Center’s John Theurer Cancer Center, were the first manufacturer-designated Qualified Treatment Centers for the gene therapy treatment in New Jersey, following FDA approval of the treatment.

To date, Joseph M. Sanzari Children’s Hospital has performed five gene therapy procedures for inherited blood disorders — three for sickle cell and two for beta thalassemia. A young girl with beta thalassemia was the first to be treated with Zynteglo in early 2025.

The process of treating the first Sanzari patient with Lyfgenia began last year when the patient’s stem cells were harvested. The blood cells were then sent to a lab where they were modified. During the patient’s most recent month-long hospital stay, the cells were returned to his body following chemotherapy that prepared the bone marrow for the re-engineered cells, which will enable his body to produce healthy red blood cells.

Lyfgenia treatment is only available to patients cared for at Sanzari.

“Sickle cell is a disease that affects every organ in a patient’s body,” said Stacey Rifkin-Zenenberg, D.O., the pediatric hematologist/oncologist and section chief, Pain and Palliative Care, Joseph M. Sanzari Children’s Hospital, who has been a lead investigator of the largest clinical trials for curative treatments for sickle cell. “It has a tremendous impact not only on the patient, but also the family.”

In addition to gene therapy treatment, Joseph M. Sanzari provides curative bone marrow stem cell transplants in partnership with the Adult Stem Cell Transplantation and Cellular Therapy program at the John Theurer Cancer Center. This joint program — which serves adults and children, as does the Lyfgenia gene therapy program — is accredited by the Foundation for the Accreditation of Cellular Therapy (FACT).