FDA Approves Gene Editing Technology As Potential Sickle Cell Cure, Hackensack University Medical Center Using CRISPR Technology To Treat Patients With Sickle Cell Disease

The Food and Drug Administration (FDA) approved two gene editing therapies for the treatment of sickle cell disease. Experts hope it’s a cure, the first, for the inherited blood disorder that affects more than 100,000 Americans with extreme pain.

In a historic moment, the FDA says CRISPR, a gene editing technology, whose inventors won the Nobel Prize in 2020, can be used to edit the genetic mutation that causes sickle cell for patients 12 and older, hopefully curing patients of the disease long term. The FDA estimates that about 20,000 patients who are 12 and older and have episodes of debilitating pain will be eligible for the therapy.

Sickle Cell Disease affects predominantly Black people - 1 in 365 Black babies are born with the disease nationally. 

Hackensack University Medical Center’s Joseph M. Sanzari Children’s Hospital and John Theurer Cancer Center has been evaluating gene therapy for severe sickle cell disease in adolescents and adults and is one of a limited number of centers to do so with an active program in allogeneic stem cell transplantation and gene therapy. 

Hackensack clinicians are now gearing up to offer both the newly approved gene therapy products for sickle cell disease as well as newer gene therapy products in clinical trials, while  continuing the program in stem cell transplantation, offering a suite of different advanced treatment options for people with sickle cell disease.

Before the approval of this gene editing, the only treatment for sickle cell was a complicated bone marrow transplant, which can be difficult because it requires a closely matching donor, and carries the risk of rejection. CRISPR does not require a matching donor and instead uses the patient's own cells. 

Experts with the hospital’s stem cell transplantation and cellular therapy program are available for interviews about their participation in the CRISPR trials, results and the future of sickle cell treatment.