Another Study Finds CRISPR Gene Therapy Fights Sickle Cell
18 Jun 2024
Gene TherapyClinical Study
TUESDAY, June 18, 2024 -- There's more good news in the battle against sickle cell disease, with another trial finding CRISPR gene-editing therapy delivering impressive results for patients.
“It’s encouraging that this gene-editing treatment continues to show promising efficacy for sickle cell patients,” said study lead investigator Dr. Rabi Hanna. He's chair of the division of pediatric hematology oncology and blood and marrow transplantation at Cleveland Clinic Children’s.
Sickle cell anemia is a painful, inherited genetic disorder which creates misshapen sickle-shaped red blood cells. Over 100,000 Americans are thought to have sickle cell disease, which can shorten life spans and is much more common among Black Americans.
Recent advances in what's known as CRISPR gene-editing technology helps correct the disorder by tweaking the underlying gene abnormality behind it.
The Cleveland Clinic CRISPR approach is called renizgamglogene autogedtemcel (shortened to "reni-cel"). It's am experimental one-time treatment that uses the patient's own stem cells to correct the genetic abnormality.
Two of the 18 patients in the new trial were treated at the Cleveland Clinic.
In the procedure, patients' stem cells are harvested and then sent for gene editing in the lab. Patients also underwent chemotherapy to make room for the new stem cells to be infused into their bone marrow.
"Following treatment, all patients successfully regained their white blood cells and platelets," according to a clinic news release. "Importantly, all patients have remained free of painful events since treatment, and those followed for five months or greater have seen their anemia resolve."
The treatment also appeared to have no serious side effects, Hanna's group reported.
"These latest results offer hope that this new experimental treatment will continue to show progress and get us closer to a functional cure for this devastating disease," he said.
The findings were presented Thursday at the European Hematology Association 2024 Hybrid Congress (EHA) in Madrid. Such findings should be considered preliminary until published in a peer-reviewed journal.
The trial was funded by gene-editing company Editas Medicine.
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