Stem cell therapy delivers potential for sickle cell disease
A Novartis’ Phase I/II study for sickle cell disease trialling the gene therapy OTQ92 is the first treatment to target a new genetic area and use cryopreserved stem cells, according to new paper published in NEMJ.
New research published in the New England Journal of Medicine has indicated that stem cell gene therapy could provide a curative treatment for sickle cell disease (SCD).
Based on new data from the Novartis Phase I/II clinical trial, which used the first therapy to target a new genetic area and use cryopreserved stem cells, trial participants reported a decrease in vaso-occlusive events.
It is the second therapy to use CRISPR-Cas9 technology for sickle cell disease. In comparison, other gene therapy studies have used lentiviruses as a treatment.
Dr LaBelle added that data from this trial “supports bringing on similar gene therapies for sickle cell disease and for other bone marrow-derived diseases.”
At present, two other gene therapies for sickle cell disease are awaiting approval from the US Food and Drug Administration (FDA) in 2023.
Preliminary results from another stem cell gene therapy using CRISPR gene-editing was announced in June 2023. It was the first time the technology demonstrated capability in altering a defective gene in a human study.
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