05 September 2023

A new gene therapy aimed at haematopoietic stem cells has shown promise in the treatment of sickle cell disease, according to the latest results of a clinical trial.

The findings, published in the New England Journal of Medicine, add to the growing body of evidence that support gene therapy for treating the painful condition, researchers say.

The University of Chicago Medicine Comer Children’s Hospital was one of three sites to enrol patients in the clinical trial, which used CRISPR-Cas9 to edit specific genes in haematopoietic stem cells taken from each patient.

The therapy was the first to target a new area of the genome, and use cryopreserved stem cells. The edits increased the cells’ production of foetal haemoglobin (HbF), which can replace unhealthy, sickled haemoglobin in the blood and protect against the complications of sickle cell disease.

The patients received their own edited cells as therapeutic infusions, and they all reported a decrease in vaso-occlusive events.

Senior author Dr James LaBelle, director of the paediatric stem cell and cellular therapy program at UChicago Medicine, said: “The biggest take-home message is that there are now more potentially curative therapies for sickle cell disease than ever before that lie outside of using someone else’s stem cells, which can bring a host of other complications.”

He said the study is important as part of the growing body of evidence supporting the viability of gene therapy as a treatment for sickle cell disease. Two other gene therapies for the disease are awaiting FDA approval this year.

“The data from this trial supports bringing on similar gene therapies for sickle cell disease and for other bone marrow-derived diseases. If we didn't have this data, those wouldn’t move forward,” he added.


Sharma A, Boelens JJ, Cancio M, Hankins JS, Bhad P, Azizy M, Lewandowski A, Zhao X, Chitnis S, Peddinti R, Zheng Y, Kapoor N, Ciceri F, Maclachlan T, Yang Y, Liu Y, Yuan J, Naumann U, Yu VWC, Stevenson SC, De Vita S, LaBelle JL. (2023) “CRISPR-Cas9 Editing of the HBG1/HBG2 Promoters to Treat Sickle Cell Disease.” New England Journal of Medicine, doi: 10.1056/NEJMoa2215643

Link: https://www.nejm.org/doi/10.1056/NEJMoa2215643 

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