26 June 2024

A European conference has heard of the latest, promising results from a trial of a new gene editing treatment for sickle cell.

18 patients have been taking part in the RUBY study. Researchers reported that all patients have been free of painful vaso-occlusive events, and those patients who have been followed for more than five-months have also been free of anaemia after completing treatment.

The latest findings of the RUBY study were reported to the European Hematology Association conference in June 2024 in Madrid, Spain.

The treatment is known as renizgamglogene autogedtemcel, or reni-cel, and involves the editing of the patients’ own stem cells. Haematopoietic stem cells (HSCs) are edited using an upgraded CRISPR/Cas12a gene editing technology, to reactivate the gamma-globin gene and increase the levels of foetal haemoglobin in red blood cells. The edited HSCs are then reinfused back to the patient.

The trial marks the first clinical use of the ‘AsCas12a’ enzyme in the gene editing process – a more efficient version compared to Cas9 previously used in CRISPR editing. 

Researchers said that, after treatment, all the patients successfully regained white blood cells and platelets and that, so far, all the patients have been free of painful events.

Researcher Dr Rabi Hanna, of Cleveland Clinic Children’s, Ohio, where two of the 18 patients were treated, said: “It’s encouraging that this gene-editing treatment continues to show promising efficacy for sickle cell patients.

“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.”


Hanna R, Frangoul H, McKinney C, Pineiro L, Mapara M, Dalal J, Rangarajan H, Chang KH, Jaskolka M, Kim K, Mei B, Afonja O, Walters M (2024) “Reni-cel, the first AsCas12a gene-edited cell therapy, led to hemoglobin normalization and increased fetal hemoglobin in severe sickle cell disease patients in an interim analysis of the RUBY trial.” Oral presentation at the 29th European Hematology Association Congress, 13-16 June 2024, Madrid, Spain.

Link: https://library.ehaweb.org/eha/2024/eha2024-congress/422389/rabi.hanna.reni-cel.the.first.ascas12a.gene-edited.cell.therapy.led.to.html

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