Hemoglobin Editors

A group of researchers from the Children's Hospital of Philadelphia presented new data on the experimental treatment of transfusion-dependent (TDT) and severe sickle cell anemia (SCA) at the Congress of the European Hematology Association (EHA). The one–time procedure developed by Vertex Pharmaceuticals and CRISPR Therapeutics has shown efficacy that has not decreased for three years, and the level of safety is as with autologous transplantation, and much higher than with allogeneic (donor) transplantation.

The findings are further evidence that gene therapy can be a transformative therapy for patients with sickle cell anemia and beta-thalassemia. It is necessary to continue to confirm their validity in clinical trials.

The report presented data from two clinical trials of the drug exa-cel (exagamglogene autotemcel), formerly known as CTX001, which uses the CRISPR gene editor to increase fetal hemoglobin production and correct a defective hemoglobin gene associated with both diseases.

The researchers presented the results of a survey of 75 patients: 44 with TDT and 31 with SKA. Of the 44 patients with TDT after exa-cel administration, 42 did not need transfusions during the follow-up period, which ranged from 1.2 to 37.2 months. None of the 31 patients with SKA experienced crises during follow-up, which ranged from 2 to 32.3 months.

Safety was generally consistent with myeloablative conditioning (radiation or chemotherapeutic effects leading to complete eradication of hematopoiesis) and autologous stem cell transplantation. Two of the 44 patients with TDT had serious adverse effects potentially associated with exa-cel, while none of the patients with SKA had them.

Article F.Locatelli et al. Efficacy and Safety of a Single Dose of CTX001 For Transfusion-Dependent Beta-Thalassemia and Severe Sickle Cell Disease is published in the journal EHA Library.

Aminat Adzhieva, portal "Eternal Youth" http://vechnayamolodost.ru .