10 December 2020

CRISPR-Cas9 gene editing therapy has been successfully used on patients with sickle cell disease and β-thalassaemia, a conference heard at the weekend.

Ten patients worldwide have taken part in two trials of the treatment, the annual meeting of the American Society of Hematology (ASH) heard. According to the researchers, they are the first trials to test a CRISPR-Cas9 gene editing therapy in humans for a genetic disease.

The treatment, called CTX001, aims to increase production of foetal haemoglobin by genetically modifying the patients’ own stem cells using the CRISPR-Cas9 gene editing system. All ten patients experienced “substantial and sustained” increases in foetal haemoglobin production, the conference heard. Overall, the treatment showed safety and efficacy, researchers said.

The study involved seven patients with β-thalassaemia and three with sickle cell disease. Before the treatment, the β-thalassaemia patients needed blood transfusions every three to four weeks, and the three patients with sickle cell diseases experience vaso-occlusive crises with severe pain roughly every other month.

After treatment, none of the thalassaemia patients have needed transfusions – and this has lasted for periods of two to 18 months. None of the three sickle cell patients have experienced crises since transfusion, the researchers said.

Researcher Dr Haydar Frangoul, of the TriStar Centennial Medical Center, in Nashville, Tennessee, USA, said: “There is a great need to find new therapies for beta thalassemia and sickle cell disease.

“What we have been able to do through this study is a tremendous achievement. By gene editing the patient’s own stem cells we may have the potential to make this therapy an option for many patients facing these blood diseases.”

Dr Catherine Bollard, from ASH and the George Washington University, USA, said: “Given that the only FDA-approved cure for sickle cell disease, a bone marrow transplant, is not widely accessible, having another curative option would be life-changing for a large number of the sickle cell disease population.

“While longer follow-up data are needed, this study is extremely exciting for the field.”

Source: Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9–Modified CD34+ Hematopoietic Stem and Progenitor Cells. ASH 6 December 2020

Link: https://ash.confex.com/ash/2020/webprogram/Paper139575.html


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