"Using CRISPR/Cas9 you are able to actually fix a disease, that otherwise, was not fixable," Dr. Haydar Frangoul explains the data he presented at ASH 2022.
Data from a successful clinical trial involving autotemcel (exa-cel) infusion in patients with severe sickle cell disease (SCD) demostrated the gene therapy's ability to eliminate vaso-occlusive crises (VOCs) for up to 3 years.
"What we have been able to do with this therapy is open the door to allow us to show that using your own cells, being genetically modified, using CRISPR/Cas9 you are able to actually fix a disease that otherwise was not fixable," Haydar Frangoul, MD, Medical Director, Sarah Cannon Pediatric Hematology/Oncology & Cellular Therapy at TriStar Centennial, said in an interview with HCPLive.
The study, which Frangoul presented at the American Society of Hematology (ASH) Annual Meeting and Exposition, also showed that patients were able to sustain increased levels of HbF and total Hb. Patients with sickle cell disease don't currently have options like this, and the few they have come with high risks.
"There is a complication called graft-versus-host disease (GvHD), where the donor cells attack the recipient body, and that is actually more debilitating than sickle cell disease," he explained. "We don't want to exchange a chronic disease with another chronic disease."
According to Frangoul, Vertex Pharmaceuticals and CRISPR Therapeutics are submitting the data to the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). He shared his excitement about the possibility of this treatment becoming available and changing the lives of patients in the future.