In a late-breaking abstract presented at the European Hematology Association (EHA) Congress, a group of researchers that includes Children’s Hospital of Philadelphia (CHOP) presented new data on an investigational therapy for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD). The one-time treatment, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, showed continued benefits at up to three years after administration, with a safety profile as expected for autologous transplant and potentially much safer than allogeneic transplant (from a donor).
Benefits of CRISPR-edited gene therapy for patients with blood disorders
In a late-breaking abstract presented at the European Hematology Association (EHA) Congress, a group of researchers that includes Children's Hospital of Philadelphia (CHOP) presented new data on an investigational therapy for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD). The one-time treatment, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, showed continued benefits at up to three years after administration, with a safety profile as expected for autologous transplant and potentially much safer than allogeneic transplant (from a donor).