EHA 2022: data show ongoing benefits of one-time gene therapy

Vertex Pharmaceuticals (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) have presented new data on exa-cel at the European Hematology Association (EHA) annual meeting.

Formerly known as CTX001, exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene‑edited therapy that is being evaluated for people with rare hematological disorders.

The firms said data from the CLIMB-111, CLIMB-121 and CLIMB-131 studies show the “potentially transformative profile” of the option, for people with transfusion-dependent beta thalassemia (TDT) or severe sickle cell disease (SCD).