Vertex Pharmaceuticals and CRISPR Therapeutics Rallied Today
Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP) announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials.
A patient with transfusion-dependent beta-thalassemia (TDT) received CTX001 in the first quarter of 2019. Data for this patient reflect nine months of safety and efficacy follow-up.
A patient with severe sickle cell disease (SCD) received CTX001 in mid-2019. Data for this patient reflect four months of safety and efficacy follow-up.
These studies are ongoing and patients will be followed for approximately two years post-infusion.
Several additional patients have been enrolled and have had drug products manufactured across the two studies.