FDA gives go-ahead for CRISPR-based sickle cell disease trial
Pharmaphorum Media Limited | October 11, 2018
Development of a stem cell therapy for sickle cell disease from Vertex and development partner CRISPR therapeutics can go ahead after the FDA lifted a hold on a review. The companies had applied to begin an early-stage trial of CTX001, a gene therapy derived from a patient’s own stem cells, for beta-thalassemia and sickle cell disease. Although it’s a long way from the market, the drug could be one of the first to use the revolutionary CRISPR/Cas9 gene editing technology to correct a genetic defect causing a disease.