The Phase I/II trial is in beta-thalassemia, a rare blood disorder. Another Phase I/II study of the same drug, in sickle cell disease, is expected to infuse its first patient in mid-2019.
The agency also accepted the two companies' IND to begin the Phase I/II study of CTX001 in patients with sickle cell disease, sending CRISPR shares up more than 11 percent Thursday morning.
The future looks bright for the integrated specialty pharmacy model – for health systems, providers, and most importantly, for patients.
In a deal worth up to $25 million for ViaCyte, the companies will work together to create allogeneic stem cell therapies to treat the disease.
FDA slaps clinical hold on Crispr Therapeutics and Vertex Pharmaceuticals' upcoming gene-editing trial.