Posted by Michael Wonder on 06 Jan 2019
CRISPR Therapeutics and Vertex announce FDA fast track designation for CTX001 for the treatment of sickle cell disease
4 January 2019 - CRISPR Therapeutics and Vertex Pharmaceuticals today announced that the U.S. FDA has granted fast track Designation for CTX001 for the treatment of sickle cell disease.
CTX001 is an investigational, autologous, gene-edited haematopoietic stem cell therapy for patients suffering from severe haemoglobinopathies.
In October 2018, CRISPR and Vertex announced the FDA acceptance of the investigational new drug application for CTX001 for the treatment of sickle cell disease (SCD), and enrolment in a Phase 1/2 trial in SCD is currently underway in the U.S. The companies are also evaluating CTX001 for the treatment of β-thalassaemia, and enrolment in a Phase 1/2 trial in β-thalassaemia is currently open at multiple clinical trial sites in Europe.
