CRISPR Therapeutics and Vertex Pharmaceuticals announced that the U.S. FDA has lifted the clinical hold and accepted the Investigational New Drug application for a new treatment for sickle cell disease.
The IND was submitted to the FDA in April, and a month later, Vertex announced the hold “pending the resolution of certain questions that will be provided by the FDA as part of its review.”
CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies. It is the first collaborative project in the Vertex-CRISPR partnership started in 2015. Vertex paid $105 million upfront to enter into the collaboration.
In addition to the acceptance of the IND, CRISPR and Vertex previously announced that they had obtained approvals of Clinical Trial Applications for CTX001 in multiple countries outside the U.S. for both β-thalassemia and SCD
Read the press release