FDA halts Vertex, CRISPR sickle-cell trial

May 31, 2018

CRISPR Therapeutics and Vertex Pharmaceuticals announced that the U.S. FDA has placed a clinical hold on the new-drug application for a drug used for the treatment of sickle-cell disease, “pending the resolution of certain questions that will be provided by the FDA as part of its review.”

The IND was submitted to the FDA in April to support the planned initiation of a Phase 1/2 trial for CTX001, being tested in adult patients with sickle cell disease. CTX001 is an investigational, gene-edited autologous hematopoietic stem cell therapy for patients suffering from β-thalassemia and sickle cell disease (SCD).

CTX001 is the first collaborative project in the Vertex-CRISPR partnership started in 2015. Vertex paid $105 million upfront to enter into the collaboration.

It is unclear specifically what the delay is about, but CRISPR and Vertex said they expect to obtain additional information on the FDA’s questions in the near future and will work rapidly with the FDA toward a resolution.

Read the press release