BRIEF—Sickle cell disease candidate on track for regulatory review

In the USA, Vertex Pharmaceuticals and CRISPR Therapeutics have been granted a rolling review for their sickle cell disease (SCD) candidate exagamglogene autotemcel (exa-cel).

Vertex will undertake its regulatory submission for exa-cel, a one-time and potentially curative therapy, over an approximately five-month period beginning in November.

The firms are seeking approval for the CRISPR/Cas9 gene-edited therapy for both SCD and transfusion-dependent beta thalassemia.

The company said it was also on track to submit for approval in the UK and Europe by the end of 2022.