Vertex Pharmaceuticals and CRISPR Therapeutics's gene editing therapy, exagamglogene autotemcel (exa-cel), for the treatment of sickle cell disease in patients aged 12 and older, has successfully passed through the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee, reportedly the last step before the FDA's final decision. The FDA is expected to make a decision on the therapy by December 8.

The therapy, which aims to restore normal red blood cell function and alleviate symptoms, has proved to be safe and effective in preventing painful episodes associated with the disease. However, there are concerns about potential long-term safety issues.

Exa-cel is the first therapy developed by the duo, which entered into a partnership in October 2015. If approved by the FDA, it will be the first gene editing therapy available for SCD patients.