CRISPR Therapeutics and Vertex Pharmaceuticals have been collaborating to develop a new treatment option for Sickle Cell Disease. It’s called CTX001, a type of stem cell therapy.

This drug has just received Fast Track Designation by the FDA, bringing even more hope about its potential as a therapy for this population of patients.

Fast Track Designation

The FDA offers Fast Track Designation to help accelerate the development of new drugs which may help serve patient populations whose needs are currently unmet. There are various benefits that may accompany this designation. Perhaps most importantly, it gives the drug developers increased communication with the FDA, helping to expedite review. It also increases the chances that the drug will receive Priority Review or Accelerated Approval.

CTX001

CTX001 is an ex vivo gene-edited therapy. CRISPR and Vertex believe that this therapy has the potential to help the body produce more fetal hemoglobin, or HbF (a form of oxygen carrying hemoglobin), in the red blood cells. HbF normally is depleted after infancy, but for Sickle Cell Disease, high levels of this form of hemoglobin could significantly improve the symptoms of the disease and ultimately the quality of life of patients.

Back in October of 2018, CTX001’s Investigational New Drug application was accepted by the FDA as a potential new treatment option for Sickle Cell disease.

CRISPR and Vertex are also working on CTX001 as a potential treatment option for β-thalassemia and they are currently enrolling a Phase 1/2 trial for the condition in Europe.