By Reuters News
Oct 31 (Reuters) – A panel of advisers to the U.S. health regulator said on Tuesday Vertex Pharmaceuticals (VRTX.O) and CRISPR Therapeutics (CRSP.BN) could assess potential safety risks of their sickle cell disease gene therapy after approval.
If the therapy is approved, Vertex has proposed a 15-year follow up of patients to evaluate the safety outcomes of the therapy.
The inherited red-blood-cell disorder causes the cells to become sickle-shaped due to abnormal levels of hemoglobin in the body. A vaso-occlusive crisis occurs when sickled red blood cells block blood flow to the point that tissues become deprived of oxygen and causes pain.