(Reuters) – The U.S. Food and Drug Administration’s staff reviewers on Friday asked the regulator’s advisory panel to consider the need for additional studies for Vertex Pharmaceuticals and CRISPR Therapeutics’ sickle cell disease gene therapy.
The FDA’s staff reviewers said it was not clear if the limited donor cells used for assessment were adequate to evaluate the potential safety risks of the therapy.
The gene editing technology has the potential to produce unintended genomic alterations, the staff said in the briefing documents.
The comments come ahead of a panel meeting of the agency’s outside advisers, who provide non-binding recommendations to the FDA, slated on Tuesday to discuss the therapy.
Vertex and CRISPR Therapeutics are seeking the health regulator’s nod for the single-dose gene-editing cell therapy, which is based on Nobel Prize-winning CRISPR technology, for the inherited red-blood-cell disorder that causes the cells to become sickle-shaped due to abnormal levels of hemoglobin in the body.
(Reporting by Sriparna Roy in Bengaluru; Editing by Shinjini Ganguli)
