Key Points
Oct 27 (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 (VRTX.O) and CRISPR Therapeutics' (CRSP.BN) 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...
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...
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