Is CRISPR safe? Genome editing gets its first FDA scrutiny
https://www.nature.com/articles/d41586-023-03317-7
NEWS EXPLAINER
28 October 2023
Is CRISPR safe? Genome editing gets its first FDA scrutiny
Advisors to the US regulatory agency will examine the safety profile of a CRISPR-based treatment for sickle-cell disease.
Heidi Ledford
A therapy based on the CRISPR-Cas9 genome editing system could become the first of its kind to gain approval from the US Food and Drug Administration. But the treatment designed to alleviate a painful blood condition must first face intense scrutiny by the agency and its advisors.
On 31 October, external advisors to the US Food and Drug Administration (FDA) will meet to discuss a DNA-altering therapy for sickle-cell disease, a genetic condition that can cause misshapen blood cells and sometimes debilitating pain. The advisors discussions are likely to be laser-focused on safety data submitted by the treatments developers, Vertex Pharmaceuticals in Boston, Massachusetts, and CRISPR Therapeutics in Zug, Switzerland.
The key to this is safety, says Mark Walters, a paediatrician at the University of California, San Francisco, who has served on a steering committee advising the two companies on the clinical development of the treatment, called exagamglogene autotemcel (exa-cel), Thats the question that could really affect decision making, and the safety information is still quite limited.
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