"CRISPR Breakthrough: Approved Gene-Editing Treatment Poses Infertility Risk for Sickle Cell Patients"
Vertex Pharmaceuticals and CRISPR Therapeutics have received regulatory approval in the UK for their gene therapy, exa-cel, intended to treat sickle cell disease. However, new research published in Nature Medicine suggests a potential risk associated with the treatment. Patients treated with a similar gene therapy approach exhibited a higher proportion of stem cells with driver mutations associated with certain blood cancers. While the risk is not guaranteed, it raises concerns about the therapy unintentionally acting as a filter that removes healthy cells and leaves cells with dangerous mutations intact. The outcome could range from no negative consequences to additional studies being demanded by regulators or, in the worst-case scenario, the therapy being pulled from the market. Investors should be prepared for potential losses and monitor regulatory developments.