The FDA approved two gene-editing treatments for patients aged 12 years or older with severe sickle cell disease, marking the first cell-based gene therapies for the condition. Exa-cel uses CRISPR gene-editing technology and demonstrated success in a clinical trial, while Lovo-cel uses a different technology and also showed positive results. Both treatments were found to be largely effective and have potential side effects. Access to these treatments is seen as a potential concern due to high costs. The approval of these gene therapies represents a significant step for the sickle cell community despite potential issues related to accessibility.
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FDA OKs Two Gene-Editing Therapies for Sickle Cell Disease
