The U.S. Food and Drug Administration has approved the first gene-editing treatment for adults with severe sickle cell disease, a milestone that could change care for patients with limited options. The therapy uses CRISPR technology to target the genetic mutation behind the inherited blood disorder.

Sickle cell disease can cause intense pain, organ damage, strokes and repeated hospitalizations. For adults with severe cases, existing treatments often manage symptoms rather than address the underlying cause. Regulators said the new therapy offers a different approach by editing cells to help reduce the disease’s impact.

The approval marks a major step for CRISPR-based medicine, a field that has moved from laboratory research into clinical use after years of development and debate over safety, access and cost. Health experts say the treatment’s long-term effectiveness and affordability will be closely watched as it becomes available to eligible patients.

While the decision is expected to bring hope to many families affected by sickle cell disease, access may remain limited at first because of the complexity of the procedure and the resources required to deliver it. The approval nonetheless represents a significant advance in the effort to treat a painful and often life-limiting condition.