US FDA Approves First Crispr-Based Therapy for Sickle Cell Disease
In late 2023, the US Food and Drug Administration approved the first Crispr gene editing therapy for sickle cell disease, developed by Crispr Therapeutics and Vertex Pharmaceuticals. This therapy targets a single-letter mutation in the genetic code that causes sickle cell disease, a condition long neglected by research. The treatment has shown positive results, with one patient remaining symptom-free for four years post-treatment. While promising, the therapy costs over $2 million per patient and is only available at a limited number of US facilities. Experts emphasize the need for affordable, accessible gene-editing therapies in the coming years, as market forces alone cannot ensure affordability. The therapy highlights the rapid progress in gene editing since 2012, but underscores the importance of equitable access to life-saving treatments.