The U.S. Food and Drug Administration (FDA) is expected to approve a groundbreaking gene-editing treatment for sickle cell disease by the end of this week. The treatment, called exa-cel, uses CRISPR technology to edit a patient’s DNA and alleviate the symptoms of the inherited blood disorder. Currently, sickle cell disease affects about 100,000 people in the U.S., primarily those of African descent. The only cure is a bone marrow transplant, often difficult to find. Exa-cel offers hope as a new genetic treatment that could provide relief without the need for a donor. However, the treatment process takes months and is expected to cost around $2 million per patient.
Vertex Pharmaceuticals, in collaboration with CRISPR Therapeutics, developed exa-cel, and anticipates a multibillion-dollar opportunity if approved. The company plans to focus on the approximately 32,000 individuals in the U.S. and Europe with severe forms of the disease. However, Wall Street remains skeptical about exa-cel’s potential profitability, estimating $1.2 billion in sales for Vertex in 2028. The FDA is also reviewing another gene therapy from Bluebird Bio called lovo-cel, which could be approved later this month.
For patients like Joe Tsogbe, who underwent the exa-cel trial in 2021, the treatment has been life-changing. Tsogbe, a sickle cell disease sufferer from Togo, experienced frequent hospitalizations until receiving exa-cel two years ago. Since then, he has not experienced a pain crisis and has been able to pursue his passions and lead a fulfilling life. However, the high cost and limited availability of the treatment could pose challenges for many patients, as well as resistance from insurers. Despite these issues, exa-cel represents a significant milestone in the field of gene therapy and offers hope for sickle cell disease patients.