Gene therapy has had a rocky history in moving from bench to bedside, especially after a young patient died in one of the first gene-therapy trials in 1999. But the discovery of a more refined way to manipulate genes, called CRISPR, has for the past decade renewed interest in developing ways to modify genes responsible for a number of diseases.
Among the first patients to benefit are those with sickle cell anemia, which results from a single change in the DNA in a gene that codes for part of the hemoglobin protein. That mutation causes red blood cells to collapse in on themselves and jam up narrow vessels, causing intense pain. CRISPR Therapeutics, whose CEO is Samarth Kulkarni, and Vertex Pharmaceuticals earned the first U.S. Food and Drug Administration (FDA) approval for any CRISPR-based treatment in the U.S. in 2023—for the gene therapy Casgevy, which essentially cures patients of sickle-cell disease. In 2024, the FDA also approved Casgevy for the blood disorder beta thalassemia. Yet while Casgevy is the first potentially curative treatment for these disorders, its high cost keeps it out of reach for many.
Kulkarni is now focused on applying CRISPR to more common diseases and driving down treatment costs by developing gene-editing therapies that work inside of patients, rather than requiring that the genetic manipulations be done in a lab. In May, the company announced promising early results in addressing genetic changes that cause high levels of LDL cholesterol and triglycerides that contribute to heart disease. “We are moving to address common diseases like cardiovascular disease, autoimmune diseases, oncology, and diabetes that are big killers,” says Kulkarni. “This marks a shift for the company to bring medicines that are scalable and in an affordable manner to patients.”