First CRISPR Trial for Patients with Severe Sickle Cell Disease to Begin

Scientists at the University of California (UC) Berkeley, the University of California San Francisco (UCSF), and the University of California Los Angeles (UCLA) have received approval from the U.S. Food and Drug Administration (FDA) to launch a clinical trial of CRISPR_SCD001, a CRISPR/Cas9-based gene therapy for sickle cell disease (SCD).

CRISPR_SCD001 was developed at Innovative Genomics Institute (IGI), a joint initiative between UC Berkeley, UCSF, and UCLA founded by Nobel Prize winner Jennifer Doudna, PhD. Dr. Doudna approached researchers at UCSF Benioff Children’s Hospital Oakland with the idea for the treatment in 2014.

Beginning this summer, the four-year trial will enroll six adults and three adolescents with severe SCD in Oakland and Los Angeles. The investigators will use non–virally delivered CRISPR gene editing to attempt to correct the faulty beta-globin gene in the patients’ cells.

“This therapy has the potential to transform SCD care by producing an accessible, curative treatment that is safer than the current therapy of stem cell transplant from a healthy bone marrow donor,” said principal investigator Mark Walters, MD, a professor of pediatrics at UCSF. “If this is successfully applied in young patients, it has the potential to prevent irreversible complications of the disease.”

“Gene therapy and gene editing allow each patient to serve as their own stem cell donor,” explained Donald Kohn, MD, who will lead the study at UCLA. “In theory, these approaches should be much safer than a transplant from another person and could become universally available because they eliminate the need to find the needle in a haystack that is a matched stem cell donor.”

Source: University of California press release, March 30, 2021.