MDI Biological Laboratory
MDI Science Café

Gene Therapy: A Potential Cure for Sickle Cell Disease

  • March 11, 2024
  • 5:00 - 6:00 pm
  • Online-only Zoom event

Edward Benz Jr., M.D., directs the NIH's Cure Sickle Cell Initiative. Also CEO emeritus of the Dana-Farber Cancer Institute and Vice-Chair of the MDI Bio Lab Board of Trustees, he will speak about how the promise of gene therapies is being realized today.

In 2018, the National Institutes of Health launched an effort to speed the development of cures for sickle cell disease, headed by the Vice-Chair of the MDI Bio Lab Board of Trustees, Edward Benz Jr., M.D.

In December 2023, the U.S. Food & Drug Administration approved the first cell-based CRISPR-Cas9 gene therapy, called CASGEVY, for the treatment of sickle-cell disease in patients 12 years and older. The FDA approval heralds the long-sought use of CRISPR gene-editing technology in medical treatments or therapeutics.

And it’s a potentially life-changing development for patients living with sickle-cell disease, whose therapeutic options are otherwise limited. A heritable, life-long, and often lethal blood cell disorder, sickle-cell disease affects an estimated 100,000 individuals in the United States, most of whom are African Americans.

Dr. Benz will outline and explain how CRISPR can improve the drug development process by directly increasing or decreasing a specific gene’s activities, to determine which gene products are responsible for a given disease and targetable for therapy. With CASGEVY, genes that affect the function and shape of red blood cells are edited in a way that appears to cure sickle cell disease for a year or more.

The Cure Sickle Cell Initiative is a collaborative, patient-focused research effort designed to accelerate promising genetic therapies. It considers non-traditional ways to advance research, bringing  together patients, advocates, caregivers, providers, researchers, industry, and others.

Dr. Benz is president and CEO emeritus of Dana-Farber Cancer Institute and principal investigator emeritus of the Dana-Farber/Harvard Cancer Center. He is the Richard and Susan Smith Distinguished Professor of Medicine, Professor of Pediatrics and Professor of Genetics at Harvard Medical School.

An active clinician and teacher, Benz is an internationally acclaimed hematologist and an NIH-funded investigator on the molecular mechanics of blood disease; he is also an active clinician and teacher. He is co-author of three textbooks, two of which have won international awards. He has served as president of the American Society of Clinical Investigation, the American Society of Hematology, the Association of American Cancer Institutes, the American Clinical and Climatological Association, and the Friends of the National Institute of Nursing Research.

Dr. Benz received his A.B. cum laude from Princeton University, his M.A. from Yale University, and his M.D. magna cum laude from Harvard Medical School. He has been elected to fellowship in the American Association for the Advancement of Science, the American Association of Physicians, the Institute of Medicine of the Academy of Sciences, and the American Academy of Arts and Sciences. He serves on numerous advisory boards at academic institutions, National Institutes of Health, and foundations.