Contributing to a Cure for Sickle Cell Disease

April 6, 2021 / by Thomas Lynch, Ph.D.

Every year, an estimated 300,000 children are born globally with a severe form of sickle cell disease. This is a genetic disease that causes red blood cells to be a sickle shape, leading to episodes of pain and anaemia, along with the potential of stroke or kidney damage.

In the United States, the median lifespan of a sickle-cell patient is in the mid-40s. However, in Africa, where a majority of sickle-cell cases are found, the median lifespan is only five years.

Last month, we were excited to host a virtual celebration of our lab facility expansion in Madison, Wisconsin. Leading the ribbon-cutting ceremony was Matthew Porteus, MD, PhD, a Stanford professor and academic founder of Graphite Bio, who presented his impressive work on a cure for sickle-cell disease.

Dr. Porteus is using gene editing with CRISPR-Cas9 to correct a single mutation in the stem cells of sickle-cell patients. Having successfully tested his work on purified cells in culture, and in a study on genetically engineered mice, he’s now planning to start recruiting his first human patients into a clinical trial this spring.

The trial will use Aldevron’s GMP-grade high-fidelity Cas9 protein.

You can watch Dr. Porteus' 10-minute presentation below!