Aldevron Breakthrough Blog

Contributing to a Cure for Sickle Cell Disease

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!

 

 

About the author

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Thomas Lynch

Thomas Lynch

Dr. Lynch is the Vice President of Client Services at Aldevron, where he leads the commercial activities for Aldevron’s Protein Business Unit. Dr. Lynch has over 25 years of combined sales and laboratory experience and holds a Ph.D. in biochemistry from the University of Illinois and a B.S. from the University of Minnesota. His postdoctoral research at the University of Chicago centered on the structural determination and protein analysis: nucleic acid complexes. Before rejoining Aldevron in 2017, Dr. Lynch served as Vice President of Sales for DNASTAR and held positions of increasing responsibility within Business Development & Client Relations at Aldevron.

Topics: Gene Editing, Protein, Video