Markus Mapara News Today

Casgevy and Lyfgenia were both approved by the FDA to treat sickle cell disease. Both treatments also cost millions of dollars.

The U.S. Food and Drug Administration is expected to approve exa-cel for sickle cell disease in what would become the first marketed medicine to use CRISPR.

The U.S. Food and Drug Administration is expected to approve exa-cel for sickle cell disease in what would become the first marketed medicine to use CRISPR.

BioPharma

Two new therapies—one which uses the gene-editing technology—treat sickle cell anemia.

The U.S. Food and Drug Administration is expected to approve exa-cel for sickle cell disease in what would become the first marketed medicine to use CRISPR.

The U.S. Food and Drug Administration is expected to approve exa-cel for sickle cell disease in what would become the first marketed medicine to use CRISPR.

For people with sickle cell disease, their red blood cells usually look like a C-shaped farm tool called a sickle.

 E-Mail University of Illinois Chicago is one of the U.S. sites participating in clinical trials to cure severe red blood congenital diseases such as sickle cell anemia or Thalassemia by safely modifying the DNA of patients blood cells. The first cases treated with this approach were recently published in an article co-authored by Dr. Damiano Rondelli, the Michael Reese Professor of Hematology at the UIC College of Medicine. The article reports two patients have been cured of beta thalassemia...