The U.S. Food and Drug Administration (FDA) has approved two new gene therapies, Casgevy and Lyfgenia, marking a significant milestone in the treatment of sickle cell disease. The announcement represents a breakthrough for gene therapy, with Casgevy standing out as the first FDA-approved treatment employing CRISPR/Cas9, a cutting-edge genome editing technology.

Sickle cell disease, a group of inherited blood disorders affecting over 100,000 Americans, predominantly of African and African American descent, is characterized by a mutation in hemoglobin that distorts red blood cells into a sickle shape, leading to severe pain and organ damage. 


Casgevy, a cell-based gene therapy, targets patients ages 12 and older and treats sickle cell disease by increasing the production of fetal hemoglobin (HbF), preventing the sickling of red blood cells. Lyfgenia, the other cell-based gene therapy, uses a gene-therapy derived hemoglobin that mimics normal adult hemoglobin.

Both therapies use patients’ own blood stem cells, delivered as a one-time infusion after myeloablative conditioning. Sickle cell disease occurs when an individual inherits two sickle cell genes, one from each parent. 

Dr. Melissa Creary, assistant professor in health management policy and global public health at the University of Michigan School of Public Health, talks with host Stephen Henderson about the pros and cons of the new gene-editing therapy.

Dr. Creary, who lives with sickle cell disease herself, describes the excitement generated by this unprecedented treatment and how it addresses a population that has been historically neglected from a medical perspective. Henderson and Dr. Creary also discuss the possible barriers to the therapy such as cost, accessibility and the complexity of the treatment.

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Watch American Black Journal on Tuesday at 7:30 p.m. and Sunday at 9:30 a.m. on Detroit Public TV, WTVS-Channel 56.