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Breakthrough Gene Therapy Promises Possible Cure for Sickle Cell Disease

ATLANTA – Sickle Cell Foundation of Georgia Executive Director Tabatha McGee on Wednesday appeared on Scripps Live, a national award-winning news program, concerning a recent FDA advisory committee decision giving preliminary approval to gene therapy to treat and possibly cure sickle cell disease. 

The treatment, called exa-cel, received a positive critique of its safety from an important FDA advisory panel during a 7-hour meeting on Nov. 1st. Exa-cel is a gene-editing treatment that changes the body’s process for making red blood cells.  

“This is a monumental step forward in the treatment of sickle cell disease, as the only cure to date is a bone marrow transplant, which is a cost-prohibitive and selective option,” said McGee. “But we must proceed with caution. There are many unanswered questions on the after-effects on the patient. Also, what also troubles me is the cost for this procedure is as much as $1.8 million.” 

Unlike with a bone marrow transplant, no donor is required for the one-time gene therapy, which Vertex Pharmaceuticals and CRISPR Therapeutics make. This new treatment involves permanently changing DNA in a patient’s blood cells. This would be the first gene therapy on the U.S. market based on CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020. 

The FDA is set to make a crucial decision on a new sickle cell gene therapy in early December.  

Sickle cell disease is a debilitating disorder that affects millions of people globally, causing excruciating pain, stroke, organ damage, and other complications due to the malformed cells.  

The new gene therapy aims to add functional copies of a modified gene that helps red blood cells produce "anti-sickling" hemoglobin, which can prevent or reverse the misshapen cells. This groundbreaking treatment promises to revolutionize sickle cell treatments and bring relief to millions of patients worldwide, according to its makers, Vertex Pharmaceuticals and CRISPR Therapeutics. 

In late December, the FDA is also expected to decide on another sickle cell gene therapy later this month, which is set to work differently from the first.  

“We eagerly await the FDA's decision and hope that these gene therapies will be the true breakthrough that the sickle cell community has been waiting for,” McGee said.