FDA approves gene-editing treatment developed in Nashville for pediatric sickle cell patients


SOURCE: WBIR.COM
JUL 15, 2026

13-year-old Rickey Buggs became the first commercial patient to receive a gene-editing treatment for sickle cell disease at TriStar Centennial Children's Hospital.

Credit: TriStar Centennial Children's Hospital

Author: Sam Perez

Published: 4:17 PM EDT July 15, 2026

Updated: 4:17 PM EDT July 15, 2026

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NASHVILLE, Tenn. — A 13-year-old Nashville boy has become the first commercial patient at TriStar Centennial Children's Hospital to receive a newly approved gene-editing treatment for sickle cell disease, following recent approval from the U.S. Food and Drug Administration.

The treatment is considered a potential cure for the blood disorder, which affects about 100,000 Americans, including approximately 3,500 people in Tennessee.

For 13-year-old Rickey Buggs, living with sickle cell disease meant years of pain and limitations.

"It's like this constant heavy burden that's on you all of the time," Buggs said. "You're always getting held back from something that you want to do. You're always getting held back from constant pain. You have to take constant breaks over and over and over again, and you never get to have that freedom to do anything that you want to do, because you're constantly in pain..."

His mother, Lametra Scott, said learning of Rickey's diagnosis when he was 8-weeks-old was devastating.

"Getting that information was almost as if somebody’s knocking the wind out of you," Scott said.

Since then, she has watched her son struggle with the disease.

"As a mom, you will go to the ends of the earth to do whatever is necessary to ensure that your child is okay as best as you can," Scott said.

Scott said she quickly began trying to better understand the disease. Scott says she understood that she was a sickle cell trait carrier, as was Rickey's father, but she didn't know much about the disease and wasn't aware her son was born with it until she received a call from the health department following her son's birth.

"If I didn't know that I was a sickle cell trait carrier, and I would say I was a more mature age when I had Rickey, then that meant there was a lot of people out in the community who may not know about sickle cell disease," Scott said. "And also, within the healthcare community itself, not all healthcare providers are well-versed in sickle cell disease. So that let me know that I needed to do a little bit of self-education."

As a pharmacist, she said research and education came naturally to her. Scott used her family's experience as a way to educate and empower others, while raising awareness and fundraising for sickle cell research and treatment. She started Breaking the Sickle Cell Cycle Foundation to further that work.

Scott's journey led her to meet Dr. Haydar Frangoul, director of the pediatric transplant program at TriStar Centennial Children's Hospital in Nashville. Dr. Frangoul said he's worked with children who have sickle cell disease for nearly 30 years.

"Sickle cell disease is a devastating disease," Dr. Frangoul said. "It affects all the organs in the body, so individuals with sickle cell disease suffer from recurrent pain crises. They can have strokes. They can have damage to their lungs, kidneys and bones, and we know that individuals with sickle cell disease have shortened life expectancy."

In 2019, Frangoul helped develop a clinical trial for gene-editing therapy to treat sickle cell disease. At the end of June, the FDA approved the treatment for patients older than two years old.

Buggs is now the first commercial patient to receive the treatment at TriStar Centennial Children's Hospital.

"I wanted to give it a try, but I was kind of skeptical about it at first because I didn't know how it was going to be or how this treatment would go, but I still took that leap of faith and went to and tried it and we got a very successful treatment," Buggs said. "It's basically like a golden opportunity. That's what it's like, and we have never had this opportunity before, and the fact that I've got this opportunity granted to me was incredible, that I even had the chance to try this."

After spending 42 days in the hospital undergoing blood transfusions and testing, Buggs is now back home and on the road to recovery.

"The future will be great," Buggs said. "That's what I think it is because I finally have this limitation off of me and I'm finally not dragging this heavy burden around with me and I'm finally not having to worry about ‘Am I going to be in pain?"

There is another treatment for sickle cell disease, although it might not be a good option for many patients, Dr. Frangoul says.

"The best results are if you have a match in the family, meaning a brother or a sister that's a match," Dr. Frangoul said. "Unfortunately, only around 18% of people with sickle cell disease have a match within their family, so that means 80% of individuals with sickle cell disease don't have a match in the family."

But with this new gene-editing therapy, Dr. Frangoul says the patients' own cells are edited and then given back to them. He said this eliminates the need to find a match, and there's no chance of rejection as there is with a bone marrow transplant.

"I think this is very exciting to the field," Dr. Frangoul said. "There is no chance that those cells will attack their body, so they don't need to take immune suppression like a regular bone marrow transplant. So it's actually ... I think it's a game changer in my mind for the treatment of sickle cell disease."

Dr. Frangoul said he feels confident the treatment is curative; however, he said because only seven years have passed since the first clinical trial patient received the therapy, longer follow-up is needed to officially confirm that conclusion.

While Buggs has received the treatment and now only needs to go back for follow-ups, his mother continues to share the message about sickle cell disease, the people it impacts, and the need for people to donate blood.

"It's so important for everybody to understand the importance of why we need blood, but especially why we need blood in the sickle cell community," Scott shared. "When we talk about the demographic of people who is impacted by sickle cell disease, it is mostly minority populations. That also lends to why we need people who are of minority descent to donate blood. It just goes hand in hand."

To learn more about sickle cell disease, you can visit BTSSCycle.org.