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Wednesday, September 10, 2025

St. Louis Children’s Hospital pioneers new gene therapy for sickle cell disease

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Tim Alexander, Senior Director, Business Research & Analysis | LinkedIn

Tim Alexander, Senior Director, Business Research & Analysis | LinkedIn

A new gene therapy for sickle cell disease is being introduced at St. Louis Children’s Hospital, making it one of the first in the nation to offer this potentially curative treatment. This initiative provides an alternative to existing treatments that only manage symptoms.

Approved by the Food and Drug Administration (FDA) in 2023, the therapy aims to significantly improve the lives of patients by going beyond mere survival to enhancing their quality of life.

St. Louis Children’s Hospital is at the forefront of implementing this innovative medical therapy, setting a precedent in the treatment of sickle cell disease. The condition impacts approximately 100,000 individuals in the United States.

The development marks a significant step forward in the medical field, with potential implications for global treatment standards for sickle cell disease.

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