Texas Children’s Hospital Delivers Novel Gene Therapy for Rett Syndrome in Pediatric Patients

Texas Children’s Hospital Makes History with First Gene Therapy Treatment for Rett Syndrome

Texas Children’s Hospital, in collaboration with Baylor College of Medicine, has achieved a groundbreaking milestone by delivering a novel gene therapy to treat Rett syndrome in pediatric patients. This innovative treatment, known as NGN-401, was administered to two female patients with Rett syndrome, making them the first children worldwide to receive this promising therapy.

Rett syndrome is a rare neurodevelopmental disorder that primarily affects girls, causing progressive regression in motor and verbal skills, as well as other severe impairments. The discovery of the underlying genetic mutation responsible for Rett syndrome, MECP2, was made by Dr. Huda Zoghbi and her team at Baylor College of Medicine 25 years ago.

The gene therapy trial, led by Dr. Bernhard Suter at Texas Children’s Hospital, aims to provide the right amount of MECP2 protein to improve clinical outcomes without causing toxicity. NGN-401 is designed to maximize therapeutic activity while avoiding toxicities associated with transgene overexpression, and it is administered as a one-time treatment via the intracerebroventricular route.

Initial results from the first two patients who received NGN-401 show no serious side effects, offering hope for a more effective treatment for Rett syndrome. Dr. Zoghbi expressed excitement about the potential of this gene therapy to revolutionize the therapeutic landscape for Rett syndrome patients.

The doctors at the Blue Bird Rett Center at Texas Children’s Hospital are optimistic about the future of this treatment and express gratitude to the patients and families who have inspired their research efforts. This groundbreaking gene therapy trial represents a significant step forward in addressing the unmet need for better treatments for Rett syndrome.

Source: Texas Children’s Hospital