A new investigational gene therapy, NGN-401, for Rett syndrome

Texas Children’s Hospital, an internationally recognized, top-ranked children’s hospital and pediatric research center affiliated with Baylor College of Medicine, is the first to deliver a novel gene therapy to treat Rett syndrome in pediatric patients. Two female patients with Rett syndrome were the first children worldwide to receive this promising treatment.

This exciting milestone is part of an ongoing first-in-human Phase I/II trial of a new investigational gene therapy, NGN-401, conducted by Neurogene Inc., a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases. Texas Children’s is currently the first clinical trial site to recruit and dose patients in the U.S. for this multicenter study.

Rett syndrome is a rare neurodevelopmental disorder that primarily affects girls, most of whom develop normally until 6-18 months of age when they begin to experience progressive regression in acquired motor and verbal skills and develop constant hand-wringing behavior. Eventually, this condition causes severe impairments that affect nearly every aspect of their daily lives, including their ability to speak, walk, eat, and breathe.

In 1999, a team led by Dr. Huda Zoghbi, a distinguished service professor at Baylor College of Medicine, founding director of the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital and Howard Hughes Medical Institute investigator, made the transformational discovery that mutations in methyl cytosine binding protein 2 (MECP2) gene causes Rett syndrome.

It is, therefore, particularly momentous that 25 years following that discovery, the first gene therapy trial for Rett syndrome is now underway in the same site under the guidance of Dr. Bernhard Suter, associate professor of Pediatrics and Neurology at Baylor College of Medicine and medical director of the Blue Bird Circle Rett Center at Texas Children’s.

https://www.texaschildrens.org/about-us/news/releases/rett-patients-receive-new-gene-therapy-treatment-texas-children%E2%80%99s-hospital-and-baylor-college-0