FDA clears IND application for gene therapy to treat children with Rett syndrome

Neurogene Inc. announced the FDA has granted clearance to its investigational new drug application for NGN-401, an adeno-associated virus gene therapy candidate for treating Rett syndrome in female pediatric patients.

According to a company press release, the FDA clearance allows the initiation of a phase 1/2 study in 2023 to assess safety, efficacy and tolerability of the novel MECP2 gene product. The open-label, single-arm, multicenter clinical trial will evaluate a single dose of NGN-401 delivered via a one-time intracerebroventricular procedure, which achieved safety and efficacy outcomes in multiple preclinical trials, the company stated.

“FDA clearance of NGN-401 represents a significant milestone for Neurogene and the Rett syndrome community and underscores our commitment to turn devastating neurological diseases into treatable conditions and improve the lives of patients and families impacted by these rare diseases,” Rachel McMinn, PhD, Neurogene CEO and founder, said in the release.

The company stated that NGN-401 is the first investigational adeno-associated virus gene therapy candidate for children using its proprietary Expression Attenuation via Construct Tuning (EXACT) gene regulation technology, which can deliver a narrow range of transgene expression, compatible with both viral and non-viral delivery platforms. This is critical for the treatment of Rett syndrome, which requires the safe regulation of MECP2 transgene expression while avoiding toxicities associated with overexpression, the release stated.

“Rett syndrome is a debilitating disease with a devastating impact on children and their families, with no disease-modifying treatments available,” Bernhard Suter, MD, assistant professor of pediatrics and neurology at Baylor College of Medicine and neurologist at Texas Children’s Hospital. “The upcoming clinical study of NGN-401, which has a mechanism of action aimed at addressing the root cause of disease, offers hope for improving the lives of those suffering from Rett syndrome.”