First patients dosed in clinical trial of Rett syndrome gene therapy

The first two female pediatric patients with Rett syndrome have been dosed in an ongoing phase 1/2 trial of investigational AAV9 gene therapy NGN-401, according to the manufacturer.

According to a release from Neurogene Inc., NGN-401 was purposefully designed and administered to maximize therapeutic activity while averting transgene overexpression toxicities. The novel therapeutic delivers the full-length human methyl cytosine binding protein 2 (MECP2) gene, providing an optimal gene replacement approach.

In non-clinical studies with NGN-401 at clinically relevant doses, cardinal features of Rett syndrome were ameliorated and no overexpression toxicity was observed, Neurogene said.

NGN-401 had previously been granted orphan drug designation, rare pediatric disease designation and fast track designation by the FDA.

“NGN-401 has been well tolerated to date in the first two patients dosed, consistent with the wide safety margins established in non-clinical studies conducted in disease models,”

Bernhard Suter, MD, principal study investigator and associate professor of pediatrics and neurology at Baylor College of Medicine and Texas Children’s Hospital, stated in the release.