Reopening the Developmental Window in Rett Syndrome with a Gene Therapy

Rett syndrome is a rare neurodevelopmental disorder that disrupts a child’s ability to purposely use their hands, communicate, and move around. It creates a lifelong caregiving burden for families, and there are still no treatments that truly change the course of the disease. Neurogene is developing a one-time gene therapy that has shown promising early results, with children gaining new skills and continuing to make developmental progress over time. We spoke with Rachel McMinn, CEO of Neurogene, about Rett syndrome, the company’s technology for controlling gene expression, and the encouraging data they’ve seen so far.

You can listen to Reopening the Developmental Window in Rett Syndrome with a Gene Therapy online on Radio and Podcast. Open the player on this page to stream the available audio.

Reopening the Developmental Window in Rett Syndrome with a Gene Therapy is an episode from RARECast by RARECast.

This episode is 00:40:52 long.

This episode was published on Mar 4, 2026.

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