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Note: This story was updated Dec. 5, 2025, to correct the primary outcome in the Embolden study. NGN-401, an experimental gene therapy for Rett syndrome, led to functional improvements in all the girls who received the treatment in an early clinical trial, according to new data announced by its…

Fibroblasts from a Rett syndrome patient with a severe MECP2 mutation (T158M) showed markedly low levels of coenzyme Q10 (CoQ10), along with signs of mitochondrial redox imbalance and dysfunction, a lab study shows. CoQ10 helps mitochondria — the cell’s energy-producing “powerhouses” — function properly. Supplementing the fibroblasts with ubiquinol, a…

The Rett Syndrome Research Trust (RSRT) is teaming up with Profluent Bio to use artificial intelligence (AI) to design personalized gene-editing therapies for Rett syndrome. Profluent will leverage its AI tools to engineer molecules that can correct mutations in the MECP2 gene, the most frequent cause of Rett syndrome.

The Embolden clinical trial, a registrational study testing Neurogene‘s gene therapy NGN-401 in people with Rett syndrome, has begun dosing participants. “Dosing the first participant in the Embolden registrational trial of NGN-401 marks a major milestone in advancing a gene therapy for Rett syndrome,” Rachel McMinn, PhD, Neurogene’s…

Ferroptosis, a form of cell death marked by the accumulation of iron, may contribute to cellular dysfunction in Rett syndrome, according to a study. Cells from Rett patients exhibited hallmarks of ferroptosis, including oxidative damage to lipids, or fats, and abnormal mitochondria, the cell’s powerhouses. Incubation with a…

Researchers in Italy described a rare case of 27-year-old identical twin sisters with Rett syndrome who, despite sharing the same disease-causing mutation, developed symptoms of different severity. Identical (monozygotic) twins develop from a single fertilized egg that divides into two embryos, so they are genetically identical. “The clinical differences…

After expatriation of a partnership agreement with Astellas, Taysha Gene Therapies regained full rights to TSHA-102, its experimental gene therapy being developed to treat Rett syndrome. “We are thrilled to regain full global rights to our lead TSHA-102 Rett syndrome program, which enables full strategic flexibility and optionality…

Neurogene expects dosing for a registrational clinical trial of its investigational gene therapy NGN-401 for people with Rett syndrome will start by the end 2025. The U.S. Food and Drug Administration (FDA) agreed to key elements of the trial design over the summer, and Neurogene reports it…

A gene therapy showing potential to restore lost developmental milestones in people living with Rett syndrome has earned breakthrough therapy status from the U.S. Food and Drug Administration (FDA). The experimental therapy, TSHA-102, is being developed by Taysha Gene Therapies. A breakthrough therapy designation…

October is Rett Syndrome Awareness Month, and supporters are going purple to bring attention to the rare genetic condition. “There’s no one right way to raise awareness,” the International Rett Syndrome Foundation (IRSF) says on its website. “Whether you’re posting on social media, sharing your story, or just starting…