Taysha and Neurogene close in on first Rett syndrome gene therapy as 2027 readouts loom

Two companies, Taysha Gene Therapies and Neurogene, are nearing the final stage of development for gene therapies targeting Rett syndrome, a rare neurodevelopmental disorder with limited treatment options.

Both firms expect to present pivotal readout data in 2027, and the Rett community is hopeful that the results could be as transformative as Novartis’s Zolgensma for spinal muscular atrophy.

Monica Coenraads, CEO of the Rett Syndrome Research Trust, expressed optimism, likening potential outcomes to children who would have died by age two now taking ballet lessons, underscoring the high expectations.

While experts caution against calling it a cure, the strong preclinical data and upcoming FDA submissions suggest the therapies are on the brink of a major milestone.