Gene therapy hope for toddlers with rett syndrome

NCT ID NCT07480564

Recruiting now Disease control Sponsor: Taysha Gene Therapies, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a one-time gene therapy called TSHA-102 in girls aged 2 to 4 with Rett syndrome, a severe genetic disorder. The therapy is given as a single injection into the spine. The main goal is to check if it is safe and tolerable, and to see early signs of whether it helps control the disease. Only 3 participants will be enrolled in this early-stage trial.

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Conditions

The condition(s) this trial relates to.

autism, susceptibility to, X-linked 2 Congenital, Hereditary, and Neonatal Diseases and Abnormalities intellectual disability nervous system disorder neurodevelopmental disorder Neurologic Manifestations Pathologic Processes Rett syndrome X-linked disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

RETT SYNDROME

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

Boston Children's Hospital
RECRUITING

Boston, Massachusetts, 02115, United States

Contact Email: •••••@•••••

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