One-Shot gene therapy trial offers hope for devastating neurological disorder
Disease control
Recruiting now
This study is testing a single injection of an experimental gene therapy called TSHA-102 for females with Rett syndrome. The goal is to see if the treatment is safe and can help improve developmental skills by targeting the genetic root cause of the disease. The study will follow…
Phase: PHASE3 • Sponsor: Taysha Gene Therapies, Inc. • Aim: Disease control
Last updated Mar 27, 2026 12:40 UTC