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An Open-label Phase 1/2/3 Study Consisting of a Phase 1/2 Safety and Dose-escalation and Phase 3 Dose-expansion Study to Evaluate Safety and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy in Females With Rett Syndrome

Trial Profile

An Open-label Phase 1/2/3 Study Consisting of a Phase 1/2 Safety and Dose-escalation and Phase 3 Dose-expansion Study to Evaluate Safety and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy in Females With Rett Syndrome

Status: Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 10 Jul 2026

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At a glance

  • Drugs TSHA 102 (Primary)
  • Indications Rett syndrome
  • Focus Adverse reactions; First in man; Registrational; Therapeutic Use
  • Acronyms REVEAL; REVEAL Pivotal Study
  • Sponsors Taysha Gene Therapies

Most Recent Events

  • 26 Jun 2026 According to a Taysha Gene Therapies media release, data form this results will be presented at the 2026 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting, taking place in Prior Lake, MN, from June 29 - July 1, 2026.
  • 22 Jun 2026 According to a Taysha Gene Therapies media release, Taysha management will host a live conference call and webcast today at 8:30 a.m. ET to discuss the longer-term data from the REVEAL Phase 1/2 trials
  • 22 Jun 2026 Results published in the Taysha Gene Therapies Media Release

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