Taysha Gene Therapies, Inc. ((TSHA)) announced an update on their ongoing clinical study.
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The REVEAL Adult Study, officially titled ‘An Open-label, Randomized, Dose-escalation- and Dose-expansion Study of the Safety and Preliminary Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, in the Treatment of Adult Females With Rett Syndrome,’ aims to assess the safety and preliminary efficacy of TSHA-102 in adult females with Rett syndrome. This study is significant as it explores a potential gene therapy treatment for a condition with limited therapeutic options.
The intervention being tested is TSHA-102, a genetic therapy utilizing a recombinant, non-replicating, self-complementary AAV9 vector encoding the miniMECP2 gene. It is administered once intrathecally and is designed to address the genetic root of Rett syndrome.
This Phase 1/2 interventional study is open-label and randomized, with a sequential intervention model. It has no masking and its primary purpose is treatment. The study evaluates two dose levels of TSHA-102 in different cohorts.
The study began on October 28, 2022, and is currently recruiting. The primary completion and estimated study completion dates are not specified, but the last update was submitted on June 5, 2025. These dates are crucial for tracking the study’s progress and potential market entry.
The market implications of this study are significant for Taysha Gene Therapies, Inc., as successful results could enhance their stock performance and attract investor interest. The gene therapy market is competitive, with several companies exploring similar avenues, making this study’s outcomes particularly impactful.
The study is ongoing, with further details available on the ClinicalTrials portal.