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TSHA-106

Development stage: Discontinued
Lead developer: Taysha Gene Therapies
Modality: Gene Therapies
Administration: Intrathecal

Overview

TSHA-106 is an investigational gene therapy being developed as a potential treatment for Angelman syndrome, a severe neurodevelopmental disorder most often caused by loss or malfunction of the UBE3A gene in the brain. TSHA-106 is designed as a UBE3A gene therapy, aiming to restore normal function by targeting the underlying genetic cause of the disorder. It was initially developed by The University of Texas Southwestern Medical Center, with ongoing development by Taysha Gene Therapies. This program is in preclinical or early clinical development stages.

Targets

UBE3A (Ubiquitin-protein ligase E3A)

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