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TSHA-114

Development stage: Preclinical
Lead developer: Taysha Gene Therapies
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration: Intrathecal

Overview

TSHA-114 is an investigational adeno-associated virus (AAV) vector-based gene therapy being developed as a one-time gene replacement therapy for the treatment of Fragile X syndrome, the most common single-gene cause of autism spectrum disorder and intellectual disability. The therapy is designed to deliver a functional copy of the gene encoding fragile X mental retardation protein (FMRP) in patients with the disorder, aiming to restore normal function by directly addressing the underlying genetic cause of Fragile X syndrome.

Targets

FMR1 (Fragile X messenger ribonucleoprotein 1)

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