Drug intelligence / Profile preview

TSHA-110

Development stage
Preclinical
Lead developer
Taysha Gene Therapies
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intrathecal
01

Overview

TSHA-110 is an AAV-based gene therapy being developed by Taysha Gene Therapies for the treatment of KCNQ2 Developmental and Epileptic Encephalopathy (KCNQ2-related disorders). Taysha obtained an exclusive option from the University of Texas Southwestern Medical Center (UTSW) to develop this therapy. KCNQ2 Developmental and Epileptic Encephalopathy is a monogenic central nervous system disorder characterized by seizures typically presenting in the first week of life, often associated with developmental impairment. As a gene therapy, TSHA-110 aims to deliver a functional KCNQ2 gene to address the underlying genetic cause of the disorder.

02

Targets

KCNQ2 (Potassium voltage-gated channel subfamily KQT member 2)

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