Drug Profile

CLN7 disease gene therapy - Taysha Gene Therapies/University of Texas Southwestern Medical Center

Latest Information Update: 28 Nov 2025

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At a glance

Originator University of Texas Southwestern Medical Center
Developer Taysha Gene Therapies; University of Texas Southwestern Medical Center
Class Gene therapies
Mechanism of Action Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

28 Nov 2025 No recent reports of development identified for preclinical development in Neuronal ceroid lipofuscinosis in USA (Intrathecal)
05 Oct 2021 University of Texas Southwestern Medical Center and Taysha Gene Therapies agree to co-develop next gereration CLN7 disease gene therapy for Neuronal ceroid lipofuscinosis
05 Oct 2021 Preclinical trials in Neuronal ceroid lipofuscinosis in USA (Intrathecal)

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