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BGT-INAD

Development stage: Preclinical
Lead developer: Bloomsbury Genetic Therapies
Modality: Gene Therapies
Administration: Intrathecal, Intracerebroventricular, Intracisternal, Intravenous

Overview

BGT-INAD is an adeno-associated virus serotype 9 (AAV9) gene therapy candidate being developed by Bloomsbury Genetic Therapies for the treatment of Infantile Neuroaxonal Dystrophy (INAD). INAD is a rare, severe neurodegenerative disorder caused by biallelic mutations in the PLA2G6 gene, which leads to a deficiency in the calcium-independent phospholipase A2 group VI enzyme. BGT-INAD is designed to deliver a functional copy of the human PLA2G6 gene to the central nervous system, aiming to restore enzyme activity and address the underlying genetic cause of the disease. The program is currently in the preclinical stage of development.

Targets

PLA2G6 (iPLA2β / PLA2G6)Gal (Terminal N-linked galactose)

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