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AGIL-501

Development stage: Preclinical
Lead developer: PTC Therapeutics
Modality: Gene Therapies

Overview

AGIL-501 is an AAV-based gene therapy developed by Agilis Biotherapeutics for the treatment of GM1 gangliosidosis, a rare central nervous system (CNS) disease. The therapy aims to deliver a functional gene to address the underlying genetic defect responsible for the condition. Agilis Biotherapeutics, along with its gene therapy programs including AGIL-501, was acquired by PTC Therapeutics in August 2021.

Targets

GLB1 (Galactosidase Beta 1)

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