Drug intelligence / Profile preview

AXO-AAV-ALS

Development stage: Discontinued
Lead developer: Benitec Biopharma
Modality: Gene Therapies
Administration: Intravenous, Intrathecal, Intranasal, Intraparenchymal, Intracerebroventricular, Intrapleural, Intralingual

Overview

AXO-AAV-ALS is a gene therapy candidate that was being developed for neurological disorders, specifically amyotrophic lateral sclerosis (ALS) and frontotemporal dementia. It originated from a research collaboration between Benitec Biopharma and Axovant Sciences, aiming to deliver genetic material to address these conditions.

Targets

SOD (Manganese Superoxide Dismutase)

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