uniQure N.V. announced on October 15, 2024, the dosing of the first patient in its Phase I/II clinical trial of AMT-162 for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1). This marks the advancement of uniQure's third gene therapy program into clinical development, targeting a debilitating, degenerative, and fatal disease.
The EPISOD1 trial is a multi-center, open-label study in the United States, featuring three dose-escalating cohorts to assess the safety, tolerability, and exploratory signs of efficacy of AMT-162. AMT-162 is an investigational AAVrh10-based gene therapy designed to knock down the expression of the mutated SOD1 protein, which is toxic to motor neurons.
AMT-162 has previously received both Orphan Drug status and Fast Track designation from the U.S. Food and Drug Administration. The trial aims to generate proof-of-concept data using established biomarkers like neurofilament light chain and SOD1 protein, offering a potential one-time, intrathecally administered approach to slowing or halting SOD1-ALS progression.
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