uniQure doses first patient in Phase I/II trial of AMT-162 gene therapy for SOD1-ALS

uniQure N.V.

First patient dosed in EPISOD1 Phase I/II open-label trial of AMT-162 for SOD1-ALS, a rare inherited progressive motor neuron disease.
Trial has three dose-escalating cohorts; up to four patients per cohort receiving intrathecal AMT-162 with immunosuppression.
AMT-162 is an AAVrh10-based gene therapy expressing miRNA to knock down mutated SOD1 protein; granted Orphan Drug and Fast Track FDA designations.
Four active U.S. sites; plans to activate seven additional sites by Q1 2025.
Trial will assess safety, tolerability, and exploratory efficacy via neurofilament light chain and SOD1 protein biomarkers.

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