Voyager Therapeutics announced FDA clearance of its Investigational New Drug application for VY1706 on 1 June 2026, enabling a multi-site, open-label, dose-escalation clinical trial of a one-time IV gene therapy in adults with early alzheimer’s disease; dosing is expected to begin in the second half of 2026, according to the company.
The trial will enroll up to 18 participants across three cohorts, with a highest dose not to exceed 5E13 vg/kg, and will evaluate safety and tolerability as the primary endpoint, with secondary assessments of CSF tau biomarkers and tau PET imaging.
VY1706 comprises a vectorized siRNA targeting MAPT mRNA encapsulated in a Voyager TRACER AAV capsid that leverages the ALPL receptor to deliver payload to the brain following a single IV dose.
The company reports comprehensive preclinical data, including GLP toxicology in non-human primates showing no adverse clinical pathology or histopathological findings up to 5E13 vg/kg at 13 weeks, and dose-dependent reductions in MAPT mRNA and tau protein of up to 75% in key brain regions.
Voyager positions this IND as the first for a tau-targeted gene therapy and highlights liver de-targeting as a potential safety advantage.
