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Voyager ASGCT Late Breaker: Single IV Dose of VY1706 Well Tolerated, Reduced Tau in 3-Month GLP Toxicology Data; Clinical Trial in Alzheimer’s Disease Expected H2 2…

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Voyager ASGCT Late Breaker: Single IV Dose of VY1706 Well Tolerated, Reduced Tau in 3-Month GLP Toxicology Data; Clinical Trial in Alzheimer’s Disease Expected H2 2…

Voyager Therapeutics presented GLP toxicology data showing its investigational tau-silencing gene therapy VY1706 was well tolerated and reduced tau protein by up to 64% in non-human primates after a single IV dose, with plans to file an FDA IND application in Q2 2026 and begin clinical trials in Alzheimer’s patients by H2 2026. The company also showcased advancements in its TRACER platform, including muscle-targeted AAV9 capsids with significantly improved transduction efficiency in both mice and non-human primates.

Voyager Therapeutics announced late-breaking data from its ASGCT 2026 Annual Meeting, highlighting progress in its tau-silencing gene therapy VY1706 for Alzheimer’s disease. In a 3-month GLP toxicology study, VY1706 demonstrated a favorable safety profile with no adverse findings up to the highest dose tested (5E13 vg/kg). The therapy reduced tau protein levels by 48-64% in key brain regions of non-human primates 13 weeks after a single intravenous dose, using ALPL as its blood-brain barrier receptor. The company’s IND application process for VY1706 is on track for submission to the FDA in Q2 2026, with clinical trials in Alzheimer’s patients expected to begin in the second half of 2026. Todd Carter, Voyager’s Chief Scientific Officer, emphasized the potential of VY1706 as the first gene therapy to target tau, a protein central to Alzheimer’s pathology. Voyager also presented advancements in its TRACER platform, including engineered AAV9-based capsids with significantly improved muscle tropism. In mice, the top muscle-targeted capsid achieved 100X higher expression in skeletal muscle and 10X in heart muscle compared to standard AAV9. In non-human primates, the capsid showed 25-30X higher expression in skeletal muscle and 13X in heart muscle, while maintaining low liver exposure. Additionally, Voyager demonstrated a bi-functional AAV gene therapy designed to reduce endogenous ApoE4 and express ApoE2 in ApoE4 humanized mice, achieving physiological levels of ApoE2 after a single dose. The company also developed a next-generation ‘stealth’ capsid capable of evading pre-existing neutralizing antibodies, improving immunogenicity profiles for future therapies. The data underscores Voyager’s progress in gene therapy innovation, particularly for neurological diseases, with multiple programs advancing toward clinical evaluation.

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