VY1706 for Treatment of Alzheimer Disease Shows Tau Reductions and Favorable Tolerability

Voyager Therapeutics shared 6-month toxicology data for intravenous VY1706 to treat Alzheimer disease, showing sustained tau protein reduction and favorable tolerability.¹ VY1706 is an investigational gene therapy targeting extracellular tau which showed positive tau reductions in nonhuman primate data, presented at the Alzheimer’s Association International Conference.²

In 3- and 6-month good laboratory practice toxicology studies in nonhuman primates, VY1706 demonstrated favorable tolerability overall. The studies showed no adverse clinical pathology or histopathological findings in the central nervous system, dorsal root ganglia, and peripheral organs up to the highest dose tested (5e13 vg/kg). The drug has been tested in mice and cynomolgus macaque monkeys.

A single dose of VY1706 in nonhuman primates showed durable and dose-dependent central nervous system delivery with up to 75% lowering of microtubule-associated protein tau (MAPT) mRNA and tau in Alzheimer-relevant brain regions through 6 months. Alkaline phosphatase, a vasculature endothelial receptor, was seen to mediate blood-brain barrier transport for VY1706, supporting potential for cross-species translation of the drug. VY1706 showed none of the typical adeno-associated viruses (AAV) liver transaminase elevations at any dose level throughout 6 months. Plasma neurofilament levels were generally stable with no dose-related increases, and there were no cellular immune activations. Biodistribution and pharmacology analyses corroborated this liver-sparing profile, with amiRNA expression and MAPT mRNA reduction substantially lower in liver than in brain, and an approximately 30-fold decrease in liver biodistribution relative to a comparable dose of wild-type AAV9 expressing an alternative transgene.

“The data we are presenting at AAIC continue to reinforce the compelling pharmacology and safety profile and durability we have observed to date with VY1706, which is the first tau-targeted gene therapy with an [Investigational New Drug designation] cleared by the [US Food and Drug Administration],” said Alfred W. Sandrock Jr, MD, PhD, chief executive officer of Voyager. “We continue to view tau as a potentially transformational target in Alzheimer’s disease, and we look forward to initiating dosing of adults with early Alzheimer’s disease in the second half of the year.”

VY1706 is an investigational gene therapy for Alzheimer disease targeting tau proteins. The main element of the drug is a vectorized siRNA that targets MAPT mRNA to decrease intra- and extracellular tau. This core is contained in an AAV capsid, leveraging ALPL to deliver the vectorized siRNA via an intravenous dose.

VY1706 received Investigational New Drug clearance from the FDA in June 2026, enabling a clinical trial in adults with early Alzheimer disease. The trial is expected to begin dosing in the second half of 2026.

References

1. Voyager demonstrates single IV dose of VY1706 well tolerated, reduced tau in 6-month GLP toxicology study; initiation of clinical trial in Alzheimer’s disease expected H2 2026. Press release. July 13, 2026. Accessed July 13, 2026. https://www.globenewswire.com/news-release/2026/07/13/3326162/36461/en/voyager-demonstrates-single-iv-dose-of-vy1706-well-tolerated-reduced-tau-in-6-month-glp-toxicology-study-initiation-of-clinical-trial-in-alzheimer-s-disease-expected-h2-2026.html

2. Arora V, Sivasankaran R, Ghosh J, et al. IND-enabling GLP NHP study of VY1706, a BBB-crossing AAV gene therapy targeting tau in Alzheimer’s disease. Accessed July 13, 2026. https://www.voyagertherapeutics.com/wp-content/uploads/2026/07/POSTER_Voyager_AAIC_Arora_Sivasankaran_July_2026_FINAL.pdf