Recent preclinical data has shown that a single intravenous dose of VY1706 achieved up to 75 percent reduction in MAPT mRNA and tau protein across Alzheimer’s-relevant brain regions over six months, with no adverse central nervous system or peripheral organ findings.
Voyager Therapeutics has announced new six-month preclinical data showing its investigational Alzheimer’s gene therapy achieved sustained reductions in tau protein while maintaining a favourable safety profile, as the company prepares to begin human dosing later this year.
The biotechnology company presented the latest good laboratory practice (GLP) toxicology findings for VY1706 during a late-breaking Developing Topics poster session at the Alzheimer’s Association International Conference (AAIC), held in London from 12-15 July 2026.
The data showed that a single intravenous dose of the VY1706 gene was well tolerated in non-human primates over six months and reduced tau protein by up to 75 percent in key brain regions associated with Alzheimer’s disease.
Clinical trial set to begin
The latest findings follow the US Food and Drug Administration’s Investigational New Drug (IND) clearance for VY1706 in June, allowing Voyager to launch its first clinical trial in adults with early Alzheimer’s disease. Patient dosing is expected to begin during the second half of 2026.
The company had previously presented three-month GLP toxicology data for the therapy at the American Society of Gene & Cell Therapy’s 2026 Annual Meeting in May.
“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 IND cleared by the FDA,” said Dr Alfred Sandrock Jr, 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.”
Positive safety and efficacy findings
According to Voyager, the six-month GLP toxicology study demonstrated a favourable tolerability profile, with no adverse clinical pathology or histopathological findings identified in the central nervous system, dorsal root ganglia or peripheral organs, including the liver, at doses up to 5E13 vg/kg.
Researchers also reported that a single intravenous dose achieved broad, durable and dose-dependent delivery throughout the central nervous system. This resulted in reductions of up to 75 percent in both MAPT mRNA and tau protein across brain regions considered relevant to Alzheimer’s disease over the six-month study period.
The company said VY1706 uses ALPL, a receptor found in brain blood vessel endothelial cells, to transport the therapy across the blood-brain barrier, with the hope that this mechanism could support translation across different species.
Targeting tau in Alzheimer’s disease
The therapy combines a vectorised small interfering RNA (siRNA), which reduces production of tau by targeting MAPT messenger RNA, with Voyager’s proprietary TRACER AAV capsid technology. The treatment is intended to be delivered as a one-off intravenous infusion.
Voyager says its wider preclinical programme has demonstrated both sustained tau reduction and reduced liver targeting, an important consideration as liver-related adverse events have been observed with some systemically administered gene therapies.
The company is now preparing to evaluate VY1706 in adults with early Alzheimer’s disease as it advances the programme from preclinical development into human clinical trials.
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