Voyager Therapeutics, Inc. (VYGR) reported new late-breaking data showing that a single intravenous dose of its investigational gene therapy VY1706 was well tolerated and produced meaningful reductions in tau protein in a three-month GLP toxicology study in non-human primates.
The findings, presented at the 2026 ASGCT Annual Meeting, reinforce the therapy's potential as a first-in-class approach to lowering tau, a key driver of Alzheimer's disease pathology.
According to the company, VY1706 showed no adverse clinical pathology or histopathology findings at doses up to 5E13 vg/kg and achieved reductions of up to 64% in tau protein across key brain regions thirteen weeks after a single IV administration. The therapy also delivered broad CNS distribution of a vectorized siRNA targeting MAPT, resulting in dose-dependent reductions of 51-75% in MAPT mRNA and 48-64% in tau protein.
Voyager said its IND application remains on track for Q2 2026, with the first-in-human Alzheimer's disease trial expected to begin in the second half of 2026, pending FDA clearance.
Chief Scientific Officer Todd Carter noted that the new data are consistent with the company's broader preclinical package and support advancing VY1706 into clinical development.
The company also highlighted progress across its TRACER capsid discovery platform, presenting eight additional ASGCT abstracts that showcased advances in muscle-targeted, neuromuscular, immune-evasive, and AI-designed AAV capsids. These next-generation capsids demonstrated significantly higher expression in muscle and CNS tissues compared with AAV9, while also showing reduced liver exposure and improved antibody evasion. Voyager said these innovations are intended to expand the reach of gene therapy to more patients and more neurological and neuromuscular conditions.
Voyager's broader pipeline includes programs in Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, amyotrophic lateral sclerosis (ALS), and other central nervous system disorders, many of which leverage TRACER-derived capsids designed for high brain penetration following IV dosing. Several programs are advancing through partnerships with Alexion, AstraZeneca Rare Disease, Novartis, and Neurocrine Biosciences.
VYGR has traded between $2.65 and $5.55 over the past year. The stock closed Wednesday's trading at $4.15, up 0.73%. In pre-market trading the stock is at $4.40, up 6.02%.
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