Voyager Therapeutics Inc

Voyager Therapeutics presented six-month GLP toxicology data for its investigational Alzheimer's gene therapy VY1706 at the AAIC conference, showing the treatment was well tolerated and reduced tau protein by up to 75% in key brain regions of non-human primates following a single intravenous dose. The company received FDA IND clearance in June, enabling initiation of a clinical trial in adults with early Alzheimer's disease, with dosing expected to begin in the second half of 2026. VY1706 is the first tau-targeted gene therapy to receive IND clearance from the FDA. The therapy uses a vectorized siRNA encapsulated in a TRACER AAV capsid that crosses the blood-brain barrier via the ALPL receptor, achieving broad and durable CNS delivery. No adverse findings were observed in the central nervous system, dorsal root ganglia, or peripheral organs up to the highest dose tested.

Voyager Therapeutics received FDA clearance for its Investigational New Drug application for VY1706, a gene therapy targeting tau in Alzheimer's disease. The approval allows the company to begin clinical trials in individuals with early Alzheimer's symptoms, with dosing expected to start in the second half of 2026. CEO Alfred W. Sandrock called it the first IND clearance for a tau-targeted gene therapy, noting that VY1706 is designed to durably reduce tau protein levels after a single intravenous administration. The therapy uses siRNA targeting MAPT mRNA, delivered via a TRACER AAV capsid that utilizes ALPL to direct it to the brain.