NEW YORK – Voyager Therapeutics on Wednesday said it is developing a tau-silencing gene therapy, VY1706, for treating Alzheimer's disease and plans to seek regulatory permission to start testing it in clinical trials in the US and Canada in 2026.
VY1706 is meant to be delivered intravenously. The gene therapy uses capsids designed to penetrate the blood-brain barrier and deliver an siRNA construct there that aims to decrease expression of tau, a hallmark of Alzheimer's.
In addition to VY1706, Voyager is also conducting a Phase I trial of VY7523, a monoclonal antibody designed to treat Alzheimer's by inhibiting the spread of pathological tau, in healthy volunteers. Toby Ferguson, chief medical officer at Voyager, said in a statement that the company's decision to clinically advance VY1706 and the Phase I trial of VY7523 already underway "demonstrate our conviction that tau is an important target in Alzheimer's disease."
"We have seen third-party data indicating that both a tau antibody and a tau knockdown approach can impact tau accumulation in a human brain, and that this may correlate with clinical benefit," Ferguson said.
In a preclinical study of nonhuman primates, a single dose of the VY1706 gene therapy candidate reduced tau mRNA levels in the range of 50 percent to 73 percent across the cerebral cortex.
Lexington, Massachusetts-based Voyager developed the capsids used in VY1706 with its adeno-associated virus capsid discovery platform, called Tropism Redirection of AAV by Cell-type-specific Expression of RNA, or TRACER for short. VY1706 is the fifth gene therapy candidate for a neurological condition that Voyager has picked to take into clinical trials that it developed using its TRACER capsids.