Voyager Selects Tau Silencing Gene Therapy Development Candidate for Alzheimer’s Disease

Voyager Therapeutics, Inc. recently announced it has selected a lead development candidate, VY1706, for its tau silencing gene therapy program in Alzheimer’s disease. The company anticipates filing an investigational new drug (IND) application with the U.S. Food and Drug Administration and a clinical trial application (CTA) with Health Canada for VY1706 in 2026.

VY1706 combines a potent siRNA construct to decrease the expression of tau with an IV-delivered, blood-brain barrier-penetrant TRACER capsid. In a non-human primate (NHP) study, a single dose of VY1706 resulted in reductions in tau mRNA levels of 50% to 73% across the cerebral cortex, including in areas of the brain where tau accumulates during progression of Alzheimer’s disease. Voyager expects to present these and additional data at an upcoming scientific conference.

“The nomination of this tau silencing development candidate VY1706, along with the ongoing clinical trial of our anti-tau antibody VY7523, demonstrate our conviction that tau is an important target in Alzheimer’s disease,” said Toby Ferguson, MD, PhD, Chief Medical Officer of Voyager. “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. We will continue to assess and learn from emerging data in this field as we efficiently progress our differentiated tau antibody and tau gene therapy programs towards human proof-of-concept.”

Alzheimer’s disease is a progressive neurodegenerative disease estimated to affect 7 million people in the US and up to 416 million people globally. The disease causes memory loss and may escalate to decreased independence, communication challenges, behavioral disorders such as paranoia and anxiety, and lack of physical control. In 2023, the total cost of caring for people living with Alzheimer’s and other dementias in the US was estimated at $345 billion.

Voyager’s TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of novel AAV capsids to enable gene therapy. Voyager has leveraged TRACER to create multiple families of novel capsids that, following intravenous delivery in preclinical studies, harness the extensive vasculature of the central nervous system (CNS) to cross the blood-brain barrier and transduce a broad range of CNS regions and cell types. In cross-species preclinical studies (rodents and multiple non-human primate species), intravenous delivery of TRACER-generated capsids resulted in widespread payload expression across the CNS at relatively low doses, enabling selection of multiple development candidates in Voyager’s wholly-owned and partnered gene therapy programs for neurologic diseases.

Voyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more information, visit www.voyagertherapeutics.com.