Voyager Receives FDA IND Clearance for VY1706, First Gene Therapy Approach to Reducing Tau Production in the Brain for Alzheimer’s Disease

- Dosing of adults with early Alzheimer’s disease in clinical trial expected H2 2026 -

- Comprehensive preclinical program showed compelling pharmacology and safety profile for VY1706 -

- VY1706 uses an IV-delivered, brain-targeted capsid to reduce intracellular and extracellular tau -

LEXINGTON, Mass., June 01, 2026 — Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to leveraging genetics to treat neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for VY1706, the Company’s investigational gene therapy targeting intracellular and extracellular tau for Alzheimer’s disease (AD). The IND clearance enables initiation of a clinical trial of VY1706 in adults with early AD; dosing is expected to begin in the second half of the year.

“The IND clearance for VY1706 is the first for a tau-targeted gene therapy and follows a comprehensive preclinical program demonstrating a compelling pharmacology and safety profile,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager. “Recent third-party data continue to suggest that tau is the next critical target in Alzheimer’s disease, and that reducing tau production holds promise. We view VY1706 as leading the next generation of tau targeting treatments; it is designed to durably reduce tau protein levels in key brain regions following a single IV administration.”

The core of VY1706 is a potent, vectorized siRNA that targets MAPT mRNA to decrease levels of both intracellular and extracellular tau in the brain. This core is encapsulated in a Voyager TRACER™ AAV capsid that leverages ALPL, a well-conserved, novel receptor identified by Voyager, to deliver the siRNA into the brain following a one-time, intravenous (IV) dose. The efficacy and safety of VY1706 have been assessed in a comprehensive preclinical program spanning multiple species. VY1706 has been demonstrated to reduce tau protein up to 75% in key brain regions relevant to AD and to de-target the liver, a source of adverse events associated with other systemically administered gene therapies.

Clinical Trial Design

Voyager is initiating a multi-site, open-label, dose-escalation clinical trial of VY1706 administered as a one-time IV dose to adult participants with early AD who have evidence of tau pathology in the brain, confirmed by PET imaging. The study will enroll up to 18 patients across three cohorts, with the highest dose not exceeding 5E13 vg/kg, the top dose tested in the good laboratory practice (GLP) toxicology study. The trial’s primary endpoint is to evaluate the safety and tolerability of VY1706. Secondary endpoints will assess VY1706’s effect on tau biology, including changes in cerebrospinal fluid (CSF) biomarkers of tau, as well as changes in tau pathology measured by tau PET imaging.

Preclinical Data Supporting VY1706

Three-month GLP toxicology data for VY1706 demonstrated a favorable tolerability profile with no adverse clinical pathology or histopathological findings up to the highest dose tested (5E13 vg/kg) at 13 weeks following a single IV dose in non-human primates (NHPs). Treatment with VY1706 resulted in dose-dependent reductions of up to 51-75% MAPT mRNA and 48-64% tau protein in key brain regions implicated in AD, such as the entorhinal cortex, frontal cortex, temporal cortex, and hippocampus.1 These findings were consistent with findings from a previous NHP study.2 Further, murine studies have demonstrated robust and dose-dependent reductions in pathological tau protein levels following a single IV dose in the P301S mouse tauopathy model.3

About VY1706

VY1706 is an investigational gene therapy for Alzheimer’s disease (AD) that targets tau, a protein associated with neurodegeneration and cognitive decline in AD. The core of VY1706 is a potent, vectorized siRNA that targets MAPT mRNA to decrease levels of both intracellular and extracellular tau in the brain. This core is encapsulated in a Voyager TRACER™ AAV capsid that leverages ALPL, a well-conserved, novel receptor identified by Voyager, to deliver the siRNA into the brain following a one-time, intravenous (IV) dose. The efficacy and safety of VY1706 have been assessed in a comprehensive preclinical program spanning multiple species. VY1706 has been demonstrated to reduce tau protein up to 75% in key brain regions relevant to AD and to de-target the liver, a source of adverse events associated with other systemically administered gene therapies. Voyager is initiating a clinical trial of VY1706 administered as a one-time IV dose to adults with early AD.

About Alzheimer’s Disease (AD)

AD is a fatal neurodegenerative disease characterized by severe memory impairment and loss of the ability to carry out everyday tasks as the disease progresses4; it impacts approximately 7.2 million people in the U.S.5, and 32 million people globally are estimated to have AD dementia6. After disease onset, patients experience a profound decline in quality of life, and death occurs within an average of four to eight years of diagnosis5. Tau pathology correlates strongly with neurodegeneration and cognitive decline in AD7, and a high unmet medical need remains for disease-modifying therapies that address tau pathology and further slow disease progression. In 2025, the total cost of caring for people living with Alzheimer’s and other dementias in the U.S. was estimated at $384 billion5.

About the TRACER™ Capsid Discovery Platform

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.

About Voyager Therapeutics

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, Friedreich’s ataxia, Parkinson’s disease, amyotrophic lateral sclerosis (ALS), 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; and Neurocrine Biosciences, Inc. For more information, visit http://www.voyagertherapeutics.com.

Voyager Therapeutics® is a registered trademark, and TRACER™ and Voyager NeuroShuttle™ are trademarks, of Voyager Therapeutics, Inc.

Contacts

Trista Morrison, NACD.DC, [email protected] Investors: Sarah McCabe, [email protected] Media: Adam Silverstein, [email protected]

1 Arora et al. ASGCT 2026. 2 Sivasankaran et al. ADPD 2026. 3 Bao et al. ASGCT 2025. 4 Masters CL, Bateman R, Blennow K, et al. Alzheimer’s disease. Nat Rev Dis Primers. 2015;1:15056. 5 2025 Alzheimer’s disease facts and figures. Alzheimers Dement. 2025;21(5). 6 Gustavsson A, Norton N, Fast T, et al. Global estimates on the number of persons across the Alzheimer’s disease continuum. Alzheimers Dement. 2023;19:658-70. 7 Braak H, Braak E. Neuropathological staging of Alzheimer-related changes. Acta Neuropathol. 1991;82(4):239-59.