Voyager Therapeutics unveils APOE-targeting Alzheimer’s therapy program

LEXINGTON, Mass. - Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene therapy company whose stock has declined over 45% year-to-date according to InvestingPro data, on Wednesday announced a new gene therapy program targeting apolipoprotein E (APOE), expanding its Alzheimer’s disease franchise. Despite recent market challenges, the company maintains a strong balance sheet with more cash than debt, positioning it for continued research and development initiatives.

The program utilizes Voyager’s proprietary intravenous TRACER capsid technology to deliver a bifunctional payload designed to reduce expression of the high-risk APOE4 variant while simultaneously delivering the protective APOE2 variant in carriers. With a current market capitalization of $170 million and a healthy current ratio of 6.1, InvestingPro analysis suggests the company has sufficient liquidity to advance its development programs.

In preclinical studies, a single intravenous injection of the therapy demonstrated significant reductions of APOE4 in key brain regions of test mice while increasing APOE2 expression to maintain overall APOE levels. The company plans to present early data on this program at a scientific meeting later this year.

"The Voyager team is leveraging our deep expertise in Alzheimer’s disease biology and drug development to advance multiple programs against what we believe to be the three most-promising targets: tau, amyloid, and APOE," said Alfred W. Sandrock, Jr., Chief Executive Officer of Voyager, according to the press release.

With this addition, Voyager’s Alzheimer’s disease portfolio now consists of four wholly-owned assets. These include VY7523, a pathologic-specific anti-tau antibody currently in clinical trials with initial data expected in the second half of 2026; VY1706, an intravenous tau silencing gene therapy advancing toward investigational new drug application in 2026; a vectorized anti-amyloid beta antibody gene therapy; and the newly announced APOE program.

The APOE gene is considered the strongest genetic risk factor for Alzheimer’s disease, with the APOE4 variant strongly linked to higher risk and the APOE2 variant associated with lower risk.

Voyager’s TRACER platform is designed to enable gene therapies that can cross the blood-brain barrier following intravenous delivery, potentially allowing for widespread central nervous system treatment at relatively low doses.

In other recent news, Voyager Therapeutics has reported a significant scientific breakthrough in the delivery of drugs to the brain. The company announced the development of a novel adeno-associated virus (AAV) capsid, named VCAP-102, which shows a dramatic increase in gene transfer efficiency across multiple brain regions in both rodents and non-human primates. This advancement, detailed in the journal Molecular Therapy, highlights the use of alkaline phosphatase (ALPL) as a receptor to traverse the blood-brain barrier, a crucial step in enhancing gene therapy for neurological diseases. The research underscores the potential for more effective treatments, as the new capsid has demonstrated improved brain transduction and immune-evasion capabilities. Voyager Therapeutics is leveraging its TRACER capsid discovery platform to develop these next-generation capsids, which have shown promising results in preclinical studies. The company is advancing towards Investigational New Drug filings for two of its gene therapy programs this year, marking a significant milestone in the field. Voyager’s Chief Scientific Officer emphasized the strategic importance of a multi-modality approach to address unmet needs in neurological disease treatment. The company’s progress in this area is supported by collaborations with several industry partners.

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