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LEXINGTON, Mass. - Voyager Therapeutics, Inc. (NASDAQ:VYGR), a biotechnology firm focused on genetic treatments for neurological diseases with a market capitalization of $176.5 million, today reported a significant scientific advancement in drug delivery to the brain. According to InvestingPro analysis, the company appears undervalued at current levels, maintaining a strong liquidity position with a current ratio of 6.1. The company’s research, recently published in the journal Molecular Therapy, demonstrates how a novel adeno-associated virus (AAV) capsid, using alkaline phosphatase (ALPL) as a receptor, can traverse the blood-brain barrier (BBB).
The peer-reviewed publication details the creation of a new AAV capsid named VCAP-102, which has shown a 20- to 400-fold increase in gene transfer efficiency across multiple brain regions in both rodents and non-human primates compared to the standard AAV9. The identification of ALPL as the primary receptor for VCAP-102 crossing the BBB could be a pivotal step in gene therapy, potentially enabling more effective treatment of neurological diseases. While the company holds more cash than debt on its balance sheet, InvestingPro data indicates it’s currently burning through cash rapidly, with analysts anticipating a sales decline in the current year.
Voyager’s Vice President of Gene Therapy, Mathieu Nonnenmacher, Ph.D., highlighted the foundational role of understanding ALPL in the company’s gene therapy programs. He noted that this discovery has led to the development of next-generation capsids with improved brain transduction and liver de-targeting, as well as immune-evading capabilities.
The company has leveraged its TRACER capsid discovery platform to generate these novel capsids, which have demonstrated widespread CNS payload expression at relatively low doses in preclinical studies. This has enabled the selection of multiple development candidates for Voyager’s gene therapy programs.
Voyager’s Chief Scientific Officer, Todd Carter, Ph.D., emphasized the strategic importance of a multi-modality approach, which includes both viral and non-viral CNS delivery methods, to meet the unmet needs in neurological disease treatment.
The company has previously presented data on next-generation and stealth-capsid at the American Society of Gene & Cell Therapy’s annual meeting, showcasing high levels of neuronal and glial transduction across various brain regions in non-human primates.
Voyager Therapeutics, which has collaborations with several industry partners, is advancing towards Investigational New Drug (IND) filings for two of its gene therapy programs this year.
This scientific breakthrough, based on a press release statement, could pave the way for more effective treatments for patients with neurological disorders and represents a significant milestone in the field of gene therapy. With the next earnings report due on July 31, 2025, investors seeking deeper insights can access comprehensive analysis through InvestingPro’s detailed research reports, which provide extensive coverage of Voyager Therapeutics and over 1,400 other US stocks.
In other recent news, Voyager Therapeutics has unveiled promising preclinical data for its Alzheimer’s disease therapies, VY1706 and VY7523. The company reported that a single intravenous dose of VY1706 in non-human primates significantly reduced tau mRNA and protein levels in the brain, with effects lasting up to three months. Additionally, the VY7523 antibody showed potential in targeting pathological tau tangles in murine studies, with plans for further trials underway. Voyager intends to file an Investigational New Drug application for VY1706 in 2026. Meanwhile, Canaccord Genuity has maintained a Buy rating for Voyager Therapeutics but adjusted its price target from $14 to $12, citing changes in the VY9323 program. H.C. Wainwright reaffirmed its Buy rating with a $30 price target, highlighting Voyager’s strong financial position and strategic partnerships. Truist Securities also maintained a Buy rating with an $18 price target, noting the company’s progress in its gene therapy programs. Citi adjusted its price target to $11, maintaining a Buy rating, while emphasizing the clean safety profile of VY7523.
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