Voyager Next-Generation CNS Capsids Featured at ASGCT 28th Annual Meeting

Rhea-AI Summary

Voyager Therapeutics announced eight presentations at the upcoming American Society of Gene & Cell Therapy's (ASGCT) annual meeting in New Orleans, May 13-17, 2025. The company will showcase significant advances in gene therapy for neurological diseases.

Key highlights:

• VY1706, their tau silencing gene therapy, achieved up to 73% knockdown of tau mRNA in non-human primates after a single IV dose
• Their TRACER capsids demonstrated impressive results, with 43-98% neuron transduction and 87-99% astrocyte transduction across brain regions
• Presentations will cover anti-amyloid gene therapy and immune evasion developments

The company expects to file two Investigational New Drug (IND) applications this year and another next year. The presentations span multiple areas, including:

• Anti-tau and anti-amyloid therapies for Alzheimer's disease
• Reducing immunogenicity of capsids
• Enhancing manufacturing processes

Positive

• VY1706 demonstrated strong tau mRNA knockdown of up to 73% in non-human primates with a single IV dose
• TRACER capsids showed high neuron transduction rates of 43-98% and astrocyte transduction of 87-99% across brain regions
• Company expects to file two INDs this year and another next year, showing clear clinical development progress
• Development of immune-evading capsids could expand the potential patient population
• Multiple presentations showcase technological advances in both anti-tau and anti-amyloid therapies for Alzheimer's disease

Negative

• All current data is from preclinical studies only, with no human trial results yet
• High viral doses required (1.3e13 to 3e13 vg/kg) which could impact manufacturing costs and treatment expenses

Insights

Biotechnology Analyst

Voyager's gene therapy shows 73% tau reduction in primates with upcoming INDs signaling clinical progression toward Alzheimer's treatment.

Voyager Therapeutics is showcasing promising preclinical data for their gene therapy programs targeting Alzheimer's disease at the upcoming ASGCT meeting. The headliner, VY1706, has demonstrated up to 73% knockdown of tau mRNA in non-human primates after a single intravenous dose—a significant achievement given tau's central role in Alzheimer's pathology.

What truly differentiates Voyager's approach is their proprietary TRACER capsid technology, which enables exceptional blood-brain barrier penetration. The data shows remarkable transduction rates of 43-98% of neurons and 87-99% of astrocytes across brain regions after a single IV administration at 3e13 vg/kg. These percentages represent extraordinary target engagement that could translate to therapeutic efficacy.

The company's progression toward clinical validation is evidenced by their expected two IND filings this year and another in 2026. These regulatory submissions represent crucial derisking events for their pipeline. The company is also developing immune evasion technologies for their capsids, which could potentially expand the treatable patient population by addressing pre-existing neutralizing antibodies to AAV vectors.

Neuroscience Expert

Voyager's dual-targeting Alzheimer's gene therapies show exceptional brain penetration with single-dose administration, advancing toward clinical trials.

Voyager's approach to Alzheimer's targets both major pathological hallmarks: tau and amyloid-beta. Their tau silencing therapy (VY1706) addresses neurofibrillary tangles while their vectorized anti-amyloid antibody targets amyloid plaques—a comprehensive strategy aligned with current understanding of Alzheimer's pathophysiology.

The 73% tau mRNA knockdown achieved in non-human primates represents a significant advantage over current approaches. Most tau-targeting therapies face challenges with CNS penetration and typically require repeated administrations. A single IV dose achieving this level of knockdown could offer superior patient convenience and compliance compared to existing treatment paradigms.

Their TRACER capsid platform directly addresses the critical challenge of CNS delivery. The blood-brain barrier has historically limited neurological therapeutic efficacy, but Voyager's capsids demonstrate remarkably high transduction rates across multiple brain regions. The immune evasion technology being developed further enhances this platform by potentially circumventing pre-existing immunity to AAV vectors—a known limitation for gene therapy approaches. With multiple INDs expected within the next two years, Voyager is poised to bring these technologies into human testing, representing meaningful progression for their Alzheimer's disease pipeline.

- Oral presentation on tau silencing gene therapy VY1706, which has previously shown up to 73% knockdown of tau mRNA in NHPs in the CNS following a single IV dose of 1.3e13 vg/kg -

- Featured data also include anti-amyloid gene therapy for Alzheimer’s disease, as well as multiple presentations on Voyager’s continued enhancements to its highly BBB penetrant novel capsids -

LEXINGTON, Mass., April 28, 2025 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to leveraging genetics to treat neurological diseases, today announced eight oral and poster presentations at the upcoming American Society of Gene & Cell Therapy’s (ASGCT) 28th annual meeting taking place in New Orleans, May 13-17, 2025.

“Voyager continues to raise the bar with our TRACER capsids. In multiple studies utilizing a variety of payloads, our capsids have transduced 43%-98% of neurons and 87-99% of astrocytes broadly across brain regions following a single intravenous 3e13 vg/kg dose in non-human primates,” said Todd Carter, Ph.D., Chief Scientific Officer of Voyager Therapeutics. “Our new data at ASGCT build on our strong foundation in developing gene therapies for Alzheimer’s disease: our tau silencing gene therapy VY1706, which will be featured in an oral presentation, has previously shown up to 73% knockdown of tau mRNA in NHPs following a single IV dose of 1.3e13 vg/kg, and we will also present data from our anti-amyloid gene therapy program. Our data also feature continued enhancements such as immune evasion to potentially increase the percentage of the population who could benefit from these treatments. With two INDs expected this year and another next year, we look forward to assessing and hopefully validating the performance of our capsids in humans.”

Anti-Tau and Anti-Amyloid Gene Therapies for Alzheimer’s Disease

• Oral Presentation: Intravenous delivery of VY1706, a CNS penetrant AAV gene therapy for Alzheimer’s disease, provides broad tau lowering in NHP. Rajeev Sivasankaran, Ph.D., VP, Head of Neuroscience. Thursday, May 15, 2025, 8:50 a.m. – 9:15 a.m. CT
• Cross-species BBB-penetrant IV-delivered AAV gene therapy provides broad and robust CNS tau lowering in tauopathy mouse models and non-human primate (#559). Hechen Bao, Ph.D., Scientist II, Neuroscience. Tuesday, May 13, 2025, 6:00 p.m. – 7:30 p.m. CT
• One-time delivery of a vectorized anti-amyloid antibody for increased and sustained CNS expression and target engagement (#541). Cassandra Retzlaff, Ph.D., Scientist II, Neuroscience. Tuesday, May 13, 2025, 6:00 p.m. – 7:30 p.m. CT
  

Reducing Immunogenicity and Enhancing Developability and Manufacturing of Capsids

• Oral Presentation: Discovery of AAV9-derived CNS capsids evading pre-existing neutralizing antibodies. Damien Maura, Ph.D., Senior Scientist II, Capsid Discovery. Wednesday, May 14, 2025, 2:15 p.m. – 2:30 p.m. CT
• Machine-learning for AAV9 mutant-capsid screening for both production and ALPL-mediated transduction efficiency (#1911). Daniel Cox, Ph.D., Senior Scientist, Data Sciences. Thursday, May 15, 2025, 5:30 p.m. – 7:00 p.m. CT
• Assessment of two HEK293 cell line cloning strategies to improve AAV yield (#1954). Hung-Lun Hsu, Ph.D., Scientist II, Process Development. Thursday, May 15, 2025, 5:30 p.m. – 7:00 p.m. CT
• Enabling large-scale implementation of anion exchange chromatography for full capsid enrichment of a novel adeno-associated viral vector (#1455). Tom Elich, B.S., Senior Engineer II, Process Development. Wednesday, May 14, 2025, 5:30 p.m. – 7:00 p.m. CT
• An alternative to detergent lysis: Promoting rAAV release to media by optimizing osmolality, pH and harvest timing (#1477). Christian Gagnon, M.S., Senior Associate Engineer, Process Development. Wednesday, May 14, 2025, 5:30 p.m. – 7:00 p.m. CT
  

Presentations will be available on Voyager’s website at: https://www.voyagertherapeutics.com/science-publications/.

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™ is a trademark, of Voyager Therapeutics, Inc. 

Forward-Looking Statements
This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “will,” “expect,” “potential,” “believe,” “could,” or “continue,” and other similar expressions are intended to identify forward-looking statements.

For example, all statements Voyager makes regarding Voyager’s ability to advance its AAV-based gene therapy programs such as tau mRNA knock-down program with VY1706 and its anti-amyloid gene therapy program, including expectations for Voyager’s achievement of preclinical and clinical development milestones for its potential development candidates for treating Alzheimer’s disease;  the potential for Voyager’s novel TRACER capsids to achieve desired results in humans, including achievement of a higher therapeutic index and increased patient eligibility to receive AAV gene therapies by immune evasion; Voyager’s expectation to advance gene therapy product candidates through IND filings under its internal and partnered programs; and the ability of Voyager’s improvements in manufacture to enable increased yields and large-scale development of AAV gene therapies are forward looking.

All forward-looking statements are based on estimates and assumptions by Voyager’s management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain and subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the expectations and decisions of regulatory authorities; the timing, initiation, conduct and outcomes of Voyager’s preclinical and clinical studies; the availability of data from clinical trials; the availability or commercial potential of product candidates under collaborations; the success of Voyager’s product candidates; the willingness and ability of Voyager's collaboration partners to meet obligations under collaboration agreements with Voyager; the continued development of Voyager’s technology platforms, including Voyager’s TRACER platform and its non-viral platform technology; Voyager’s scientific approach and program development progress, and the restricted supply and increased costs of critical research components; the development by third parties of capsid identification platforms that may be competitive to Voyager’s TRACER capsid discovery platform; Voyager’s ability to create and protect intellectual property rights associated with the TRACER capsid discovery platform, the capsids identified by the platform, and development candidates for Voyager’s pipeline programs; the possibility or the timing of Voyager’s receipt of program reimbursement, development or commercialization milestones, option exercise, and other payments under Voyager’s existing licensing or collaboration agreements; the ability of Voyager to negotiate and complete licensing or collaboration agreements with other parties on terms acceptable to Voyager and the third parties; the success of programs controlled by third-party collaboration partners in which Voyager retains a financial interest; the ability to attract and retain talented directors, employees, and contractors; and the sufficiency of Voyager’s cash resources to fund its operations and pursue its corporate objectives.

These statements are also subject to a number of material risks and uncertainties that are described in Voyager’s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Contacts
Trista Morrison, NACD.DC, tmorrison@vygr.com
Investors: Sarah McCabe, smccabe@jpa.com
Media: Brooke Shenkin, brooke@scientpr.com

FAQ

What percentage tau reduction did Voyager's (VYGR) VY1706 gene therapy achieve in NHP studies?

Voyager's VY1706 gene therapy demonstrated up to 73% knockdown of tau mRNA in non-human primates (NHPs) following a single intravenous dose of 1.3e13 vg/kg.

How effective are Voyager's (VYGR) TRACER capsids in neuron and astrocyte transduction?

Voyager's TRACER capsids showed 43%-98% neuron transduction and 87-99% astrocyte transduction across brain regions after a single intravenous 3e13 vg/kg dose in non-human primates.

When will Voyager (VYGR) present their Alzheimer's gene therapy data at ASGCT 2025?

Voyager will present their tau silencing gene therapy VY1706 data on Thursday, May 15, 2025, from 8:50-9:15 a.m. CT at the ASGCT annual meeting in New Orleans.

How many IND submissions is Voyager (VYGR) planning for 2025-2026?

Voyager Therapeutics expects to submit two INDs in 2025 and another one in 2026, totaling three IND submissions over the next two years.

What are the key programs Voyager (VYGR) is presenting at ASGCT 2025?

Voyager is presenting data on their tau silencing gene therapy VY1706, anti-amyloid gene therapy for Alzheimer's disease, and improvements to their BBB-penetrant novel capsids, including immune evasion capabilities.