Voyager Presents Robust Preclinical Data from Tau Targeting Gene Therapy and Antibody Programs at AD/PD™ 2025
Voyager Therapeutics (VYGR) has presented new preclinical data for its tau-targeting programs in Alzheimer's disease treatment at AD/PD™ 2025. The company's tau silencing gene therapy VY1706 demonstrated significant results in non-human primate studies, showing 44-73% reduction in tau mRNA and 27-55% reduction in tau protein levels after a single intravenous dose, with effects lasting up to three months.
The therapy achieved broad brain distribution while showing 30-fold liver de-targeting compared to wild type AAV9. VY1706 is advancing toward IND filing expected in 2026. Additionally, their anti-tau antibody VY7523 showed selectivity for pathologic tau tangles in preclinical studies. The company is currently conducting a multiple ascending dose trial of VY7523 in early Alzheimer's patients, with initial tau PET imaging data expected in second half of 2026.
Voyager Therapeutics (VYGR) ha presentato nuovi dati preclinici per i suoi programmi mirati al tau nel trattamento dell'Alzheimer durante l'AD/PD™ 2025. La terapia genica di silenziamento del tau VY1706 ha mostrato risultati significativi negli studi su primati non umani, con una riduzione del 44-73% dell'mRNA tau e una riduzione del 27-55% dei livelli di proteina tau dopo una singola dose endovenosa, con effetti che durano fino a tre mesi.
La terapia ha raggiunto una distribuzione ampia nel cervello, mostrando un de-targeting epatico 30 volte superiore rispetto al tipo selvatico AAV9. VY1706 sta avanzando verso la richiesta di IND prevista per il 2026. Inoltre, il loro anticorpo anti-tau VY7523 ha mostrato selettività per i grovigli patologici di tau negli studi preclinici. L'azienda sta attualmente conducendo uno studio di dosaggio ascendente multiplo di VY7523 in pazienti con Alzheimer precoce, con i dati iniziali di imaging PET tau attesi nella seconda metà del 2026.
Voyager Therapeutics (VYGR) ha presentado nuevos datos preclínicos para sus programas dirigidos al tau en el tratamiento de la enfermedad de Alzheimer en el AD/PD™ 2025. La terapia génica de silenciamiento del tau VY1706 demostró resultados significativos en estudios con primates no humanos, mostrando una reducción del 44-73% en el ARN mensajero tau y una reducción del 27-55% en los niveles de proteína tau después de una única dosis intravenosa, con efectos que duran hasta tres meses.
La terapia logró una amplia distribución en el cerebro mientras mostraba un desvío hepático 30 veces mayor en comparación con el AAV9 tipo salvaje. VY1706 avanza hacia la presentación de IND esperada para 2026. Además, su anticuerpo anti-tau VY7523 mostró selectividad por los enredos patológicos de tau en estudios preclínicos. La compañía está llevando a cabo actualmente un ensayo de dosis ascendente múltiple de VY7523 en pacientes con Alzheimer temprano, con datos iniciales de imagen PET tau esperados para la segunda mitad de 2026.
보이저 테라퓨틱스(VYGR)는 AD/PD™ 2025에서 알츠하이머병 치료를 위한 타우 표적 프로그램의 새로운 전임상 데이터를 발표했습니다. 회사의 타우 침묵 유전자 치료제 VY1706는 비인간 영장류 연구에서 44-73%의 타우 mRNA 감소와 27-55%의 타우 단백질 수준 감소를 보여주며 단일 정맥 주사 후 효과가 최대 3개월 동안 지속되었습니다.
이 치료법은 뇌에서 넓은 분포를 달성했으며, 야생형 AAV9에 비해 간에서 30배의 비표적화가 나타났습니다. VY1706은 2026년 IND 제출을 목표로 진행 중입니다. 또한, 그들의 항타우 항체 VY7523는 전임상 연구에서 병리학적 타우 엉킴에 대한 선택성을 보였습니다. 회사는 현재 초기 알츠하이머 환자에서 VY7523의 다중 상승 용량 시험을 진행 중이며, 초기 타우 PET 이미징 데이터는 2026년 하반기에 예상됩니다.
Voyager Therapeutics (VYGR) a présenté de nouvelles données précliniques pour ses programmes ciblant le tau dans le traitement de la maladie d'Alzheimer lors de l'AD/PD™ 2025. La thérapie génique de silençage du tau VY1706 a montré des résultats significatifs dans des études sur des primates non humains, avec une réduction de 44-73% de l'ARNm tau et une réduction de 27-55% des niveaux de protéine tau après une seule dose intraveineuse, avec des effets durables jusqu'à trois mois.
La thérapie a atteint une large distribution dans le cerveau tout en montrant un dé-targeting hépatique 30 fois supérieur par rapport au type sauvage AAV9. VY1706 avance vers le dépôt d'IND prévu pour 2026. De plus, leur anticorps anti-tau VY7523 a montré une sélectivité pour les enchevêtrements pathologiques de tau dans des études précliniques. L'entreprise mène actuellement un essai de dose croissante multiple de VY7523 chez des patients atteints d'Alzheimer précoce, avec des données initiales d'imagerie PET tau attendues dans la seconde moitié de 2026.
Voyager Therapeutics (VYGR) hat auf der AD/PD™ 2025 neue präklinische Daten für seine tau-zielgerichteten Programme zur Behandlung der Alzheimer-Krankheit präsentiert. Die tau-silencing Gentherapie VY1706 zeigte signifikante Ergebnisse in Studien mit nicht-menschlichen Primaten, mit einer Reduktion von 44-73% des tau-mRNA und einer Reduktion von 27-55% der tau-Proteinwerte nach einer einzelnen intravenösen Dosis, wobei die Effekte bis zu drei Monate anhielten.
Die Therapie erreichte eine breite Verteilung im Gehirn und zeigte eine 30-fache De-Targeting der Leber im Vergleich zu Wildtyp AAV9. VY1706 schreitet auf die IND-Anmeldung zu, die für 2026 erwartet wird. Darüber hinaus zeigte ihr Anti-Tau-Antikörper VY7523 in präklinischen Studien eine Selektivität für pathologische tau-Fibrillen. Das Unternehmen führt derzeit eine multiple aufsteigende Dosisstudie von VY7523 bei frühen Alzheimer-Patienten durch, wobei erste tau-PET-Bildgebungsdaten für die zweite Hälfte von 2026 erwartet werden.
- Single IV dose of VY1706 achieved 44-73% tau mRNA reduction and 27-55% tau protein reduction
- VY1706 showed broad brain distribution with 30-fold liver de-targeting
- Effects sustained for 3 months post-dosing
- VY7523 demonstrated positive selective binding to pathologic tau tangles
- IND filing for VY1706 not expected until 2026
- Initial VY7523 imaging data won't be available until second half of 2026
Insights
Voyager's preclinical data represents significant progress in their tau-targeting programs for Alzheimer's disease. The two-pronged approach – combining gene therapy and antibody technologies – strategically addresses tau pathology through complementary mechanisms.
The VY1706 gene therapy data is particularly compelling, demonstrating 44-73% tau mRNA knockdown and 27-55% protein reduction in non-human primates with a single IV dose. This level of target engagement is impressive, especially considering the sustained effect over three months. The broad brain distribution coupled with 30-fold liver de-targeting suggests an optimized delivery profile that maximizes efficacy while potentially reducing off-target effects.
For VY7523, the antibody's demonstrated selectivity for pathological tau tangles is noteworthy. The preclinical mouse model validation is especially valuable when contrasted with competitors – their data showing effectiveness where N-terminal tau antibodies (which previously failed in clinical trials) showed no significant effect in the same model. This suggests Voyager's C-terminal targeting approach may overcome limitations of previous tau-directed therapies.
While both programs remain in early development stages (VY1706 approaching IND in 2026; VY7523 in dose-escalation with imaging data expected H2 2026), these data strengthen the company's scientific positioning in the competitive tau-targeting landscape. The dual-modality approach provides strategic risk diversification across different therapeutic approaches targeting the same pathology.
Voyager's preclinical data release represents meaningful de-risking for both tau-targeting programs. The company's dual-platform approach provides multiple shots on goal against a validated Alzheimer's target, potentially creating significant long-term value.
The gene therapy data offers three critical differentiation points from competing approaches: 1) sustained effects from a single administration, 2) substantial tau reduction across multiple brain regions, and 3) engineered liver de-targeting that may improve the safety profile. This addresses key delivery challenges that have historically CNS-directed gene therapies.
For the antibody program, Voyager's mechanistic validation against previously failed approaches provides important competitive positioning. By directly comparing their C-terminal antibody against N-terminal antibodies that failed clinically, they've demonstrated a potential explanation for why their approach may succeed where others haven't.
While both assets remain years from potential commercialization (IND filing for gene therapy in 2026; clinical imaging data for antibody in H2 2026), these preclinical results validate Voyager's scientific approach. For a company with $214M market cap, having two differentiated programs against a major neurodegenerative disease target provides substantial upside potential if either program continues showing positive data through development.
The scheduled webcast indicates management's confidence in the data package and willingness to engage investors on these early-stage programs, suggesting they view these results as meaningful catalysts for the company's scientific and strategic positioning.
- Single IV administration of tau silencing gene therapy VY1706 significantly reduced tau mRNA and protein levels, with broad brain distribution and liver de-targeting, in NHP study -
- Preclinical murine data strengthen case for specifically targeting pathologic forms of tau with the clinical-stage anti-tau antibody VY7523 -
- Voyager to host live webcast on April 7 recapping key AD/PD™ 2025 data -
LEXINGTON, Mass., March 31, 2025 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to leveraging genetics to treat neurological diseases, today reported new data from its two preclinical programs targeting tau for the treatment of Alzheimer’s disease (AD). Data on tau silencing gene therapy VY1706 and anti-tau antibody VY7523 will be presented at the upcoming 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD™ 2025), taking place April 1-5, 2025, in Vienna. Additionally, Voyager announced it will host a live webcast recapping key AD/PD™ 2025 data at 4:30 p.m. ET on Monday, April 7, 2025.
“Emerging data continue to strengthen our conviction that tau is the next critical target in Alzheimer’s disease, which supports Voyager’s advancement of both antibody and gene therapy approaches,” said Toby Ferguson, M.D., Ph.D., Chief Medical Officer of Voyager Therapeutics. “When I look across the design of these two molecules, the preclinical data each has generated, and the emerging clinical profile for our antibody, I believe our anti-tau antibody and gene therapy each have the potential to be transformative for patients.”
VY1706 tau silencing gene therapy:
CROSS-SPECIES BBB-PENETRANT IV-DELIVERED AAV GENE THERAPY PROVIDES BROAD AND ROBUST CNS TAU LOWERING IN TAUOPATHY MOUSE AND NON-HUMAN PRIMATE. Wencheng Liu, Ph.D., ID:419.
New preclinical data from a non-human primate (NHP) study show that a single, intravenous (IV) 1.3E13 vg/kg dose of Voyager’s tau silencing gene therapy VY1706 resulted in dose-dependent knockdown of tau mRNA (
VY7523 anti-tau antibody:
DISCRIMINATION OF ANTI-TAU ANTIBODIES TARGETING DIFFERENT TAU EPITOPES BY A P301S MOUSE HIPPOCAMPAL SEEDING MODEL OF TAUOPATHY. Wencheng Liu, Ph.D., ID:1403.
New preclinical data show that the murine version of Voyager’s VY7523 demonstrates selectivity for binding pathologic tau tangles. Additionally, Voyager assessed several anti-tau antibodies using a P301S mouse hippocampal seeding model of tau spread. Antibodies that bind the N-terminal of tau and previously failed to achieve their primary endpoints in clinical studies also failed to significantly reduce tau spread in the model. Conversely, the murine version of Voyager’s VY7523, which targets the C-terminal of tau, reduced tau spread in the model, as did a third-party mid-domain antibody that has been shown to reduce tau accumulation in a clinical trial. Voyager is currently assessing VY7523 in a multiple ascending dose trial in patients with early AD and expects initial tau positron emission tomography (PET) imaging data in the second half of 2026.
The posters can be accessed on Voyager’s website at: https://www.voyagertherapeutics.com/science-publications/.
Dr. Ferguson will be speaking on two panels at AD/PD™ 2025:
- Challenges and Opportunities for Anti-Tau Therapies in Clinical Trials – How Can We Make Hopes Come True? Saturday, April 5, 2025, at 2:10 p.m. in Hall A
- Drug Development and Biomarkers in Rare CNS Diseases (ALS, FTD): From Basics to Approval – How to Define Success? Saturday, April 5, 2025, at 4:40 p.m. in Hall A
Live Webcast
Voyager will host a live conference call and webcast at 4:30 p.m. ET on Monday, April 7, 2025, to discuss data from AD/PD™ 2025. A live webcast of the call will be available on the Investors section of the Voyager website at https://ir.voyagertherapeutics.com/, and a replay of the call will be available at the same link approximately two hours after its completion. The replay will be available for at least 30 days following the conclusion of the call.
About VY1706
VY1706 is an IV-administered gene therapy for Alzheimer’s disease that combines a potent siRNA construct to decrease the expression of tau with an IV-delivered, blood-brain barrier-penetrant TRACER™ capsid. In preclinical non-human primate (NHP) studies, a single IV administration of VY1706 was well-tolerated and resulted in dose-dependent knockdown of tau mRNA (
About VY7523
VY7523 is an IV-administered, recombinant, humanized IgG4 monoclonal antibody developed to inhibit the spread of pathological tau, which is closely correlated with disease progression and cognitive decline in Alzheimer’s disease. VY7523 is selective for pathological tau and targets a specific C-terminal epitope of tau. In preclinical in vivo studies, the murine version of VY7523 inhibited seeding and spread of pathological tau by approximately
About Alzheimer’s Disease
Alzheimer’s disease is a progressive neurodegenerative disease estimated to affect 7 million people in the U.S.I and up to 416 million people globallyii. The disease causes memory loss and may escalate to decreased independence, communication challenges, behavioral disorders such as paranoia and anxiety, and lack of physical controliii. In 2023, the total cost of caring for people living with Alzheimer’s and other dementias in the U.S. was estimated at
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,” “believe,” “anticipate,” “potential,” “may,” 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 and tau antibody program, including expectations for Voyager’s achievement of preclinical and clinical development milestones for its potential development candidates such as the IND and CTA filings, the initiation of clinical trials, clinical trial enrollment, and the generation of clinical data; Voyager’s plans to present scientific data at future conferences; the commercial potential for VY7523 and VY1706; the importance of tau as a target for the treatment of AD; and the potential for third-party clinical data to inform Voyager’s clinical development plans 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 antibody screening technology; Voyager’s scientific approach and program development progress, and the restricted supply 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
References:
i. Alzheimer’s Association. 2024 Alzheimer’s Facts and Figures. Available at: https://www.alz.org/media/Documents/alzheimers-facts-and-figures.pdf. Accessed January 9, 2025.
ii. Gustavsson A, Norton N, Fast T, et al. Global estimates on the number of persons across the Alzheimer’s disease continuum. Alzheimer’s Dement. 2023; 19: 658–670. doi: 10.1002/alz.12694.
iii. Penn Medicine. The 7 Stages of Alzheimer’s Disease. Available at: https://www.pennmedicine.org/updates/blogs/neuroscience-blog/2019/november/stages-of-alzheimers. Accessed January 9, 2025.
iv. USAgainstAlzheimer’s. The Alzheimer’s Disease Crisis – By the Numbers. Available at: The Alzheimer’s Disease Crisis – By the Numbers | UsAgainstAlzheimer’s (usagainstalzheimers.org). Accessed: January 9, 2025.
FAQ
What were the key results of VYGR's VY1706 gene therapy in the NHP study?
When will Voyager Therapeutics (VYGR) file IND for VY1706?
What are the initial results of VY7523 in preclinical studies for VYGR?
When will VYGR release tau PET imaging data for VY7523?
