VY1706 / Voyager - LARVOL DELTA

Pharmacology and Safety Assessment of VY1706, a BBB‑Crossing Tau‑Lowering AAV Gene Therapy for Alzheimer's Disease, in a GLP Toxicology Study in NHPs (ESGCT 2026) - No abstract available

Clinical • Gene therapy • Alzheimer's Disease • CNS Disorders • Gene Therapies

An Open Label Dose Escalation Study of VY1706 in Participants With Early Alzheimer's Disease (clinicaltrials.gov) - P1 | N=18 | Recruiting | Sponsor: Voyager Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • First-in-human • Alzheimer's Disease • CNS Disorders

Second Quarter 2026 and Recent Highlights (The Manila Times) - "VY7523 (anti-tau antibody): Voyager expects tau positron emission tomography (PET) imaging efficacy data in Q4 2026 from the ongoing multiple ascending dose (MAD) clinical trial in participants with Alzheimer’s disease (AD)..VY1706 (tau silencing gene therapy)...Dosing is expected to begin Q4 2026.In July, Health Canada cleared Voyager’s Clinical Trial Application (CTA), enabling the inclusion of Canadian clinical trial sites in the study....Early 2027: Potential for initial acute safety data with VY1706, pending enrollment; H2 2027: Potential for initial biomarker-based data for VY1706, pending enrollment."

New trial • P1/2 data • Alzheimer's Disease

An Open Label Dose Escalation Study of VY1706 in Participants With Early Alzheimer's Disease (clinicaltrials.gov) - P1 | N=18 | Not yet recruiting | Sponsor: Voyager Therapeutics

First-in-human • New P1 trial • Alzheimer's Disease • CNS Disorders

First-in-Human Study Design of VY1706, a Systemically Administered Blood-Brain Barrier (BBB)- Penetrant Tau Lowering Gene Therapy in Early Alzheimer's Disease (CTAD 2026) - No abstract available

First-in-human • Gene therapy • P1 data • Alzheimer's Disease • CNS Disorders • Gene Therapies

IND‑Enabling GLP NHP Study Of VY1706, A BBB‑Crossing AAV Gene Therapy Targeting Tau In Alzheimer's (AAIC 2026) - No abstract available

Gene therapy • Alzheimer's Disease • CNS Disorders • Gene Therapies

Voyager’s Tau-Targeted Gene Therapy VY1706 for Alzheimer’s Disease to Be Featured in Developing Topics Poster Presentation at AAIC 2026 (GlobeNewswire) - "The Developing Topics poster presentation will feature VY1706, Voyager’s investigational gene therapy targeting intracellular and extracellular tau for Alzheimer’s disease (AD). Earlier this month, Voyager received U.S. Food and Drug Administration (FDA) Investigational New Drug (IND) clearance for VY1706, enabling initiation of a clinical trial in adults with early AD, with dosing expected to begin in the second half of the year."

New trial • Preclinical • Alzheimer's Disease

Voyager Receives FDA IND Clearance for VY1706, First Gene Therapy Approach to Reducing Tau Production in the Brain for Alzheimer’s Disease (The Manila Times) - "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...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."

IND • New trial • Alzheimer's Disease

Intravenous Delivery of VY1706, a CNS Penetrant AAV Gene Therapy for Alzheimer’s Disease, Demonstrates Compelling Pharmacology and Safety in a 3-Month GLP Toxicology Study in NHPs (ASGCT 2026) - P1 | "The trial design incorporated standard conditioning regimens such as myeloablative conditioning (MAC) or reduced-intensity conditioning (RIC) as well as GvHD prophylaxis anchored in post-transplant cyclophosphamide (PTCy) plus mycophenolate mofetil (MMF) and sirolimus. Conclusion TRX103 is a novel, off-the-shelf, engineered Tr1 Treg cell therapy that promotes reprogramming of the post-HSCT immune system towards a tolerogenic state. This immunological reprogramming offers a proof-of-mechanism strategy to mitigate the incidence of complications, such as graft-versus-host disease (GvHD) and infections, in high-risk patients undergoing HSCT for hematological malignancies."

Clinical • First-in-human • Gene therapy • Late-breaking abstract • Alzheimer's Disease • Bone Marrow Transplantation • CNS Disorders • Gene Therapies • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • CD4 • FOXP3 • IFNG • IL10 • IL2 • NGFR

Allogenic Off-the-Shelf, Engineered Tr1 Treg cells (TRX103) Exhibit Dose-Dependent Persistence and Establish a Tolerogenic Environment for the Prevention of GvHD in Patients Undergoing HLA-Mismatched Hematopoietic Stem Cell Transplantation (ASGCT 2026) - "Introduction Tau pathology progression in the brain is strongly correlated with cognitive decline in Alzheimer’s disease (AD). These results build on previously described pharmacology and safety data and highlight the potential of VY1706 as a one-time, well- tolerated treatment strategy for AD and other tauopathies, demonstrating the promise of combining potent tau-targeting pri-amiRNAs with novel BBB-penetrant delivery systems. Conclusion Building on prior pharmacology and safety data, these findings position VY1706 as a well- tolerated, single-dose therapeutic strategy for AD and other tauopathies, enabled by potent tau-targeting pri-amiRNAs and BBB-penetrant delivery systems."

Clinical • Late-breaking abstract • Alzheimer's Disease • Bone Marrow Transplantation • CNS Disorders • Gene Therapies • Graft versus Host Disease • Immunology • Transplantation

Voyager ASGCT Late Breaker: Single IV Dose of VY1706 Well Tolerated, Reduced Tau in 3-Month GLP Toxicology Data; Clinical Trial in Alzheimer’s Disease Expected H2 2026 (GlobeNewswire) - "Data presented showed that VY1706 was well tolerated, with no adverse clinical pathology or histopathological findings up to the highest dose tested (5E13 vg/kg), and reduced tau protein up to 64% in key brain regions of non-human primates (NHPs) at 13 weeks following a single IV dose...Voyager’s U.S. Food and Drug Administration (FDA) investigational new drug (IND) application process for VY1706 is on track for Q2 2026 to support projected first-in-human dosing in AD patients in H2 2026...Treatment with VY1706 resulted in dose-dependent reductions of up to 51-75% MAPT mRNA and 48-64% tau protein in key brain regions at 13 weeks in NHPs."

First-in-human • IND • Late-breaking abstract • New trial • Preclinical • Alzheimer's Disease

First Quarter 2026 and Recent Highlights (The Manila Times) - "Multiple presentations at ASGCT 2026, including late breaker on VY1706 3-month GLP tox data...VY1706 (tau silencing gene therapy): Voyager completed investigational new drug (IND)-enabling good laboratory practice (GLP) toxicology in Q1 2026. The U.S. Food and Drug Administration (FDA) IND application process is on track for Q2 2026 to support projected first-in-human dosing in Alzheimer’s disease (AD) patients in H2 2026; VY7523 (anti-tau antibody): Voyager continues to expect tau positron emission tomography (PET) imaging efficacy data in H2 2026 from the ongoing multiple ascending dose (MAD) clinical trial in AD patients."

First-in-human • IND • New trial • P1/2 data • Preclinical • Alzheimer's Disease

Intravenous Delivery of VY1706, a CNS Penetrant AAV Gene Therapy for Alzheimer’s Disease, Demonstrates Compelling Pharmacology and Safety in a 3-Month GLP Toxicology Study in NHPs (ASGCT 2026) - No abstract available

Clinical • Gene therapy • Late-breaking abstract • Alzheimer's Disease • CNS Disorders • Gene Therapies

Voyager IV-Delivered CNS Gene Therapies Featured in Multiple Presentations at ASGCT 2026, Including Late Breaker on Tau-Targeted VY1706 for Alzheimer’s Disease (GlobeNewswire) - "Voyager’s investigational tau-silencing gene therapy VY1706 for Alzheimer’s disease, for which Voyager anticipates submitting an investigational new drug (IND) application in Q2 2026 to support projected first-in-human dosing in the second half of 2026, will be featured in a late-breaking presentation of three-month good laboratory practice (GLP) toxicology data....Intravenous delivery of VY1706, a CNS penetrant AAV gene therapy for Alzheimer’s disease, demonstrates compelling pharmacology and safety in a 3-month GLP toxicology study in NHPs."

First-in-human • IND • Preclinical • Alzheimer's Disease

INTRAVENOUS DELIVERY OF VY1706, A CNS PENETRANT AAV GENE THERAPY FOR TAUOPATHIES, PROVIDES BROAD TAU LOWERING IN NHP (ADPD 2026) - "The extensive biodistribution of VY1706 into AD and PSP relevant brain regions, together with its robust pharmacological activity, supports its potential as a promising one-time, IV treatment strategy for these Tauopathies."

Gene therapy • Alzheimer's Disease • CNS Disorders • Gene Therapies • Movement Disorders • Progressive Supranuclear Palsy

Pipeline updates (Yahoo Finance) - "VY1706 (tau-silencing gene therapy): Voyager expects completion of a good laboratory practice (GLP) toxicology study in Q1 2026, investigational new drug (IND) application submission in Q2 2026, and first-in-human dosing in H2 2026. Following a pre-IND communication with U.S. Food and Drug Administration (FDA) in Q1 2025, Voyager completed a Type C communication with FDA in Q1 2026 and believes there to be a path to IND...H2: Tau PET imaging data expected in MAD clinical trial of VY7523 in AD."

First-in-human • IND • P1/2 data • Preclinical • Alzheimer's Disease

The primary focus for the upcoming year centers on two Alzheimer's programs, both scheduled for the second half of 2026 (AD HOC NEWS) - "Clinical Phase 1 initiation is planned for H2 2026 for VY1706, a gene therapy candidate; Study data for the Alzheimer's antibody VY7523 is expected in H2 2026."

New P1 trial • P1/2 data • Alzheimer's Disease

Anticipated Upcoming Milestones (GlobeNewswire) - "(i) 2025-2026: Potentially informative data read-outs expected for tau-targeting agents from multiple third parties; (ii) 2026: U.S. IND/Canadian CTA submissions + clinical trial initiation anticipated for VY1706 in AD; (iii) 2026: Neurocrine indicates potential clinical trial initiations for FA and GBA1 programs; (iv) H2 2026: Initial tau PET imaging data expected in MAD clinical trial of VY7523 in AD."

Clinical data • New trial • Alzheimer's Disease • Friedreich ataxia

Second Quarter 2025 and Recent Highlights (GlobeNewswire) - "VY7523 (anti-tau antibody): initial tau positron emission tomography (PET) data from clinical trial in Alzheimer’s disease (AD) patients expected in the second half of 2026...Potential for data from multiple gene therapy programs: Neurocrine-partnered FA and GBA1 programs anticipated to enter clinical trials in 2026; VY1706 tau silencing gene therapy anticipated to enter a clinical trial in 2026; Critical third-party data from large Phase 2 studies of an anti-tau antibody and tau knockdown program expected in 2026, which have the potential to further validate tau as a target....VY1706 (tau silencing gene therapy): IND-enabling work is underway to support an IND and clinical trial initiation expected in 2026."

Clinical data • IND • New trial • Alzheimer's Disease • Friedreich ataxia

Cross-Species BBB-Penetrant IV-Delivered AAV Gene Therapy Provides Broad and Robust CNS Tau Lowering in Tauopathy Mouse Models and Non-Human Primate (ASGCT 2025) - "In the present study, we utilized a second-generation, cross-species BBB-penetrant TRACER™-derived capsid to intravenously deliver a vectorized primary artificial microRNA (pri-amiRNA) containing a potent tau siRNA, VY-1706, in tauopathy mouse models as well as non-human primates (NHP)...These findings demonstrate that the combination of a potent and well-tolerated tau targeting pri-amiRNA with a novel cross-species BBB-penetrant capsid holds promise as a one-time, IV treatment option for AD and other tauopathies. Disease Focus of Abstract:Central Nervous System Disorders"

Gene therapy • Preclinical • Alzheimer's Disease • CNS Disorders • Gene Therapies

Intravenous Delivery of VY1706, a CNS penetrant AAV gene therapy for Alzheimer's Disease, Provides Broad Tau Lowering in NHP (ASGCT 2025) - "Supported by the Neurologic and Opthalmic Committee"

Gene therapy • Alzheimer's Disease • CNS Disorders • Gene Therapies

Voyager Reports First Quarter 2025 Financial and Operating Results (GlobeNewswire) - "Advanced tau silencing gene therapy VY1706 for AD: Completed a pre-IND interaction with the U.S. Food and Drug Administration (FDA). IND-enabling work is ongoing to support U.S. IND and Canadian clinical trial application (CTA) submissions expected in 2026."

FDA event • IND • New trial • Alzheimer's Disease

Voyager Reports Fourth Quarter and Full Year 2024 Financial and Operating Results (GlobeNewswire) - "Development candidate selected for tau silencing gene therapy program VY1706: In a three-month non-human primate (NHP) study, a single 1.3E13 vg/kg dose of VY1706 delivered intravenously (IV) resulted in reductions in tau mRNA levels of 50% to 73% across the cerebral cortex, including in areas of the brain where tau accumulates during progression of Alzheimer’s disease (AD). Voyager expects to present additional data from the NHP study of VY1706 at the International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (ADPD) April 1-5, 2025....Anticipated Upcoming Milestones:...2026: U.S. IND and Canadian clinical trial application (CTA) filings anticipated with tau silencing gene therapy VY1706 for AD."

IND • New trial • Preclinical • Alzheimer's Disease

Voyager Provides Update on SOD1 ALS Gene Therapy Program (GlobeNewswire) - "Voyager Therapeutics... today announced it has decided to assess alternate payloads related to its gene therapy program for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS)....Voyager no longer anticipates filing an investigational new drug (IND) application for VY9323 in mid-2025. Voyager’s cash runway is now expected to extend into mid-2027...We continue to expect IND filings in 2025 for our gene therapy candidates for GBA1 and FA, and in 2026 for VY1706."

IND • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • CNS Disorders