Voyager Therapeutics, Inc. · NASDAQ:VYGR
Voyager Therapeutics — Technical Presentation (May 2026)
16 slides from Voyager Therapeutics's technical presentation, May 2026
Slide 1: Intravenous Delivery of VY1706, a CNS Penetrant AAV Gene Therapy for 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 Presenter: Todd Carter, Chief Scientific Officer Authors: Vik Arora, Joydeep Ghosh, Yasuhiro Ito, Charlotte Chung, Jeffr
Slide 2: Disclosures
Disclosures • Todd Carter is a full-time employee of Voyager Therapeutics, Inc. 2 |
Slide 3: Tau Accumulation is Associated with Alzheimer's Disease Progression
Tau Accumulation is Associated with Alzheimer’s Disease Progression Accumulation of Tau measured by Tau PET • Alzheimer’s disease affects ~ 7M in the U.S.1 imaging is associated with neuropathological • Progressive neurodegenerative disease, staging of disease progression4 causing relentless memory
Slide 4: Single IV Dose of BBB-crossing Tau Lowering AAV Gene Therapy to Treat Alzheimer's Disease
Single IV Dose of BBB-crossing Tau Lowering AAV Gene Therapy to Treat Alzheimer’s Disease Vectorized MAPT mRNA Tau Protein Neurofibrillary Slowing of pri-amiRNA Lowering Lowering Tangle Lowering Neuronal Loss amiRNA Slowing of AD Progression & Improvement of Tau mRNA Quality of Life Pri-amiRNA conta
Slide 5: BBB-crossing Gen2 TRACER Capsids Demonstrate Enhanced CNS Tropism and Liver De-targeting
BBB-crossing Gen2 TRACERTM Capsids Demonstrate Enhanced CNS Tropism and Liver De-targeting Across Species TRACER Capsids: Pan-Species CNS Targeting via Gen2 Capsids: Increased Brain Tropism and Liver De-targeting in NHP ALPL Binding AAV9 Gen 1 Gen 2 Capsid Cortex Liver ALPL Gen2 Capsids: 50-75% of C
Slide 6: VY1706 Demonstrates Dose-dependent Reduction of Total and Pathological Tau in P301S Mice
VY1706 Demonstrates Dose-dependent Reduction of Total and Pathological Tau in P301S Mice Study Scheme IV Injection i.v. dosing Necropsy P301S Mouse Age 12 wks 20 wks Male + Female Tau Protein Pathological Tau Cortex Hippocampus Cortex Hippocampus VY1706 VY1706 VY1706 VY1706 6 |
Slide 7: VY1706 Shows Robust Tau Protein Knockdown in Neurons Across AD-relevant Brain Regions in NHP
VY1706 Shows Robust Tau Protein Knockdown in Neurons Across AD-relevant Brain Regions in NHP DRF Study Neuronal Tau protein lowering Vehicle VY1706 VY1706 de-targets the liver ~30-fold decrease relative to similar AAV9 dose Tau Protein Expression in SMI311+ve Neurons at 11 weeks: SMI311 (neurons); T
Slide 8: GLP 3-Month Toxicology Demonstrates Favorable Safety, Tolerability Profile
VY1706 GLP 3-Month Toxicology Demonstrates Favorable Safety, Tolerability Profile with No Adverse Findings Across Tested Dose Range NUMBER OF Study design: ANIMALS GROUP Delivery Duration: 3-month MALES FEMALES ( ) ( ) Species: Cynomolgus macaques Vehicle 2 2 Animals were seronegative prior to dosin
Slide 9: VY1706 Shows No AAV-Typical Liver Transaminase Elevations at All Doses Tested
VY1706 Shows No AAV-Typical Liver Transaminase Elevations at All Doses Tested 5E13 vg/kg 9 | Timepoints: Predose, Day 3, Day 5, Week 2, Week 4, Week 13
Slide 10: VY1706 Demonstrates Stable Plasma NfL with No Dose-related Elevations
VY1706 Demonstrates Stable Plasma NfL with No Dose-related Elevations Vehicle Low Dose Mid Dose 1 Mid Dose 2 5E13 vg/kg 10 |
Slide 11: Dose-Dependent Increase in miRNA Level and Lowering of MAPT mRNA in NHP
VY1706 Shows Dose-Dependent Increase in miRNA Level and Lowering of MAPT mRNA in AD-relevant Brain Regions in NHP amiRNA MAPT mRNA Copies/µg of RNA % of Vehicle VY1706 demonstrated broad CNS biodistribution and robust dose-dependent MAPT mRNA lowering of up to 51-75% in AD-relevant brain regions GLP
Slide 12: Dose-Dependent Lowering of Tau Protein in AD-Relevant Brain Regions in NHP
VY1706 Shows Dose-Dependent Lowering of Tau Protein in AD-Relevant Brain Regions in NHP % of Vehicle VY1706 demonstrated robust dose-dependent Tau protein lowering in the range of up to 48-64% in AD-relevant brain regions GLP Tox Study (3 month); n=4/group; Plots show mean + SD 12 | EC: Entorhinal C
Slide 13: VY1706 Capsid Demonstrates Cross-species Translatability of Capsid-ALPL Interaction
VY1706 Capsid Demonstrates Cross-species Translatability of Capsid-ALPL Interaction Transduction Assay Binding Assay (SPR) ALPL Binding Model 13 |
Slide 14: VY1706 Capsid Engages an Endothelial Receptor Distinct from Published Capsida Vector
VY1706 Capsid Engages an Endothelial Receptor Distinct from Published Capsida Vector • We examined the receptors mediating BBB transport of the two capsids: • VY1706 uses ALPL as its primary BBB receptor • Identification of CAP receptor as ADAM15, not the patent reported PI16 • No receptor cross-rea
Slide 15: Conclusions
Conclusions • Single IV dose of VY1706 achieved broad CNS delivery of vectorized MAPT siRNA in multiple nonclinical species • ALPL, a well conserved endothelial receptor, mediates VY1706 blood-brain barrier transport, supporting cross-species translatability • VY1706 was safe and well-tolerated in t
Slide 16: Team Acknowledgements
Team Acknowledgements Key Contributors: Rajeev Sivasankaran Mathieu Nonnenmacher Kevin Nguyen Elena Ratti Wencheng Liu Tyler Moyer Krishanu Mathur Raj Rajagovindan Dinah Sah Brett Hoffman Kumar Dhanasekharan Vik Arora Hechen Bao Cansu Colpan Marina Feschenko Aadhar Mahajan Maneesha Paranjpe Jeron Ch
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