Voyager Therapeutics (VYGR) Canaccord Genuity 44th Annual Growth Conference & Private Company Showcase 2024 summary

Leadership and team evolution

• Recent additions to the executive team bring deep neurology and drug development expertise, including a new CFO and CMO with strong backgrounds in neuroscience and neurology drug development.

• The CEO is recognized for assembling a high-caliber team and leading a strategic pivot in 2021 to focus on IV-delivered, brain-penetrant gene therapies.

• The company now has 13 partnered and 4 wholly owned programs, with the lead program already in the clinic.

Technology and innovation

• Developed TRACER technology enabling IV delivery of AAVs across the blood-brain barrier, addressing a major challenge in CNS drug development.

• Identified ALPL as a key receptor for non-viral CNS delivery, opening potential for broader neurogenetics applications.

• Demonstrated robust preclinical data in multiple species and identified human-relevant receptors, supporting clinical translatability.

Pipeline and clinical development

• Lead anti-tau antibody program targets a novel tau epitope, with preclinical data showing 70% reduction in tau spread; clinical data expected in the first half of next year, with tau PET data in late 2026.

• Three gene therapy programs (SOD1 ALS, GBA1, Friedreich's ataxia) are tracking toward INDs in 2025, with initial patient data anticipated soon after trial initiation.

• SOD1 ALS program leverages biomarker endpoints (SOD1, neurofilament) for rapid clinical readouts, aiming for a one-time gene therapy solution.