Voyager Therapeutics (VYGR) TD Cowen Genetic Medicines & RNA Summit summary

Company overview and strategic priorities

• Focused on developing transformative therapies for severe neurological diseases, including ALS and Alzheimer's, with a robust pipeline and strong cash position extending into 2027.

• Advancing three gene therapy programs toward IND filings in 2025, including a wholly owned ALS program.

• Emphasizes four pillars: pipeline, platform, partnerships, and potential, leveraging a proprietary TRACER platform for blood-brain barrier-penetrant capsids.

• Maintains 13 partnered programs, providing non-dilutive revenue and risk mitigation.

• Seeks to balance wholly owned programs with strategic partnerships to maximize value and manage risk.

Technology platform and innovation

• TRACER platform enables discovery of novel capsids with enhanced CNS delivery and reduced peripheral exposure, validated across multiple species.

• Second-generation capsids achieve robust transduction (50%-98%) in key brain regions and cell types with a single IV dose.

• Iterative screening and optimization process allows continuous improvement of capsid performance.

• Platform supports both internal and partnered development candidates, with three moving toward INDs in 2025.

Pipeline and R&D approach

• Prioritizes programs with highly validated targets, high unmet need, and efficient de-risking pathways.

• Focuses on rapid proof-of-concept studies to minimize risk and resource expenditure.

• Lead program VY-TAU01 is a monoclonal antibody targeting the C-terminus of tau, differentiated from prior failed approaches.

• Tau silencing gene therapy uses BBB-penetrant capsids to deliver siRNA, aiming for a one-time IV treatment; IND expected in 2026.

• SOD1 gene silencing program for ALS mirrors the approach of recently approved tofersen, with IND planned for 2025.