Voyager Therapeutics (VYGR) Stifel 2026 Virtual CNS Forum summary

Key events and strategic focus for 2026

• 2026 is highlighted as the "Year of Tau" with two major tau-directed assets: an antibody and a gene therapy tau knockdown, both expecting key data readouts this year.

• First clinical use of newly discovered blood-brain barrier (BBB)-penetrant capsids, with two assets entering the clinic, including a Neurocrine-partnered Friedreich's ataxia program.

• Plans to present additional data on the shuttle platform, an extension of the capsid discovery platform.

Scientific rationale and disease insights

• Tau pathology, rather than amyloid, is strongly implicated as the driver of dementia progression, supported by human cohort studies and biomarker staging.

• Amyloid abnormalities precede tau accumulation, but the spread of tau correlates more closely with cognitive decline.

• The spread of pathological tau, not its initial misfolding, is linked to disease progression in Alzheimer's.

Development and regulatory strategy

• Ongoing close interactions with the FDA, including pre-IND and Type C meetings, to ensure safe and effective study design for tau knockdown gene therapy.

• Initial clinical focus is on demonstrating tau reduction in the brain using tau PET imaging, mirroring successful approaches in prior trials.

• IND filings for tau knockdown gene therapy and the Friedreich's ataxia program are planned for 2024, with clinical entry expected in the second half of the year.