Translating ALPL-Mediated BBB Transport into the Clinic: Next-Generation Capsid Engineering for CNS Gene Therapy

• Translate ALPL-mediated BBB transport from platform discovery into clinical reality, supported by early human data and safety readouts
• Position ALPL as a differentiated, cross-species BBB target with improved translational relevance beyond legacy approaches such as TfR1
• Demonstrate how strong NHP validation of ALPL-targeting capsids enables progression into clinical CNS programs, including tau knockdown in Alzheimer’s disease
• Showcase next-generation capsid engineering strategies that enhance brain-wide transduction, reduce off-target effects, and improve systemic gene therapy delivery

Speakers:

Mathieu Nonnenmacher

Vice President, Gene Therapy

Voyager Therapeutics