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Brain-penetrant AAV capsid progress at ASGCT 

ARTICLE | Discovery & Translation

Companies and academics showcase two approaches to make AAV capsids with enhanced brain targeting and suppressed liver targeting

By Danielle Golovin, Senior Biopharma Analyst

May 18, 2024 12:16 AM UTC

Gene therapies could transform treatment of many neurological diseases, if the viral vectors were better at getting into the brain. At this year’s ASGCT meeting, at least a dozen research teams presented progress in engineering capsids to cross the blood-brain barrier.

Whether by delivering a functional copy of a mutant gene, or delivering a DNA-encoded antibody or protein therapy, AAV-mediated gene therapy promises new solutions to both rare and common CNS diseases. …