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Voyager Therapeutics’ TRACER™ Capsids Demonstrate Enhanced CNS Transduction Across Species; New Preclinical Results Bolster GBA1, Tau, SOD1 ALS Gene Therapy Programs
VCAP-102 capsid achieved respective 60-fold and 50-fold improvements in brain transduction in non-human primates and mice compared to conventional AAV9
About this update from Voyager Therapeutics, Inc.
[{"type":"text","content":"VCAP-102 capsid achieved respective 60-fold and 50-fold improvements in brain transduction in non-human primates and mice compared to conventional AAV9 capsids with IV dosing Data demonstrate potential of multiple TRACER capsids to target CNS disorders, including certain diseases that may benefit from glial cell targeting Preclinical data demonstrate potential to ameliorate disease in models of GBA1, tauopathies, and SOD1 ALS Results presented at the American Society of Gene and Cell Therapy 25th Annual Meeting CAMBRIDGE, Mass., May 19, 2022 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy company developing life-changing treatments and next-generation adeno-associated virus (AAV) capsids, today is scheduled to present new preclinical data on a family of AAV9-derived TRACER capsids demonstrating cross-species central nervous system (CNS) transduction. The Company is also scheduled to report updated results from preclinical gene therapy programs in GBA1, tauopathies, and SOD1 ALS. These results are being presented at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting in Washington, D.C. Family of AAV9-Derived Capsids Enhanced Brain Transduction Across Non-Human Primates and Mice Voyager’s TRACER capsid discovery platform has identified a novel AAV9-derived capsid, VCAP-102, which demonstrated 50-fold better transduction in mice and 60-fold better transduction in non-human primates (NHPs) versus conventional AAV9 capsids, following intravenous (IV) administration. While other TRACER capsids have shown strong neuronal delivery, preclinical data in this study also demonstrated that VCAP-102 and other TRACER capsids showed preferential tropism for glial cells in mice. “These TRACER findings are important because demonstrating equivalent cross-species functionality is critical to increasing a capsid’s potential for translation into humans,” said Mathieu Nonnenmacher, Ph.D., Vice President of Capsid Discovery at Voyager. “Furthermore, the glial tropism observed in this AAV9-derived capsid family may allow us to address CNS indications that would benefit from non-neuronal cell transduction.” Key Results Top variants from the capsid family exhibited increased blood brain barrier (BBB)-penetrance across NHP species and mice.Top variants showed preferential transduction of glial cells in ...