VCAP-Gen2

VCAP-Gen2 is a proprietary, second-generation adeno-associated virus (AAV) capsid developed by Voyager Therapeutics, specifically engineered to cross the blood-brain barrier (BBB) with high efficiency after intravenous administration. This BBB-penetrant capsid platform is designed to enable the delivery of genetic payloads to the central nervous system (CNS) for the treatment of neurological disorders. In preclinical studies for Alzheimer's disease, VCAP-Gen2 has been used to deliver a bi-functional gene therapy that simultaneously knocks down the expression of the risk-associated ApoE4 allele using artificial microRNAs and expresses the protective ApoE2 isoform, aiming to achieve therapeutic levels of ApoE2 in key brain regions.