AAV-hfCas13Y-gBACE1

AAV-hfCas13Y-gBACE1 is an experimental gene therapy designed to treat Alzheimer's disease by targeting and degrading the mRNA of beta-site amyloid precursor protein cleaving enzyme 1 (BACE1). Developed by HuidaGene Therapeutics, the therapy utilizes a high-fidelity CRISPR-Cas13Y (hfCas13Y) system delivered via an adeno-associated virus (AAV) vector. Unlike traditional CRISPR-Cas9 systems that edit DNA, Cas13Y targets RNA, providing a precise and potentially reversible approach to reducing amyloid-beta (Aβ) production without permanently altering the host genome. Preclinical studies in 5xFAD mouse models and non-human primates have demonstrated significant reductions in BACE1 expression and cerebrospinal fluid Aβ levels, alongside improvements in cognitive function.