EBT-001

EBT-001 is an investigational AAV9-based CRISPR-Cas9 gene-editing therapy developed by Excision BioTherapeutics for the treatment of simian immunodeficiency virus (SIV) in non-human primates. It serves as the preclinical precursor to EBT-101, which is being developed for HIV-1 in humans. EBT-001 utilizes a Staphylococcus aureus Cas9 (SaCas9) nuclease and dual guide RNAs (gRNAs) to target and excise integrated proviral DNA from the host genome. Specifically, it targets the viral long terminal repeats (LTRs) and the Gag gene. In preclinical studies using rhesus macaques, the therapy demonstrated broad biodistribution to key viral reservoirs and successfully excised viral DNA without evidence of off-target effects or significant toxicity.