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EBT-103

Development stage: Preclinical
Lead developer: Excision BioTherapeutics
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration: Intravenous

Overview

**EBT-103** is an investigational AAV-based gene therapy developed by Excision BioTherapeutics for the potential functional cure of HIV infections and progressive multifocal leukoencephalopathy (PML). It employs CRISPR/Cas9 delivered via adeno-associated virus (AAV) vectors with guide RNAs targeting specific viral genome sites, such as JC virus for PML or HIV proviral DNA, to excise integrated viral sequences from host genomes, aiming to eliminate latent reservoirs and prevent viral replication.[3][17]

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