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EBT-101

Development stage
Phase 1
Lead developer
Excision BioTherapeutics
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration
Intravenous
01

Overview

EBT-101 is an in vivo CRISPR-based gene therapy developed as a potential functional cure for chronic HIV infection. It uses an adeno-associated virus (AAV) vector to deliver the CRISPR-Cas9 system and two guide RNAs, which target three distinct sites within the integrated HIV genome. This multiplexed editing approach excises large portions of the proviral DNA from infected cells, aiming to prevent production of new intact virus and minimize viral escape. The therapy is administered as a single intravenous infusion and is designed to remove latent HIV reservoirs that are not eliminated by standard antiretroviral therapies. EBT-101 has been evaluated in Phase 1/2 clinical trials for safety, tolerability, biodistribution, and immunogenicity in adults with HIV-1 who are suppressed on antiretroviral therapy[1][3][4][5][8].

02

Targets

HIV-1 RNA (HIV-1 RNA genome)

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