EBT-101

A Dual-guide RNA Treatment for HIV

EBT-101 is an in vivo CRISPR-based therapeutic candidate designed to excise human immunodeficiency virus (HIV) pro-viral DNA from HIV-infected cells. Dosing has been completed in the EBT-101 clinical trial, and participants are in follow-up. The trial met the endpoints of safety and tolerability, and Excision has developed a new vector with increased activity and manufacturability that could be pursued with a partner in a new clinical trial.

Our Pipeline

$23 Billion Market Opportunity

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EBT-101’s Editing Strategy

Featuring Two gRNAs Targeting Three Sites Within the HIV Genome

EBT-101 utilizes an adeno-associated virus (AAV) to deliver a one-time treatment intended to functionally eradicate HIV infections. The investigational program employs CRISPR-Cas9 and two guide RNAs which target three sites within the HIV genome, thereby excising large portions of the HIV genome and minimizing potential viral escape.

Preclinical Development

In preclinical studies, EBT-101 demonstrated the ability to excise HIV pro-viral DNA in multiple cell lines such as human primary cells and multiple animal models, including non-human primates. 

Human Cell Lines

• CRISPR Construct Delivery
• HIV DNA Excision
• HIV DNA Efficacy

Mice/Small Animal Models

• CRISPR Construct Delivery
• HIV DNA Excision
• HIV DNA Efficacy in Humanized Mouse Model - No viral rebound

Primates

• CRISPR Construct Delivery
• SIV DNA Excision
• In vivo CRISPR Safety & Biodistribution

The Innovative Technology That Drives Our Programs

Our cutting edge CRISPR gene therapy platform is built on technology licensed from the Doudna Lab at UC Berkeley and the Khalili Lab at Temple University.

Research & Publications

View our library of publications for more information on the research behind our CRISPR gene-editing technology.