A Dual-guide RNA Treatment for HIV
Excision BioTherapeutics’ lead program, EBT-101, is an in vivo CRISPR-based therapeutic candidate designed to excise human immunodeficiency virus (HIV) pro-viral DNA from HIV-infected cells. EBT-101 is being developed as a potential curative treatment for patients with chronic HIV and is rapidly advancing toward clinical trials.
Our Pipeline
Other
EBT-101
HIV AAV9 Cas9 dual gRNA Therapeutic
| Discovery/Validation | Preclinical | Cleared IND | Clinical Trials |
|---|---|---|---|
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Discovery/Validation Phase complete
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Preclinical Phase complete
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Cleared IND Phase in progress
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Clinical Trials Phase not started
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$23 Billion Market Opportunity
Prevalence
~1 million HIV cases in the U.S.1
~38 million HIV cases worldwide1
Target Population
Stable on Anti-Retroviral Therapy (ART)
Standard of Care
Antiretrovirals (non-curative)
1) AIDS. 2017 Jan 28; 31
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
Clinical Trials
On September 15, 2021, the U.S. Food and Drug Administration (FDA) accepted the Investigational New Drug (IND) application for EBT-101, enabling the program to commence a first-in-human Phase 1/2 clinical trial to evaluate the safety, tolerability, and efficacy of EBT-101 in individuals with HIV.
View Clinical Trial for EBT-101Upcoming Milestones
Initiating Phase 1/2 clinical trial in the second half of 2021.
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.
More About Our TechnologyResearch & Publications
View our library of publications for more information on the research behind our CRISPR gene-editing technology.
View Publications