- EBT-101 achieves HIV excision without detectable off-target effects
- Poster presentation to take place today from 5:30 – 6:30 PM ET
SAN FRANCISCO, May 17, 2022 (GLOBE NEWSWIRE) — Excision BioTherapeutics Inc., a biotechnology company developing CRISPR-based therapies intended to cure viral infectious diseases, today announced that preclinical and bioinformatic data showing robust and specific editing of HIV proviral DNA with EBT-101 will be featured in an upcoming poster presentation at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting, which is taking place at the Walter E. Washington Convention Center in Washington, D.C.
EBT-101 is a unique, clinical-stage, in vivo CRISPR-based therapeutic designed to functionally cure HIV infections after a single intravenous infusion. It employs an adeno-associated virus (AAV) to deliver CRISPR-Cas9 and dual guide RNAs targeting three sites within the HIV genome. Bioinformatic analyses featured in the ASGCT poster compare these EBT-101 target sites against the human reference genome and show no identical matches. Moreover, there are no candidate off-target sites in the human reference genome with only one or two differences from EBT-101 target sites, and only a single candidate off-target site with three differences. These findings predict a lack of off-target activity for EBT-101, which has been confirmed by in vitro and in vivo studies showing HIV excision without unintended editing.
“Taken together, the data being presented at ASGCT highlight the key advantages of Excision’s novel therapeutic approach,” said TJ Cradick, Ph.D., Chief Scientific Officer of Excision and lead author on the poster. “By leveraging our in-silico design platform, we carefully crafted EBT-101 to minimize viral escape and off-target effects by cutting at three target sites that are specific to the HIV genome. Latent viral reservoirs were eradicated in in multiple disease models and resulted in functional cures in transgenic mice. We are now working to build on these results through the advancement of EBT-101’s recently initiated Phase 1/2 clinical trial, and look forward to discussing our program with the scientific community at the upcoming ASGCT meeting.”
The ASGCT poster, entitled, EBT-101 achieves robust CRISPR-based editing of HIV proviral DNA without detectable off-target effects, will be presented today from 5:30 – 6:30 PM ET during the “Hematologic and Immunologic Disease II” poster session (Board No. TU-205). The poster was authored in collaboration with researchers from institutions including Temple University and Dartmouth-Hitchcock Medical Center. A copy of the poster will be posted to the Excision’s website following its presentation at the meeting.
EBT-101 is a unique, in vivo CRISPR-based therapeutic designed to functionally cure HIV infections after a single intravenous infusion. It employs an adeno-associated virus (AAV) to deliver CRISPR-Cas9 and dual guide RNAs targeting three sites within the HIV genome. This novel approach enables the excision of large portions of the HIV genome, thereby minimizing potential viral escape. EBT-101 is being evaluated in clinical trials and is the only therapeutic candidate in history to achieve functional cures of HIV in animal models.
About Excision BioTherapeutics, Inc.
Excision BioTherapeutics, Inc. is a biotechnology company developing CRISPR-based therapies as potential cures for viral infectious diseases. Excision unites next-generation CRISPR nucleases with a novel gene editing approach and has become the first and only company in history to cure HIV with a therapeutic in an animal model. EBT-101, the company’s lead program intended to cure HIV with a single IV infusion, is being evaluated in clinical trials. Excision’s pipeline includes one-time potential cures for Herpes Virus, JC Virus, which causes PML, and Hepatitis B virus. The Company’s foundational technologies were developed in the laboratories of Dr. Kamel Khalili at Temple University and Dr. Jennifer Doudna at University of California, Berkeley. For more information, please visit www.excision.bio.
Robert Flamm, Ph.D.
Burns McClellan, Inc.
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