Excision BioTherapeutics Initiates Phase 1/2 Trial Evaluating EBT-101 as a Potential Cure for HIV

  • EBT-101 is a CRISPR-based therapeutic being developed as a potential functional cure for people living with HIV
  • First-in-human trial is now open for enrollment

SAN FRANCISCO, Jan. 27, 2022 (GLOBE NEWSWIRE) -- Excision BioTherapeutics, Inc., a clinical-stage biotechnology company developing CRISPR-based therapies intended to cure viral infectious diseases, today announced the initiation of a first-in-human (FIH) Phase 1/2 clinical trial of EBT-101 in individuals living with human immunodeficiency virus type 1 (HIV). The FIH study is a multicenter, open-label, single ascending dose study designed to evaluate the safety, tolerability and efficacy of EBT-101. The trial is supported by a robust preclinical dataset that includes both positive long-term non-human primate safety data and efficacy data in transgenic mice showing functional cures from HIV with EBT-101 treatment.

“The initiation of this trial is a landmark accomplishment for Excision and an important step towards our goal of developing a cure for patients with HIV,” said Daniel Dornbusch, Chief Executive Officer of Excision. “We endeavor to provide a one-time curative treatment option to people living with HIV. EBT-101 is the first of a unique class of therapeutics with the potential to alleviate a major burden on patients and their families by eradicating viral infections. We sincerely thank all of our stakeholders for their support to advance Excision’s novel therapeutics and look forward to the trial’s progress and results.”

EBT-101 is a unique, in vivo CRISPR-based therapeutic designed to excise large portions of HIV proviral DNA. The investigational program employs CRISPR-Cas9 and dual guide RNAs which target three sites within the HIV genome, thereby excising large portions of the HIV genome and minimizing potential viral escape. EBT-101 utilizes an adeno-associated virus (AAV), to deliver a one-time intravenous infusion intended to functionally cure HIV infections.

Lisa Danzig, M.D., Chief Medical Officer of Excision, commented, “While there are treatments to control HIV, they must be taken for life. Forty years into this pandemic there is still no curative regimen. With the initiation of the EBT-101 Phase 1/2 trial, we are now working to validate our preclinical findings in the clinic by generating safety, pharmacodynamic and preliminary efficacy data. We believe these data will inform the design of future studies and demonstrate EBT-101’s potential to address the unmet needs of patients with HIV.”

Rachel M. Presti, M.D., Ph.D., Associate Professor of Medicine at Washington University School of Medicine in St. Louis and Principal Investigator of the Phase 1/2 trial added, “This trial will evaluate a novel strategy – CRISPR/Cas9 gene editing – to remove HIV DNA from the cells of infected people. We’ll be looking at the safety and tolerability of CRISPR in removing fragments of viral DNA from the genome of infected cells and tissues that are known to be reservoirs of HIV. The existence of such reservoirs has been a major hurdle in our efforts to cure HIV.”

The Phase 1/2 trial is an open-label, multicenter trial designed to evaluate EBT-101 for the treatment of HIV in approximately nine study participants. The primary objective of the trial is to assess the safety and tolerability of a single dose of EBT-101 in study participants with undetectable viral load on antiretroviral therapy (ART). Pharmacokinetic, pharmacodynamic, and efficacy assessments will also be made. Following the initial 48-week first-in-human study, all patients will be enrolled into a long-term follow up study.  For more information, see ClinicalTrials.gov identifiers NCT05144386 (Phase 1/2 trial) and NCT05143307 (long-term follow up trial).

About Excision BioTherapeutics, Inc.

Excision BioTherapeutics, Inc., is a clinical-stage biotechnology company developing CRISPR-based therapies intended to cure viral infectious diseases. The Company focuses on improving the lives of chronically ill patients by excising viral genomes from infected individuals. By using CRISPR in novel ways, Excision's lead program demonstrated the first and only cure in multiple animal models of HIV. The Company's platform technology was initially developed in the Khalili lab at Temple University and the Doudna lab at U.C. Berkeley. Excision is located in San Francisco, California and is supported by ARTIS Ventures, GreatPoint Ventures, Norwest Venture Partners, Adjuvant Capital, Cota Capital, Celesta Capital, IndusAge Partners, Loreda Holdings, Olive Tree, Anzu Partners, SilverRidge Venture Partners, Oakhouse Ventures, and Gaingels. For more information, please visit www.excision.bio.


John Fraunces
LifeSci Advisors

Robert Flamm, Ph.D.
Burns McClellan, Inc.
212-213-0006 ext. 364