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

Excerpt from the Press Release:

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.

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