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

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.

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