Excision BioTherapeutics, Inc., the developer of CRISPR-based therapies intended to cure viral infectious diseases, announced that the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application for EBT-101, a CRISPR-based therapeutic candidate in development as a potential functional cure for chronic HIV. The IND clearance enables Excision to initiate a first-in-human Phase 1/2 clinical trial to evaluate the safety, tolerability, and efficacy of EBT-101 in individuals living with human immunodeficiency virus type 1 (HIV).

“The clearance of our IND application for EBT-101 represents an important milestone for Excision and is the result of years of commitment to developing a functional cure for individuals living with HIV,” said Daniel Dornbusch, Chief Executive Officer of Excision. “Although antiviral treatments can manage HIV infection, they require life-long treatment, cause side effects, and do not provide the possibility of a functional cure. We are grateful for the FDA’s engaged review and acceptance of the IND for EBT-101 and look forward to initiating the Phase 1/2 clinical trial later this year.”

EBT-101, an in vivo CRISPR-based therapeutic designed to excise HIV proviral DNA, is a unique gene therapy that leverages CRISPR’s evolutionary history as a viral defence system in bacteria. In preclinical studies, it has demonstrated the ability to excise HIV proviral DNA in multiple cell lines: human primary cells as well as multiple animal models including non-human primates. EBT-101 utilizes an adeno-associated virus (AAV) to deliver a one-time treatment intended to functionally cure 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.

Lisa Danzig, MD, Excision’s Chief Medical Officer, added, “EBT-101 has demonstrated removal of proviral DNA in multiple animal models and offers an opportunity for individuals living with HIV to potentially cease life-long therapies. The Excision team looks forward to this important collaboration with our principal investigators, scientific advisors and regulators, to conduct a safe and informative trial with this first-in-class approach to a viral disease target previously considered to be incurable.”