SAN FRANCISCO, Oct. 19, 2023 (GLOBE NEWSWIRE) — Excision BioTherapeutics Inc., a clinical-stage biotechnology company developing CRISPR-based therapies to cure viral infectious diseases, today announced that interim clinical safety and biodistribution data will be presented at the 30th European Society of Gene and Cell Therapy (ESGCT) 2023 Annual Congress, which will be held from October 24-27, 2023, in Brussels, Belgium.
Dr. Rachel Presti of Washington University School of Medicine, a principal investigator for the clinical evaluation of EBT-101, will offer the first look at interim safety and biodistribution data from the EBT-101-001 Phase 1/2 clinical trial. EBT-101 is being evaluated in a first-in-human clinical study to assess its safety and efficacy in people with HIV on antiretroviral therapy.
The presentation details are listed below, and the full program is available on the ESGCT website.
Session: 5A Infectious Diseases & Vaccines
Date/Time: Wednesday, October 25, 2023, 14:30-16:30 pm (CEST)
Title: First-in-human trial of systemic CRISPR-Cas9 multiplex gene therapy for functional cure of HIV (OR31)
Room: Shed 2A Parallel
EBT-101 is a first-in-class in vivo CRISPR-based therapeutic designed to cure HIV infection after a single intravenous infusion. EBT-101 employs an adeno-associated virus (AAV) to deliver CRISPR-Cas9 and dual guide RNAs, enabling a multiplexed in vivo editing approach that simultaneously targets two distinct and conserved sites within the latent HIV genome distinct from the human genome. This allows for the excision of large portions of the HIV genome, thereby minimizing potential viral escape.
About the EBT-101 Clinical Program
The EBT-101-001 Phase 1/2 trial is an open-label, multi-center, single ascending dose study designed to evaluate the safety, tolerability, biodistribution and pharmacodynamics of EBT-101 in approximately nine participants with HIV-1 with an undetectable viral load on antiretroviral therapy. Preliminary efficacy assessments will also be conducted. Participants in EBT-101-001 will be followed for 48 weeks post EBT-101 administration. All eligible participants will be assessed for sustained viral suppression off their background ART, in an analytical treatment interruption (ATI) starting at Week 12. After EBT-101-001, participants will be enrolled in a long-term follow up study, EBT-101-002. For more information, see ClinicalTrials.gov identifiers NCT05144386 (Phase 1/2 trial) and NCT05143307 (long-term follow up study).
The EBT-101 Phase 1/2 clinical trial is supported by a grant from the California Institute for Regenerative Medicine (CIRM). For more information on CIRM go to www.cirm.ca.gov.
About Excision BioTherapeutics, Inc.
Excision BioTherapeutics, Inc. is a clinical-stage biotechnology company developing CRISPR-based therapies as potential cures for viral infectious diseases. EBT-101, the Company’s lead program, is an in vivo CRISPR-based therapeutic designed to cure HIV infection after a single intravenous infusion. Excision’s pipeline unites next-generation CRISPR nucleases with a novel gene editing approach to develop curative therapies for Herpes Virus, JC Virus, which causes PML, and Hepatitis B Virus. Excision’s foundational technologies were developed in the laboratories of Dr. Kamel Khalili at Temple University and Dr. Jennifer Doudna at the University of California, Berkeley. For more information, please visit www.excision.bio.