SOUTH SAN FRANCISCO, Calif. — June 7, 2024 — Epicrispr Biotechnologies, a leading epigenetic editing company that plans to have its FSHD program enter the clinic this year, today announced its participation in the upcoming FSHD International Research Congress being held June 13-14, 2024, in Denver, Colo.
In an oral presentation at the conference, the company will present the highlights of the IND-enabling data package for lead candidate EPI-321, an epigenetic therapy for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Data to be presented include updated efficacy and safety data that were recently presented at the 2024 ASGCT Annual Meeting.
In addition, Epicrispr Biotechnologies will engage in panel discussions at the conference alongside members of the FSHD community.
Full details of the oral presentation are as follows:
Presentation Title: “Efficacy and Safety of an Investigational Single Dose Epigenome Editing Therapy, EPI-321, Targeting D4Z4 in Facioscapulohumeral Muscular Dystrophy (FSHD)”
Session: Disease Mechanisms & Interventional Strategies
Presenter: Alexandra Collin de l’Hortet
Date & Time: Thursday, June 13, 2024 at 12:20 p.m. MDT
Abstract Number: 7
Location: Hilton Denver City Center, Denver, Colo.
About EPI-321
EPI-321 is an investigational epigenetic therapy that aims to address the underlying molecular mechanisms of FSHD by restoring methylation to the D4Z4 region of chromosome 4 and halting toxic expression of the DUX4 gene. EPI-321 is delivered to muscle tissue within a single AAV vector (AAVrh74) which has been clinically validated for muscle delivery. Preclinical studies on EPI-321 have demonstrated its ability to robustly suppress pathological expression of the DUX4 gene and rescue muscle cell survival.
About Epicrispr Biotechnologies
Epicrispr Biotechnologies is a leading epigenetic editing company, leveraging the power of CRISPR without cutting DNA. The company’s proprietary Gene Expression Modulation System (GEMS) includes the smallest Cas protein known to work in human cells, enabling in vivo or ex vivo delivery via a single viral vector. Epicrispr plans to initiate a clinical trial of its lead program — EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD) — in 2024; additional programs seek to address alpha-1 antitrypsin deficiency (A1AD), heterozygous familial hypercholesterolemia (HeFH), and other indications. Visit www.epicrispr.com for more information or follow us on X and LinkedIn.
Investor Contact
Benson Cheng
benson.cheng@epic-bio.com
Media Contact
Lisa Raffensperger
Ten Bridge Communications
lisa@tenbridgecommunications.com