Epicrispr Biotechnologies Announces FDA Clearance of IND Application for EPI-321, A First-in-Class Epigenetic Therapy for FSHD

- U.S. IND clearance follows recent approval of CTA for EPI-321 in facioscapulohumeral muscular dystrophy (FSHD) in New Zealand

- Global Phase 1/2 clinical trial expected to commence in 2025

Epicrispr Biotechnologies, a biotechnology company focused on developing curative therapies, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for EPI-321, a first-in-class epigenetic therapy for the treatment of facioscapulohumeral muscular dystrophy (FSHD), a genetic neuromuscular disease.

IND clearance in the US is part of a global development strategy for EPI-321 and follows the recent approval of its clinical trial application by New Zealand’s Medsafe. The company plans to initiate a global Phase 1/2 clinical trial in 2025.

EPI-321 is an investigational one-time gene-modulating therapy designed to silence aberrant expression of DUX4, a gene that is incorrectly activated in FSHD and leads to progressive muscle degeneration. Delivered systemically via a clinically validated AAV vector, EPI-321 has demonstrated robust suppression of DUX4 expression and protection of muscle tissue in preclinical models. EPI-321 has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations.

“For too long, individuals with FSHD have faced a progressive disease with no treatment options,” said Amber Salzman, Ph.D., Chief Executive Officer, Epicrispr Biotechnologies. “EPI-321 is the first therapeutic candidate designed to silence DUX4 through epigenetic reprogramming, offering a potentially transformative approach to halting disease progression in FSHD. We look forward to initiating our study in the US and bringing EPI-321 to patients who urgently need therapies.”

“FDA clearance of EPI-321 is a significant milestone for the FSHD community,” said Russell Butterfield, M.D., Ph.D., Associate Professor in the Department of Neurology and Pediatrics, University of Utah. “Patients and families have long lacked options for this progressive neuromuscular disorder. EPI-321's innovative approach targeting the root cause of FSHD offers new hope for treatment. We are optimistic about the upcoming U.S. clinical trial and the potential benefits this therapy could bring to individuals living with FSHD.”

About Epicrispr Biotechnologies

Epicrispr Biotechnologies is a biotechnology company pioneering gene-modulating therapies, leading with treatments for neuromuscular diseases. The company’s proprietary Gene Expression Modulation System (GEMS) enables precise, durable control of gene expression, unlocking first-in-class treatments for previously untreatable conditions. Epicrispr’s lead program, EPI-321 is in clinical trials for FSHD, and the company is advancing additional gene-modulating therapies. Epicrispr also has a research collaboration with Kite Pharma to develop next-generation CAR T-cell therapies. Learn more at www.epicrispr.com or follow us on LinkedIn.

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