Interim data demonstrated a favorable safety profile and early evidence of disease modification, including statistically significant increases in lean muscle volume and biomarker changes consistent with DUX4 suppression
Additional clinical data expected to be presented at the World Muscle Society Annual Congress in September 2026
SAN FRANCISCO–(BUSINESS WIRE)–Epicrispr Biotechnologies, a clinical-stage company pioneering epigenetic therapies, today announced the completion of enrollment and dosing in the dose-escalation portion of its first-in-human clinical trial evaluating EPI-321 for facioscapulohumeral muscular dystrophy (FSHD). All 12 patients have now been enrolled and dosed across two dose cohorts.
The milestone follows the company’s recently reported interim clinical data demonstrating a favorable safety profile and early evidence of disease modification. Among evaluable patients treated with a single dose of EPI-321, Epicrispr observed statistically significant increases in whole-body lean muscle volume measured by MRI, favorable changes in circulating biomarkers consistent with DUX4 suppression, favorable strength and functional outcomes, and a manageable safety profile.
“Completing enrollment and dose escalation marks an important milestone for the EPI-321 program,” said Amber Salzman, Ph.D., Chief Executive Officer of Epicrispr Biotechnologies. “We completed enrollment as rapidly as the study protocol allowed in this complex first-in-human AAV gene therapy trial, and our recently reported interim data provide encouraging early evidence that EPI-321 may modify the underlying biology of FSHD. We look forward to presenting additional clinical data at the World Muscle Society Annual Congress in September and completing 12-month follow-up across all participants next year.”
The ongoing open-label, dose-escalation study is evaluating the safety, tolerability, and preliminary biological and clinical activity of EPI-321 in adults with FSHD. The completed dose-escalation portion enrolled six patients at each of two intravenous dose levels (2 × 1013 and 4 × 1013 vg/kg).
Additional clinical data from the study are expected to be presented at the World Muscle Society Annual Congress in September 2026.
FSHD is a progressive genetic neuromuscular disease affecting approximately one million people worldwide, according to the FSHD Society. The disease is caused by inappropriate expression of the DUX4 gene, leading to progressive skeletal muscle degeneration, weakness, and disability. There are currently no approved therapies for 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 and durable epigenetic modulation 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. Learn more at www.epicrispr.com or follow us on LinkedIn.
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