Epicrispr Reports Early Clinical Activity and Favorable Safety Profile in First-in-Human Epigenetic Editing Study for FSHD
Company to present at J.P. Morgan Healthcare Conference on Tuesday, Jan 13, 2026
Excerpt from the Press Release:
SAN FRANCISCO–(BUSINESS WIRE)–Epicrispr, a clinical-stage company pioneering gene-modulating therapies, today reported early clinical data from its ongoing first-in-human, open-label study evaluating EPI-321, an investigational epigenetic therapy for facioscapulohumeral muscular dystrophy (FSHD).
Epicrispr holds the first and only open epigenetic editing Investigational New Drug (IND) authorization in the United States, with additional Clinical Trial Application (CTA) clearances in New Zealand and Australia. The ongoing open-label study is primarily designed to evaluate safety and tolerability, with multiple exploratory efficacy endpoints. The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to EPI-321 for the treatment of FSHD.
To date, three participants have completed their three-month follow-up visits. Across these participants, Epicrispr has observed favorable performance, including improvements across a broad range of evaluable strength and functional measures. Performance across these measures generally exceeded outcomes observed in an external comparator cohort, providing early evidence of biological activity.
Importantly, no serious adverse events and no severe adverse events have been reported to date among 4 dosed participants.
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