Company to present at J.P. Morgan Healthcare Conference on Tuesday, Jan 13, 2026
Epicrispr, a clinical-stage company pioneering gene-modulating therapies, 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).
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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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The external comparator is drawn from ReSolve, a prospective, longitudinal, multi-national natural history study conducted over two years in more than 240 symptomatic adult FSHD patients. Of these, 171 patients meet the enrollment criteria for Epicrispr’s EPI-321 first-in-human study, creating a robust and well-matched comparator arm for contextualizing early clinical observations.
“FSHD is a progressive disease with currently no treatment options,” said Amber Salzman, Ph.D., CEO, Epicrispr. “The consistency of improvement we’re seeing across these participants, together with a clean safety profile and comparison against a high-quality natural history dataset, gives us confidence that EPI-321 is engaging relevant disease biology. While these data are early, they reinforce the promise of epigenetic editing as a fundamentally new therapeutic approach.”
Epicrispr’s epigenetic editing platform is designed to activate or repress gene targets, either transiently or durably, without cutting DNA, offering the potential for a differentiated safety and durability profile relative to nuclease-based approaches. EPI-321 targets the underlying genetic driver of FSHD by suppressing pathogenic gene activity at its source.
The study remains ongoing, and Epicrispr plans to continue enrolling participants and evaluating safety and exploratory efficacy outcomes over time.
Epicrispr CEO Amber Salzman, Chief Executive Officer, will deliver a company presentation followed by a Q&A session at the 44th Annual J.P. Morgan Healthcare Conference on Tuesday, January 13, 2026, at 5:30 pm PT.
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Source- businesswire
