A Phase 1/2 Open-label Dose-escalation Study to Evaluate the Safety, Tolerability, and Biological Activity of EPI-321, an AAVrh74-delivered Epigenetic Editing Therapy in Adult FSHD Patients
Latest Information Update: 02 Feb 2026
At a glance
- Drugs EPIC 321 (Primary)
- Indications Facioscapulohumeral muscular dystrophy
- Focus Adverse reactions; First in man
- Sponsors EPIC BIO; Epicrispr Biotechnologies
Most Recent Events
- 13 Jan 2026 According to an Epicrispr Biotechnologies media release, the company reported early clinical data from an ongoing first-in-human, open-label study evaluating EPI-321 for facioscapulohumeral muscular dystrophy (FSHD). To date, three participants have completed their three-month follow-up visits. The study remains ongoing, with plans to continue enrolling participants and to further evaluate safety and exploratory efficacy outcomes over time.
- 13 Jan 2026 According to an Epicrispr Biotechnologies media release, company received approval for CTA application for phase I/II trial in facioscapulohumeral muscular dystrophy in New Zealand and Australia before January 2025.
- 08 Jan 2026 Results presented in an Epicrispr Biotechnologies media release