Epicrispr and Forge Biologics partner on AAV manufacturing for EPI-321 FSHD gene therapy

Edited by Robert McTiernan··2 min read

Epicrispr Biotechnologies and Forge Biologics have announced an AAV manufacturing partnership to support the clinical development of EPI-321, an investigational gene therapy for facioscapulohumeral muscular dystrophy (FSHD), according to a press release issued May 5, 2026.

Under the agreement, Columbus, Ohio-based Forge Biologics is providing San Francisco-based Epicrispr with AAV process development, current Good Manufacturing Practice (cGMP) manufacturing, and analytical development services. Epicrispr is also accessing Forge’s proprietary Fuel platform, which includes HEK293 suspension Ignition Cells, the pEMBR 2.0 adenovirus helper plasmid, and an optimized AAVrh74 rep/cap plasmid. Financial terms were not disclosed.

EPI-321 and FSHD gene therapy program

EPI-321 is an investigational, single-dose therapy designed to silence aberrant DUX4 expression in skeletal muscle, the molecular driver of progressive muscle degeneration in FSHD. The candidate is delivered intravenously via an AAVrh74 vector and is positioned within Epicrispr’s Gene Expression Modulation System (GEMS) platform, which the company describes as enabling durable control of gene expression through epigenetic regulation rather than permanent DNA alteration.

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The program has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the US FDA. EPI-321 is currently being evaluated in a first-in-human Phase I/II study (NCT06907875) across sites in the United States, New Zealand, and Australia. Material manufactured by Forge at its 200,000-square-foot Hearth facility in Columbus is being used to supply that ongoing trial.

Early data from the study, as cited in the announcement, indicate that EPI-321 demonstrated favorable clinical activity and was well tolerated, with no serious adverse events reported to date. Patients treated at the six-month assessment point showed gains in muscle volume compared to baseline, a result the company contrasts with declines typically observed in natural history datasets for FSHD. Improvements in strength and function, increased lean muscle volume on whole-body MRI, and reductions in circulating DUX4 biomarkers were also cited.

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