Epicrispr Biotechnologies has raised $90 million in financing to support late-stage clinical testing of EPI-321, the company’s epigenetic treatment candidate for facioscapulohumeral muscular dystrophy (FSHD).
“This financing marks a pivotal milestone for Epicrispr as we advance EPI-321 and the next generation of programmable epigenetic medicines,” Amber Salzman, PhD, CEO of Epicrispr, said in a company press release.
Epicrispr said the financing will support pivotal clinical testing, clinical studies aimed at proving the therapy is effective and forming the basis for regulatory approval.
FSHD is a genetic disease marked by abnormal activation of the DUX4 gene in muscle cells. This abnormal gene activation damages muscles, leading to muscle weakness and wasting, particularly in the face, shoulder, and upper arm muscles.
Ongoing trial evaluates safety
EPI-321 uses epigenetics — molecular modifications that change how DNA is packaged within cells — to essentially shunt the DUX4 gene into storage, deactivating the gene to prevent its toxic effects. The therapy delivers its genetic payload to cells using a viral vector, a virus that’s been engineered so it can be used for medicine instead of causing infection. The company has a partnership with Forge Biologics, which is supporting the manufacturing of the vector for EPI-321.
A Phase 1/2 clinical trial (NCT06907875) testing EPI-321 in adults with FSHD is currently underway. The main goal is to evaluate safety, and according to Epicrispr, results have so far been favorable. Early biomarker data from the trial indicate that EPI-321 treatment increased lean muscle volume and suppressed DUX4 gene activity.
The new financing was led in part by the investment firm Octagon Capital. As part of the financing, Octagon analyst Anran Li, PhD, will join Epicrispr’s board.
“Epicrispr has established itself as a leader in the field of programmable epigenetic medicine,” Li said. “The team has demonstrated exceptional execution by translating a differentiated platform into encouraging early clinical data for EPI-321 in a remarkably short period of time. We believe Epicrispr’s proprietary technology, strong leadership team and expanding clinical pipeline position the company to define an entirely new therapeutic modality, and we are excited to partner with the team as they advance this important work.”
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