Epicrispr Biotechnologies, a US-based clinical-stage biotechnology company developing epigenetic therapies, has received an $8 million grant from the California Institute for Regenerative Medicine (CIRM) to support clinical development of EPI-321, its investigational treatment for facioscapulohumeral muscular dystrophy (FSHD).
Announced on October 19, 2026, the grant was awarded through CIRM’s Clinical Trial Stage Projects program and will support the company’s ongoing first-in-human Phase 1/2 clinical trial.
EPI-321 is designed as a one-time epigenetic therapy that suppresses abnormal expression of the DUX4 gene, which contributes to the progressive muscle degeneration associated with FSHD. The treatment aims to regulate gene activity without changing the underlying DNA sequence.
The ongoing open-label clinical study is evaluating EPI-321’s safety, tolerability and preliminary biological and clinical activity in adults with FSHD, a genetic neuromuscular disorder that causes progressive muscle weakness and disability.
Epicrispr reported updated six-month clinical results on October 5, 2026, from the first six patients evaluated in its Phase 1/2 study. According to the company, the preliminary findings showed increases in lean muscle volume and improvements in muscle strength following treatment. Further clinical evaluation remains necessary to establish the therapy’s safety and effectiveness.
Founded by Stanford University bioengineer Stanley Qi, Ph.D., Epicrispr develops gene-regulating medicines using its proprietary Gene Expression Modulation System (GEMS). The platform is designed to activate or suppress specific genes through epigenetic mechanisms without permanently altering DNA.
The South San Francisco-based company is led by Chief Executive Officer Amber Salzman, Ph.D., and focuses on developing therapies for genetic diseases, including neuromuscular disorders.
In August 2026, Epicrispr closed a $90 million Series C financing round co-led by Octagon Capital and Janus Henderson Investors. The financing was intended to support further clinical development of EPI-321, expand the company’s therapeutic pipeline and strengthen its manufacturing capabilities.
The additional $8 million CIRM grant will support Epicrispr’s ongoing clinical investigation of EPI-321 as the company advances its development program for FSHD.
