Epicrispr Biotechnologies, a clinical-stage company pioneering epigenetic therapies, today announced that it has been awarded an $8 million grant from the California Institute for Regenerative Medicine (CIRM) to support the ongoing first-in-human clinical trial of EPI-321, the company’s lead investigational epigenetic therapy for facioscapulohumeral muscular dystrophy (FSHD).

The grant, awarded through CIRM’s Clinical Trial Stage Projects program, will support clinical development of EPI-321, a first-in-class, one-time therapy designed to durably silence expression of DUX4, the genetic driver of FSHD, without altering the underlying DNA sequence.

“This $8 million award from CIRM represents important validation of our innovative epigenetic editing approach and the potential of EPI-321 to transform treatment for patients living with FSHD,” said Amber Salzman, Ph.D., Chief Executive Officer, Epicrispr Biotechnologies. “Our recent clinical data demonstrated encouraging increases in lean muscle volume and strength following a single administration of EPI-321, reinforcing our confidence in its potential to address the underlying cause of this devastating disease. We are grateful for CIRM’s support as we continue advancing EPI-321 through clinical development and toward our goal of delivering the first disease-modifying therapy for people living with FSHD.”

The award follows Epicrispr’s recent presentation of positive six-month clinical data from its ongoing Phase 1/2 study at the 31st Annual Congress of the World Muscle Society. Results demonstrated increases in lean muscle volume across total-body, upper-body and lower-body composite measures, accompanied by improvements in muscle strength, including in muscle groups commonly affected early in FSHD. These findings provide encouraging preliminary evidence of biological and clinical activity following a single administration of EPI-321.

“CIRM is committed to accelerating the development of innovative therapies that have the potential to transform the lives of patients with serious unmet medical needs,” said Maria Chiuchiolo, PhD, a science officer in clinical development at CIRM. “FSHD is a devastating progressive neuromuscular disease with no approved disease-modifying treatments.”

The ongoing open-label study is evaluating the safety, tolerability and preliminary biological and clinical activity of EPI-321 in adults with FSHD, a progressive genetic neuromuscular disease characterized by the inappropriate expression of DUX4, a gene that is normally silenced in healthy skeletal muscle. Aberrant DUX4 expression leads to progressive muscle degeneration, weakness and disability. There are currently no approved therapies for FSHD.

About CIRM

The California Institute for Regenerative Medicine (CIRM) is a state agency created by California voters to accelerate stem cell and gene therapies for people with unmet medical needs. Since 2004, Californians have entrusted CIRM with $8.5 billion to accelerate promising discoveries through clinical trials, train a regenerative medicine workforce, strengthen the state’s biotechnology economy, and expand access to transformative treatments. Today, CIRM is pioneering new models of therapy development and accelerating medical breakthroughs that change lives — in California and around the world.

About EPI-321

EPI-321 is an investigational epigenetic therapy that aims to address the underlying molecular mechanisms of FSHD with a one-time dose. Following intravenous administration, EPI-321 is directed to muscle tissue within a single AAV vector, which has been clinically validated for muscle delivery. Preclinical studies on EPI-321 have demonstrated its ability to robustly suppress pathological expression of the DUX4 gene and reduce muscle cell death. EPI-321 previously reported interim data from the Phase 1/2 trial, including statistically significant increases in whole-body lean muscle volume measured by MRI, favorable changes in circulating biomarkers consistent with DUX4 suppression, favorable strength and functional outcomes, and a manageable safety profile.

About Facioscapulohumeral Muscular Dystrophy (FSHD)

Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common forms of muscular dystrophy, affecting more than an estimated 1 million people worldwide. FSHD is a progressive genetic neuromuscular disease characterized by the gradual weakening and loss of skeletal muscle, often beginning in the face, shoulders, and upper arms before progressing to other parts of the body. The disease is caused by aberrant expression of DUX4, a gene that is normally silenced in healthy muscle tissue but becomes activated in individuals with FSHD, leading to muscle damage, inflammation, and progressive muscle degeneration. There are currently no approved disease-modifying therapies for FSHD.

About Epicrispr Biotechnologies

Epicrispr Biotechnologies is a biotechnology company pioneering gene-modulating therapies, leading with treatments for neuromuscular diseases. The company’s proprietary Gene Expression Modulation System (GEMS) enables precise and durable epigenetic modulation of gene expression, unlocking first-in-class treatments for previously untreatable conditions. Epicrispr’s lead program, EPI-321 is in clinical trials for FSHD, and the company is advancing additional gene-modulating therapies. Learn more at www.epicrispr.com or follow us on LinkedIn.

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