The Pharmaletter

One To Watch

Epicrispr Biotechnologies

An American biotech company developing gene-modulating therapies, particularly for neuromuscular diseases.

Epicrispr Biotechnologies, a South San Francisco-based biotech company, focuses on developing gene-modulating therapies, particularly for neuromuscular diseases. The company's proprietary Gene Expression Modulation System (GEMS) enables precise and durable control of gene expression, offering potential treatments for previously untreatable conditions. ​

In March 2025, Epicrispr secured a $68 million Series B financing led by Ally Bridge Group, with participation from SOLVE FSHD and other investors. The funds will support the clinical development of EPI-321, a first-in-class, disease-modifying therapy for facioscapulohumeral muscular dystrophy (FSHD). EPI-321 is an investigational one-time gene-modulating therapy designed to silence aberrant expression of the DUX4 gene, which is incorrectly activated in FSHD and leads to progressive muscle degeneration. Delivered systemically via a clinically validated AAV vector, EPI-321 has demonstrated robust suppression of DUX4 expression and protection of muscle tissue in preclinical models. The therapy has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations. ​

In October 2023, Epicrispr entered a research collaboration and license agreement with Kite, a Gilead Company, to develop next-generation cancer cell therapies. This partnership allows Kite to leverage Epicrispr's gene regulation platform to potentially enhance CAR T-cell functionality. ​

Want to Update your Company's Profile?


More Epicrispr Biotechnologies news >