Epicrispr Biotechnologies has been awarded an $8 million grant from the California Institute for Regenerative Medicine to support the ongoing first-in-human Phase 1/2 clinical trial of EPI-321, its investigational epigenetic therapy for facioscapulohumeral muscular dystrophy, or FSHD.
The funding was awarded through CIRM’s Clinical Trial Stage Projects program and will support continued clinical development of EPI-321. The therapy is being developed as a one-time treatment designed to durably silence expression of DUX4, which Epicrispr identifies as the genetic driver of FSHD, without altering the underlying DNA sequence.
EPI-321 remains investigational and has not been approved for commercial use. The ongoing open-label Phase 1/2 study is evaluating its safety, tolerability, and preliminary biological and clinical activity in adults with FSHD.
FSHD is a progressive genetic neuromuscular disease associated with inappropriate expression of DUX4, a gene normally silenced in healthy skeletal muscle. Aberrant DUX4 expression is associated with progressive muscle degeneration, weakness, and disability. There are currently no approved disease-modifying treatments for FSHD.
The CIRM award follows Epicrispr’s presentation of six-month clinical data from the ongoing study at the 31st Annual Congress of the World Muscle Society. The company reported increases in lean muscle volume across total-body, upper-body, and lower-body composite measures, along with improvements in muscle strength, including in muscle groups commonly affected early in FSHD.
Epicrispr characterized the results as encouraging preliminary evidence of biological and clinical activity following a single administration of EPI-321. Because the study remains ongoing and early-stage, further clinical evaluation will be required to establish the treatment’s safety and efficacy.
Epicrispr is developing epigenetic therapies designed to regulate gene expression without directly changing the DNA sequence. EPI-321 is the company’s lead clinical-stage program and is intended to address FSHD by targeting DUX4 expression at its source.
CIRM is a California state agency created to accelerate development of stem cell and gene therapies for serious unmet medical needs. Since 2004, California voters have authorized $8.5 billion to support research, clinical development, workforce training, and efforts to expand access to regenerative medicine therapies.
KEY QUOTES:
“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. 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.”
Amber Salzman, Ph.D., Chief Executive Officer Of Epicrispr Biotechnologies
“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. FSHD is a devastating progressive neuromuscular disease with no approved disease-modifying treatments.”
Maria Chiuchiolo, Ph.D., Science Officer In Clinical Development At CIRM
