A San Francisco Bay Area biotechnology company has secured $68 million in Series B funding to advance its innovative treatment for
facioscapulohumeral muscular dystrophy (FSHD), a rare neuromuscular condition affecting approximately 870,000 people worldwide. The funding round was spearheaded by Ally Bridge Group, with participation from Solve FSHD, an advocacy organization established by Lululemon Athletica founder Chip Wilson.
Epicrispr Biotechnologies is developing EPI-321, a pioneering genetic treatment that employs CRISPR technology to suppress the expression of DUX4, a gene linked to FSHD progression. The company plans to initiate Phase 1 clinical trials in New Zealand this year.
FSHD causes progressive muscle deterioration, initially affecting the face, back, and upper arms, potentially leading to wheelchair dependency and severe pain and fatigue. While no approved treatments currently exist, several pharmaceutical companies have focused their efforts on targeting the DUX4 gene, whose abnormal expression triggers muscle degeneration.
The field has seen both setbacks and ongoing development efforts. A notable disappointment occurred last year when Fulcrum Therapeutics and Sanofi’s oral medication failed in Phase 3 trials. However, multiple companies, including Avidity Biosciences, Novartis, Arrowhead Pharmaceuticals, and Dyne Therapeutics, continue to pursue
DUX4-targeting treatments. The FSHD Society reports over twelve active drug programs targeting this gene.
Epicrispr’s approach stands out for its use of epigenetic editing, which modifies gene expression without directly altering DNA sequences. The company’s technology uses CRISPR tools to create specific chemical modifications that prevent DUX4 protein production while avoiding the risks associated with DNA cutting.
CEO Amber Salzman, who has extensive experience in genetic disorder research and advocacy, reports promising preclinical results showing the treatment’s potential to impact muscle function and prevent DUX4 protein leakage. The company’s strategy targets what Salzman describes as “the absolute root cause” of the disease.
Salzman’s leadership of Epicrispr is deeply personal, shaped by her family’s experiences with rare genetic conditions. While at GSK, her son and two nephews were diagnosed with adrenoleukodystrophy (ALD). This led her to collaborate with gene therapy expert Jim Wilson and establish the Stop ALD Foundation. Though one nephew succumbed to ALD in 2004, her son and other nephew received successful treatment with what later became Skysona.
The connection to FSHD through her husband’s family influenced Salzman’s decision to join Epicrispr, formerly known as Epic Bio, in 2021. The company’s potential to address limitations in genetic medicine she had encountered throughout her career particularly appealed to her.
Beyond FSHD, Epicrispr is developing treatments for various
conditions, including heterozygous familial hypercholesterolemia, alpha-1 antitrypsin deficiency, specific eye diseases, and certain blood cancers. All programs remain in preclinical stages.
The company’s scientific foundation stems from co-founder Stanley Qi’s work at Stanford University, where he collaborated with CRISPR pioneer Jennifer Doudna at UC Berkeley. This latest funding builds upon a $55 million Series A round raised in 2022, positioning Epicrispr to advance its innovative epigenetic editing platform toward clinical development.
The continued investment in Epicrispr reflects growing interest in novel approaches to genetic medicine, particularly those that might offer safer alternatives to traditional gene editing methods. As the company moves toward clinical trials, it joins a broader ecosystem of biotechnology firms working to address previously untreatable genetic conditions.