Epicrispr secures $68M for FSHD epigenetic editing trial

Amber Salzman vividly recalls a time when she felt powerless to aid her cousin-in-law suffering from facioscapulohumeral muscular dystrophy (FSHD), a progressive muscle disorder. Despite her success as a pharmaceutical executive in developing a groundbreaking gene therapy for her son, who battled a severe neurological disease, she found herself without a promising solution for FSHD due to the limited understanding of its cause at the time. “I’m a drug developer. I’m not a basic research scientist,” Salzman expressed, highlighting her frustration and lack of direction in combating FSHD.

Today, Salzman leads Epicrispr Biotechnologies, formerly Epic Bio, a company that recently secured $68 million in Series B funding to explore epigenetic editing treatments for FSHD. This form of muscular dystrophy primarily affects the muscles of the shoulders, arms, and face. Epicrispr has gained approval from New Zealand regulatory bodies to initiate a clinical trial for their novel epigenetic editing treatment in adults with FSHD, with plans to administer the first dose within the year.

Over the past two decades, significant scientific advancements have paved the way for this innovative therapy. Researchers discovered that the root cause of FSHD is the abnormal activation of the DUX4 gene, which produces toxic molecules that harm muscle tissue. Additionally, the advent of CRISPR-based editing tools has revolutionized the approach to tackling genetic diseases, offering new pathways for treatment development.

Epicrispr, established by Stanley Qi, a former member of Jennifer Doudna’s lab and now a professor at Stanford Medicine, has developed an experimental treatment named EPI-321. This one-time treatment aims to suppress the expression of the DUX4 gene. It employs viral vectors known as AAVs to deliver the therapy, which functions by methylating the DNA to silence the gene expression without cutting the DNA itself. “What’s really exciting is we are actually going after the root cause,” Salzman stated, emphasizing the significance of their approach targeting the fundamental genetic issue.

The Series B funding was spearheaded by Ally Bridge Group and included participation from SOLVE FSHD, a venture fund initiated by Lululemon founder Chip Wilson, who himself has been diagnosed with FSHD. This highlights the persistent interest and investment in the field of neuromuscular diseases, even amidst a general decline in the gene therapy and gene editing sectors.

This sustained investor enthusiasm is reflected in recent industry movements, such as Novartis’s acquisition of Kate Therapeutics, a muscular dystrophy gene therapy developer, for $1.1 billion in November. Concurrently, Sarepta entered into a $500 million upfront agreement with Arrowhead Pharmaceuticals to collaborate on a range of experimental treatments for rare diseases, including FSHD.

Epicrispr’s endeavor marks a promising stride in addressing FSHD, offering hope for those affected by the disease. With considerable backing and groundbreaking technology, the company is poised to make significant advancements in understanding and treating this challenging condition. As Salzman and her team move forward, they continue to push the boundaries of what is possible in genetic medicine, paving the way for future breakthroughs in the treatment of muscular dystrophies and other genetic disorders.