Epicrispr raises $68M for epigenetic editing in rare muscle disease testing

Epicrispr Biotechnologies, a company based in the San Francisco Bay Area, has successfully secured $68 million in funding to develop a pioneering genetic medicine targeting a rare neuromuscular disorder known as facioscapulohumeral muscular dystrophy (FSHD). The company's main initiative, EPI-321, leverages CRISPR technology to inhibit the errant expression of a gene associated with this muscle-wasting disease. Epicrispr plans to initiate a Phase 1 clinical trial in New Zealand later this year, as announced in a statement released on Wednesday. The recent Series B funding round was led by Ally Bridge Group and included participation from Solve FSHD, an advocacy organization established by Chip Wilson, the founder of Lululemon Athletica.

FSHD is a rare neuromuscular condition estimated to affect approximately 870,000 individuals globally. It is characterized by the progressive weakening of muscles, initially manifesting in the face, back, and upper arms, which can lead to significant disability, pain, and fatigue, often confining individuals to wheelchairs. Currently, there are no approved treatments for FSHD. Recent research has focused on the DUX4 gene, as its overexpression due to a genetic anomaly leads to muscle degeneration and atrophy. Various biotech companies are exploring different methods—including small molecule drugs and gene therapies—to mitigate this issue.

Despite setbacks, such as the failure of an oral drug developed by Fulcrum Therapeutics and Sanofi in Phase 3 trials last year, numerous companies like Avidity Biosciences, Novartis, Arrowhead Pharmaceuticals, and Dyne Therapeutics continue to work on potential treatments for FSHD. According to the FSHD Society, there are currently over a dozen active drug programs targeting DUX4.

Epicrispr distinguishes itself by utilizing CRISPR technology not to alter DNA directly, but to regulate gene expression by turning genes on or off. In the context of FSHD, the company is employing CRISPR to bind to a specific region of the DUX4 gene and induce a chemical modification, with the aim of halting the expression of the encoded protein while avoiding the risks associated with DNA modification.

CEO Amber Salzman, who has extensive experience in genetic disorders as both a biotech executive and patient advocate, remarked on the uniqueness of Epicrispr's approach. Her personal connection to genetic conditions is profound—her son and two nephews were diagnosed with adrenoleukodystrophy (ALD) while she worked at GSK years ago. This connection led her to collaborate with gene therapy researcher Jim Wilson and establish the nonprofit Stop ALD Foundation. Although one of her nephews succumbed to ALD in 2004, her son and second nephew received a treatment that was eventually approved as Skysona.

Salzman’s journey through various biotech startup roles, including at Adverum Biotechnologies, led her to Epicrispr in 2021. By that time, the startup was already researching FSHD, which also affected her husband’s family—a factor that motivated her to join the company. Epicrispr's innovative approach to epigenetic editing and its broad potential applications across diverse diseases convinced her to take on the leadership role.

In addition to the Series B funding, Epicrispr had earlier raised $55 million in a Series A round in 2022. Beyond FSHD, the company is working on developing treatments for heterozygous familial hypercholesterolemia, alpha-1 antitrypsin deficiency, a couple of eye diseases, and certain unspecified blood cancers, all of which are still in the preclinical phase. Epicrispr was co-founded by Stanley Qi, a researcher from Stanford who collaborated closely with gene editing pioneer Jennifer Doudna at UC Berkeley.